TIF Magazine - Issue 67

TIFMAGAZINE

Regional Conference 2nd Pan-Asian Conference on Haemoglobinopathies, Vietnam, 26-27 September 2015

PUB L I S HE D BY THE TH AL A S SAE M I A I NTE R NAT I O NAL FE D E R AT I O N

Precious Rubies

TIF awarded!

International Thalassaemia Day 2015

TIF received WHO's 2015 Dr Lee Jongwook Memorial Prize for Public Health

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Foreword

from TIF’s President and Executive Director

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n behalf of the Board of Directors of the Thalassaemia International Federation (TIF), we are delighted to present you issue 67 of TIF Magazine.

This has been a year of unique and commendable successes, as well as unprecedented challenges.

Over the course of 2015, TIF delegations visited countries around the globe, including India, China, Egypt, Vietnam, the Maldives, Albania, Germany, the Kingdom of Saudi Arabia, Algeria and Morocco. TIF also took part in a range of meetings and conferences, in the US, Switzerland, Germany, the UK and Spain. At the same time, courses and projects proceed apace, such as the MSc on Inherited Haemoglobin Disorders: Thalassaemia and Sickle Cell Syndromes (Blended), the Thal e-course, the e-ENERCA project and TIF’s electronic applications for smartphones, with further developments expected in 2016.

Most importantly, TIF has been awarded the World Health Organisation (WHO)’s prestigious Dr Lee Jong-wook Memorial Prize for Public Health, for people or organisations that have made an outstanding contribution to the field.

Being recognised in this way reaffirms our conviction that with hard work, courage and determination, our dreams are within reach: no challenge can deter us from achieving our goal of quality healthcare for every patient, anywhere in the world (read more on page 16). TIF

has also been awarded a prize by the University of Nicosia, an internationallyrecognised academic institute, based in Cyprus.

These successes act as a bridge to an even more exciting and challenging phase. As 2016 approaches, TIF’s objectives focus still more on improving the education of patients/parents, strengthening their capacity to manage their disease more effectively and to become equal and valued stakeholders in decision/policymaking. TIF strives to move beyond being patient-centred towards patient inclusiveness.

TIF’s educational programme had an extremely successful year.

The 2nd Pan-Asian Conference on Haemoglobinopathies organized in Hanoi, Vietnam between the 26th and the 27th of September 2015 was an exciting event focused on regional needs and expectations, offering hundreds of patients a forum to interact with international health experts from a range of disciplines.

As ever the 8th of May—International Thalassaemia Day—was marked with great zeal by associations everywhere. In Cyprus, TIF organised a Musical Evening and Gala Dinner for the Ambassadors of TIF’s member countries. The event, entitled "Precious Rubies", was held at the Presidential Palace in Nicosia, under the auspices of His Excellency the President of the Republic of Cyprus, Mr Nikos Anastasiadis, and the Honourable First Lady, Mrs Andri Anastasiadou. A dedicated section of this issue offers

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detailed coverage of the International Thalassaemia Day events around the world.

However, much remains to be done.

There are still countries where thalassaemia is prevalent but which lack national prevention and treatment programmes or reimbursement policies. There is a shortage of drugs in many areas, and the provision of multi-disciplinary care is limited. No more than 30% of patients worldwide receive the optimal care recommended by international experts (as per TIF’s Guidelines for the Management of

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Transfusion Dependent Thalassaemia, 3rd Edition, 2014, available here: http://goo.gl/MlceyR TIF is, therefore, redoubling its focus on networks in 2016, pressing for greater collaboration with national and international health authorities and organisations to achieve its mission of quality care for all. Our driving force remains the determination and courage of our patients, who inspire, guide and motivate us to address the numerous challenges they face.

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CONTENTS 2nd Pan-Asian Regional Conference on Haemoglobinopathies

TIF’s receipt of the World Health Organizatio

(WHO’s) Dr Lee Jong-wook Memoria

International Thalassaemia Day 2015 Precious Rubies

Patient’s Story

Cover Photograph “Awareness about Thalassaemia” by Mr Dipayan Bhar (India), winner of the first prize of TIF's 2015 Photography Contest (read more on page 18).

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Chief Editor Dr Androulla Eleftheriou TIF Editor Liana Prastiti

on’s al Prize

Design and Layout Liana Prastiti Images and Graphics StockInDesign Freepik Pixabay

Medical Corner

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Proof Reading Helen Perry Lily Cannon

Foreword from TIF President and Executive Director TIF activities around the world

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International Collaborations

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TIF’s receipt of the World Health Organization’s (WHO’s) Dr Lee Jongwook Memorial Prize for its outstanding contribution to Public Health

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International Thalassaemia Day 2015

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TEAM

2nd Pan-Asian Regional Conference on Haemoglobinopathies TIF Publications Member's News Patient Story

TIF BOARD

MEMBERS Panos Englezos, President Shobha Tuli, Vice President Loizos Pericleous, Secretary George Constantinou, Assistant Secretary Riyad Elbard, Treasurer Her Highness Sheikha Sheikha Bint Seif Al-Nahyan Anton Skafi Christina Stephanidou Duru Malyali Fatemeh Hashemi Ivan Dimitrov Ivanov Loris Angelo Brunetta Mouna Haraoui Ramli Mohd Yunus Robert (Bob) Ficarra Saeed Jafaar Al-Awadhi Gabriel Theophanous

TIF's Projects TIF's Smart Phone Applications TIF’s Thal e-Course TIF's Video Challenge Competition Calendar and upcoming Events MSc Course in Inherited Haemoglobin disorders (Blended) Medical and Scientific Update News and Updates

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Technical specifications for photographs 1. Photographs submitted for TIF Magazine should be in JPEG format (although .PDF and .TIFF are also accepted).

2. Photos should be “print quality”. This means:

• The largest size possible. Please do NOT reduce the images! Send them either one by one via email, or alternatively on a CD.

• High-resolution (at least 300 dpi). Please ensure that when transferring photos from a digital camera, a high resolution is selected (How this is done depends on the camera model.)

3. All photos should have captions - a short text explaining what is happening in the photo and the names of the people in the photo.

4. It is the responsibility of the person submitting photos to ensure that any person(s) featured in them has given their permission to publish the photo.

Disclaimer: Reproduction of material published in TIF Magazine for educational purposes is encouraged, provided it is accompanied by the following attribution “…according to TIF magazine, the official newsletter of the Thalassaemia International Federation”. The contents of any scientific article or presentation of any material by manufacturers does not imply the expression of any opinion on the part of Thalassaemia International Federation. The mention of specific companies or products does not imply that they are endorsed or recommended by TIF in preference to others. The contents express the opinions of the authors who alone are responsible for the views expressed. TIF does not accept any legal responsibility for their contents.

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TIF around the world of Laboratory Science in Cairo between the 18th and the 20th of April 2015.

Eastern Mediterranean Region (EMR) TIF sets the pace for Iran Iran’s programme for the prevention and treatment of haemoglobinopathies is a model for the region.

The provision of optimum care for all, however, will require greater accessibility to treatment, as well as the harmonisation of care to ensure a multidisciplinary approach across the entire country.

In this context, TIF conducted a delegation visit to Iran between the 16th and the 18th of September 2015. The objectives of the delegation visit were to map the current situation with regard to issues of concern to patients/parents and health providers alike. These include the use of locally manufactured copy and generic drugs, a shortage of infusion pumps, and inconsistency in standards of, and approaches to, multi-disciplinary care between regions and specialised units, particularly in rural areas. TIF is now better able to assist and support national efforts to instigate further improvements to existing services, including the establishment of reference centres, and satellite treating centres in rural areas.

TIF also participated in the Iranian Paediatric Haematology/Oncology Society (IPHOS) Congress.

Egypt: A need to move forward.... building on past success...

Since 2011, Egypt’s national health authorities have faced considerable challenges. The patients’ movement had been a consistent compelling element in instigating changes. In support of these efforts, TIF attended the Egyptian Thalassaemia Association’s workshop on Laboratory Medicine: Digging for More Treasures, at the Egyptian Society

TIF delegates also met with leading medical experts and discussed the Memorandum of Understanding (MOU) drawn up between TIF and Egypt’s Ministry of Health.

Once signed, the agreement will enable TIF to draw up a tailored framework of activities in the country. A planned second visit is expected to result in an MOU for the implementation of prenatal services and treatment, in particular the provision of drugs free-of-charge to patients over the age of 18.

Kingdom of Saudi Arabia (KSA): Reactivation and re-empowerment TIF has embarked on a process of reinforcing lines of communication with important stakeholders in KSA, with the aim of ensuring uniformity in the quality of care across the country. This effort

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A TIF delegation visit to Jeddah between the 6th and 7th November 2015 aimed to revisit incomplete activities, with a view to moving on to new projects and programmes. A key success was the work towards the adoption of national guidelines for the clinical management of thalassaemia, which would help to address regional variations in levels of care, particularly in rural areas. The delegation visit also presented a great opportunity for TIF to strengthen its network of experts in the country, as well as to investigating ways to support patient and parent associations in their quest to build their capacities and skills.

At the same time, high-calibre academic resources will facilitate dramatic advances in the care of patients with haemoglobinopathies in KSA. A workshop organized by TIF in collaboration with the Saudi Thalassaemia Friends’ Society, entitled Moving Forward in Saudi Arabia: Status of health care services and beyond in Jeddah on the 7th of November 2015, was held under the auspices of the King Abdulaziz University and the King Fahd Medical Research Centre. Participants discussed the need to establish thalassaemia reference centres and the development of nationwide multi-disciplinary care.

Following the delegation visit, it is hoped that a consensus document that will be drawn up and shared with national health authorities. A possible follow-up visit in 2016 would consolidate the outcome of the earlier delegation visit, and could include the organisation of a large educational event for patients/parents as part of the International Thalassaemia Day celebrations.

Laying the ground for success in Pakistan Thalassemia is the most prevalent genetic blood disorder in Pakistan. As a result, thalassaemia constitutes a major challenge to the national healthcare system, as well as a significant

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psychological and financial burden on affected families.

In the context of promoting the development and implementation of a comprehensive national policy on thalassaemia, an official delegation visit was conducted between the 27th and the 30th of November 2015, in Karachi and Lahore, Pakistan.

Furthermore, TIF was invited to participate in the faculty of speakers at 10th National Thalassaemia Conference.

The objectives of the visit were to strengthen collaboration between TIF and Pakistan’s national associations, sprearheaded by the Thalassaemia Federation of Pakistan (TFP), and to meet with the ministries of health of Sindh and Punjab provinces.

The Karachi workshop had a very positive impact, updating medical specialists and parents/ patients on latest advances in the prevention and management of thlassaemia, while working to sensitise policymakers to the importance of developing a comprehensive provincial/ national strategy to address haemoglobinopathies in Pakistan.

Kuwait: Steadily advancing towards change...

An official delegation from Kuwait’s Ministry of Health visited the Nicosia (Cyprus) on the 20th of April 2015, as part of on-going efforts to strengthen collaboration with TIF and the Ministry of Health of Cyprus.

The purpose of the visit was to exchange information and experiences of the prevention and management of thalassaemia, and to update TIF on current approaches to the control (prevention and management) of haemoglobinopathies in Kuwait.

Western Pacific Region (WPR) China’s continued fight for better care!

Over the years TIF has established valuable relationships with key organisations in China, and has visited the country on numerous occasions since 2002.

Collaboration between TIF, the National Family Planning Commission (NFPC) and China’s medical community, combined with good planning by provincial governments, has resulted in the implementation of effective prevention

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marks a continuation of TIF’s valuable work to improve the situation regarding thalassaemia in KSA.

10 African Region (AFR) Algeria: committed to moving forward...

TIF’s work to establish links with Algeria’s medical and patient communities, as well as its hard-won success in building partnerships with national health authorities, is an important and on-going effort.

programmes in many affected provinces, with other provinces now starting to follow suit.

Over the last year, TIF has also made a concerted effort to work closely with local patient associations.

Members of one such organisation— the Angel Mom Foundation—accompanied

A workshop and press conference organised by TIF in collaboration with Algerian stakeholders in May 2015, to coincide with International Thalassaemia Day, underscored a shared commitment to effectively address inherited haemoglobinopathies in the country. The meeting was organised by the Algerian Ministry of Health, local health authorities and the El Tarf Hospital, and took place in Willaya of El Kala, in the northeast of Algeria where both thalassemia and sickle cell disorders are prevalent.

The meeting was attended by at least 150 participants, including the Director and Deputy Director of the Prevention and Promotion Department of the Ministry of Health, representatives of local health authorities, several eminent physicians from Algiers and Annaba, as well as patients and parents.

The event constituted an important part of TIF’s effort to further awareness of thalassaemia in Algeria, and to further sensitise the local health authorities as well as the community about the disorder.

European Region (EUR) Promising times ahead for Azerbaijan

representatives of the Chinese Philanthropy Research Institute and the NFPC on a visit to Cyprus, holding discussions with TIF management and the Minister of Health of Cyprus, as well as visiting the WHO Thalassaemia Collaborating Centre and the Thalassaemia Research Unit at the Cyprus Institute of Neurology and Genetics.

TIF plans to participate in the 2ndThalassaemia Alliance Conference in Guilin City, Guangxi Province between the 13th and the 14th December 2015.

Provincial Ministry of Health officials have been invited to attend.

A TIF delegation to Baku between the 16th and the 18th of November 2015 has further strengthened partnerships with local patient/parent and medical/ scientific communities in Azerbaijan, as well as with the country’s Ministry of Health. The TIF delegation was comprised of Dr Anver Kuliev, Ex-head of the WHO’s Hereditary Diseases Programme, and TIF Medical Adviser, Dr Michael Angastiniotis, who held meetings with medical specialists, patients/ parents and officials from Azerbaijan’s Ministry of Health.

Within the framework of this visit, TIF was able to map approaches to the prevention and management of haemoglobinopathies, including multi-disciplinary monitoring, with a view to developing a national policy. Following the visit, TIF aims to relaunch close communication with Azerbaijan’s Ministry of Health, to support national efforts for the implementation of a prevention programme.

TIF plans a follow-up delegation visit to support the organisation of a National Thalassaemia

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11 collaboration between stakeholders has facilitated improvements in basic services, particularly blood transfusion and iron chelation. However, a number of key issues remain, especially in terms of the adequate provision of supplies and regional inconsistencies in the quality of and accessibility to treatment, as well as a lack of multi-disciplinary care, including the use of MRI T2*.

Germany prepares for a brighter future

A TIF delegation visit to Tirana between the 25th and the 26th of June 2015 aimed to further strengthen

TIF is committed to further strenghening its communication and collaboration with stakeholders in Germany, a country with a robust health infrastructure and a tradition of high quality medical practice.

In response to great interest from members of the patient community in Germany, TIF visited Hamburg, Germany between the 24th and the 27th of September 2015, to learn more about the provision of patient services, including multidisciplinary care.

In the course of the visit, the TIF delegate met patient/parent representatives in Ratzburg, taking part in a roundtable ‘brainstorming’ meeting organised with a view to improving the cooperation and consolidation of patient groups, strengthening and increasing their capacity for advocacy.

TIF reiterates its commitment to supporting patient association in Hamburg in as many ways as possible, including translating key TIF publications into German. An official TIF delegation is likely to visit Berlin between the 21nd and the 23rd of April 2016, coinciding with the Pan-German Thalassaemia Meeting.

Successful steps in Albania!

TIF delegates have visited Albania on several occasions since 2004, establishing a collaborative relationship with national health authorities and supporting the participation of health professionals, patients and parents in a variety of TIF educational and training programmes. This

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collaboration with Albanian stakeholders, including meetings with Ministry of Health officials responsible for thalassaemia and representatives of patients’ associations. The aforementioned weaknesses in treatment were discussed, enabling the TIF team to propose methods to improve the effectiveness of patient care and prevention in Albania.

TIF plans to develop a joint plan of action for 201618, along with a follow-up visit in 2016 to monitor progress.

Exciting new chapter unravels for Romania!

Romania is a country which has been improving steadily its health services over recent years on the back of rising health expenditure per capita, helping to reduce the infant mortality ratio to 10/1000 live births. Despite these advances, however, significant gaps in the treatment and prevention of thalassaemia remain.

A TIF delegation visit to the capital, Bucharest, in early 2016 aims to promote TIF’s activities and goals in Romania, and to find out the latest developments and policies regarding health care services for rare and chronic disorders through meetings with the country’s Ministry of Health. TIF reiterates its aim to further support national efforts to address the control of thalassaemia, and to identify solutions to improve thalassaemia patients’ quality of life and access to quality healthcare.

The aims of the delegation visit are to promote its activities and achieve its goals in Romania and to learn about any new developments / policies in the field of health and especially regarding promoting health care services for the rare disorders and chronic disorder patients. In addition, TIF aims to further support national efforts in addressing the issue of control of thalassaemia, highlight gaps and identify solutions to improve the quality of life and access to quality healthcare of thalassaemia patients.

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Conference, to update medical specialists on advances in the prevention and treatment of thalassaemia, as well as build the capacity for selfadvocacy among patient/parent groups, through education on thalassaemia. TIF is also considering offering support for the training of two medical professionals and a laboratory scientist through its training fellowship programme.

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International Collaborations

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IF at the European Patients’ Forum (EPF) Annual General Meeting (AGM) in Brussels TIF Executive Director, Dr Androulla Eleftheriou represented the Federation at the European Patients’ Forum (EPF) Annual General Meeting (AGM) in Brussels on the 19th of May 2015. The AGM provided an excellent opportunity to learn about what has been achieved in 2014, discuss and adopt strategic priorities for the year to come, meet colleagues from around Europe, exchange ideas of common interest and celebrate being part of the European patients’ movement.

Elections to the EPF board were also held.

The AGM was followed by a two-day conference entitled Empowered Patients are an Asset to Society, held between the 20th and the 21st of May 2015, marking the launch of a major campaign on patient empowerment that will run until June 2016. The empowerment of patients is currently a key theme at the EU level, with a number of initiatives underway to support patients to be more active in the management of their own health. This campaign aims to move from discussion towards concrete practical and political action, led by the patient community in partnership with all relevant stakeholders.

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13 TIF is proud to have received a prestigious prize—the University of Nicosia Outstanding Contribution Award, granted annually to individuals or institutions in recognition of their contribution to society. The award The award is a welcome recognition of TIF’s work. However, it is also recognition of the broader importance of efforts aimed at the control and clinical management of the haemoglobinopathies—conditions which, until a few decades ago, were fatal, with a life expectancy of little more than 10 years. TIF’s work has played a key role in raising awareness of such conditions, and increasing their visibility in the wider community. was accepted by TIF President, Mr Panos Englezos and TIF Executive, Director Dr Androulla Eleftheriou, during the University’s Graduation Ceremony held on the 25th of June 2015.

We are grateful for the encouragement inherent to this award, which deepens our commitment to continue our work and motivates our future endeavours.

EURORDIS Membership Meeting (EMM 2015)

Participation in the 20th European Hematology Association (EHA) Congress

TIF Operations Manager, Ms Lily Cannon attended the EURORDIS Membership Meeting (EMM), held in Madrid between the 29th and the 30th of May 2015. The annual EMM provides over 200 patients, patient organisations, policy makers and other stakeholders with networking opportunities and capacity-building workshops to improve the lives of those living with a rare disease.

TIF delegates attended the 20th European Haematology Association (EHA) Congress held in Vienna, Austria between the 11th and the 14th of June 2015, represented by Mr George Constantinou, TIF Assistant Secretary, Dr Androulla Eleftheriou, TIF Executive Director and Ms Lily Cannon, TIF Operations Manager.

Mr Constantinou participated in the Patient Advocacy Track, with an immensely informative presentation on ‘Access to Quality Healthcare: Challenges and successes of the UK experience’.

EMM workshops enable patients to develop their knowledge and ability to advance policies and services for rare disease in their countries and communities. The Meeting also provides an opportunity for EURORDIS members to share best practice and to discuss issues integral to member organisations and to the rare disease community and environment in different countries.

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A pioneer in the field of non-malignant haematology, TIF continues its efforts to raise awareness amongst haematologists in Europe and across the world.

The Federation has assumed a

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TIF was granted the Outstanding Contribution Award of the University of Nicosia, Cyprus!

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leading role in the EHA Patient Advocacy Track, contributing to its formulation and participating in two sessions, which this year focused on ‘Collaborating with patients for successful haematology research and for assessment of optimal benefits and risks’ and ‘Partnership and evidence: Key elements to improving access to treatment’.

In addition to the presentation by TIF Board Member George Constantinou, TIF educational material was available from a dedicated exhibition booth overseen by TIF Operations Manager, Ms Lily Cannon. Over 500 copies of TIF’s the Clinical Guidelines on Transfusion-Dependent Thalassaemia (2014) were distributed.

to be incorporated in the School’s MSc in Family Medicine.

It has also been announced that the Medical School and TIF are to jointly award the biannual Panos Englezos Prize to an individual who has made an outstanding international contribution to the field of haemoglobinopathies or public health. The Prize was established by the TIF Board of Directors and its International Advisory Panel in 1997, in honour of TIF President, Panos Englezos, whose immense, steadfast and voluntary work to promote and safeguard the rights of patients around the world, irrespective of religion, social and cultural differences, continues to this day.

The event also marked EHA’s 25th Anniversary and the publication of a commemorative booklet entitled Fresh Blood, which includes an interview with Dr Androulla Eleftheriou.

The Congress provided an excellent opportunity for further discussions concerning the development of a Road Map on European Haematology Research—a project which begun in June 2014. The Road Map aims to inform and engage stakeholders with a direct interest in haematology, and to promote research into blood disorders in the context of the EU’s Horizon 2020 funding scheme.

TIF has been directly involved in the drafting and reviewing of several chapters pertaining to haemoglobinopathies.

University of Nicosia Medical School signs Memorandum of Understanding (MOU) with TIF The Medical School of the University of Nicosia, Cyprus and TIF have signed a memorandum of understanding (MOU) to promote collaboration in the field of haemoglobinopathies. Among other actions and activities, the Medical School with the support of TIF, will develop and offer an elective module on anaemias and rare disorders,

Collaboration between TIF and the European Medicines Agency (EMA) opens up new opportunities!

TIF European Countries Officer, Mr Stelios Elia, attended the European Medicines Agency (EMA) Training Session for Patients’ and Consumers’ Organisations, held between the 25th and the 26th of November 2014 in London, England.

The event included an overview of the Agency, its procedures, how patients are involved in drawing up scientific advice, the Scientific Advisory Group (SAG) meetings, the review of information to patients, and pharmacovigilance.

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2nd Global Innovation for Patients’ Forum TIF Executive Director, Dr Androulla Eleftheriou participated in the 2nd Global Innovation for Patients Forum, which took place in Basel, Switzerland, on the 1st of July 2015.

This Forum was attended by 63 patient-advocates from 25 countries worldwide, representing 20 disease areas. Its purpose was to enhance the continuing dialogue between patient leaders, to deepen the understanding of patients’ experiences and strengthen interactions with patient organisations (PO) around research. This included informing participants about the progress on the patient perspective in all activities.

Specifically, trends for patient engagement in research represent an opportunity of collaboration through the worldwide Patient Research Exchange

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(PRE) platform, which was co-created with patient advocates, showcased for patient-advocates’ feedback. Successful PO initiatives around patientbased evidence, and the correlation with trends in real-world evidence were presented, and learnings and opportunities for other POs were discussed.

Across the many discussions during the day, a number of points were frequently made, which shows their relevance to the dialogue and enhancement of patients’ roles in research, and to the on-going conversation between stakeholders in the healthcare continuum.

Among the key points raised were the need for POs to be more involved in the generation and communication of real-world evidence: registry data in particular are key tools for a constructive dialogue with governments. Further, it was underlined that long-term planning and continuity of engagement by pharmaceutical companies is essential to successful collaborations. At the same time, processes are needed to ensure POs and patient-opinion leaders (POLs) remain independent.

This conference was very fruitful as it stimulated interaction between participants and supported long-term innovation. It also allowed TIF to network with other partners.

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The workshop served to reiterate EMA’s commitment to engage in dialogue with European patients and consumers, enabling patients and organisation representatives to engage with the Agency on key issues, highlighting the real-life implications of regulatory decisions and thereby improving the decision-making process.

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World Healh Organization (WHO) ONE OF THE MOST PRESTIGIOUS HONOURS OF WHO HAS BEEN GRANTED TO TIF!

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IF is proud to announce that it has been awarded the WHO’s Dr Lee-Jong Wook Memorial Prize for Public Health.

The WHO has presented the Dr Lee Jongwook Memorial Prize for Public Health to TIF at the 68th World Health Assembly on the 21st of May 2015 in Geneva, Switzerland.

Since its establishment in 1987, TIF has worked to improve the lives of patients everywhere, supporting patient/parent organisations and building partnerships with national health authorities to establish and strengthen policies aimed at the prevention and clinical management of thalassaemia, working towards the goal of high quality care for all.

The holistic management and prevention of genetic diseases such as haemoglobinopathies crosses many disciplines. At the same time, these conditions represent a considerable social and economic burden.

TIF therefore promotes not just disease-specific policies and programmes, but also those oriented at broader public-health issues.

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The empowerment of patients and parents across geographic, cultural, religious, social and linguistic borders, and their transformation into equal partners with national and international health bodies is one of TIF’s major success stories, and remains the main focus of its work.

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Another important part of our activities is our work with the WHO. TIF is privileged to be have been in official relations with the WHO since 1996, promoting and contributing to many of its programmes, both disease-specific and public health.

A key objective of international public health-related institutions such as the WHO is the development of patient-centred healthcare systems and the achievement of health equity. As such, partnerships with non-governmental organisations such as TIF is of paramount importance to both sides.

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TIF has long recognised the instrumental role played by patient/parent associations in the development and implementation of disease-specific control programmes, both in supporting existing programmes and in motivating new initiatives.

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Precious Rubies

Special focus on the International Thalassaemia Day on the 8th of May 2015!

The evening of the 6th of May 2015 was marked in Nicosia by an exquisite Musical Evening and Gala Dinner, organized by TIF to honour International Thalassaemia Day. The event, entitled “Precious Rubies”, took place at the Presidential Palace in Nicosia, under the auspices of His Excellency, the President of the Republic of Cyprus, Mr Nicos Anastasiades, and the Honourable First Lady, Mrs Andri Anastasiadou.

The event was also attended by His Highness Sheikh Dr Sultan Bin Khalifa Al Nahyan of the United Arab Emirates (UAE), Adviser to the President of the UAE and President of the Sultan bin Khalifa Al-Nahyan Humanitarian and Scientific Foundation, and Her Highness Sheikha Sheikha bint

Seif Al-Nahyan, Vice-President of the Foundation, Chairperson of Emirates Thalassemia Society and TIF Board Member. The royal couple were joined by ministers, ambassadors, and international health experts, along with representatives of patient organisations and the international scientific and medical communities.

Opening speeches were made

by President Anastasiadis and TIF President, Panos Englezos, during which the award of the WHO’s Dr Lee Jong-wook Memorial Prize for Public Health to TIF was officially announced.

The award is the highest honour in the field of public health, granted to an international NGO for outstanding contributions to the field.

The musical programme began with the Cyprus Young Strings Soloists with piano accompanist Jana Drhov, under Prof Matheos Kariolou, Artistic Director of the Ministry of Education and Culture’s Musical Talent Development Programme.

Paris-based singer/songwriter Vakia Stavrou, accompanied by pianist Argyro Christodoulides continued with a blend of brio and sweet nostalgia, showcasing work by Edith Piaf, Nat King Cole, Manos Hadjidakis, Yves Montand, Rita Hayworth, Frank Sinatra and many more.

The evening ended with the first-ever performance of the official anthem for thalassaemia, composed by world-renowned composer George Theofanous with lyrics by Stavros Stavrou, and sung by the Musical Workshop children’s choir, under

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the direction of Mr Theofanous.

thalassaemia across the world.

TIF’s International Photography Contest inspires participants worldwide!

The theme for the TIF International Thalassaemia Day 2016 will be:

TIF has received the enthusiastic response from participants from all over the world who took part in the International Photography Contest organised by TIF in the context of the International Thalassaemia Day 2015.

Access to safe and effective health care in thalassaemia

The aim of the contest was to raise awareness about thalassaemia, through the year’s theme “Enhancing partnership towards patient-centred health systems: Good health adds life to years”. A total of 125 entries were received by the deadline of the 18th of April, with the names of the winners announced through social media.

The winners of TIF’s International Photography Contest are: First Prize of $500: Dipayan Bhar

TIF’s Activities for 2016: TIF has many events planned for International Thalassaemia Day 2016 and a detailed programme will be uploaded on our website soon!

Toolkit for International Thalassaemia Day Materials available include: International Thalassaemia Day poster International Thalassaemia Day logo International Thalassaemia Day video

International Thalassaemia Email banner

Second Prize of $300: Pushkar Vyas

Third Prize of $100: Victor Hugo Casillas Romo

TIF’s Photography Contest to mark International Thalassaemia Day 2016

Congratulations to all those who took part, helping to spread awareness about the rights of patients with

The 3rd TIF International Photography Contest has been launched, with the aim of building awareness about thalassaemia in the context of International Thalassaemia Day, on the 8th of May 2016.

For more information, visit: http:// goo.gl/WHSkZP Share your event!

Associations, organisations, health professionals and academics are encouraged to share their events with us. Send details of your activities to thalassaemia-lp@thalassaemia.org.cy

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SPECIAL HIGHLIGHTS

OF THE 2ND PAN-ASIAN CONFERENCE ON HAEMOGLOBINOPATHIES, 26-27 SEPTEMBER 2015, HANOI, VIETNAM The 2nd Pan-Asian Conference on Haemoglobinopathies The scientific programme was a major event, held in Hanoi, Vietnam between the 26th and the 27th of September 2015.

The majority of presentations were given by experts in thalassaemia in Asia, with additional specialist topics The conference gathered together over 700 also covered. participants—some 500 health professionals and 200 patients/parents from 30 countries in the region along with Vietnam’s Deputy Minister of Health, Dr Session on treatment updates Pham Le Tua, and WHO South East Asia Regional Office (SEARO) representative, Dr Aparna Singh Shah. Overviews of transfusion-dependent and nontransfusion dependent thalassaemias were provided

by Prof Chi Kong Li (Hong Kong) and Dr Anuja Premawazdehena (Sri Lanka), covering epidemiology, pathophysiology, and clinical features. In a study of 109 patients from Sri Lanka with HbE/beta-thalassaemia, a strong association between Erythropoietin (EPO) levels and Hb was found, as well as declining EPO response to anaemia with age.

Haematopoietic stem cell transplantation (HSCT) in Asia was reviewed by Dr Suradej Hongdeng (Thailand), The conference was divided into a scientific conference who noted that in his country the probability of for regional health professionals and a patient/ finding a matched related or unrelated donor was parent conference for patients, families and support 30-60%, and that disease survival levels were 70associations. Patient participation was supported 90%. New conditioning regimens have been tried through TIF’s education sponsorship programme. using Treosulfan, which has reduced toxicity, and also The conference was chaired by TIF President, Panos the use of pre-transplant immunosuppression using Englezos and Prof Nguyen Anh Tri, Director of Fludarabine and dexamethasone followed by the the National Institute of Haematology and Blood conditioning regimen. Haploidentical dononations Transfusion and the Vietnam Thalassaemia Association. have achieved 88% disease-free survival.

The scientific committee was chaired by Prof Suthat Fucharoen, Professor of Haematology and Head of the Thalassaemia Research Centre at the Institute of Science & Technology for Research of Mahidol University, Salaya Campus, Thailand.

Symposium on Blood Safety During this symposium, blood transfusion issues in Asia were reviewed by WHO representative,

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21 Vietnam, Myanmar, and Sri Lanka.

Satellite symposium

During the satellite symposium, optimal management of iron overload, was discussed by Dr Vip Viprakasit, (Thailand), who emphasised the need for Liver Iron Concentration (LIC) and myocardial iron measurements. Comprehensive thalassaemia care programme, was another topic discussed by Dr Dr Ngo Manh Quan, (Vietnam) proceeded in Hishamshah Imbrahim (Malaysia). Malaysia has 6,246 examining the situation in the country. In Vietnam, 480,000 blood units go to thalassaemia patients; paid registered patients, of which 42% have thalassaemia major, 33% have HbE/beta-thalassaemia, 10% have donations fell to 1.13% in 2014 while replacement donations are at 2.6%. Viral hepatitis transmissions are beta-thalassaemia and 11% have HbH disease. This low—on average below 1%—although Hepatitis B Virus large burden of disease is not evenly distributed (HBV) is still 2.6%. Dr Erica Wood (Australia) described across the country (25% reside in Sabah). Planning of services should therefore be guided by epidemiology, blood safety practices in Australia. Transfusion in thalassaemia was reviewed by Dr Chi Kwong Lee (Hong to develop a comprehensive programme with an integrated, multi-disciplinary approach.

Kong) who emphasised the need for extended red blood cell (RBC) antigen typing. Prof Yesim Aydinok (Turkey), then, discussed mitigating infectious risks in Opening Ceremony transfusion practice

Free Oral Presentations and Country Reports The programme continued with a session of free oral presentations. Subjects covered included: Comparison of Deferasirox in single or twice-daily

dosage, concluding that single-dosing regimes are more effective than divided doses (Dr Shruti The first day opened with an official ceremony, with Kakkar, India) a-thalassaemia: the Maldivian story, presented by welcoming addresses from the co-chairmen, Panos Englezos and Prof Nguyen Anh Tri, each of whom Dr Zileena Zahir Sanguinate as a therapeutic agent for thalassaemia emphasised the importance of the conference to was presented by Dr Kittiphong Paiboonsukwong the countries of the Far East. The Deputy Minister of Health of Vietnam welcomed participants on behalf of Thailand. This is a new compound, active in of the Vietnamese government, followed by Keynote oxygen transfer which also enhances blood flow presentations of Dr Androulla Eleftheriou, TIF Executive Evaluation of iron overload in thalassaemia patients was presented by Dr Nguyen Thi Thu Ha Director, on access to care and global approaches to prevention and of Dr Philippe Leboulch (France) who of Vietnam A review of endocrinopathies following HSCT was presented. It was concluded that performing HSCT at a young age with better pre- and posttransplant chelation reduces gonadal dysfunction and hypogonadism. Busulfan dosage showed correlation with the incidence of hypogonadism.

Presented by Wing Shan Queenie See, Hong Kong HSCT results from northern Iran were presented by Dr Tamaddoni Quality of life of transfusion dependant discussed recent results of gene therapy trials. thalassaemia (TDT) patients in Sri Lanka was presented by Dr RM Mudiyanse, Sri Lanka The ceremony ended with a performance of traditional Microparticles inducing platelet activation in splenectomised HbE/beta-thalassaemia patients, Vietnamese music. presented by Dr Saovaros Svasti, Thailand The second day began with a session including a A study from Australia showed dysregulation of state-of-the-art presentation on iron overload and innate immune responses in beta-thalassaemia. its management. Speakers were Dr Vip Viprakasit Reduced chemotaxis and neutrophil maturation (Thailand) on iron metabolism and iron load, Prof John may be related to genes that regulate phagocytosis and ROS production (Dr Jim Vadolas) Porter (UK) on iron chelation in TDT, and Dr Ibrahim Mohamed Hishamshah (Malysia) on iron chelation in non transfusion dependant thalassaemia (NTDT) Country reports were presented in a separate session, providing epidemiological and other information from The second session of the second day concerned complications in thalassaemia syndromes. Liver disease Malaysia, India, Indonesia, Philippines, Bangladesh, related to viral hepatitis, presented by Dr Dimitrios

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Dr Aparna Singh Shah. The South East Asia region has 4,151 blood centres and national policies in almost every country. There are 16 million annual collections, 88% from voluntary donors. In the Western Pacific region, 17 of 37 countries have a national blood policy, with 21.7 million units collected each year. However, in some countries less than 1% of the population donates blood.

22 Kountouras (Greece), who highlighted the good results of current Hepatitis C Virus (HCV) treatment, despite high costs. Cardiovascular complications, reviewed

by Dr Athanasios Aessopos (Greece), emphasising the prevention of congestive heart failure by good iron chelation. Endocrinopathies, reviewed by Dr Shau Yin Ha (Hong Kong), who emphasised the need for regular monitoring. He also drew attention to the need for holistic care through a coordinated multi-disciplinary team. Infections in thalassaemia, reviewed by Prof Suthat Fuchareon (Thailand), who also drew attention to specific conditions indigenous to the region, such as fungal infections including Pythiosis. He noted that innate immunity dysfunction may play a role and so prevention is necessary through vaccinations, alerting families that fever may signal a serious infection and to seek early medical advice. Broad-spectrum antibiotics should be used with adequate supportive care. Silent cerebral ischaemia in TDT, described by Dr Mehran Karimi (Iran). In a study of 40 TDT patients, 15 (37.5%) were found to have asymptomatic infarcts (compared to 28% of NTDT). There was no significant correlation with serum ferritin

of which collaborating with strong patient support groups will make requests more effective.

The patient/parent programme

Under the title “Empowering patients”, the patient programme ran in parallel with the scientific conference. As the title implies, the objective was to empower patients to become partners in their care A session on new advances also took place. Dr Paul through knowledge of the protocols and choices that Kwo (Hong Kong) reported on the results of the new combination of Grazoprevir and Elbasvir in chronic HCV they may have in their treatment, supporting and being supported by their physicians. In addition, patients infection. MRI as a tool for monitoring iron overload are expected to support their physicians in advocacy, was reviewed by Dr Tim St Pierre (Australia) and Dr which will prompt health officials to upgrade services Pairash Saiviroonporn (Thailand), who described the aimed at improved survival and quality of life. With this Thai experience. Prof John Porter (UK) described the philosophy in mind, the programme was designed to novel erythropoiesis stimulating agents with the end include all subjects covered in the scientific sessions, of the phase-2 clinical trials. HSCT practices in South China were described by Dr Huaying Liu. In the next session screening and counselling were discussed by Dr Roshan Colah (India), who described the practices and difficulties faced in prevention in India and Asia, Dr Chanane Wanapirak (Thailand), who reviewed obstetric approaches to prevention, and Dr Wirawit Piyamongkol (Thailand), who reviewed the role of pre-implantation genetic diagnosis.

Public health issues were also a part of the debate. Dr Fred Piel noted that severe HbH disease in this region affected 9,568 births annually, while 5,183 pregnancies were affected by alpha-thalassaemia hydrops fetalis.

The beta-thalassaemia births were estimated at 22,989, while HbE/beta thalassaemia births reached 19,129.

He pointed out that these estimates are inaccurate because of lack of data but they do demonstrate that haemoglobinopathies are a global health burden.

Health Technology Assessment (HTA) was described by Prof Ying Yao Chen of China as a multi-disciplinary field of policy analysis aiming to establish safe, effective, cost-effective, ethical, legal and social practices in providing health care services. Lastly, generic and substandard drugs were reviewed by Dr Mahmood Hadipour Dehshal of Iran.

with the same speakers presenting their subjects in a manner suitable for a lay audience.

In addition to tailored material presented in the scientific programme, the session also covered more patient-orientated subjects, including sexual development and problems of adolescence, quality and efficiency of life-saving drugs, psychosocial support, the role of Associations in education and advocacy, organising an association – building up infrastructure, a day in the life of a patient, generic and sub-standard drugs, understanding clinical trials and the patient’s perspective and patient advocacy and update on recent gene therapy trials.

During the closing ceremony the two conference chairpersons emphasised that the scientific session was not just an update on new developments but also, for many Asian medical practitioners, a starting point for introducing optimum and evidence-based care. This requires advocacy to health authorities, in respect

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23

The following summary represents data from 65 Patient/ Parent questionnaires and 93 Healthcare Professional questionnaires.

Future Participation

In both groups, most attendees reported that they would like to attend another TIF event in the future, but only if the costs of travel and accommodation were covered. Many attendees Reasons for Participation stated that they would also come if their expenses were only partially covered, or not covered at all. Only 3 attendees In both the patient/parent and healthcare professional groups, of all 158 that completed questionnaires reported that out they the most reported reason for participation was the need to be would not attend another TIF event in the future. updated with reliable information on haemoglobinopathies.

Future Topics

General Quality and Utility

Patients and parents reported that the quality of the scientific presentations and the utility to their associations’ work exceeded relevance to their concerns and reasons for their participation. These were all reported to be better than the opportunity to develop collaborations/partnerships/networks and the format of the conference. For healthcare professionals, the quality of the scientific presentations and the relevance of the conference to their concerns and the reasons of their participation exceeded the utility to their professional work, opportunities to develop collaborations and partnerships, and the format of the conference. Therefore, future conferences may require a more organised format and more networking opportunities in order to meet the interests of patients, parents and healthcare professionals.

Attendees from both sections provided suggestions for future conference topics. Patients and parents suggested the psychological side of care, transitioning into adult care, patients’ experiences, inspirational sessions, fertility and pregnancy, employment and human rights of patients, exercise, and universal treatment and prevention. Healthcare professionals recommended sessions on national screening programmes, different chelation regimes, ‘Meet the Experts’, the results of HSCT, pregnancy, NTDT, endocrinology, sickle cell disease, country profiles, haemoglobin typing, ferritin, splenectomy, and severe iron overload.

Organisational Satisfaction

The questionnaires indicate that attendees were generally satisfied with organisational aspects of the conference. Patients Content and parents were most satisfied with the accommodation, conference staff, conference location and registration process.

Patients and parents reported that they would prefer more However, they noted that improvements could be made Q&A sessions and small group discussions, while the lectures the terms of conference materials, transport arrangeme in nts, and case studies may have been adequate or excessive. The and the exhibition area (which was not open to patients/ results of the healthcare professionals’ questionnaires indicate parents). Healthcare professionals were most pleased by the that they would have liked more case studies and Q&A audio-visual and translation facilities, accommodation, sessions, while less lectures and small group discussions would conference location. Improvements were suggested inand terms have pleased them. of conference materials and the exhibition area. While most

aspects of the conference were commended, future events Speakers should also focus on improving conference materials provided to attendees, the exhibition area, and transport arrangements.

The speakers of both the patient/parent and healthcare

professional sessions were well received. Patients and parents General Recommendations rated all speakers between 3.76 and 4.67 out of 5, while healthcare professionals rated all speakers between 3.98 and Beyond suggestions referred to above, attendees who 4.72 out of 5. completed the questionnaires provided a number of recommendations to improve the overall quality of the Expectations conference. These include making presentations available to everybody, using simpler language, more group discussions Most of the attendees reported that the conference met and focus sessions, less overlap between presentations, their expectations. Patients and parents said that this was true opportunities for doctors and patients to interact (patientbecause they received useful information, and healthcare doctor dinner), presentations from patients from different professionals stated that this was true because they updated countries, less density by adding another day or reducing their professional knowledge. the amount of material presented, new topics, and more information on chelators.

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Analysis of the Patient/Parent and Healthcare Professional Questionnaires of the Conference

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TIF’s Publications OUT NOW! PUBLICATIONS

Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT), 3rd Edition (2014), English ▀▀

TRANSLATIONS OF PUBLICATIONS

A Guide for the Haemoglobinopathy Nurse (2013), Greek ▀▀

COMING SOON PUBLICATIONS

Shorter Version of the Guidelines for the Management of TDT (2014), English, Turkish ▀▀ Shorter Version of the Guidelines for the Management of Non Transfusion Dependent Thalassaemia (NTDT) (2013), English, Turkish ▀▀ Update of the Guidelines for the Management of NTDT (2013), English ▀▀

TRANSLATIONS OF PUBLICATIONS

A Guide for the Haemoglobinopathy Nurse (2013),Farsi, Myanmar ▀▀ Guidelines for the Management of TDT 3rd Edition (2014),French, Turkish, Greek, Arabic, Chinese ▀▀ Sickle Cell Disease (2008), Arabic ▀▀

IN THE PIPELINE! POSITION PAPERS

It is TIF’s policy to present its position on key issues of concern to patient groups, health authorities, professionals and society as a whole. Position papers are curently being prepared on the following topics: Liver disease in thalassaemia - liver disease is emerging as a major cause of morbidity and mortality in thalassaemia patients. TIF therefore maintains that its prevention, monitoring and case management must be a priority for thalassaemia services. ▀▀

Effective monitoring of patients - TIF is concerned that few centres follow full monitoring protocols, with the result that only a minority of patients are benefiting from early identification of and protection from potential threats to their health. ▀▀

Adherence to treatment – the need for strict adherence to chelation and other aspects of the thalassaemia treatment protocol, as per international guidelines, is a subject that must be revisited, especially in view of new therapies that may lead to a final cure, but from which patients may only benefit if they maintain good overall health ▀▀

The first of these position papers will be released before the end of 2015, with the remaining two due out in early 2016.

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25 T I F M AGA Z I N E

ADVERT

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Members’ News West Pacific Region (WPR) Philippines The patient group of the Balikatang Thalassemia Association in the Philippines has sent news of many exciting developments.

The Association has forged a successful partnership with the Philippine Blood Centre, which invited members to their annual Dugong Bayani (Blood Heroes) event, at which thalassaemia patients met with blood donors to thank them.

Following this event, a blood donation camp was held on the 14th of June 2015, as part of Blood Donor’s Day celebrations. A good percentage of the blood donated was provided to thalassemia patients free of charge. The Balikatang Thalassemia Association also participated in a blood donation activity held in July.

The Association is in the process of establishing regular blood donation activities in collaboration with a local university specialising in thalassaemia.

Congratulations to members of the Balikatang Thalassemia Association. We hope they continue their efforts with the same zeal!

China The 1st Hunan Thalassemia Congress, organised by the Beijing New Sunshine Charity Foundation New Sunshine Foundation and its branch in Hunan province to mark International Thalassemia Day, was held on the 9th of May 2015 in Changsha City, capital of Hunan province. The event was co-hosted by the Hunan Thalassemia Patients’ and Parents’ Volunteer Team, the Hunan Women’s Federation, the Hunan

Children’s Hospital and the China Democracy Alliance.

The Deputy Mayor of Changsha City, the Changsha Chief of the China Democracy Alliance and the Secretary-General of the Hunan TV Charity Fund joined around 400 participants, including thalassemia patients from south China and their families, medical professionals, government officials, and academia.

Australia The Thalassaemia Society of Australia marked the 8th of May with an information drive, setting out a stall at the Monash Medical Centre in Clayton to reach out, engage and inform the community about thalassaemia and sickle cell anaemia.

Another important event was the 9th Australasian Gene and Cell Therapy Society (AGCTS) Conference, held at the University of Melbourne between the 29th and the 30th of April and on the 1st of May 2015.

The Conference, which was sponsored by Thalassaemia Australia, featured excellent speakers whose exciting presentations reflected the huge effort made by the local organising committee.

Speakers covered a range of topics relative to thalassaemias and other associated conditions, but perhaps the one that drew most attention was the Update on Gene Therapy Clinical Trials for the Treatment of beta-haemoglobinopathies

by Prof Philippe Leboulch (Director, Institute of Emerging Diseases and Innovative Therapies, the bbu Institut national de la santé et de la recherche médicale (INSERM), University of Paris, France).

A detailed report on Conference proceedings will be available shortly.

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Turkey Members of the Mevlana Solidarity Association for Thalassemia Patients and their Families in İzmir (Mevlana Talasemi Hastaları ve Aileleri Yardımlaşma Derneği ile İzmir) held a friendly football match in the city on the 9th August 2015, with the goal—pun intended—of raising awareness about thalassaemia.

After the match, members joined the President of the Association to discuss thalassemia. But perhaps the most important message lay in the determination they had shown taking part in the match: that appropriate and regular treatment improved their health and quality life.

Pakistan The Hamza Foundation Welfare Thalassaemia Hospital and Blood Transfusion Services in Peshawar, Pakistan organised a Blood Donation Awareness Event to mark World Blood Donor Day on the 14th of June 2015, with guest of honour Prof Abaseen Yousafzay, Chairman of the Pushtu Department of the University of Peshawar and a well-known figure throughout Pakistan.

The theme of World Blood Donor Day 2015 was “Thank you very much for saving my life”. The main objective of the event was, therefore, to give thanks, to encourage volunteers to donate blood and to create awareness about blood donation.

The event was followed by an awareness-raising walk from the Peshawar Press Club, attended by doctors, journalists, students, social workers, politicians, patients and their families.

The Thalassaemia Society of Pakistan celebrated the International Thalassaemia Day, on the 8th of May 2015, with an awareness-raising campaign, organised in collaboration with major educational institutions of the city. Lectures, seminars and a walk were organised at the Liberty Market roundabout, a commercial hub of Peshwar.

A large number of patients, parents, social workers and volunteers gathered in the area to enjoy a

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colourful programme of performances, with gifts, certificates, prizes and toys distributed to children.

Morocco The Development Association for the Support of Patients with Blood Disorders (Association de Développement pour l’Aide aux Malades du Sang - ADAMS) in Morocco organised an event to mark International Thalassaemia Day, on the 23th of May 2015, with the aim of raising awareness of thalassaemia and other haemoglobinopathies.

The event was a great success, drawing together doctors, patients, families and other relevant stakeholders.

Egypt In Egypt, the 16th National Thalassemia Conference was organised to commemorate International Thalassaemia Day, held between the 6th and the 7th May 2015, in Cairo.

National and international scientists examined issues regarding thalassaemia, including clinical management, care, prevention and gene therapy.

The Conference also had a social programme, providing an opportunity to experience the culture and sights of Hurgada and Sharm El Sheikh.

South East Asian Region (SEAR) Myanmar The Myanmar Thalassaemia Association held an event at the Yangon Children Hospital in Myanmar attended by over 100 thalassaemia patients and their families.

The event included patient workshops run by haematologists and representatives of the National Blood Centre, as well as free bone mineral density measurement, free Hb antigen testing and free hepatitis B vaccination.

TIF’s cartoon booklet “All about Thalassaemia” (Myanmar translation) was distributed to all participants, along with handmade toys for the children present at the event.

Indonesia The Indonesian Thalassaemia Foundation organised a celebratory event in honour of International Thalassaemia Day, on the 8th of May 2015. This memorable event enriched the minds and hearts of all those who attended.

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Eastern Mediterranean Region (EMR)

28 India

In India the following activities were held: • The Federation of Indian Thalassaemics (FIT) observed International Thalassemia Day at a special event organised

at Sri Sathya Sai International Centre on the 8th of May, with chief guest Maneka Sanjay Gandhi, Union Minister for Women and Children Development. The celebrations included dancing and singing performances by the talented Swati Tuteja and the Bollywood children’s dance group from Shamak Davar’s Institute of Dance. Two brilliant authors—Jyoti Arora from Delhi and Chandan Das from Orissa—were also present to speak about their books Lemon Girl and The Road Taken.

The event was a big success, with over 500 guests including doctors, senior officials, social workers and blood bank staff.

• The Thalassemia & Sickle Cell Society of India celebrated the day on the 10th of May 2015, with activities, including amusements for children, that gained wide media coverage.

• The National Thalassemia Welfare Society (NTWS) observed the 21st International Thalassemia Day at the Constitution Club of India, Rafi Marg, New Delhi on 8 May 2015. Around 250 thalassemia patients, parents and thalassemia experts gathered to discuss the problems faced by thalassaemics, and to find means to solve those problems.

Many health officials were present at this event, where thalassaemia patients performed the Hindi version of Florilegium of Thalassemia, which was released on the 8th May 2015 by Sh Satyender Jain,

Minister of Health of the government of National Capital Territory of Delhi.

• The International Thalassemia Day 2015 was celebrated in Nagpur (Maharashtra) by the Thalassemia Society of Central India. An academic session was organised, followed by a cultural programme that included singing and dancing performed by thalassemia patients. In order to create awareness about thalassaemia, a poster contest workshop was organised by the Gunjan Organisation for Community Development ,in collaboration with the Students Welfare Office, National Service Scheme Unit, under the aegis of the State Institute of Health and Family Welfare, Parimehal.

• In Punjab on the 7th of May 2015, a voluntary blood donation camp was organised at the Dayanand Medical College and Hospital Ludhiana. At the Christian Medical College Ludhiana, a Seminar on Comprehensive Care for Thalassaemics was conducted, entitled “Way to Well-being”.

• The Blood Patients Protection Council (BPPC) organised an event to commemorate World Sickle Cell Day and World Blood Donor day.

The event took place on the 14th of June 2015 at the Regional Science Center and Planetarium, in Calicut.

Bangladesh In Bangladesh International Thalassaemia Day was honoured with the organisation of a celebratory event on the 8th of May at the Thalassaemia Welfare Centre of Bangladesh, Chittagong.

The event comprised of many fun activities for patients, including lottery draws and discussion sessions. The activities revolved mainly around spreading awareness and knowledge about thalassaemia.

The Bangladesh Thalassaemia Society marked International Thalassaemia Day with a roundtable discussion at the Centre on Integrated Rural Development for Asia and the Pacific (CIRDAP) Conference Hall, covering awareness, prevention and treatment. The discussion was presided over by Omar Golam Rabbany, Chairman of the Bangladesh Thalassaemia Society. The event was attended by haematologists, paediatricians, neonatologists, surgeons and eminent expert medical practitioners, as well as representatives of

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29 the Youth Club of Bangladesh, the Medicine Club of Bangladesh and other patients’ associations.

American Region (AMR) United States of America (USA) Many events and activities have been held across the US: • The Painted Turtle Camp was held on the 17th of July, in Los Angeles, California, where children with medical conditions celebrated just being kids!

Through innovative, camp-based programmes that offer great fun and support, children with more than 30 medical conditions visited the Painted Turtle, reclaiming the joys of childhood.

Opened in 2004, the Painted Turtle has offered thousands of children and their families hope and encouragement through its Summer and Family Weekend camps.

• Cooley’s Anemia Foundation (CAF) organised its 2015 Patient/Family Conference between the 26th and the 28th of June at the Wyndham Hamilton Park Hotel and Conference Center in Florham Park, New Jersey.

Entitled “Celebrating Self-Empowerment”, the 2015 Conference was a unique opportunity for individuals with thalassaemia and their families to meet other members of the community and learn important information that can have a positive impact on their health and lives.

• The Lighthouse at Chelsea Piers sported a special glow on 4 June, as Cooley’s Anaemia Foundation (CAF) descended upon the venue for its 2015 Gala.

“This year’s Gala looks to be a very special evening,” said Anthony J. Viola,

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Argentina The Thalassemia Association of Argentina in collaboration with the Network of Transfusion Medicine (GCABA) coorganised a Campaign to encourage voluntary blood donation, to commemorate International Thalassemia Day on the 8th of May 2015. The event was also supported by the National Blood Plan Department of Argentina’s Ministry of Health.

Blood donation awareness brochures were distributed during the event, underlining the importance of donating blood and informing the community about thalassemia. The campaign was a great success, with more than 90 individuals donating blood.

European Region (EUR) Portugal International Thalassaemia Day was celebrated in Portugal with many events organised by the Associação Portuguesa de Pais e Doentes com Hemoglobinopatias, in collaboration with other patient organisations and health professionals of the Alentejo region, in the south of the country.

A blood donation camp was organised in the city of Beja, along with a series of workshops at schools in the region, which has the highest prevalence of thalassaemia carriers in the country. The workshops informed students about haemoglobinopathies, while some sessions incorporated information on health services.These educational activities were an immense success, spreading awareness about thalassaemia across the region.

United Kingdom (UK) Family, friends and pets from across the UK and beyond joined the 2nd International Thalassaemia Day Walk, on the 10th of May 2015 of the UK Thalassaemia Society (UKTS) for a scenic stroll at Alexandra Palace, raising funds to support the Society’s invaluable work.

Read more, here: http://ukts.org/events/walk.html.

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The Association is pleased to report that the day was also marked by the President of the Republic of Bangladesh, along with the Prime Minister and the Minister of Health, and the Secretary and Director-General of the Health Ministry, all of whom made public statements of support for policies aimed at the prevention of thalassaemia.

CAF National President. “ • The Thalassemia Research at Children’s Hospital (UCSF) in Oakland organised its World Thalassemia Day 14th Annual Blood Drive on the 7th of May 2015.

This was followed on the 9th of May by the Root in the Boot Walkathon at Lafayette Reservoir in Lafayette, California. The event raised funds from sponsored walkers for Thalassemia Research at Children’s Hospital, Oakland.

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Patient’s Story Thalassemia in Iran: One Man’s Triumph: Mohammad Hosein Nikopour’s Story

W

By Josephine Bila

hat was it like growing up with Thalassemia in Iran?

I was diagnosed with thalassemia major when I was six months old, so I have spent my entire life with this condition. In all of these years I have come to accept that thalassemia is not a disorder, but a perpetual companion, perhaps my most loyal friend.

What do you think of blood donors?

I think that blood donors are an example of the most generous people on Earth. No words can ever express their sacrifice and dedication to help save lives.

How do you chelate?

I was among those thalassemia patients who were lucky enough to use Desferal shortly after my diagnosis — largely thanks to my family’s pursuit. When I was younger, I used to resist treatment with Desferal because of the pain that I had to endure. During my adolescence and early adulthood, I realised the significance of this drug in keeping me alive. Today I consider this drug and its infusion pump as my close companion that never leaves my side.

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31 do not affect my work. But if that is not possible, I get permission from my office.

How did your family treat you as you were growing up, compared to the other children with thalassemia in your clinic?

I was fortunate in that until my adolescent years I never felt alone in my trips to the hospital for treatment, because I was always accompanied by either my mother or uncle. They understood my situation very well and took very good care of me. It was useful for us to share our experiences with other thalassaemia families.

Through their compassion, my family always sought to create a caring environment for me. Despite having a good understanding of my condition and always being worried about me, my family sought not to transfer their worries to me.

If you could give advice to someone in India, Afghanistan, Iran or Pakistan about how to achieve the wonderful things you have achieved what would you say?

I recommend that by setting up NGOs with the support of patients who are highly motivated to participate in group efforts, they solve their problems, and together go through the treatment of thalassaemia, as healthy and normal people do.

Although a person with thalassemia may experience insufficiencies, he or she has capabilities from which others can benefit.

Thalassaemia patients should instead thrive on their high intelligence and hard work, while at the same time suffering from this blood condition.

What type of challenges did you face in school?

During those years in which thalassaemia had not yet been well enough introduced to the public, because of their lack of knowledge, sometimes teachers and kids did not act appropriately towards me and at times they even mocked me.

I remember that in elementary school, one of my teachers thought that when my parents took me out of school for treatment, they were actually lying to take me to the park or other adventures!

Wall Tile Mosaic by Mohammad Hosein Nikopour

How did you become a photojournalist?

I became interested in photography while I was enrolled in training courses for filmmaking. My passion for photography led me to engage with press circles, and my work was recognised and published in smaller publications. This experience eventually led me to enter professional media, and over time I became experienced in professional journalism.

How do you manage working and going to the hospital?

Usually I try to schedule my hospital visits and treatments on weekends so that they

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Beautiful Scene by Mohammad Hosein Nikopour

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Tell us how you stay positive and move forward to achieve your goals.

Although I, like everyone else, sometimes get overwhelmed with life’s difficulties, I try to escape them through my work, which I love and enjoy. I try to distract myself from anxieties and fatigue by reading books and articles on art and fictional stories, or by reading the newspaper.

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distinctive innovative unique

TIF’S PROJECTS TIF Renzo Galanello Fellowship The Renzo Galanello Fellowship, launched in 2013 under TIF’s Educational Programme, honours the late Prof Renzo Galanello, a pioneer in the field of thalassaemia research and management.

The Fellowship is offered to physicians and specialists in the field of haematology, paediatrics or internal medicine, usually from outside Europe where haemoglobinopathies are more prevalent. This programme was developed in collaboration with the Whittington Hospital National Health Service Trust in London, England.

As part of our effort to collaborate with other reference centres, training will now be offered through University College Hospital London, (UCLH) under the supervision of Prof John Porter, Professor of Haematology, and Dr Perla Eleftheriou, Consultant Haematologist.

chronic disorders are suboptimal or inadequate in many countries and health policy interest is diverted to other health priorities. Improving knowledge towards holistic care in thalassaemia and sickle cell disease has been a TIF priority since its establishment and this need has no geographical boundaries.

Improving policies for the prevention and management of these disorders constitutes a vast and invaluable contribution towards ameliorating national, regional and international public health and social burden. Since these diseases are genetic and polyorganic in nature, with lifelong dependency on blood, they bring considerable social and economic repercussions.

The Renzo Galanello Fellowship was until now offered to two candidates, covering a training period of two to four months, according to their needs. Our vision for the upcoming period, however, is to offer this training to as many candidates as possible, in order to educate more health professionals and so contribute to efforts to offer quality health care in as many countries as possible. We are therefore particularly pleased to have secured funding for four candidates, coming from Egypt, Maldives, Nigeria and Pakistan to follow the two-month training course.

Objectives Many countries lack trained medical staff and as result, the healthcare of haemoglobinopathy patients is less than optimum or even rudimentary. The lack of specialisation is partly due to a lower level of specialist Next training period: interest in non-malignant haematological disorders, The course begins on the 30th November 2015 but also because these disorders are regarded as rare and, as such, are not given priority or integrated into national strategies or programmes. Policies for rare and DECEMBER 2015 www.thalassaemia.org.cy

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We are overjoyed to share with you that TIF has attended the SITA Awards ceremony, which took place at the Emirates Palace in Abu Dhabi, United Arab Emirates, on the 19th November 2015.

During this event, Mr Panos Englezos, President of TIF was also honoured with ‘the special Medal of Honour for his longtime and global contribution in the field of thalassemia. This important distinction is of great value and reflects the timeless, voluntary and selfless contribution of Mr Englezos.

The Sultan Bin Khalifa International Thalassemia Awards scheme is a personal initiative of H.H. Dr Sheikh Sultan Bin Khalifa Al Nahyan, established in 2013 by His Highness Sheikh Sultan Bin Khalifa Al Nahyan Humanitarian and Scientific Foundation (SBKF) in partnership with TIF. The Award recognises scientists, medical professionals, researchers and patient associations that have made a significant, internationally recognised contribution to the benefit of patients with thalassaemia across countries and regions of the world. It also aims to bring together, motivate and empower regional and global medical, academic, scientific and research thalassaemia communities to facilitate still more improvements and achievements in the field.

TIF is actively involved in this initiative through the participation of TIF President, Panos Englezos, who is Co-Chairman of the Board of Trustees of Sultan Bin Khalifa International Thalassemia Award, and of TIF Executive Director Dr Androulla Eleftheriou as Head of the Higher Scientific Committee (HSC) of the SITA International Awards.

2015 SITA International Awards The Higher Scientific Committee (HSC) of the SITA International Awards has reviewed all applications and nominations. The work of the members of the HSC was very challenging, as they had to choose amongst strong candidates of high academic calibre and expertise.

The winners are: 1. SITA Grand International Award – Prof George Stamatogiannopoulos and Prof Maria Domenica Cappellini 2. SITA International Awards for Lifelong Achievement - Prof Sir David Weatherall.

3. SITA International Award for Clinical Centre for Excellence - ULCH, Dr John Porter 4. SITA International Award for Innovative Medical Research -ProfStefanoRivella 5. SITAInternationalAwardforThalassaemiaSocietyof Excellence-UKTS 6. SITAInternationalAward forTranslationalResearchin Thalassaemia-YuanhanMo

TIF’s new endeavour: Clinical Laboratory Preceptorships The development of clinical and laboratory preceptorships (mentoring) is a new venture of TIF, which is expected to become an integral part of its educational programme for health professionals dealing with haemoglobinopathies. A series of preceptorships are planned for 2016.

Preceptorships provide opportunities for professionals to work closely for a short period of time with an expert in their field of interest, in order to benefit from their practical experience. The objective is to help raise standards, as well as to increase collaborations and networking.

The first clinical and laboratory preceptorship, which will take place in the early part of the year, will be for a laboratory scientist, and will take place in Palermo, Italy under the supervision of Prof Aurelio Maggio, Director of the Department of Haematology and Oncology at the Villa Sofia-Cervello Hospital in Palermo. Expert centres in Asia have also been identified, and will host regional candidates during 2016.

The main impact of this initiative will be to increase expertise in laboratory diagnosis and research, as well as in clinical care, at centres across the world, ultimately benefiting patients in terms of better diagnosis and management.

TIF plans to make this programme a permanent part of its Educational Programme.

e-ENERCA project: fullfiling TIF’s commitment for epidemiological surveillance of major rare anaemias in Europe!

The objective of this project is to provide a tool to gather information on the prevalence of rare anaemias in Europe, and to form a geocode. This will support the development of policies and services, including the recognition of reference centres and networking across the EU and beyond, in order to best serve the needs of patients in expert diagnosis and clinical care.

The project is the main activity in fulfilling TIF’s commitment to complete epidemiological surveillance of major rare anaemias in Europe. A secondary objective is to educate patients on the value of registries and to know their rights concerning the ownership and protection of their personal data.

TIF is leading a group of European organisations from Spain, the Netherlands and Italy as well as Cyprus, in the development of a European electronic registry for rare anaemias. TIF’s project partner is the Department of Informatics of the University of Cyprus, headed by Prof Christos Schizas, which is providing technical support for the development of the registry platform.

TIF Medical Adviser Dr Michael Angastiniotis represented TIF at the ENERCA Executive Committee Meeting held in Barcelona, Spain, between the 28th and the 29th of April 2015, to discuss the e-ENERCA project, which started in September 2013. During this meeting, a number of key topics were presented and discussed, including monitoring the progress of the e-ENERCA project, aligning joint efforts, and identifying and planning future action.

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Honouring great achievements: Sultan Bin Khalifa International Thalassemia Awards (SITA) !

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The registry prepared by TIF and its partners is now ready for piloting in two centres. The principles of registry development were presented by TIF at the 6th European Symposium on Rare Anaemias, held in Amsterdam between the 21st and the 22nd November 2015.

Preliminary data on epidemiology and other outcomes of the registry will be presented at the European School of Haematology (ESH)-ENERCA training course on diagnosis and management of rare anaemias, which will be held in Lisbon, Portugal between the 29th and the 30th of January 2016.

Apart from the obvious benefits of identifying rare anaemia patients and where they are located, in support of rational policy planning for service development, the registry will also help to keep track of increases in cases due to migration currently occuring in Europe from the Middle East and Africa, which are increasing the numbers of haemoglobinopathies in European regions with historically low prevalence of such disorders, as well as other rare anaemias from countries with traditional consanguineous marriage customs. Policy adjustments may be required in European countries to accommodate the needs of congenital and chronic diseases that require specialised services.

Generic Drugs Project: A blessing that can turn into a curse? Ensuring this doesn’t happen!

Roundtable meeting: 29 July 2015 – Beirut, Lebanon Iron chelators are by far the most important drugs that contribute to the survival of lifelong blood transfusion-dependent thalassaemia patients. Such treatment is also key to other forms of thalassaemia that may not require blood transfusions from a very young age, but which may become clinically necessary at a later point in their lives, in which case iron chelation drugs will also constitute an important component of effective management of the disease.

iron from the body, i.e. iron chelators.

Our priorities can be summed up in four simple words: to increase accessibility, availability, safe and effective use of drugs for patients around the world. In the context of national governments, and of regional and international efforts to establish equity in quality health for all, the use of cheaper generic drugs is becoming a major tool for achieving accessibility and availability of drugs to patients across and between countries of the globe, a need that is also recognised

by TIF. However, the use of cheaper generic/copy drugs should under no circumstances jeopardise their quality and effectiveness. TIF has grave concerns that weaknesses in some countries’ drug regulatory systems mean that adherence to good manufacturing practices is not universally promoted.

“Nothing is truly more expensive, in the end, than a treatment failure from an ineffective drug or from a drug that causes side and adverse effects, leading to poor adherence, increase of morbidities and even fatalities”, noted TIF President Panos Englezos in a message to patients and health professionals.

TIF organised its first Roundtable Meeting on the use of generic drugs in thalassaemia on the 29th of July 2015, at the Chronic Care Centre in Beirut, Lebanon—a regional Thalassaemia Reference Centre.

The meeting, which mapped the situation in the Middle East and North Africa (MENA) region, was coordinated by Dr Androulla Eleftheriou, Executive Director of TIF, and Dr Angastiniotis, TIF Medical Adviser, under the leadership of Prof Ali Taher, a member of TIF’s International Advisory Board and Head of its Regional Advisory Committee.

The meeting took the form of a ‘brainstorming’ session, focusing on the quality and safety of drugs, particularly generic drugs. Participants included key physicians from MENA region countries, with whom TIF hopes to form a strong and active network of health professionals, who actively exchanged information and experiences. The meeting also heard from expert patients, including Mahmood Hadipour Dehshal, a pharmacist and Member of the Iranian Thalassaemia Association in Tehran, and Sarunas Narbutas, President of the Lithuanian Cancer Patient Coalition (POLA). A report on the meeting has now been finalised and will soon be circulated among the WHO, the EU, MENA region Ministries of Health, TIF collaborators and General, Voting and Associate Members of TIF. TIF’s position is that ‘Drug Efficacy and Patient Safety’ should always outweigh the costs!

In light of the above, TIF has embarked on a new initiative, as part of its continuous efforts to safeguard the quality of health care of patients with thalassaemia all over the world. This initiative focused specifically on the education of patient/parent and health professional communities on the use of safe and effective drugs in the management of thalassaemia and, more importantly those that remove excess

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TIF’s digital library App finally launched! ThaliMe, TIF’s groundbreaking smartphone application TIF has finally completed its exciting project to develop a mobile application for iOS and Android devices, providing users with a powerful platform containing all TIF publications and informing them of TIF news and events.

This application offers users a simple way to access TIF publications from any device, anywhere, at any time.

It consists of a digital bookshelf and enables users to select a publication, download and read it, providing some stunningly-looking interactive features that harness the touch controls of mobile phones.

The application also allows users to catch up on the latest news and events of TIF, through push notifications, alerts and a digital calendar.

TIF has now launched the app on the IOS and Android markets! Access iOS and Android apps via the links below: Apple store for IOS: https://goo.gl/JtSXXF Google Play for Android: https://goo.gl/I2hf4x

The ThaliMe app is a unique, exciting new mobile application that promises to improve the lives of people living with thalassaemia, and will soon be available for download from the Apple Store and as a web device.

ThaliMe gives people living with thalassaemia, their families and caregivers, a private mobile support network and a set of tools to simplify daily management and monitor overall health.

ThaliMe is easy to use, personalised, and provides users with helpful tools to manage everything from medication reminders to appointment scheduling, from mood and mobility levels to transfusion dates and access to the latest research.

Among its many features are data visualisation tools that present health tracking in a visual format, thus providing an easy and motivating way to chart personal health. Medication and appointment reminders are also available to encourage adherence and timely care.

ThaliMe also helps to connect thalassaemia community members worldwide, including their families and carers. Through ThaliMe, patients are linked to private, peer-to-peer and peer-to-caregiver support networks to reduce isolation and improve patients’ sense of support. Stay tuned! Soon available for download from the Apple store!

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rt t develop ment in TIF programmean Programme.was the creation in 201’s world-renowned educa componen The name is self-expla 1 of the Expert Patient tional natory: the a and its matnis to establish patients im of this a a g em en t, in discussion transformin s experts on their disea g se s th a n em into equa d decisions re effect on thei l partners lating to thei r h ea lt h a nd quality of patients. r d ease and life, and this s eir rights ait s A major ad be the establivance in the Expert Pati wants every shment of an electronicent programme will patient, wher ucationa to transform ever he or shed e may live,ltoplatform. TIF disease. The themselves into an ex focus on pati pert advocate be able been a core act ent and fam r their iv ily educatifo it y for TIF since in 1986, with on has it s es co through new nstant efforts to u tablishment pgrade our w approaches. ork

The Scope of

the Platform

The platfor will utilise a medical edum critical mass of educational cational content repres active learninapproaches (e.g. convenenting various address diffe g, e-learning and ble tional teaching, nded learnin rent audience g) s, languages and cultures.

The overall a to im is to impr grand challen ov e a n d en navigate and ge is to empower the le hance the experience of arner with k meaning ll e learner. The resources ava nowledge ath y interact wit ilable, as wfu n d skills h th e vast wealth ell as to conn of educationto ect with other The learner is al learners. personalised therefore encouraged please visit: collaborative, learningto adopt active learnin g, in environment. academy.tha For more infoa self-directed, lassaemia.org rmation, .cy

DECEMBER 2015 www.thalassaemia.org.cy

T I F M AGA Z I N E

T hal ours ar t o Patiene-tC fT s Proger, apm me IF’s Exper t An impo

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POSITIVITY

STRENGTH

OPTIMISM

Living with thalassaemia: The Video Challenge competition continues for a second year!

Join the 2016 Video Challenge competition and let your imagination flow!

TIF is holding its special “Living with Thalassemia Video Challenge” for a second consecutive year, asking members of the thalassaemia community to share videos relating to thalassaemia that will spread a positive, hopeful, or inspiring message.

The TIF video-sharing platform, designed with a primary focus on connecting thalassaemia patients around the world, constitutes one of TIF’s most powerful projects. The aim is to motivate patients, providing them with an incentive to upload videos in a contest that will culminate with the publication of the winning videos during May, the month of the International Thalassaemia Day!

More information will be available soon on social media and TIF’s website. So stay tuned—the winning entries will be selected, announced, and shared on social media and in the next issue of TIF magazine.

To view entries to last year’s competition, please visit: http://goo. gl/1Lh4Mh

© olly - Fotolia.co

Participants are asked to upload a 2-5 minute video about their experiences living with thalassaemia. Participants can unleash their creative spirit with short films, fiction stories or animation videos about thalassaemia. The possibilities are endless!

By sharing stories, patients and their families will help to create a virtual community where they can connect and gain support from others.

The deadline for the contest is on the 6th of April 2016, with the selection procedure taking place in early May.

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39 T I F M AGA Z I N E

om

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Master’s Degree

www.unic.ac.cy

MSc in Inherited Haemoglobin Disorders: Thalassaemia and Sickle Cell Syndromes (Blended) DECEMBER 2015 www.thalassaemia.org.cy

41 T I F M AGA Z I N E

GENERAL Inherited haemoglobin disorders are the commonest monogenic disorders affecting about 7% of the global population, with thalassemia spreading over the Mediterranean basin and Asia, and sickle cell syndromes being extremely frequent in Africa and the Indian subcontinent, areas where malaria used to be a devastating condition.

Throughout the years, migration and free population movement have caused these diseases to spread from the indigenous populations to almost every country of the world, impacting public health, and leading to economic and social repercussions that contribute significantly to the national and regional disease burden. Moreover, their proper management is often suboptimal, because of lack of resources and infrastructure and also because of inadequate medical knowledge and expertise.

In countries with lower incidence of thalassaemia and sickle cell disease, possibly introduced through population movements, inherited haemoglobin disorders are on the list of rare diseases and, as such, they seldom attract the due priority for the implementation of the necessary specific control programmes.

As a result, knowledge and expertise both on treating but also on globally managing these disorders remains extremely limited and patients are often misdiagnosed or undertreated, while the public health impact inevitably grows at a rapid rate.

Within this context, the limited expertise, in both the countries with high prevalence and those where the haemoglobin disorders are rare, has resulted in a long term request for a more specific education of medical specialists and paramedical personnel in order to improve patient management and provide guidance to policy makers at the national level to develop appropriate programmes for prevention.

It is our belief that this request can be satisfactorily met by the joint educational activity of the University of Nicosia and the Thalassaemia International Federation, formally validated as a University of Nicosia Programme - a blended/hybrid programme, the MSc in Inherited Haemogobin Disorders: Thalassaemia and Sickle Cell Syndromes. To our knowledge this is a unique programme with no other similar in Cyprus or abroad.

The University of Nicosia (UNic) is the largest private university in Cyprus, and it is an independent, co-educational, equal opportunity institution of higher education. International in philosophy, the University provides a multicultural learning environment, promoting friendship, cooperation and understanding. The University pursues excellence in education through research and high teaching standards, in a continually improving academic environment.

The Thalassaemia International Federation (TIF) is the official world body of patients and parents collaborating closely and officially with WHO since 1996, and with many other official and professional This request is fully in keeping with the position of health bodies, the EU, the industry and patients’ both the WHO and the EU, who, by recognising the organizations, in order to promote programmes for the importance of the inherited haemoglobin disorders, control and management of these disorders across encourage member states to promote the control οf the world. Today TIF has 117 member associations Thalassaemia and other Haemoglobinopathies by from 57 countries of the world, and there is a vast offering technical and other support, and to develop network of health professionals in this field attached national plans to this effect (WHO 118th Executive to TIF with vast experience and knowledge to support Board Meeting, EB118.R1, «Thalassaemia and Other the development of management and control Haemoglobinopathies», May 2006; WHO 59th World programmes in «affected» countries, who will also Health Assembly, WHA59.20, «Sickle Cell Anaemia», support the development and realisation of this MSc May 2006; EU Recommendation on Rare Diseases, June 2009. 2015 www.thalassaemia.org.cy programme.

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OBJECTIVES OF THE PROGRAMME

LEARNING OUTCOMES

The overall aim of the programme is to increase access of quality health care to patients with haemoglobin disorders (thalassaemia and sickle cell syndromes) through strengthening education, training and skills of medical specialists, as well as to support the development of effective management and control strategies globally through prevention and treatment.

Upon successful completion of this programme, the graduates should be able to:

The specific objectives of the programme are: 1. To develop an educational programme at the postgraduate level of Master of Science to cover the holistic care of haemoglobin disorders, addressed to medical specialists globally.

2. To utilise the knowledge, experience and expertise of a diverse range of medical and scientific disciplines from the different parts of the world, particularly Europe, where successful control programmes have been established and where wide research programmes have been conducted for developing the programme curriculum.

3. To bring the control of haemoglobin disorders, including the components of prevention and treatment, to the forefront of the agendas of health authorities at the national level and the regional and international level, including official bodies such as the World Health Organisation (WHO) and the European Union (EU), and to support their integration into established official programmes including non-communicable diseases or prevention of birth defects or rare diseases programmes in Europe.

4. To bring to the forefront of the agendas of professional health bodies, industry and research, the needs to allocate more resources and focus on further improvement in the care and total cure mainly of these diseases.

1. Demonstrate in-depth knowledge on how to treat patients with haemoglobin disorders with confidence.

2. Fully describe and utilise the knowledge on the genetics and pathophysiology as well as the natural history of these disorders in order to achieve the best possible outcomes; 3. Confidently interpret laboratory, radiological and other results both for the purposes of accurate diagnosis but also for the follow up of patients under treatment.

4. Evaluate the impact of the inherited haemoglobin disorders on the economical and public health aspects, understand their social repercussions and promote effective control programmes including effective prevention and appropriate clinical management.

5. Work and collaborate closely and guide policy makers on developing effective control programmes with official bodies, including WHO and the EU, and in promoting to Member States the implementation of the specific resolutions adopted by the WHO Executive Board Meeting and the World Health Assembly (WHA) in 2006 and the EU Recommendation of 2009, to Member States and/or their integration into their established programmes of non-communicable diseases or prevention of birth defects or rare diseases.

CAREER PROSPECTS There has been a long-term request from many countries around the world on the needs of education of medical specialists in the field to allow the promotion of these disorders on the national health agendas, and on the guidance of policy makers at the national level to develop appropriate programmes.

Therefore, graduates of this programme would have employment opportunities in universities, specialised health centers, government health offices, national associations and international offices promoting worldwide health (such as the WHO or international associations).

ACCESS TO FURTHER STUDIES We expect that a significant percentage of medical specialists who will acquire this MSc degree will have the interest, the motivation and the medical prerequisites to continue to further research and PhD.

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ADMISSIONS CRITERIA

Specific: (if applicable) 1. Medical specialists who have not obtained their academic qualification from an English-speaking university must satisfactorily pass the IELTS (6.5) or TOEFL examination, or any other examination from an internationally approved body.

2. Basic computer skills.

Priority will be given to medical specialists already involved in the treatment of haemoglobin disorders in their country in the public sector.

DURATION OF STUDIES Three semesters plus a minimum of one week (40 hours) of clinical practice in various cities/countries around the world according to the place of residence of students at an accredited Reference Centre for Haemoglobin Disorders.

T I F M AGA Z I N E

General: 1. A University degree in Medicine, preferably with specialisation in the following disciplines: haematology or paediatric haematology or paediatrics or internal medicine.

OR 2. A University degree in Nursing, preferably with specialisation in a haemoglobin disorders or having demonstrated experience in haematological area /red cell pathology.

STRUCTURE OF THE PROGRAMME To successfully complete the programme the student must either complete 90 ECTS of structured sessions or complete 70 ECTS of structured sessions and write a thesis (20 ECTS). In addition, the student must complete a minimum of 1 week (40 hours) of ad-hoc clinical practice at an accredited Reference Centre for Haemoglobin Disorders.

LANGUAGE OF THE PROGRAMME English

ACADEMIC PATHWAY

ECTS

MAJOR REQUIREMENTS

70

THAL-511DL THAL-512DL THAL-513DL THAL-521DL THAL-522DL THAL-531DL THAL-532DL

10 10 10 10 10 10 10

Inherited Haemoglobin Disorders I Inherited Haemoglobin Disorders II Laboratory Aspects; Epidemiology; Statistical Methods Thalassaemia Clinical I Thalassaemia Clinical II Sickle Cell Syndromes Prevention and Cure

MAJOR ELECTIVES THAL-523DL THAL-533DL THAL-590DL

20 New Advances and Patient-Centred Services Key Issues Related to the Quality of Life of Patients with Haemoglobin Disorders Master’s Thesis

10 10 20

INFORMATION

DECEMBER 2015 www.thalassaemia.org.cy

UNIVERSITY OF NICOSIA Please contact: Distance Learning Unit Tel.: +357 22842016, +357 22842015, For telephone calls from Greece: 800 100 1212 Email: DL.admissions@unic.ac.cy Website: www.unic.ac.cy/DL

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Medical

and Scientific Update

bluebird bio presented LentiGlobin® BB305 Clinical Data and bb2121 Preclinical Data at Annual Meeting of the American Society of Haematology (ASH) bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies, announced that data from its ongoing clinical studies of LentiGlobin BB305 in beta-thalassemia major and severe sickle cell disease (SCD) will be highlighted in oral and poster presentations at the 57th Annual Meeting of the American Society of Hematology (ASH). The company also presented preclinical data from its lead oncology program, bb2121, in three posters at ASH. bluebird bio is developing bb2121 as an anti-BCMA oncology therapy in collaboration with Celgene Corporation. Preliminary data from all six of these abstracts became available on the ASH conference web site “Our expanding clinical experience with LentiGlobin

continues to show promising clinical benefit in patients with beta-thalassemia major and severe sickle cell disease,” said David Davidson, M.D., chief medical officer, bluebird bio.

“We are pleased to see a median HbAT87Q level of 5.2 g/

dL in the seven Northstar patients with beta-thalassemia major of all genotypes followed for six months or more, as this represents a substantial proportion of their total heamoglobin. It is exciting that transfusion independence has been achieved in all of our patients with beta-thalassemia major with non-β0/β0 genotypes followed for at least six months in Northstar and HGB-205. Varying degrees of transfusion reduction have been observed in patients with β0/β0 genotypes. As would be expected, longer follow-up is required to assess the extent of HbAT87Qproduction and the impact on transfusion requirements in these patients with β0/β0 genotypes since they produce no functional betaglobin at baseline. Turning to sickle cell disease, our first treated patient is producing increased levels of HbAT87Q since we last reported on this study in June. HbAT87Q represented 48 percent of total haemoglobin at nine months postinfusion, and the patient remains transfusion independent without sickle cell-related adverse events as of the data cutoff. These results support the transformative potential of gene therapy, and we look forward to sharing more data at ASH.” "The three accepted oncology abstracts represent the great progress we have made building our immuno-oncology business,” said Rob Ross, M.D., head of oncology, bluebird

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45 T I F M AGA Z I N E

bio. “These posters describe the encouraging pre-clinical

data supporting the development of bb2121 in multiple myeloma, including our robust manufacturing platform, and key scientific insights into engineering more potent CARs that are applicable across our planned oncology portfolio.”

LentiGlobin Presentations

Universitaire Necker – Enfants Malades, Paris, France Abstract Results, as of 31st July Data Cut-Off: The subject with severe SCD is producing approximately 51.5% anti-sickling haemoglobin (48 percent HbAT87Q, 1.8 percent HbF, 1.7 percent HbA2) at nine months post-infusion. ▀▀

The subject with severe SCD remains free of transfusions.

Update of Results from the Northstar Study (HGB204): A Phase 1/2 Study of Gene Therapy for BetaThalassemia Major via Transplantation of Autologous Haematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)

▀▀

Presenter: Mark C. Walters, M.D., UCSF Benioff Children’s Hospital, Oakland, CA

▀▀

Abstract Results, as of 31st July Data Cut-off: ▀▀ Seven subjects have been monitored for at least six

months post-infusion: three of the β0/β0genotype and four of the non-β0/β0 genotype.

The subject with severe SCD has not had a posttreatment hospitalization for a disease-related event despite ceasing chronic transfusions on Day +88. ▀▀

Both subjects with beta-thalassemia major have remained transfusion-free for at least 15 months postinfusion, with consistent expression of HbAT87Q – both subjects are β0/βE genotype. ▀▀One additional subject with beta-thalassemia major

had one month follow-up post-infusion. ▀▀

▀▀ The median level of HbAT87Q expression

among these seven subjects is 5.2 g/dL (range 1.9 to 8.2 g/dL), with total haemoglobin ranging from 8.5 to 11.1 g/dL at last visit.

Initial Results from Study HGB-206: A Phase 1 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease

▀▀ All four non-β0/β0 subjects have been

transfusion-free for at least 90 days, with a median of 287 days transfusion-free (range: 171 to 396 days).

▀▀ Two of the β0/β0 subjects have

received a single transfusion postdischarge, and one remains transfusiondependent.

Presenter: John F. Tisdale, M.D., National Institutes of Health, Bethesda, MD

▀▀ All subjects engrafted. ▀▀ The

safety profile was consistent with autologous transplantation. No Grade 3 or higher drugproduct related adverse events have been observed, and there is no evidence of clonal dominance after a median follow-up of 198 days (range: 65 to 492 days).

Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major via Transplantation of Autologous Haematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector Presenter: Marina Cavazzana, M.D., Ph.D., Hôpital

DECEMBER 2015 www.thalassaemia.org.cy

No subject has experienced a drug product-related adverse event, and there is no evidence of clonal dominance.

Abstract Results, as of 31st July Data Cut-Off: ▀▀ LentiGlobin

BB305 drug product has been manufactured for 2 subjects with severe SCD and 1 subject has been infused. ▀▀ The safety profile has been consistent with autologous

transplantation, with no Grade 3 or higher drug productrelated adverse events.

46 Novel stem cell transplantation-based regimen as curative treatment option for thalassemia major with minimal risk of GVHD and better immune protection Beta thalassemia syndromes are a group of hereditary disorders characterized by a genetic deficiency in the synthesis of beta-globin chains. In the homozygous state, beta thalassemia (i.e. thalassemia major) causes severe, transfusiondependent anemia within the first year of life and if left untreated, patients will have a life expectancy of no more than three years. Management of beta thalassemia still largely depends on supportive care, with lifelong red blood cell transfusions and iron chelation. In economically poor countries, affected individuals still die as children, and even in developed countries, most patients with beta thalassemia still have a reduced life expectancy and often experience a relatively poor quality of life.

Allogeneic haematopoietic stem cell transplantation (HSCT) is the only curative treatment option that would not rely on gene therapybased approaches to correct the haematological manifestations in patients with thalassemia major, but is limited by matched donor availability and the risk of graft-versus-host disease (GVHD). Although positive results are obtained in those with a fully HLAmatched sibling donor, use of matched unrelated or partially matched donors has been complicated by GVHD, poor engraftment, excessive regimen-related toxicity, high infection rates, and high risks for mortality and morbidity. It is clear that there is still a great need for new, alternative, and effective therapeutic strategies for treatment

of this life-limiting disease to render patients transfusion-independent.

While gene therapy-based approaches are emerging and being tested in clinical trials, it is unclear whether effects would last life-long and which risks the genetic manipulations could have over time.

Kiadis Pharma N.V., a clinical stage biopharmaceutical company developing innovative T-cell immunotherapy treatments for blood cancers and inherited blood disorders, is developing ATIR201, a personalized T-cell immunotherapy based on family donor immune-cell preparations selectively depleted of those T-cells that might attack the patient and elicit GVHD (figure). Briefly, GVHD causing T-cells from the donor are allowed to attack patient tissue in the petri dish. Thereafter cells are treated with Kiadis’ compound, TH9402, which is selectively retained in activated T-cells. Subsequent lightexposure eliminates these activated T-cells but preserves all other immune cells.

In a first step of a transplantation procedure in which ATIR201 would be administered, the diseased bone marrow of the β thalassemia patient would be replaced by a pure stem cell transplantation from a healthy heterozygous family donor in order to restore the proper production of haemoglobin. In the second step of the procedure, ATIR201 would be administered to the patient shortly after

New developments

the transplantation, providing large numbers of functional, mature immune cells. These cells help protect the patient against infections without the risk of severe acute GVHD due to the specific elimination of the cells that would trigger GVHD. Therefore, the patient also would not need to take prophylactic immune suppressants.

In prior and ongoing clinical trials for treatment of blood cancers, Kiadis Pharma confirmed the safety and efficacy of a related product designated ATIR101; in those trails no grade III-IV (life-threatening) acute GVHD occurred, despite the fact that no prophylactic immune suppressants were used.

Furthermore, only limited severe infections and relapses were reported in those studies evidencing the potential of the product to make curative HSCT a viable option to many more patients in need of stem cell transplantation.

ATIR201 is expected to enter clinical development for thalassemia with a Phase I/II trial in the first half of 2016, and is expected to address the key risks and limitations of HSCT in inherited blood disorders such as GVHD, opportunistic infections and limited donor availability.

The phase 2 trials for the Luspatercept and Sotatercept drug have been completed successfully and phase 3 trials are expected to start in January 2016.

in several centres across Europe and North America, it was demonstarted that Luspatercept was able to raise the total Hb level by 1.5g/dl in non-transfusion dependent thalassaemia, and reduce blood transfusions at least 40% over the same 12 week period (see charts provided by Prof Antonio Piga in the European Haematology Association Conference in June 2015, page 47, at the top of the page)

What are these drugs?

How are these substances given?

They are substances which affect Ineffective erythropoiesis, which characterizes the thalassaemia syndromes. Ineffective erythropoiesis is characterized by elevated proteins such as the TGF-β superfamily ligands. These substances are recombinant fusion protein containing a modified extracellular domain of the activin receptor. This binds to GDF11 and other ligands, and promotes late-stage erythroid differentiation.

They are administered subcutaneously every 3 weeks

Dr Angastitniotis M, TIF Medical Adviser

Their effect is an increase in haemoglobin levels demonstrated initially in healthy volunteers. In the phase 2 study in adults with beta-thalassemia, which was conducted

No major side effects were demonstrated during the phase 2 study TIF believes that these new drugs are major breakthrough in the management of the thalassaemias and they will reduce the iron loading since they will reduce the frequency of blood transfusions. The impact on the quality of life and reduction of complications is expected to be significant and so we may be near a major revolution in patient care.

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Development of a new disease severity scoring system for patients with non-transfusiondependent thalassemia Eur J Intern Med. 2015 Nov 3. pii: S0953-6205(15)00332-5.

doi: 10.1016/j.ejim.2015.10.003.

Cappellini MD, Porter JB, Musallam KM, Kattamis A, Viprakasit V, Galanello R, Taher AT.

Background Patients with non-transfusion-dependent thalassemia (NTDT) present with a spectrum of disease severities.

Since there are multiple pathophysiologies in such patients, tailoring treatment remains essential. Therefore, one simple, reliable tool would be beneficial to assess disease severity and tailor therapy, particularly for internal medicine specialists who may treat a variety of NTDT patients with a multitude of complications.

This would allow for standardization of assessments leading to timely interventions and prevention of complications.

Methods A working group of NTDT experts was formed to develop a new disease severity scoring system for adult and pediatric patients with NTDT, based on parameters considered to be most pertinent in defining disease severity.

Results 20 parameters were selected for inclusion in the disease severity scoring system. An additional six parameters, largely related to growth and development, were selected specifically for pediatric patients (≤16years of age). Consensus of expert opinion was used to establish the selected methods of assessment for each parameter, based on feasibility and availability of technology, cost containment, and avoidance of patient risk.

Conclusion We propose that this new disease severity scoring system for adult and pediatric NTDT patients could be developed into a practical tool for widespread clinical use.

Read this article here: www.ncbi.nlm.nih.gov/ pubmed/26545830

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Thalassemia 2016: Modern Medicine Battles an Ancient Disease Rund D. Thalassemia was first clinically described nearly a century ago and treatment of this widespread genetic disease has greatly advanced during this period.

DNA-based diagnosis elucidated the molecular basis of the disease and clarified the variable clinical picture. It also paved the way for modern methods of carrier identification and prevention via DNA-based prenatal diagnosis. Every aspect of supportive care, including safer blood supply, more regular transfusions, specific monitoring of iron overload, parenteral and oral chelation, and other therapies, has prolonged life and improved the quality of life of these patients.

Significant advances have also been made in allogenic bone marrow transplantation, the only curative therapy. Recently, there has been a rejuvenated interest in studying thalassemia at the basic science level, leading to the discovery of previously unknown mechanisms leading to anemia and enabling the development of novel therapies. These will potentially improve the treatment of, and possibly cure the disease. Pathways involving activin receptors, heat shock proteins, JAK2 inhibitors and macrophage targeted therapy, among others, are being studied or are currently in clinical trials for treating thalassemia. Novel types of genetic therapies are in use or under investigation. In addition to the challenges of treating each individual patient, the longer survival of thalassemia patients has raised considerations regarding worldwide control of thalassemia, since prevention is not universally implemented. This review traces a number of the original medical milestones of thalassemia diagnosis and treatment, as well as some of the most recent developments which may lead to innovative therapeutic modalities. To read the article, visit: http://www.ncbi.nlm. nih.gov/pubmed/26537527

48 ITHANET: Gene Therapy for Transfusion Dependent Betathalassemia (TIGET-BTHAL) This phase I/II clinical trial will investigate safety and efficacy of the GLOBE lentiviral vector, which encodes the human β-globin under control of native promoter and control elements. Preclinical studies by the G. Ferrari group (TIGET, Ospedale San Raffale) have given extremely encouraging results for the GLOBE vector in mouse models of β-thalassaemia and in human patientderived cells. The study is open to patients aged 3 to 64 years with transfusion-dependent β-thalassaemia and is expected to enrol ten patients over a time period of two years. The detailed study description provides extensive inclusion and exclusion criteria for potential participants in this study.

More information: https://clinicaltrials.gov/ct2/show/ NCT02453477

coordinated European multicentre and multi-disciplinary way. This is due because the RA are a complex group of RD, and according to their mechanism, prevalence and/ or relevant clinical and/or social impact in the European population, they have been classified into ten main groups (figure 1 below). From these, up to 80% are hereditary, and in, the remaining 20%, the underlying cause of RA is acquired, or, remains unknown. Probably, almost one third of RA with unknown origin might be accounted for myelodysplastic syndromes (MDS) or to complex clinical situations with multifactorial mechanisms, in general associated with systemic, non haematological, hereditary or acquired diseases 1.

ENERCA: The European Network for Patients with Rare Anaemias Joan-Lluis Vives Corrons, Maria del Mar Mañú Pereira, Journal of Rare Disorders: Diagnosis & Therapy 2015, Vol. 1 No. 1:9

Summary ENERCA intends to promote a multi-disciplinary approach to care in order to address the complex and disorders called Rare Anaemias. Gathering expertise at European level is therefore paramount to ensure equal access to accurate information, appropriate and timely diagnosis and high quality care for patients with a rare anaemia independent from their Country of origin.

Moreover, increasing Healthy Life Years by preventing disease and promoting policies that lead to a healthier way of life is important for the well-being of EU citizens and helps to meet the challenges of the Lisbon decision as regards the knowledge society and the sustainability of public finances, which are under pressure from rising health care and social security. In this way, the consolidation of a European Reference Network (ERN) of Experts Centres in Rare Anaemias, is a crucial step to improve the services for clinical management of these diseasesas well as education and social care regardless of their country of origin.

This will allow health professionals an easy access to recognized centres of expertise in each RA category and provide new opportunities to undertake innovative and useful actions based on the ERN.

Figure 1

What is ENERCA? The European Commission (EC) has been especially sensible to the rare diseases challenge, and, since 2002, has taken an active role by co-financing a large-scale network of experts and specialists working in the field of RA, called EUROPEAN NETWORK FOR RARE AND CONGENITAL ANAEMIAS (ENERCA). ENERCA has been developed through four consecutive phases with a total duration on 15 years. In all these phase developing ENERCA Projects, consecutive achievements have been nourished by all the concepts and initiatives that continuously appear in the discussion platforms; Rare Disease Task Force (RDTF) from 2004 to 2009, and European Committee of Experts in Rare Diseases (EUCERD) from 2010 to 2014. (Figure 1). ENERCA involves associated and collaborating partners from 15 different European countries (Figure 2). and most of they have been working together since 2002, . All ENERCA partners are well known and recognized experts in their respective field.

Figure 1

What Are Rare Anaemias?

Rare Anaemias (RA) are a group of rare diseases (RD), in which anaemia are the key clinical manifestation.

As all RD the RA have prevalence in Europe of less than 5 cases for 10,000 people. The existence of RA is an exciting tackling exercise for clinical and biological research, and the need of improving the quality of health services for these patients has obliged to mobilise resources that can only be efficient if done in a

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49 T I F M AGA Z I N E

Health Services Provided By Enerca and Policy During the last 10 years (20022012), ENERCA has contributed to promote two pivotal aspects for RA: 1) A specific framework for cross-border healthcare and 2) A European cooperation for providing health services such as diagnostic help, training, information, dissemination and evaluation. All these ENERCA outcomes have had a wide geographical coverage and an efficient impact as a high number of health professionals and patients were implicated. Obviously, ENERCA website (www.enerca.org) has definitely contributed to this success that allows to present, actually, a definitive European added value, as stated in the EU Health Programme 2008-2013 ( “common principles in all EU health systems aiming to ensure clarity and confidence with regard to authorities setting and monitoring healthcare standards, have to be implemented”) and proven to be a focal point for the following health services implementation. One example of this contribution is the publication of ENERCA White Book. 2 After 2013, the EC has approved co-financing ENERCA Project for a additional tree years (2014-2017), with the aim of developing and implementing the new e-health information and communication technologies (ICT) for assuring the same access to health services in RAs across Europe, independently from the place of residence.

This new Project, called e-ENERCA, is based, in part, on previous ENERCA projects achievements, but adapted to the “2011 EU Directive on patients’ rights in crossborder healthcare”. e-ENERCA will

incorporate the most innovative e-health ICT to create a panEuropean interoperable e-health platform for, electronic registry/ epidemiological health records (EHR), online teaching programes for continuous medical education , and tele-expertise/telediagnosis for medical practice. Accordingly, the and e-learning. e-Health services will be developed through the set-up of three different e-platforms endorsed by ENERCA website ( www.enerca.org) : 1) e-Registry, a Pan European registry of RAs for gathering patient’s data necessary to achieve the required sample size for epidemiological surveillance and clinical research 2) e-Learning , a teaching platform for the dissemination of knowledge ,continuous medical education, and best practices awareness and promotion through Internet, and 3) e-Medicine , a platform to provide, at distance, expertise (telexpertise) and diagnostic facilities (telediagnosis) , avoiding , when possible, the need of travelling. The ERN will identify needs and priorities for basic, clinical translational and social research in the fields of the different RA categories: a) Sickle cell disease (SCD), b) Thalassaemia syndromes and d) Very Rare Anaemias (e.g. erythropoietic failure and RBC defects, either hereditary or acquired). This will facilitate ENERCA fostering all these activities and the promotion of inter-disciplinary cooperative approaches to the complementarily addressed through national MS and Community programs. One example is sickle cell diseases (SCD).

During the last 30 years, SCD is increasing in Europe due to African immigration, leading to an important impact on health

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care burden in several countries.

Preventive programs, aiming to epidemiological control, and improvement of diagnosis and clinical management of major RA, are crucial for decreasing the affected birth rate and achieving an efficient balance between morbidity and patient’s life expectancy 3.

Accordingly, ENERCA has taken an active role for improving this situation by the following actions : a) the identification of Centres of Expertise on RAs in Europe according to the recommendations of ENERCA White Book b) the promotion of best clinical and laboratory practices and, c) the improving of continuous medical education, by organising topicspecific training courses, workshops and symposia , e) the empowerment of patients, by cooperation with Patient’s Associations, and coorganizing a bi-annual European Symposium on RAs with interactive patients-health professionals sessions.

References

1.BeatriceGulbisB,EleftheriouA,AngastiniotisM, BallS,SurrallésJ,etal.(2010)EpidemiologyofRare AnaemiasinEurope,RareDiseasesEpidemiology, AdvancesinExperimentalMedicineandBiology, 686:375-396.

2.AguilarMartinezP,AngastiniotisM,Eleftheriou A,GulbisB,MarMañúPereiraMM,etal.(2014) HaemoglobinopathiesinEurope:Health& Migrationpolicyperspectives,OrphanetJournalof RareDiseases9:97.

3.ENERCA:AWhiteBook-ProdrugMultimedia (2014)RecommendationsforCentresofExpertise inRareAnaemias

This article is available on: //www. raredisorders.imedpub.com/

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Š prochkailo - Fotolia.com #68198034

Other News and Updates

S

ince the Ninth Cooley’s Anemia Symposium was held in 2009, all areas of thalassemia research have sustained major advances.

The 2015 Symposium brought together basic scientists, clinical investigators, and clinicians, for interactions and education. Areas of focus included the biology of globin gene regulation and fetal haemoglobin induction; the evolving areas of stem cell transplant, gene therapy strategies, and gene editing; the biology of iron regulation and possible therapeutic interventions in the hepcidin regulatory system; and clinical issues in thalassemia treatment and imaging.

Leading investigators, together with physicians involved in thalassemia care from around the world, were featured speakers. Interactive poster sessions allowed trainees involved in all of these research areas to present their work.

Tenth Cooley’s Anemia Symposium Poster Abstracts Nutritional Deficiencies, by Ellen Fung 50-70% of adult patients have low bone mass. Only 18% had deficient levels of VitD. There seems to be more hypercalcuria wit normal VitD levels.

Some patients were treated with Vit K supplementation and had significant improvement in their bone mineral density. Zinc supplementation was given 25mg/day for 18 months. There was another study with 2 g Strontium at night, one with VitD and Calcium for 2 years. All three studies showed some positive findings and improvement in pain.

25% of children have growth deficit. There is a decrease in body fat related to bone density and growth. Zinc supplementation was associated with an increase in IGF1.

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John Porter An â&#x20AC;&#x2DC;Agingâ&#x20AC;&#x2122; Population of Adults with Transfusion-dependent Thalassemia (TDT) The UCH has 380 patients with SCD, 217 with beta thalassemia, 42 with TI and 19 with HbE/thal. The peak age is at 30. Chleation was avaiable since 1975, CMR was available from 1999. 132 patients with 109 having long term follow up. LIC was <5 in only 25% now 62% have LIC <5. 13 deaths. No heart failure 6 sepsis, 3 hepatic, 1 breast cancer, 1 diabetes, 1 sudden unexplained. No deaths in those born after 1980. Median age of death 49. Still have AF in patients who had previous heart failure. Endocrinopathies have a weak relationship to age. Diabetes is higher in older patients. Hypogonadism is different according to age. Hypothyroid is common in <40 year olds.

Osteoporosis is common in older patients. Spinal degeneration 47/106 patients. 60% in > 40 yo.

35% in < 35. No correlation to bone density.

Mainly back pain. Increased risk of thrombosis, renal and GIT problems. 8/120 have exocrine pancreatic problems.

Ali Taher, Intermedia

Morbidities in Thalassemia

This study looked at the clinical profile of number of TI patients from Lebanon, Thailand, Italy, India and Egypt. Morbidities increase with increasing age. Extramedullary erythropoiesis, Pulmonary hypertension and leg ulcers are major morbidities. Ineffective erythropoiesis results in increased iron absorption. Complications are worse in patients who have Hb <7. In 120 patients with no transfusions 7 had leg ulcers. In the whole group 50 patients have them. Hepatic Carcinoma is a very dangerous complication.

There are 6 patients in this group all of whom are over 50. Also there is more hepatic fibrosis. Renal dysfunction is emerging as a problem. Reducing eGFR and increasing proteinuria. 6 (4.7%) of 127 TI patients are on dialysis.

Hyper coaguability was analysed and there is a relationship to splenectomy, higher nucleated red blood cell count, increased plateletsand pulmonary hypertension. The latter is much more common in the splenectomized patients.

They are also more likely to have higher LIC and increased age. QOL is not as good as in TM. They have increased pain. Also there are silent infarcts.

Management: 325/584 have had splenectomy.

This increases risk of all complications. Worsening anemia and poor growth usually requires

transfusion. The only indication for splenectomy now is possibly worsening anemia and poor growth, hypersplenism as indicated by leukopenia and thrombocytopenia and a spleen greater than 20 cm below the costal margin. Transfusions are protective against many of the complications.

However there is a high risk of allo-immunization in the patients who start transfusions late in their lives.

HbF induction. Some patients respond to hydroxyurea treatment. It was protective against EMH, PHT , leg ulcers, hypothyroidism and osteoporosis.

Ferritin underestimates iron burden. <300 is OK. Increasing probability of increased LIC > 5 mg/g dry weight if ferritin is >800. Gave maximum dose of 20mg/kg of DFX. Keep MRI for those who have ferritin between 300-800.

Emanuele Angelucci Management of Iron Overload Before, During, and After Successful Transplantation.

BFUEs are impaired in MDS in the presence of high ferritin . There is a toxic effect of iron on the bone marrow. Iron load impairs the function of Haemopoietic stem cells both long term and with multi-lineage engraftment (in the murine model).

Therefore some thought needs to be given to reducing iron pre-transplant.

Key point is to optimize iron load.

The question remains whether short term intensive chelation may be valuable for patients before transplantation.

During Transplantation: More toxicity due to LPI. Possibly there is increased GVHD with excess Fe. Even in the old Pesaro data there was >80% risk of GVHD in the high risk patients. There is a huge release of free iron during conditioning therapy. The NTBI in thalassemia patients during conditioning is much higher than that which appears in the leukemia patients. There is one small study where intravenous DFO chelation was given during conditioning and a German trial is underway looking at this. GITMO (Italian Bone marrow transplant group) is looking at this as well.

Management after transplant could be with phlebotomy (6ml/kg every two weeks). DFO and DFX are being used as well. Phlebotomy removes iron effectively and reverses fibrosis if present.

Read more: http://www.nyas.org/Events/ Detail.aspx?cid=9c11e73d-2349-4cdd-89100d8fad184ced

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T I F M AGA Z I N E

9-19% of adults will have diabetes. There is a link between Zn deficiency and glucose metabolism disorders.

52 INDUSTRY NEWS Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias (FIRST) Information provided by (Responsible Party): ApoPharma Purpose This research is being done so that we can look at the safety and efficacy of deferiprone in people with sickle cell disease or other anemias. Deferiprone is a drug that removes iron from the body. We will be comparing deferiprone with deferoxamine, another drug that removes iron from the body.

Number of participants in each group who discontinued from the study due to AEs More information: ClinicalTrials.gov processed this record on November 09, 2015 FDA Fast Track Designation Granted to Luspatercept for the Treatment of Patients with Beta-Thalassemia Celgene Corporation (NASDAQ:CELG) and Acceleron Pharma Inc. (NASDAQ:XLRN) announced that the United States Food and Drug Administration (FDA) has granted Fast Track designations to luspatercept for two separate indications; the use of luspatercept for the treatment of patients with transfusion dependent beta-thalassemia and the use of luspatercept for the treatment of patients with non-transfusion dependent beta-thalassemia.

Celgene and Acceleron are jointly developing luspatercept.

“The FDA’s Fast Track designation for

the luspatercept development program recognizes the serious unmet medical needs of patients with beta-thalassemia and the potential for luspatercept in this area”

Further study details as provided by ApoPharma Primary Outcome Measures Change in liver iron concentration, as measured in mg/g dry weight (dw) using MRI Time Frame: Change from baseline to Week 52 , designated as safety issue: No Without effective iron chelation therapy, transfusion-dependent patients experience a progressive increase in Liver Iron Concentration (LIC). High LIC increases the risk of iron-induced toxicity such as cardiac disease, hepatic fibrosis, diabetes mellitus, and death.

[Designated as safety issue: Yes

The Fast Track program of the FDA is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. The designation also provides the opportunity for more frequent interaction with FDA over the course of the development program, and allows a sponsor to submit sections of the BLA on a rolling basis as they are finalized.

“The FDA’s Fast Track designation for

the luspatercept development program recognizes the serious unmet medical needs of patients with beta-thalassemia and the potential for luspatercept in this area,” said

Jacqualyn A. Fouse, President, Hematology/ Oncology for Celgene. “Celgene and

Acceleron are working diligently to initiate a phase 3 clinical program in 2015 to treat patients with beta-thalassemia and we look forward to continuing to work closely with health authorities and other important stakeholders to advance this program.”

For more information, please visit http://www.businesswire.com/news/ home/20150518005252/en/FDA-FastTrack-Designation-Granted-LuspaterceptTreatment

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Study

-

Beta

Information provided by Acceleron Pharma, Inc. Description Study A536-06 is an open-label extension study for patients previously enrolled in study A536-04 (ClinicalTrials.gov Identifier NCT01749540), to evaluate the safety, tolerability and pharmacodynamic effects of up to 24 months of ACE-536 treatment in adult patients with beta-thalassemia previously treated with ACE-536 for up to 3 months in study A536-04. The starting dose level in A536-06 will be 0.8 mg/kg by subcutaneous (SC) injection once every 3 weeks. Dose titration/modification rules will be followed for individual patients and will be based upon safety and efficacy data collected during the course of treatment

Primary Outcome Measures Long-term safety and tolerability of ACE536 in patients with β thalassemia who were previously enrolled in study A536-04 safety and tolerability will be assessed by recording and classification of all adverse events (clinical and laboratory) reported by study investigators in all subjects who received at least one dose of study drug Read more: https://www.clinicaltrials.gov/ct2/ show/NCT02268409

Novartis announced that the US Food and Drug Administration (FDA) has approved JadenuTM (deferasirox) tablets, a new oral formulationofExjadeÂŽ(deferasirox)tabletsfor oral suspension, for the treatment of chronic iron overload due to blood transfusions in patients 2 years of age and older, and chronic iron overload in non-transfusion-dependent thalassemia syndromes (NTDT) in patients 10 years of age and older. Jadenu is the only once-daily oral iron chelator that can be swallowed whole.

Many patients with sickle cell disease, thalassemia or myelodysplastic syndromes need repeated blood transfusions and consequently, long-term daily chelation therapy. Jadenu oral tablets can be taken in a single step, with or without a light meal, simplifying administration of treatment for chronic iron overload. Exjade is a dispersible tablet that must be mixed in liquid and taken on an empty stomach. Jadenu is approved under accelerated approval based on a reduction of liver iron concentrations and serum ferritin levels. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Chronic iron overload is a life-threatening cumulative toxicity that results from blood transfusions required to treat sickle cell disease, myelodysplastic syndromes, thalassemia and other conditions. Chronic iron overload also can occur in patients with NTDT due to increased iron absorption in the stomach and intestines. If left untreated, chronic iron overload can damage the liver and heart[.

Jadenu contains deferasirox, the same active ingredient that is in Exjade, a medicine that has been used by patients with chronic iron overload for almost 10 years. Exjade currently is the most-prescribed chelator in the United States.

Novartis has submitted additional regulatory applications for Jadenu in other countries worldwide.

Read more: https://www.novartis.com/ news/media-releases/novartis-announcesfda-approval-jadenutm-simplify-treatmentadministration

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ACE-536 Extension Thalassemia

Novartis announces FDA approval for JadenuTM to simplify treatment administration for patients with chronic iron overload

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Patient Experience by Akash Chaurasia (USA) “Taking my iron chelator used to be one of the worst parts of the day for me. I usually had mild stomach issues if I ate anything around the same time as Exjade, so I avoided this problem by taking it 30 minutes before I even started my day. My routine was to make it, as I tolerated the chalky, slurry concoction and went back to sleep. I used apple juice for it, and I never drank apple juice outside of my Exjade usage.

For this reason, I was ecstatic when my haematology clinic’s nurse practitioner informed me of the new formulation, Jadenu. I imagined all the possibilities as she went on to describe the lower dose required, the much reduced dietary restrictions, and the greater ease of use. It would be like taking a headache pill.

Although I knew taking the new formulation would signify profound changes in my daily routine, the actual results exceeded my expectations. Not only did I win back my 30 minutes of sleep each morning, but I could eat chronologically adjacent meals.”

The introduction of the new version of the drug could not have entered my life at a more ideal time. It was at the point during which I had decided to travel the world. The adventure would entail sleeping in hostels and travelling between European cities every few days. Taking Exjade, while feasible would have been a significant hindrance to my freedom to explore. I was able to eat at any time of the day, and did not have to worry about maintaining a supply of apple juice and plastic cups.

After over 4 months on Jadenu, I can assert with ease that it is a small difference in my day that results in a surprisingly cogent improvement in my quality of life. It is easy for an average person with no vested interest in medicine to overlook these miniscule advances. However, the cumulative evolution of an ancient science has tremendous potential that I have experienced in the current age".

The Patient Research Exchange (PRE) – Where Patient Centricity Helps Evolve Research Patient Research Exchange is a platform developed by patient advocates to represent patient communities. The participants should be patients, patient advocates, Patient Organizations, caregivers, healthcare professionals and other stakeholders engaged in research affecting patients and patient outcomes. The PRE is open to all those who wish to actively contribute, share experiences learn or increase capacity for patient collaboration.

The Patient Research Exchange website is not a commercial venue nor is it a place for companies or individuals to seek to sell, market, or promote

products, treatments or services for financial gain. Additionally, it is not a venue for market research, focus groups, surveys or any other type of commercially motivated research.

The Patient Research Exchange is a group of people representing many different experiences and geographies who are dedicated to exploring how the patient perspective can improve participation in and the effectiveness of research. This website is a place for people interested in active patient-centred research to exchange ideas, experiences, resources, and perspectives.

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Patients are meaningfully engaged in research at all levels with appropriate roles throughout the research process.

The value of the patient perspective and experience will be integral to any research activity that affects patients. Patients Organizations and patient advocates will be afforded the opportunities and necessary support to achieve optimal value from participation and to assure mutually beneficial experiences.

Patient engagement will ultimately lead to better outcomes for all patients.

Values PRE values research as an essential activity that has the ultimate goal of improving the well-being of those living with disease or other healthrelated condition. The PRE is based on the fundamental value of the patient as a partner in research, with

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the right and responsibility to be fully engaged in any research activity that affects his/her personal well-being as well as that of any patient community or population represented. The PRE values the patient as a whole person and believes research should take into consideration the diversity of medical needs, healthcare conditions, cultures, geographic location, and economic realities.

We believe that by sharing our ideas and perspectives, working together by building capacities and capabilities, and by participating in research actively, we can develop appropriate frameworks and promote supportive attitudes around patient active engagement in research.

Strengthening the patient position in research and development will lead to new therapies which are more targeted to patient needs and better equipped with patient relevant data.

An effective PRE will enable clinical decision makers to have a better evidence base to deliver patient centred decisions together with the patient.

By sharing our ideas and perspectives, working together by building capacities and capabilities, and by participating in research actively, we can develop appropriate frameworks and promote supportive attitudes around patient active engagement in research.

T I F M AGA Z I N E

PRE was founded by a group of patient advocates from Patient Organizations across a broad range of conditions and geographic regions to foster mutual learning and collaboration. The Steering Committee for the Patient Research Exchange came together after having exchanged ideas, experiences, and expectations concerning active patient involvement in clinical trials and generation of patient based evidence at a global patient forum in 2014. An Editorial Board comprised of members of the steering committee manages the Patient Research Exchange website.

56 PATIENT AND OTHER ORGANIZATIONS

International Alliance Patients’ Organization (IAPO): Patient Solidarity Day 2015 theme announced We are thrilled to share with you that the IAPO members voted on the theme for Patient Solidarity Day 2015. There were many responses The most popular theme among members was...

Healthcare is a human right This year’s theme matches the current health landscape and resonates with patients from different countries and cultures across the globe.

With the launch of the Sustainable Development Goals, there is greater focus on the rights of each person and what we must do to protect and sustain these rights.

Health is no exception. WHO describes ‘the enjoyment of the highest attainable standard of health as a fundamental right of every human being.’

European Organization for Rare Diseases (EURORDIS): Rare Disease Day 2016 Theme and Slogan Theme: Patient Voice Slogan: Join us in making the voice of rare diseases heard 2016markstheninthyearthattheinternational rare disease community celebrates Rare Disease Day. On the 29 February 2016, people living with or affected by a rare disease, patient organisations, politicians, carers, medical professionals, researchers and industry will come together in solidarity to raise awareness of rare diseases. th

The Rare Disease Day 2016 theme ‘Patient Voice’ recognises the crucial role that patients play in voicing their needs and in instigating change that improves their lives and the lives of their families and carers.

The Rare Disease Day 2016 slogan ‘Join us in making the voice of rare diseases heard’ appeals to a wider audience, those that are not living with or directly affected by a rare disease, to join the rare disease community in making known the impact of rare diseases. People living with a rare disease and their families are

often isolated. The wider community can help to bring them out of this isolation.

Rare Disease Day amplifies the voice of rare disease patients so that it is heard all over the world.

EURORDIS, whose mission is to be the voice of an estimated 30 million people living with a rare disease in Europe, supports patients to make their voice stronger through various initiatives and activities, including: ▀▀ The EURORDIS Access Campaign, which

invites anybody living with a rare disease to voice the difficulties they experience accessing treatment or care through a questionnaire. ▀▀ The facilitation of the participation of

patient advocates in numerous scientific committees and working parties at the European Medicines Agency and beyond. ▀▀ The EURORDIS ExPRESS Summer School,

aimed at empowering patient representatives through training in the areas of clinical research, regulatory affairs and healthtechnology assessment. ▀▀ The

EURORDIS Patient Voices Programme, created to collect patient opinions on transversal topics and include them in the policy and decision-making process and other regular consultations with patients. ▀▀ Rare Diseases International, the global

voice for rare disease patients.

▀▀ RareConnect, the online network of rare

disease communities that provides a forum for people living with or affected by rare diseases to voice their experiences and meet others living with the same rare disease.

More information ▀▀ Visit the official Rare Disease Day website.

EULAR Brussels Conference on 13 October 2015 The 6th edition of the EULAR Brussels Conference has been completed on the 13th October 2015 under the theme: “Towards more integrated health care in Europe: Strengthening patients’ access to cross-border care and enhancing health professionals’ mobility”.

The goal of the conference was to identify and

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We encourage our national societies to post this information to announce this initiative on their websites and inform all members.

European Reference Network (ERN): 2nd conference on European Reference Networks Imagine if the best specialists from across Europe could join their efforts to tackle complex or rare medical conditions that require highly specialised healthcare and a concentration of knowledge and resources.

That’s the purpose of the European Reference Networks and it’s becoming a reality.

Health systems in the European Union seek to provide high-quality, cost-effective care. This is particularly difficult in the case of conditions requiring a concentration of resources or expertise, and even more so with rare or lowprevalence complex diseases or conditions.

EU healthcare policy has furthered cooperation between EU countries, including networking. Some networks benefit from the EU’s public health and research programmes, especially in the areas of rare diseases , paediatric cancer and neurological complex diseases.

Such cooperation has been based mainly on bilateral agreements or joint projects in specific fields. Moreover, healthcare access varies widely across the EU. More efficient and coordinated sharing of resources and expertise was thus needed, and can be achieved through the creation of European Reference Networks (ERNs).

Legal background ▀▀ Directive 2011/24/EU on patients’ rights

in cross-border healthcare;

▀▀ Commission delegated decision listing

the criteria and conditions that healthcare providers and the ERNs should fulfil; ▀▀ Commission

implementing decision containing criteria for establishing and evaluating ERNs, including the exchange and dissemination of information about the ERNs. Networks implementation

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The Directive on the application of patients’ rights in cross-border healthcare requires the European Commission to support Member States in the development of European Reference Networks (ERN) and the Commission Implementing Decision establishes the process and criteria for setting the complete lifecycle of the Networks from the call for proposals to the assessment, establishment and evaluation.

ERNs & centres of expertise Several EU countries already have national models and systems for identifying and designating highly specialised healthcare providers as centres of expertise or reference.

This enables them to concentrate expertise and patient numbers in one place to tackle rare or complex diseases and conditions more effectively.

ERNs - projects & activities The EU has funded pilot networks and projects focusing on groups of rare, low-prevalence or complex diseases. A number of studies have been, or are being conducted on ERNs and the provision of highly specialised care.

About the Conference Lisbon, 8-9 October 2015 DG Health and Food Safety has organised the “2nd Conference on European Reference Networks (ERN)” between the 8th and the 9th of October 2015 in Lisbon, Portugal.

The 2nd Conference, hosted by the Ministry of Health of Portugal, was included as an event of the Luxembourgish EU Presidency.

The conference built on the success of the 1st conference on European Reference Networks, which took place in Brussels on 23rd June 2014.

The Conference can be considered as a great success in terms of participation, high quality of the speakers and presentations and substantial commitment and engagement of all the players: politicians, experts, managers, healthcare professionals and providers, patients and other stakeholders.

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recommend possible policy developments at both the EU and national levels aiming to optimise cross-border care and the mobility of patients and health professionals.

58 WORLD HEALTH ORGANIZATION NEWS

Non Communicabe Disease news The Global Cornication Mechanism/ Non Communicabe Disease (GCM/ NCD) welcomed a new staff member, Dr Guy Fones. Guy is a medical doctor by training with 25 years’ experience and achievements in the public, private and global health sectors. He joins WHO as Advisor directly from his previous post as Health Attaché for the Permanent Mission of Chile here in Geneva. Guy’s first task will be to prepare for the work of the three new working groups to be established under the WHO GCM/NCD in 2016-2017 : 1. Working Group for the Action 3.1 of the 2016-2017 GCM work plan will be established “to recommend ways and means of encouraging Member States and non-State actors to promote the inclusion of the prevention and control of NCDs within responses to HIV/AIDS and programmes for sexual and reproductive health and maternal and child health, as well as other communicable disease programmes, such as those on tuberculosis , including as part of wider efforts to strengthen and orient health systems to address the prevention and control of NCDs through people-centred primary health care and universal health coverage”.

2. Working Group for Action 3.2 of the 2016-2017 GCM work plan will be established “to recommend ways and means of encouraging Member States and non-State actors to align international cooperation on NCDs with national plans concerning NCDs in order to strengthen aid effectiveness and the development impact of external resources in support of noncommunicable diseases”.

3. Working Group for Action 3.3 of the 2016-2017 work plan will be established “to recommend ways and means of encouraging Member States and nonState actors to promote health education and health literacy for NCDs, with a particular focus on populations with low health awareness and/or literacy, and taking into account the cost-effective and affordable interventions for all Member States contained in Appendix 3 of the WHO Global NCD Action Plan 2013-2020”.

Information Note 3 sets out the process

for the creation of the three new Working Groups. Additional information is available on the GCM/NCD Working Group webpages here: http://www.who. int/global-coordination-mechanism/ working-groups/ Fifth meeting of the UN Interagency Task Force on NCDs The Fifth meeting of the NCD Task Force was held between the 26th and the 27th October 2015 and was attended by representatives from 23 UN agencies, programmes and funds. The Task Force reviewed progress reports from countries that have received Task Force programming missions. Members of the Task Force agreed that there has been good progress in moving the NCD agenda forward in these fast track countries. The Task Force also agreed that, moving forward, progress of the work of the UN Country Teams will be measured against the 10 WHO NCD Progress Monitor indicators which WHO published on 1 May 2015.

The latest achievements of the ongoing WHO/ITU Global Joint Programme on mHealth for NCDs was highlighted.

Three other Global Joint Programmes, which are under development (two on cancer and one on multisectoral action for NCDs) were discussed so that they can be finalized during the next months.

A new thematic group on the harmful use of alcohol has been established which WHO and UNDP are jointly chairing.

Blood Safety: World Blood Doner’s Day - 14 June 2015!

The theme of this year’s campaign is “Thank you for saving my life”. It focuses on thanking blood donors who save lives every day through their blood donations and strongly encourages more people all over the world to donate blood voluntarily and regularly with the slogan “Give freely, give often. Blood donation matters.” The campaign aims to highlight stories from people whose lives have been saved through blood donation, as a way of motivating regular blood donors to continue giving blood and people in good health who have never given blood to begin doing so.

WHO’s goal 2020 is the target year for all countries to obtain 100% of blood

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Campaign goals and objectives

taking place during 2011-2015.

The global meeting for countries participating in MeTA was organized ▀▀ Blood safety and availability to share experiences and to develop a deeper understanding of the strategies ▀▀ Technical resources and practices that have been successful in improving transparency and ▀▀ Blood transfusion safety accountability in the pharmaceutical ▀▀ Blood donor selection: Guidelines on sector. assessing donor suitability for blood DFID have supported this effort and the donation representatives from the MeTA countries ▀▀ Blood donor counselling: have worked hard to establish and maintain the multi-stakeholder dialogue, Implementation guidelines improve transparency and accountability Counterfeit Medicines: Medicines and to make policy recommendations Transparency Alliance Global Meeting for improving access to medicines. We hope that this report is useful in sharing 2014 some of the many achievements made Each year an estimated US$ 4 trillion is in countries through this initiative. For spent worldwide on health services. A more, visit the following link: http://apps. large proportion of this expenditure who.int/iris/bitstream/10665/176618/1/ is due to pharmaceutical expenditure. WHO_EMP_PAU_2015.1_eng.pdf Despite this, there is a continued lack of access in many countries, affecting Birth defects surveillance training: treatment outcomes of the millions of facilitator’s guide people suffering from illnesses such as malaria, pneumonia, cardiovascular World Health Organization (WHO), disease and diarrhoea. For these National Center on Birth Defects and people and others struggling to live Developmental Disabilities from the with conditions such as diabetes, high United States Centers for Disease Control blood pressure and mental illness, and Prevention (CDC), International the medicines which can be used to Clearinghouse for Birth Defects cure or manage their illnesses can be Surveillance and Research (ICBDSR) too expensive or simply not available. Overview Moreover, those medicines that are available may be of doubtful quality or The goal of this course is to provide dispensed inappropriately. participants with the foundational skills needed to begin the development, The Medicines Transparency Alliance implementation and ongoing (MeTA) is an initiative funded by the improvement of a congenital anomalies United Kingdom Department for surveillance programme, in particular International Development which for countries with limited resources. It works to improve access to medicines focuses on the methodology needed by increasing transparency and to develop either population-based or accountability in the pharmaceutical hospital based surveillance programmes. sector.

A set of congenital anomalies will be Multi-stakeholder groups have been used as examples throughout this formed to collect and share data on course. The specific examples used are the selection, procurement, quality, typically severe enough that they would availability, pricing, promotion and use probably be captured within the first of medicines. The data and evidence few days after birth, have a significant collected is analysed and used to public health impact and, for some of support policy dialogue and to advise them, have the potential for primary and recommend actions that improve prevention. access to medicines. The initiative was piloted in 2008-10 and is now in its second phase, MeTA Phase 2 which is

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supplies from voluntary unpaid donors.

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Season's Greetings!

The end of 2015 is here, a year full of challenges and rich in achievements. The year-end is an opportunity to celebrate those achievements, and to make resolutions for an even more productive 2016.

TIF wishes you and your families a very happy New Year, full of health, happiness and personal and professional fulfilment!

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