TIF Magazine - issue 65

TIFMAGAZINE OCTOBER 2014

Issue 65

Published by the Thalassaemia International Federation

www.thalassaemia.org.cy

3rd Updated Edition of the “Guidelines for the Clinical Management of Transfusion Dependent Thalassaemia�

4th Pan-European Conference on Haemoglobinopathies and Rare Anaemias Athens, 07-09 November 2014 Join Us! International Thalassaemia Day 2015: Enhancing partnership towards patient-centred health systems: good health adds life to years!

2

OCTOBER 2014 www.thalassaemia.org.cy

3

As we approach the end of 2014, it is time to review what has been achieved in 2014 to-date and plan ahead for the future. The last months have been extremely busy, exciting, and productive, with numerous activities taking place all over the world and with a huge workload on TIF Office’s shoulders.

Indeed, we have undertaken, participated in, and supported meetings and conferences in many parts of the world including Algeria, Morocco, China, India, Maldives, Palestine, United Kingdom and Greece!

An overview of all the significant events of this year to date can be found in this issue but also a sneak preview of the exciting events to come.

In 2014, the major Regional Conference, namely, the 4th Pan-European Conference on Haemoglobinopathies and Rare Anaemias, will be the driving force of TIF’s activities in the European Region. It will undoubtedly be the highlight of the year for the European patient community. This regional conference will take place between 07-09 November 2014 in Athens, Greece, thus taking advantage of the Greek beauty and hospitality but also of geographical crossroads on which Greece rests upon - joining the east with the west.

This is an event that, I am sure, we all wait with great excitement and expectations, and will be the undisputed highpoint of the year! This grand educational event is organized by TIF in collaboration with the Greek Thalassaemia Federation (EOTHA), and is held under the auspices of the Hellenic Society of Haematology and the Greek Ministry of Health. It will also and very importantly be observed by the European Commission and the European Hematology Association through the delivery of keynote speeches in the programme. It will feature a wealth of topics both for patients and health professionals as well as for policy-makers.

Based on its vision to expand its network of medical and health professionals from around the world in this field, TIF is working on a new format for its regional conferences, aiming to strengthening

the educational nature of the conference and the motivation and interest of young health specialists in the fields of haematology and paediatrics.This change has become essential to ensure that the inheritance of knowledge and experience of international experts is passed on to the younger generation.

Such moves constitute important steps forward and this conference constitutes the first in the context of TIF’s new vision. We look forward to welcoming “new blood” from the region while the support and guidance of our international experts from around the world will continue to be cherished and constitute an essential element of TIF’s educational programme.

Furthermore, we have made significant progress in consolidating our collaborative relationships with important organisations in Europe and across the world, including official health-related bodies and agencies, professional medical bodies and other (than thalassaemia) disease-oriented organisations.

A new highlight in the European Region has been the election of new members of the European Parliament and the European Commission that will give the new light and hope in improving and promoting chronic rare diseases. The new vision of the European Union (EU) will be materialised through its 3rd Health Programme (2014-2020) and will be streamlined to focus on priorities such as promoting the quality of health, protecting EU citizens from serious cross-border health threats, contributing to innovative and sustainable health systems, and facilitating access to better and safer healthcare for EU citizens.

On a global scale, the 12th Association of South East Asian Nations Health Ministers’ Meeting (12th AHMM) in Hanoi, which took place in September 2014, was set on the aim of promoting the transition of health care systems to more patient-orientated ones and on tackling effectively health inequities and the emerging threats of the common noncommunicable diseases.

Cover Photograph: “Hugs” by Truong Ngoc Son of the Vietnamese Thalassaemia Association (ViTA), winner of the first prize of the TIF Photography Contest (read more on page 17).

OCTOBER 2014 www.thalassaemia.org.cy

TIFMAGAZINE

Foreword from the TIF President and Executive Director

Panos Englezos President

Dr. Androulla Eleftheriou

4

TIF displays a strong commitment in promoting the aforementioned values as they are incorporated in its work and will form the basis for its future plans on a national, regional, and international level.

TIF’s zest was also evident during the celebrations of World Thalassaemia Day 2014, which took place on the 8th of May 2014 to honour this important day.The theme this year was “Economic Recession: Observe - Join Forces - Safeguard Health” and it aimed to highlight the health inequalities experienced by hundreds of thousands of patients across the world during the difficult times of economic austerity we are all going through. This issue of TIF Magazine contains a special selection of activities from around the world that took place to honour the day.

The year 2014 also marked the establishment of new, highly significant, and evolutionary projects such as the video challenge platform, the “My Thal” mobile application, the Sphera project, the Australian Blood Project and many more! However, the highlight of this year’s educational programme is the publication of the 3rd edition of the Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT), a publication which will continue to strengthen TIF’s impact in the education of health professionals and relevant regional bodies in this field.

OCTOBER 2014 www.thalassaemia.org.cy

In 2015, a very ambitious and challenging plan is ahead of us with many significant activities that will support and contribute to the realisation of TIF’s strategic goals for 2015-2017. Some of the goals for the next three years include: • The development of TIF’s presence globally • Further enrichment of TIF’s educational programme and strengthening of access to health programmes and policies.

• Establishment and strengthening of the collaboration with relevant International and Regional bodies which will assist TIF’s work and mission in including thalassaemia in the health priorities and programmes that receive global recognition and funding.

In realising our mission for the inclusion of thalassaemia in international health control programmes and our vision for establishing equal access for all patients to quality health and social care, we must always remember that unity and knowledge constitute our strong weapons.

We feel extremely privileged to be surrounded by distinguished Board Members, embraced with the love and support of volunteers, health professionals, and patients, and supported by a distinguished group of international, regional, and national experts who have given the opportunity to TIF to truly make an impact in the world.

5 TIFMAGAZINE

Contents Chief Editor: Dr. Androulla Eleftheriou TIF Editor: Liana Prastiti Design and Layout: Design for Life Ltd - CY Proof Reading Demetris Lambrou Lily Cannon Matthew Demetriades Printing: Cassoulides Masterprinters ISSN:1450-3093 TIF BOARD MEMBERS Panos Englezos, President Shobha Tuli Vice President Loisos Pericleous Secretary Riyad Elbard Treasurer Her Highness Sheikha Sheikha Bint Seif Al-Nahyan Anton Skafi Christina Stephanidou Duru Malyali Fatemeh Hashemi George Constantinou Ivan Dimitrov Ivanov Loris Angelo Brunetta Mouna Haraoui Nailya Guliyeva Ramli Mohd Yunus Robert (Bob) Ficarra Saeed Jafaar Al-Awadhi Gabriel Theophanous

Foreword

3

TIF Activities Around The World

6

International Collaborations

9

Fostering a strengthened collaboration with the WHO A milestone in TIF’s Educational: Upgrading the Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT)

12

4th Pan-European Regional Conference

14

Poem dedicated to Mr Panos Englezos, TIF President

15

International Thalassaemia Day! Join Us! March with us on the 8th of May 2014 !

16

TIF Publications

19

Regional News

20

Congratulations

23

Patients’ Story

25

Other News

26

Revolutionary new project: TIF video sharing platform

29

Obituaries

30

Calendar & Upcoming International Events

31

Medical Corner

32

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.

OCTOBER 2014 www.thalassaemia.org.cy

6

TIF Activities Around The World

TIF’s taskforce in India

SEAR Setting the grounds for success in India...

Groundbreaking progress in India was achieved due to the series of TIF delegation visits and meetings held over the past 18 months in the country, and the immense support and assistance of the Federation of Indian Thalassemics (FIT), and each of the involved State Taskforces.

In the context of its most recent delegation visit, which took place between 19-20 April 2014, in New Delhi, a meeting with state coordinators was held with the aim of finalising the Charter of Priorities that TIF has undertaken to develop for the Ministry of Health and Welfare (Central Government) of India and for each one of the participating States.The States covered were Punjab, Haryana, Uttar Pradesh, Maharashtra, Chandigarh, Gujarat and Delhi.

The Charters of Priorities have now officially been dispatched to the governments of each of the aforementioned States and to the Ministry of Health and Welfare of the Central Government of India with a view to address the problem of Haemoglobin Disorders in a holistic manner. These developments are of vital importance as they will serve as advocacy tools for involved stakeholders to instigate further improvements to the healthcare services provided nationally to our patients in these States and by extent to hopefully encourage other Indian states to follow.

Parallel to this meeting, and in the context of its delegation visit, TIF participated in the 7th National Thalassaemia Conference which was successfully organised by the National Thalassaemia Welfare Society (NTWS) in New Delhi, India, on 19-20 April 2014. This conference was attended by over 300 participants, mostly patients and their parents.TIF was represented

by Mrs Shobha Tuli,TIF Vice President and President of FIT. In addition, Dr Michael Angastiniotis, TIF Medical Advisor, and Ms Lily Cannon, TIF European Affairs Officer, also participated in the Conference.

This workshop offered an excellent opportunity for TIF to discuss aspects of the practical management of thalassaemia and to further interact with the national and international academic faculty.

The Maldives are welcoming growth We are pleased to share with you that TIF, represented

by Dr Michael Angastiniotis, TIF Medical Advisor, has completed a delegation visit in the Maldives, after having received a request from the Regional Office for South-East Asia(SEARO) of the World Health Organization (WHO). This request was made for the purpose of assisting the health authorities of the OCTOBER 2014 www.thalassaemia.org.cy

country in the updating their National Guidelines for the Clinical Management of Thalassaemia, mainly Transfusion Dependent Thalassaemia.

The new 3rd edition of TIF’s international Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT), will form the basis for the updating of the country’s existing version of such Guidelines.

This visit also provided an excellent opportunity for TIF’s representative to liaise with officials from the Ministry of Health of the Maldives, relative blood services of the country, and the patients associations.

EMR TIF sets the pace for Palestine Shortly before the tribulations that occurred in the Palestinian region, the Thalassaemia Patients’ and Friends’ Society (TPFS) in Palestine and the National Committee for Thalassaemia Prevention, with the collaboration of TIF in Cyprus and the Palestinian Ministry of Health, have successfully organized the 5th National Palestinian Thalassaemia Conference under the theme “Thalassaemia… New Challenges”. This Conference was held under the patronage of His Excellency the Palestinian Minister of Health, in Ramallah, Palestine between 07 and 08 May 2014.

The conference was well attended with around 400 participants and wide representation from governmental services, non-governmental organisations and other stakeholders.TIF participated in this conference, represented by Mr Riyad Elbard,TIF Board Member, and Dr Michael Angastiniotis, TIF Medical Advisor.

The 5th National Conference addressed a series of important matters which included healthcare in Palestine, the role of national institutions when it comes to no new cases of thalassaemia, the psychological and social issues of patients with thalassaemia in Palestine, and patients’ rights.

This conference contributed greatly to achieving the objective of enhancing the knowledge of local medical and health authorities and gave them the opportunity to meet and network with involved stakeholders in the field. This conference has proven

7

stakeholders, including the medical community, the Ministry of Health, and Patient and Parent Associations of the country.

Heading towards a new era in Morocco The dawn of a new era has begun in Morocco! In alignment with TIF’s efforts to strengthen collaboration with important stakeholders in Morocco, TIF, represented by one of its Board Members, Mr Loris Brunetta, has completed a delegation visit to Morocco between 09 and 13 May 2014.

In the context of this visit, Mr Brunetta participated in the Thalassaemia Day Scientific Workshop held on 9 May 2014, organised by the Service of Haematology and Paediatric Oncology in collaboration with the Faculty of Medicine and Pharmaceutics of the Casablanca University, which took place in Rabat, Morocco. Many important physicians, haematologists and paediatricians attended this event.

This workshop was very constructive as it allowed for the fruitful discussion of topics of great value and significance, and gave the opportunity for exchange and networking between important stakeholders in the field and the medical community. The diagnostics of haemoglobinopathies, their monitoring, the causes and consequences of iron overload, clinical case studies and the establishment of haemoglobinopathies’ reference centres in Morocco were among the central topics discussed at this workshop.

Furthermore, meetings with crucial supporters and stakeholders, such as members of the Moroccan Rotary Club and the Moroccan Association for Thala-ssaemia and Sickle Cell Disease, were conducted. These are important steps towards bringing change and entering a new age in Morocco regarding haemoglobinopathies!

AFR Algeria: Steadily advancing towards change...

As a result of TIF’s endeavours in Algeria and the collaboration with its Health Authorities and stemming from several delegation visits and the signing of a Memorandum of Understanding (September 2013), the Algerian Health Authorities have demonstrated great willingness to exert every possible effort to allocate resources in moving things forward in Algeria for the materialisation of the jointly agreed road map.

In the context of these endeavours, TIF, represented by Prof Dimitris Loukopoulos, TIF Medical Maghreb coordinator, participated on 7 July 2014 in a constructive meeting which was organised by the Department of Prevention of the Ministry of Health, Population and Hospital Reform of Algeria (MSPRH).

This initiative has been a stepping stone with regards to ensuring that the aforementioned meeting will constitute a great first step not only in the materialisation of the jointly agreed road map but also in supporting efforts made to improve the quality of life of patients in the country.

Iran states its commitment to progress As TIF establishes its commitment to support Iran, an official high-level follow-up visit to Iran is planned to take place within the following months of 2014.

The aim of the visit is to monitor the effective implementation of the national policies for thalassaemia that were put forward in this country in the past and review the current situation in view of the effects of the recent embargo that has been imposed on Iran by United Nations (UN) and European Union (EU) laws and which forbids Iran to export and import certain technologies that also need to be made available to patients with haemoglobinopathies.

Meetings will also be held with relevant

WPR TIF’s plans in China TIF sets a new path with Chinese Health Authorities!

As a result of joint efforts, an official delegation visit and workshop in Guilin City of Guanxi Province in China is planned for 11 October 2014. The workshop will be organised by TIF in collaboration with Chinese Health Authorities including the Division of Family Planning, the Department of Maternal and Child Health, and the National Health and Family Planning Commission of the People’s Republic of China.

The aim of this workshop is to educate medical specialists on the management of thalassaemia and to sensitize local health authorities to take decisions on the control (prevention and management) of the disease. An additional aim of the workshop is to evaluate and review the current management and prevention policies in southern China.

Vietnam prepares for a brighter future Vietnam continues the fight against thalassaemia joining hands with TIF! Following TIF’s previous delegation OCTOBER 2014 www.thalassaemia.org.cy

TIFMAGAZINE

to be of vital significance, now more than ever, on the eve of the tribulations in the region, since those affected the most by such situations are the vulnerable population groups, including thalassaemia patients.

8

visits and activities in the country, TIF is planning to realise a delegation visit to Hanoi,Vietnam, in October to meet representatives of the Ministry of Health, the Vietnamese Thalassaemia Association (ViTA), which is the National Thalassaemia Association of the county, and officials of the National Institute of Haematology and Blood Transfusion (NIHBT).

The aim of this meeting will revolve around the implementation of a National Health Programme for Thalassaemia in this country but also the around the discussion on the 2nd Pan-Asian Regional Conference on Haemoglobinopathies and Rare Anaemias, which is to be organised by TIF in the upcoming year.

Standards for the Clinical Care of Sickle Disease in adults (2008) in Greek

EUR Supporting patients with haemoglobinopathies in Greece Thrilling News! TIF has already proceeded, in collaboration with international and local experts to translate in Greek and publish the United Kingdom’s “Standards for the Clinical Care of Sickle Cell Disease in Adults”, the “Emergency Management of Thalassaemia”, and the “Guide for the Haemoglobinopathy Nurse”.

The official launching for these publications will be held at the 4th Pan-European Conference on Haemoglobinopathies and Rare Anaemias, 07-09 November 2014, in Athens and they are expected to be published in the upcoming months. These publications are of vital importance to appropriately inform the medical and paramedical community in Greece about the timely diagnosis, monitoring, and clinical management of haemoglobin diseases, including thalassaemia and sickle cell disease.

Cyprus : The patients’ voice is heard...

Workshop for the Cyprus National Health Plan under the motto “Are you ready to Learn?”

An important highlight of TIF’s activities in Cyprus will be the workshop on the Cyprus National Health Plan, which will take place at the University of Nicosia, on 4 October 2014. This workshop will be organised by the Cyprus Alliance for Rare Diseases (C.A.R.D.), of which TIF is a founding member, with the collaboration of the Cyprus Federation of Associations of Patients, and Poster of the the participation of the Cyprus Medical Association campaign for the and the Cyprus Confederation of Organisations for Workshop on the the Disabled. It will be placed under the auspices Cyprus National of the University of Nicosia and the Cyprus Health Plan and Broadcasting Corporation (CY.B.C.) will function as Rare Diseases the communication sponsor for this event.

OCTOBER 2014 www.thalassaemia.org.cy

TIF is a part of the Scientific Committee of this Conference, represented by Dr Androulla Eleftheriou, TIF Executive Director, and Dr Michael Angastiniotis, TIF Medical Advisor. The TIF representatives have significantly contributed to the compilation of the workshop programme and Dr Androulla Eleftheriou will also participate in the programme and coordinate this event.

The main objectives of this workshop will focus on familiarizing patients with rare and chronic disorders such as thalassaemia about the National Health Plan, but also on examining its provisions as regards to rare diseases including haemoglobinopathies.

The outcome of the interactive discussion will be materialised in a manifesto which will be compiled and sent to all involved stakeholders, including the Ministry of Health of Cyprus.

Strategic meeting of the Cyprus Alliance for Rare Disorders July was landmarked by a key strategic meeting organised by TIF and the Cyprus Alliance for Rare Disorders (C.A.R.D.). This meeting was held at the TIF Headquarters in Nicosia, Cyprus, on 10 July 2014 with the aim to discuss future action on the direction of rare diseases in Cyprus. It was attended by representatives of individual Patient Organisations for Rare Diseases across Cyprus.

Among the topics discussed were the promotion of joint projects involving common policies on rare diseases, the detailed lobbying of all national, regional, and international governmental and non-governmental Rare Disease Patient Organisations, Bodies and Agencies, and the establishment, and implementation of EU and national programmes.

The discussion provided a revived impetus for the materialisation of a joint action plan on rare diseases, which also include haemoglobinopathies, as they are amongst the family of rare diseases in Europe through EC Communication on Action in the field of Rare Diseases (2009/C 151/02).

Promoting Self-management: Interactive Workshop on Thalassaemia With the aim of promoting the self-management of patients with Thalassaemia, the Pan-Cyprian Thalassaemia Association in collaboration with TIF and the Cyprus Institute of Neurology and Genetics (CING) organized an Interactive Workshop on Thalassaemia on 11 May 2014, at CING, Nicosia, Cyprus.

This workshop was placed under the auspices of the Cyprus Ministry of Health.

9

Participation in the 19th Annual Congress of the European Hematology Association (EHA) Continuing its efforts to raise awareness amongst haematologists in Europe and across the world,TIF has successfully participated in the 19th Annual Congress of the European Hematology Association (EHA), held in Milan, Italy between 12 and 15 June 2014.

Taking on a pivotal role in the Patient Advocacy Track - representing the non-malignant part of haematology - TIF participated in these sessions which this year focused on generics in haematology and the challenges of young people with old peoples’ diseases. Most notably, the presentations and expert knowledge of Dr Androulla Eleftheriou,TIF Executive Director, Dr Mahmoud Hadipour Dehshal (Board Member of the Iranian Thalassaemia Society) and Mr Michael Michael (Former TIF Board Member, UK) immensely impressed the audience.

Furthermore, a dedicated exhibition booth provided the perfect opportunity to distribute TIF’s educational material. The booth was adorn with the exceptional abilities of Mr Loris Brunetta, TIF Board Member, Mr Marco Bianchi (Italy), Ms Tomassina Iorno (Italy) and Ms Lily Cannon,TIF European Affairs Officer.

Moreover, a consensus paper on European haematology research is to be developed by the European Hematology Association (EHA) with the collaboration and contribution of TIF, among other important patients’ organisations relative to the field of haematology.This paper aims to inform and engage stakeholders with a direct interest in haematology and to promote the research of blood disorders in the context of EU’s Horizon 2020 Funding Scheme.

The paper is scheduled to be finalised by the end of spring 2015.

Seeking healthcare autonomy: European Reference Networks Conference The European Reference Networks Conference, held in Brussels on 23 June 2014, was marked by TIF’s dynamic presence and active participation. Organised by the Directorate for Health and Consumers of the European Commission, this Conference brought together highly specialised healthcare providers, experts, national authorities, decision–makers,

and independent bodies with experience in the assessment and evaluation of healthcare providers.

The aim of the conference was to discuss the state of play on the organisation of highly specialised networks and their members across the EU and to look into the next steps of the deployment process, in preparation for the forthcoming call for European Reference Networks in 2015. Establishing European Reference Networks of highly specialised healthcare providers represents a clear added value for the EU and will help to provide affordable, high-quality and cost-effective healthcare to patients with conditions requiring a particular concentration of resources or expertise, and to improve these patients’ access to the best possible expertise and care available in the EU for their condition.

The Changing Face of Healthcare: High Level Meeting on the Economic Crisis and Healthcare - the case of hepatitis B and C A very significant high level meeting entitled “Economic Crisis and Healthcare - ensuring access to public health services: the case of hepatitis B and C” took place between 03-04 June 2014 in Athens.

This meeting was co-organized with the Hepatitis B and C Public Policy Association. It was hosted by the Hellenic Centre for Disease Control and Prevention (KEELPNO) and was attended by TIF Executive Director, Dr Androulla Eleftheriou.

The meeting has given a particular focus on the barriers to the provision of health services, particularly in countries where austerity programmes are in place.

EU Member States have been invited to speak of their varying national experiences aiming to identify means of increasing access to health services. The case of chronic hepatitis B and hepatitis C has served as a very apt example: the prevalence of these diseases is high, especially in the European South, with very serious lasting effects, although the means to prevent and treat them exist.

TIF has already compiled a full report on the results of this meeting which includes a full update on the treatment of hepatitis B and the cure of Hepatitis C with the new drugs already available and those upcoming. Although the report is still in the

OCTOBER 2014 www.thalassaemia.org.cy

TIFMAGAZINE

International Collaborations

At the dedicated exhibition booth of the 19th Annual Congress of the EHA

10

Greek language for the purpose of being diffused to the Cypriot government and to the Cyprus mass media, the next step is to translate it into English and distribute it to our members. When completed, this document will consist TIF’s official position on the treatment of chronic hepatitis B and C, which are considered major problems in most of the nonEuropean countries and a big problem in some Eastern European countries as well.

Participation at the EU High Level Expert Meeting on Health Emergency Management The National Health Operations’ Centre (EKEPY) of the Hellenic Ministry of Health has organised for the first time an EU High Level Expert Meeting on Health Emergency Management in Athens, Greece, between 09-10 May 2014.The meeting was held under the auspices of the Greek EU Presidency and the Hellenic Ministry of Health and focused on the role of National Health Operations’ Centres.

The purpose of this meeting was not only to discuss but to proceed further to a fruitful exchange of scientific data, experiences and followed practices in the field of emergency management in the health sector among experts at an EU level. Represented

by Ms Lily Cannon, European Affairs Officer, TIF participated in the meeting with a presentation on haemoglobinopathies in Europe as a result of population migrations.

Strengthening the Pan- Middle East Network of medical specialists and patients Progress work is also being planned for revisiting and strengthening the Pan- Middle East Network of medical specialists and patients. This network is an important component of TIF’s work, ensuring the keeping of the momentum for haemoglobin disorders across this region and the diffusion of education, knowledge and awareness among the relevant stakeholders.

Enhanced collaboration with the European Parliament strengthens TIF’s position in Europe We are delighted to announce that TIF has embarked on a new initiative that revolves around the strengthening of TIF’s collaboration with Members of the European Parliament, who are participating

in the Environment, Public Health and Food Safety Committee for the development of effective prevention, clinical management, and control strategies for haemoglobinopathies and rare anaemias in Europe.

TIF, with the opportunity of this communication, has sought to investigate the possibility of ensuring the European Parliament’s support and involvement in TIF’s activities in order to promote institutional consolidation of key matters for the benefit of patients with haemoglobin disorders.

TIF has already received the expression of interest of the President of the European Parliament Mr Martin Schulz, conveying his desire to get involved in TIF’s activities and to align in joint action. By the means of this collaboration, TIF aspires to engage in a deeper debate on health and migration policy measures that are able to adequately respond to the rise of Haemoglobinopathies in Europe. TIF’s ultimate goal is the compilation of a consensus paper on haemoglobinopathies which is to be submitted at the European Parliament and which will constitute an expected continuation of TIF’s significant work on the control of haemoglobinopathies on the global and European map.

Association of South East Asian Nations Recognizing the importance of the Association of South East Asian Nations’ (ASEAN) Health Ministers’ Meeting (AHMM), and looking to benefit from the presence of the many important stakeholders attending the meeting, TIF’s on-going effort of the last two years has been to instigate a collaboration with the Ministries of Health of the ten ASEAN countries with an interest in organising a side meeting to the AHMM. Already, the 12th AHMM took place in Hanoi, Vietnam, between 17 and 19 September 2014 and has been a major driving force for the South East Asian Region. We are looking forward to the next AHMM whose date and venue will be announced soon.

The aim of the side meeting that TIF plans to organize, will revolve around informing the Ministers of Health of each country on: • The incidence and burden of Thalassaemia in each ASEAN member state • The estimated impact of control and management programs • The impact of Thalassaemia on child mortality This will be a very big step towards spreading awareness for thalassaemia among the member states of the ASEAN.

OCTOBER 2014 www.thalassaemia.org.cy

11

New opportunities for collaboration with the World Health Organisation (WHO) We are delighted to announce that TIF has reinstated its commitment to officially consolidate its relations with the WHO. In accordance with the principles governing relations between TIF and WHO, TIF has submitted its triennial report on the implementation of the plan of collaboration between TIF and WHO. This report is the basis of an official relationship for a plan for collaboration based on mutually agreed objectives outlining activities for a three-year period.

Through the submission of this report,TIF aspires to continue and to strengthen its collaboration with the World Health Organisation (WHO) with the objective to promote the policies and strategies derived from the decisions of the governing bodies of WHO. The review offers an opportunity not only to build this collaboration but to seek agreement on a new three year collaborative plan, in this case for the period 2015-2017. The agreed activities are intended to contribute to the achievement of the WHO strategic objectives and Organization-wide expected results as set out in the WHO’s MediumTerm Strategic Plan 2009-2014.

Situation analysis for haemoglobinopathies across the WHO Regions…

TIF has undertaken an ambitious initiative revolving around preparing a situation analysis for the countries of each WHO Region which aims to identify the characteristics of the population (e.g. birth, death and fertility rates), their health needs (e.g. epidemiology of haemoglobin disorders), and the public and private health-related services currently available. This analysis forms a part of a disease burden mapping and will also include the available services and future policies to be developed.

More specifically, the aims of the situation analysis are the following: • To promote national planning for prevention and management of haemoglobinopathies and its implementation • To establish/ strengthen surveillance systems for haemoglobinopathies • To establish/ strengthen existing health services and create new ones We are looking forward to embarking on this initiative in collaboration with the WHO, which formed a part of our proposed joint plan of action.

Working together for the Compilation of Scientific papers…

Furthermore, stemming from the situation analyses of the countries of each WHO Region mentioned previously, four scientific papers will be prepared by the TIF scientific medical panel in collaboration with the WHO Regional Directors and the WHO Directors of the Non Communicable Disease (NCD) Units of the following 4 Regional Offices of WHO: • Eastern Mediterranean Regional Office (EMRO) • South-East Asian Regional Office (SEARO) • Western Pacific Regional Office (WPRO) • European Regional Office (EURO) These scientific papers are focused on the current status of control of haemoglobin disorders and in particular thalassaemia, in terms of their prevention and clinical management within and across all countries of each region. We have secured the collaboration and interest of the director of each WHO Regional Office for the preparation of these papers. These scientific papers will be finalised by the end of 2014.

OCTOBER 2014 www.thalassaemia.org.cy

TIFMAGAZINE

Fostering a strengthened collaboration with the WHO

12

A milestone in TIF’s Educational Programme: Upgrading the Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT)

We are proud to share with you that TIF through a panel of experts has finally completed the guidelines for transfusion dependent thalassaemias. We are anticipating with great excitement their official launching which will be marked by two major events, a press conference in Cyprus that will take place in October and the 4th Pan-European Conference on Haemoglobinopathies and Rare Anaemias (0709 November 2014, Athens). This conference will provide a stage for the official launching of TIF’s newest publications, including the 3rd edition’ of the ‘Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT)”.

TIF’s three previous editions of the ‘Guidelines for the Clinical Management of Thalassaemia’ (1999, 2007, 2008) have been amongst the most important undertakings of TIF, which had perhaps the greatest impact in the context of its educational programme. This book is the result of the compilation of available scientific evidence, experiences, views, practices and knowledge put together for the first time. There was no such source of information prior to these guidelines and in fact the last documents produced by WHO in a short version of an information guide dates back to the 1970s and 1990s.

TIF considers that the contribution of this book to the education of the medical community has been exemplary in addition to its use as reference material for National Health Authorities and policy makers to plan and take action on treatment/ reimbursement and implement other health polices for patients with these diseases. It has indeed given the interest and motivation to other countries’ health authorities to follow and prepare their own standards of care and national guidelines.

OCTOBER 2014 www.thalassaemia.org.cy

As with every publication and according to TIF’s policy in the frame of its educational programme, this book requires thorough revision and updating after 5 years of significant progress and advances achieved.

A team of international experts in the field has been invited to contribute in this edition. The update focused on a more comprehensive description of the available information, clinical experience, research results and evidence-based knowledge which has been accumulated through recent years on the diagnosis, prognosis, prevention, monitoring and treatment of medical complications of β-thalassaemia major.The social aspects and quality of life perspectives of patients have also been given a significant focus.

In addition, the guidelines include TIF’s data on the current status of the development of national planning and other important policies at the national, regional, and international level.

TIF, as a patient-centred organization that has been striving for decades to support the effective prevention and optimum care for patients with haemoglobinopathies across the world, wishes to thank and is greatly indebted to each and every one of the dedicated scientists and medical specialists who have offered their time and labour freely to this vast and challenging work of updating the existing Guidelines. Their knowledge and scientific judgment are now disseminated across the world and TIF will exert every possible effort to ensure that health authorities in all countries and regions of the world adopt and apply these recommendations, even in times of economic austerity, for the benefit of citizens who, despite their inherited condition, are willing and able to return the investment through their own labour and positive contribution to society, if kept healthy and with an acceptable quality of life.

13 TIFMAGAZINE

OCTOBER 2014 www.thalassaemia.org.cy

14

Join us in the historical city of Athens for a memorable experience!

The Board of Directors of the Thalassaemia International Federation (TIF) and the Greek Thalassaemia Federation (EOTHA) look forward to the 4th PanEuropean Conference on Haemoglobinopathies and Rare Anaemias with immense enthusiasm. The destination of this conference is the alluring city of Athens, birthplace of arts, sciences and modern medicine, the capital and the political and cultural centre of Greece, fusing together history, culture and magnificent beauty.

This extremely interesting and informative conference will offer a unique platform of exchanging and sharing ideas and will touch all aspects of research, concerns, successes, and expectations. Each topic is covered by national, regional or international experts in this field.

What is more, Regional Conferences, starting from this one, have now been tailored to meet the needs of patients and health professionals within and between countries geographically.

Steering Towards the Future: changing the dynamics and vision of TIF’s Regional Conferences Therefore, the 4th Pan-European Conference on Haemoglobinopathies and Rare Anaemias and of every subsequent Regional Conference revolves around the following aims: A. For Patients • To educate, empower, and create core groups of patients in each country to support and strengthen their role in monitoring their own disease • To develop the skills and capacities of patients for the advocacy and productive participation in decision-making at the country and Regional level, and • To develop a robust infrastructure for National Patients’ Associations, making them truly supportive to their patients and their families at the country level.

B. For Health Professionals • To develop a network of collaborators from each region ensuring a more tailored support by TIF at the country level.

• To develop a core group of expert medical specialists across disciplines relevant to the multidisciplinary care of haemoglobinopathies, aiming to support the pillars of TIF’s educational programme • To prepare, translate, and distribute publications and educational material

OCTOBER 2014 www.thalassaemia.org.cy

• To include scientific programmes in educational events • To develop or contribute to academic courses C. For Policy Makers • To provide a forum of interaction for patients, families, and health professionals aiming to improve the recognition of priorities and needs pertaining to haemoglobin disorders by the government in each Region.

This conference aims to conclude with a declaration that is to be delivered to the European Commission and the European Parliament, as well as to the Government of each of the European member countries, for recognizing the need of effectively addressing the prevention and clinical management of haemoglobin disorders, which is a rising public health problem in almost every European country.

This year’s new format for the Regional Conference is certain to make this conference a truly remarkable experience! More information can be found at the conference’s website: www.tif2014.org

15 A Special Man’s Journey Dedicated to Mr. Panos Englezos, President of Thalassaemia International Federation

Our eyes reflect the struggles, efforts and successes His heart feels our setbacks Swells with pride for our triumphs Breaks with the loss He understood the future needed change A world with access A better future for all thalassaemics And so it began. Over twenty-five years ago In a distant land, a group convenes Constitution and bylaws are formed Country to country Negotiations, handshakes, reaching out Every child, every country Change happens, slowly but surely A man’s journey for others Accomplished more than others in a lifetime Not for himself For his son and others who lost the fight Unselfishly carrying the memory and the light For every child and parent who now has…

HOPE Thank you for all you do, and all you are!

Written by Dawn Adler, member of TIF’s Expert Patients’ Panel

OCTOBER 2014 www.thalassaemia.org.cy

TIFMAGAZINE

Even though he only held his son for a short time He never gave up the fight In each one of us, he saw a part of him

8 of May 2014 th

INTERNATIONAL THALASSAEMIA DAY This year’s International Thalassaemia Day was honoured with zeal and enthusiasm that surpassed our greatest expectations. The slogan for 2014 was ‘Economic Recession: Observe - Join Forces - Safeguard Health’, inspiring patients everywhere to keep up the fight for fair access to healthcare, education, employment-in short, to fulfill their dreams in spite of the economic recession.

TIF’s members organised events worldwide promoting awareness and knowledge regarding haemoglobinopathies for the International Thalassaemia Day. Read more about these activities on page 20, 21 & 22.

TIF, therefore, in the context of the International Thalassaemia Day and in the context of its internationally renowned educational program, which constitutes one of the most powerful means not only in achieving its mission and vision but also in raising awareness regarding haemoglobinopathies around the world, has organised many activities in the country of its headquarters, Cyprus, leading up to the 8th of May. The events and activities for the International Thalassaemia Day 2014 in Cyprus have begun on the 2 May, with the 2nd Ambassadors’ Circle and ended on the 8 May with the Press Conference organized to honour this occasion. In the following pages, we will share these activities in greater detail.

A closer look at the International Thalassaemia Day: a day of spreading awareness and supporting those who have been affected by Thalassaemia...

Highlighting a successful 2nd Ambassadors’ Circle Meeting!

2 May 2014, Hilton, Nicosia The 2nd Official Meeting of the Ambassadors’ Circle, organised by TIF was held with great success for a second successive year!

This meeting was marked by the avid participation of renowned diplomatic personalities, Ambassadors, Consuls, former Ministers of Foreign Affairs of Cyprus, and other official Embassy Representatives of TIF’s member countries have attended this Meeting on 2 May 2014, at the Hilton Hotel in Nicosia, for an event dedicated to the International Thalassaemia Day. More than 45 participants attended the meeting and a total of 15 countries were represented by embassy delegates, namely Australia, Egypt, Malta, Iran, Philippines, France, Spain, Nepal, Sri Lanka, Thailand, Greece, Syria, Italy, Cyprus and China.

The purpose of the aforementioned meeting was to inform foreign diplomatic missions in Cyprus about TIF’s activities in each one of the affected countries as well as the management and prevention protocols for haemoglobinopathies in these countries.

Dr Valentino Martelli, former Secretary of State of Foreign Affairs of Italy, Mr Alexandros N. Zenon, Director General of the Ministry of Foreign Affairs in Cyprus, Dr Erato Kozakou Marcoullis, and Mr Markos Kyprianou, former Ministers of Foreign Affairs of Cyprus and Mr. Charalambos Bakirtzis, Director of Anastasios OCTOBER 2014 www.thalassaemia.org.cy

“ECONOMIC RECESSION: OBSERVE - JOIN FORCES - SAFEGUARD HEALTH” Leventis Foundation, were also among the distinguished guests of this meeting.

The Programme of this event was of particular interest with world-renowned speakers such as Dr. Valentino Martelli who is also the Director General of the Mediterranean Institute of Hematology (IME Foundation) of Italy, Prof. George Marcoullis, Dean of the School of Medicine of the European University of Cyprus, Dr Androulla Eleftheriou, Executive Director of TIF, and Dr. Michael Angastiniotis, Medical Advisor of TIF. This event has managed to reach a high level of publicity through media coverage.

New collaborations have been initiated and existing ones were strengthened between TIF and representations of Member Countries’ Embassies who were present at this meeting. TIF aims to continue the Ambassadors’ Circle for the next year to strive towards further progress and towards truly supporting and safeguarding the right of every patient for quality healthcare in all of TIF’s member countries.

Enhancing awareness on haemoglobinopathies in schools across Cyprus: TIF’s Pan-Cyprian Drawing Contest for Students of Junior and Senior High Schools...

6 May 2014, Journalist House, Nicosia We were overjoyed to have received so many wonderful creations through TIF’s Pan-Cyprian Drawing Contest for Students of Junior and Senior High Schools, which gave them the opportunity to demonstrate their talent and inspire the world!

For the first time TIF in collaboration with the Ministry of Education and Culture of Cyprus has organised the 1st TIF Student Drawing Contest entitled “Economic Recession: Observe - Join Forces Safeguard Health” for High Schools and

17

Honouring the International Thalassaemia Day through TIF’s successful Press Conference and Blood Donation Camp...

8 May 2014, Famagusta Gate, Nicosia Spreading the message that the responsibility of all of us is to secure the rights of patients with chronic diseases in times of economic crisis, TIF in collaboration with the Nicosia Municipality organized a press conference on the 8th of May. The Nicosia Municipality supported this Press Conference with the active participation of the Mayor, Mr Konstantinos Yiorkatzis.

In this framework, the Cyprus Blood Centre in collaboration with the Nicosia Municipality, have decided to devote the blood drives held on the 8th of May to the Thalassaemia International Federation and to the International Thalassaemia Day.

Medical Journalism at the forefront: sparking a fruitful discussion on health media coverage through TIF’s interactive workshop in Cyprus!

6 May 2014, Journalist House, Nicosia TIF has organised a workshop on medical journalism, held on Tuesday, 6 May 2014, from 10:30 a.m. to 12:30 p.m. at the Journalist House in Nicosia.

This workshop was placed under the auspices of the Union of Cyprus Journalists and the University of Nicosia. The Cyprus News Agency acted as the Communication sponsor.

The aim of the workshop was to discuss and exchange views, experiences, and knowledge on how media should cover health issues.

The main speaker at the event was the distinguished BBC journalist, Dr Patricia McNair. Among the other speakers of this workshop were Dr Demetriou, President of the Pancyprian Medical Association, and Mr Makrides, President of the Union of Cyprus Journalists. Prof Nicos Peristianis, President of the Council of the University of Nicosia acted as the coordinator of this workshop.

This event has reached a high level of publicity through media coverage in Cyprus. Seeing its great success, TIF aims to continue this workshop for the upcoming year.

Additionally, in the context of the press conference, a photography exhibition was held, displaying all the entries of the 1st Photography Contest entitled “Economic Recession: Observe - Join Forces - Safeguard Health” (immediately following).

TIF’s International Photography Contest inspires, challenges, and engages participants from all over the world!

8 May 2014, Famagusta Gate, Nicosia 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 this year’s International Thalassaemia Day. The aim of the contest was to raise awareness about thalassaemia and its subject was based on this year’s theme for

TIFMAGAZINE

Senior High Schools (students from 1217 years of age) in Cyprus. This drawing competition aimed to spread awareness about thalassaemia and sensitize students about patients’ equal rights to a quality healthcare system.

The deadline for the drawing contest was 18 April and TIF has received a total of 92 entries. The evaluation process took place on the 25th of April when the names of the winners were decided.

The first three monetary prizes have been shared to enable as many students as possible to benefit from them.The first prize of 500 Euros was shared between Georgia Iakovou, Natalia Chrysostomou, and Chrystalla Ioannou.The second prize of 300 Euros was given to Katerina Chrysostomou and Constantina Shacola. The Third Prize of 150 Euros was given to Vanessa - Nasso Christou.TIF has also given 25 non-monetary awards and honorary diplomas to students who excelled in the category of Graphic Design for Junior and Senior High Schools.

The names of the winners have been announced during an Award Ceremony on 6 May 2014 at the Journalist House in Nicosia and all the entries for this contest have been displayed at an art exhibition held on the same day.

the 21st International Thalassaemia Day: “Economic Recession: Observe - Join Forces - Safeguard Health”. The deadline of the contest was 18 April and TIF has received a total of 125 entries. The names of the winners were decided after the evaluation process which took place on the 25 of April and were announced through TIF’s social media.

The winners of TIF’s International Photography Contest are: - First Prize of 500$: Hugs - Truong Ngoc Son from Vietnam (ViTA) - Second Prize of 300$: Battling Dengue Fever - Angtuaco Susana from the Philippines - Third Prize of 100$: Joven Perfil - Silvana Andrea Cinquemani from Argentina As mentioned previously, the entries of all the contestants were exhibited at a Press Conference organised by TIF in

OCTOBER 2014 www.thalassaemia.org.cy

18

collaboration with the Nicosia Municipality which was held at the Famagusta Gate in Nicosia on the 8th of May 2014 to honour the International Thalassaemia Day. This activity has managed to spread awareness to the community at large about the rights of patients with Thalassaemia on a global scale.

Involving students across Cyprus through Project 8: School Activities for the “Red Day” 8 May 2014, Nicosia TIF continued the Project 8 for a second year, in the context of the International Thalassaemia Day, aiming to diffuse knowledge and expertise regarding haemoglobinopathies, to all social levels, particularly amongst young people. In these lines,TIF has taken steps to establish the ‘Red Day’ as a part of Project 8 at schools in Cyprus.

This year’s school visits and activities took place at many schools including

the GC School of Careers (28 students), Lykavitos Elementary School (46 students), the English School (180 students), and the Grammar School (90 students) in Cyprus on the 8th of May 2014.

The aim of these visits was to raise awareness about Thalassaemia in students between 10-12 years old. The educational visits included activities targeting to inform students about preventing and treating Thalassaemia. Furthermore, these activities aimed to promote equal access and equal rights to quality health care for all individuals.

The school activities for the day included the following: • Educational presentation about Thalassaemia especially designed for children by TIF. • Projection of the Animated Cartoon “All About Thalassaemia” • Projection of the 8 May Cartoon • Games and Quizzes • Distribution of the cartoon book “All

Consequently, this year’s 8th of May activities reached a very high level of publicity and the goals of the organized activities, which were focused on expanding knowledge and awareness regarding haemoglobin disorders to all age groups and social levels, were significantly achieved. There was a widespread participation from TIF’s member associations. Therefore, TIF, through these activities, has managed to send to the official related stakeholders the message that the Thalassaemia International Federation will overcome any obstacles that interfere with its mission, which is the establishment of equal access to quality health care for all the patients with Thalassaemia.

OCTOBER 2014 www.thalassaemia.org.cy

About Thalassaemia” in English.

The school visits were effectuated by Mr Loizos Pericleous, TIF Board Member, Dr Soteroulla Christou, Head of the Cyprus Thalassaemia Centre, Dr Irene Savvidou, Treating Physician of the Cyprus Thalassaemia Centre, and Mrs Natalia Michaelidou, former president of the Pancyprian Thalassaemia Association.

We truly believe that the above activities have helped the students of these schools to be properly informed about these diseases and to gain knowledge on how to prevent and treat them, while at the same time raising awareness on issues relating to the health system and its patients.

The theme for 2015 will be: ENHANCING PARTNERSHIP TOWARDS PATIENT-CENTRED HEALTH SYSTEMS: GOOD HEALTH ADDS LIFE TO YEARS! Healthcare is changing. Patient needs are changing. Government regulations are changing. With an evolving landscape and overwhelming expectations, the transformation of healthcare systems is becoming all the more necessary to meet the needs of this changing environment.

Providing patient-centred quality healthcare is critical to transforming health. TIF reinitiates its commitment to be involved in a variety of initiatives and collaborations to improve healthcare quality and make it more patient centred.

Collaborations with other healthcare entities, nationally, regionally, and internationally are a vital pillar of TIF’s work.

Through maintaining the quality and standards of TIF’s educational programme, TIF continues to fight for patientcentred healthcare systems that can respond to the historic realignments of our industry and economy. If we can ensure that people are living healthier as well as longer lives, the opportunities will be greater and the costs to society less.

As we react to the changing landscape and create real value for patients and their families, we serve as a model for all healthcare organizations struggling to do the same. The future presents an opportunity for us all, and TIF strives to lead the way.

Stay tuned! The website for next year’s 8th of May Day is underway and it is expected to be ready before the end of this year.

19 TIFMAGAZINE

TIF Publications In proof of the general enthusiasm and zeal which TIF invests in its Educational Programme, we refer to the vast extent of our medical publications as well as to the number and value of our periodical journals, and we are excited to share our publication news with you! TIF’s Publications are designed to reach the entire spectrum of the medical community, including patients, doctors, and medical specialists. Incrediblywell researched and assembled, TIF’s widely-read publications are renowned in the medical community and distributed free-of-charge. They are a great source of insightful content and features relevant to its audience.

TIF caters to both the professional and personal interest of its readers keeping them up to date on all news relevant to haemoglobinopathies.

To date, TIF has published 19 books and pamphlets translated into 22 languages and distributed across over 60 countries. In 2014 alone, requests for TIF publications totaled over 15,000.

OUT NOW!!!

Community Awareness Booklets on α-Thalassaemia, β-Thalassaemia & Sickle Cell Disease (2008). (English) COMING SOON Publications Guidelines for the Clinical Management of Transfusion Dependent Thalassaemia (3rd ed.) (2014). (English)

free of charge All our publications are available as PDF files on our website, completely free of charge.

Translations of Publications • Standards for the Clinical Care of Sickle Cell Disease in Adults (2008) in Greek • A Guide for the Haemoglobinopathy Nurse (2013) in Greek, in Burmese and in Farsi • Emergency Management of Thalassaemia (2012) in Greek • All about thalassaemia Cartoon Booklet (2010) in Bahasa Indonesian • Sickle Cell Disease (2008) in Arabic Translations of Videos • “All about Thalassaemia” Cartoon Animation (2013) is being translated into Bahasa Indonesian, Malaysian, Chinese, Hindi, and Urdu Position Papers Two important position papers are underway: • Position paper on Hepatitis B and C • Position paper on Blood Safety TIF’s Contribution in articles published in Journals We are delighted to announce that TIF contributed to the following publications: • Treatment Strategies, Blood & Marrow Transplantation, (2014) Cambridge Research Centre.

• Under diagnosed, under treated, under estimated, (2014) Middle East Health Journal: Advances in Imaging • The impact of Migrations on the Health Services for Rare Diseases in Europe: The Example of Haemoglobin Disorders, (2013) Scientific World Journal: Updates in Haemoglobinopathies

Magnetic Resonance Imaging (MRI) Information Material underway...

The MRI information material is in the process of being prepared by the members of the MRI consulting group of TIF who have met in Abu Dhabi in October 2013 in the context of the TIF International Congress. Dr Androulla Eleftheriou, TIF Executive Director, with her own expert group at the TIF headquarters is now reviewing the material and is in the process of finalising three types of educational/ informational material: 1.

2.

3.

MRI Information Material or Patients/parents MRI Information Material for medical specialists, haematologists, and paediatricians treating Thalassaemia MRI Information Material for users of the MRI

OCTOBER 2014 www.thalassaemia.org.cy

20

Regional News

Dr. Lawrence Faulkner, Haematologist and Head of the Cure2Children Foundation

Celebrations of the International Thalassaemia Day in India

SEAR Sri Lanka The First Bone Marrow Stem Cell Transplantation (BMT) for Thalassaemia in Sri Lanka was performed on the 4th June 2014, with the participation of the Minister of Health of Sri Lanka. An international team of experts led by Dr. Lawrence Faulkner, Haematologist and Head of the Cure2Children Foundation, and nurses from the UK, together with Sri Lankan medical specialists, were actively involved in this groundbreaking venture designed to treat most of the 3000+ thalassaemia children in Sri Lanka It was performed at a large private hospital where a Memorandum of Understanding with the Cure2Children Foundation was signed to treat six thalassaemic children with BMT, free-of-charge, in exchange for training its staff on this procedure.

Sri Lanka has a free healthcare service system and the“LankaThalassaemia Circle”,the parent organization encompassing nearly 3000 Thalassaemic children and families whose Human Leukocyte Antigens matched (doubly in New York & Germany) children that were treated, is embarking on a venture to establish a free of charge bone marrow transplant centre in Kurunegala , which houses the National Thalassaemia Centre, under the Sri Lankan Ministry of Health.

India The Blood Patients’ Protection Council (BPPC) in Kerala, India, organised an Anti-Pain Rally on the 21st June 2014 as a part of the World Sickle Cell Day commemoration. Many patients with sickle cell disease and thalassaemia as well as their families participated in this rally.

Furthermore, a workshop was organized in honour of the International Thalassaemia Day at the Regional Science Centre and Planetarium, in Calicut, on 8th of May 2014. Hundreds of thalassaemia patients and their families participated in this event with great enthusiasm.

The Minister of Education of Kerala, Mr Abdu Rabb, inaugurated this event, assuring patients and their families that active steps will be taken to help patients affected by thalassaemia and other blood disorders.

Lastly, the International Thalassaemia Day 2014 was also honoured in Nagpur (Maharashtra) by the Thalassaemia Society of Central India. An event was organised on 8 May in Nagpur which consisted of a workshop, and many other festivities including

OCTOBER 2014 www.thalassaemia.org.cy

performances, singing, and dancing by thalassaemia major patients.

EMR Egypt The Egyptian Thalassaemia Association has commemorated the 21st International Thalassaemia Day, by organising the 15th International Thalassaemia Conference on 7-8 May 2014 in Cairo at the Cairo International Conference Centre.

Cairo has provided the unforgettable setting for this meeting and gave a wealth of opportunities to network, to provide all the recent updates on thalassaemia, to exchange scientific knowledge, and to discuss policies for the health care of thalassaemia patients. This conference was attended by international and local scientists.

The key topics of this conference were management and prevention of thalassaemia, gene therapy, stem cell transplantation, iron overload and iron chelation, and pregnancy in female thalassaemic patients.The social program was designed to provide many opportunities to experience the culture, the Red Sea resorts (namely in Hurghada and Sharm El Sheikh) and the beauty of Egypt Iraq The Thalassaemia Association in Niniva in collaboration with the Guidance Unit of the Science College of the University of Mosul, held a workshop entitled “ Thalassaemia: pain and hope” on Monday, 21 April 2014, at the Hall of the Faculty of Science at the University of Mosul.

The workshop was held under the patronage of the Dean of the Faculty of Science of the University of Mosul, Dr. Imad Aldboni.The workshop was attended by a large group of teaching staff and students.

This seminar was held within the context of the prevention program activities of the Faculty of Science and was supported by the Thalassaemia Association in Niniva, in collaboration with the University of Mosul Committee for the prevention of Thalassaemia, Afghanistan Α commemoration event for the International Thalassaemia Day was held on the 10th of May 2014 in Afghanistan by the Meena Health Welfare Organization. The event was attended by representatives of the Ministry of Public Health of

21

Pakistan The Hamza Foundation Welfare Hospital and Blood Transfusion Services (HFWHBTS), in Peshawar, Pakistan organized a daylong blood donation camp at Sarhad University Peshawar, a Walk and a Press Conference/ Seminar at Peshawar Press club to mark the World Blood Donor Day on 14 June, 2014.

Furthermore, the Hamza Foundation Welfare Hospital and Thalassaemia Centre organised an event on 8 May in Pakistan for thalassaemia patients and their parents. This event also included a Marathon, aiming to raise awareness about thalassaemia.

Additionally, the International Thalassaemia Day on 8th May 2014, was also commemorated

by the Pakistan Thalassaemia Welfare Society. A special seminar was arranged for the day, which was attended by Ms Al Jourah Abdullah Alarifi, Wife of the Ambassador of Saudi Arabia in Pakistan, and many world-known health professionals such as Dr. Saeed Elahi, Chairman of Pakistan Red Crescent Society. Local representatives of the WHO were also present at this seminar. Thalassaemia patients and their parents participated in this seminar along with the elite of medical profession and local dignities.

WPR Philippines The Balikatang Thalassaemia Foundation in collaboration with the Galloner’s Club, and the Blood Donors Group, have organised a blood donation fellowship activity on 14 June for thalassaemia patients.

Moreover, the Balikatang Thalassaemia Foundation (BATHA) and the Quezon City Health Department in the Philippines have partnered with Novartis Healthcare Philippines to celebrate the International Thalassaemia Day on 8 May 2014 at the Quezon City Hall. BATHA focused on the following components, for this year’s International Thalassaemia Day, coded through BATHA’s “Thalassaemia alphabet”: A - Awareness and Screening Programs B - Blood Donation Programs C - Iron Chelation Programs D - Diagnostic Monitoring (which includes ferritin, imaging, and other laboratory tests to monitor the status of iron overload) E - Education, Engagement, Empowerment.

TIFMAGAZINE

Afghanistan, the Afghanistan Cricket Board, Patient Associations, Non-Governmental Organisations, thalassaemia patients, their parents, and media to give coverage. The event was composed of two sessions: a Workshop and a blood Drive.

Additionally, the Meena Health Welfare Organization, together with the Afghanistan Cricket Board (ACB), the Afghanistan Child Protection Foundation (ACPF), the Ihsas Social Association (ISA) and other Associations organized an event that gathered management, volunteers, and patients to commemorate the International Thalassaemia Day 2014 on 8 May in Kabul, Afghanistan.

Celebrations of the International Thalassaemia Day in Philippines

Many activities have taken place to cover the above components including recreational activities, workshops, blood donation camps and many more.

Vietnam On the occasion of the International Thalassaemia Day, the Vietnamese Thalassaemia Association (ViTA) and the National Institute of Hematology and Blood Transfusion (NIHBT) held a meeting with over 300 patients/ parents, which has received great attention from the media on 8 May 2014.

At this meeting there were psychology, prenatal diagnostic, and endocrine consulting services to attend to the needs of thalassaemia patients who were present at this event. Parallel to this event, a recreational activity was held for children with thalassaemia where cartoons were displayed to entertain them.

EUR Azerbaijan Positive news from Azerbaijan! According to the official statement of the Ministry of Health of

OCTOBER 2014 www.thalassaemia.org.cy

Participants of the meeting that took place in Vietnam, 8 May 2014

22

Azerbaijan Republic dated 09 July 2014, three patients have undergone bone marrow transplantation at the Azerbaijan Thalassaemia Centre for the first time with the supervision of doctors from the Belorussian Transplantation Centre. The operation was paid for by the state budget. The Minister of Health of Azerbaijan stated that this was a great success for this country. The health status of the patients is stable. We are very happy about this turn of events and we applaud the Ministry of Health of Azerbaijan Republic for this effort.

UK The UK Thalassaemia Society (UKTS) held its Annual General Meeting (AGM) on the 25th February 2014.

We were delighted to welcome four new Trustees to the UKTS Management Committee - Tina Bhagirath, Chris Fassis, Anand Singh Ghattaura and Raj Klair. It is always refreshing for any organisation to have new people with new ideas and perspectives and we look forward to a very productive year!

The Trustees of the Society are (in alphabetical order): - Tina Bhagirath (Committee Member) - George Constantinou (Secretary) - Chris Fassis (Assistant Secretary) - Pany Garibaldinos (Assistant Treasurer) - Anand Singh Ghattaura (Vice President) - Raj Klair (Committee Member) - Romaine Maharaj (Treasurer) - Gabriel Theophanous (President)

Third Sponsor World of the UK Thalassaemia Society, North London 4 May 2014

On behalf of the TIF President and Board of Directors, many sincere congratulations are extended to the President and each and every one of the Trustees of UKTS and many warm wishes for continuing and strengthening the extremely valuable work that UKTS has accomplished in the area of haemoglobinopathies, not only at a national but also at a European, and International level. UKTS is, indeed, a very valued collaborator of TIF. Furthermore, on Sunday 4th May 2014 the UK Thalassaemia Society, held its third sponsored walk at Alexandra Palace, North London. After the success of last year’s walk, it was decided to give participants the option of walking or running double the distance!

More than 100 people participated as well as UKTS Trustees and their family members. The event raised funds which will be a great help in supporting the Association’s work over the next year.

OCTOBER 2014 www.thalassaemia.org.cy

Greece On the occasion of the World Blood Donor Day, bicycle marathons were co-organized on 15 June 2014 by Novartis Hellas, in collaboration with the Thalassaemia Associations in Athens, Thessaloniki, Volos, Heraklion and Ioannina with the aim of creating awareness on Thalassaemia and Voluntary Blood Donation.

The bicycle marathons were open to the public in the 5 aforementioned cities of Greece. These marathons were held under the slogan “The ride is better with friends” and aimed to raise awareness about the disease and to inform citizens about the benefits of donating blood.

Furthermore, the Greek Thalassaemia and Sickle Cell Association in Larissa organized an Informative Meeting on Sickle Cell Disease on 31 May, on the occasion of this year’s World Sickle Cell Day, on 19 June.The meeting was held in the hotel Larissa Imperial, in Larissa, a city located in central Greece.

What is more, the Association forVoluntary Blood Donation and Friends of Thalassaemia Patients “Pigi Zois” (Life Spring) and the Association of Patients and Parents of Children with Thalassaemia in Thessaloniki organized an event on Thursday, 8 May 2014, to honour the International Thalassaemia Day at the pedestrian path between St Sophia Street and Tsimiskes Street in Thessaloniki.

AMR United States of America (USA) Car enthusiasts, take note! Cooley’s Anaemia Foundation (CAF) has organised “Suffolk Chapter’s 3rd Classic Car Show “Test & Tune”! The Suffolk chapter of CAF has held this event on 13 September 2014 in Hempstead, New York in conjunction with Long Island Drag Racing.

23

Congratulations to Dr Perla Eleftheriou!

We are delighted to express our warmest congratulations and pass on our most sincere greetings to Dr. Perla Eleftheriou, Medical Advisor of TIF, for having obtained the honorific title: Fellow of the Royal College of Pathologists (FRCP) of the Royal College of Pathologists of the United Kingdom. This title is the highest academic rank at the Consultant level in the specialty of haematology. The official awarding was held on 4 July, 2014.

We are extremely proud of the success of Dr. Perla Eleftheriou who has excelled in the field of haematology in the UK. We welcome this new scientist to unite with other expert haematologists who have excelled both in Cyprus and abroad. We hope that TIF will continue to have her as a friend and as a valuable collaborator.

Congratulations are in order for Dr Stephan Lobitz!

An esteemed medical specialist in Germany and valuable collaborator of TIF, Dr Stephan Lobitz, has successfully completed the MSc Course in Haemoglobinopathies at the University College London (UCL) and succeeded in the publication of two important articles, which have been accepted and published in well-known journals: • S. Lobitz, C. Frömmel et al., Incidence of sickle cell disease in an unselected cohort of neonates born in Berlin, Germany, European Journal of Human Genetics (2014) vol. 22, 1051-1053 • S. Lobitz C. Frömmel, et al., Newborn Screening for Sickle Cell Disease: Technical and Legal Aspects of a German Pilot Study with 38,220 Participants, BioMed Research International (2014) vol. 2014, 1-10

SEND US YOUR NEWS If you have important news you want to share with the world thalassaemia community, let us know!

Like our page on Facebook and share it with your friends!

Honouring the global haematology community at the 55th American Society of Haematology (ASH) Annual Meeting and Exposition...

We would like to applaud the outstanding achievements of some of the most prominent figures in the field of medicine, and more specifically in the field of haematology who were awarded for their contribution in research, their trailblazing scientific and clinical studies, and their exceptional scientific accomplishments: Wallace H. Coulter Award for Lifetime Achievement in Haematology ASH presented the Society’s highest honor, the 2013 Wallace H. Coulter Award for Lifetime Achievement in Hematology, to Professor Sir David Weatherall of the University of Oxford for his more than 50-year career in haematology combining seminal discoveries, visionary translational research leadership, and a passion for global health initiatives that have together helped to improve clinical care for thousands of individuals throughout the developing world.

E. Donnall Thomas Lecture & Prize Katherine A. High of The Children’s Hospital of Philadelphia (CHOP) was honoured for her exemplary scientific and clinical research in the bleeding disorder haemophilia when she received the 2013 E. Donnall Thomas Prize by the Society. An internationally recognized expert in the molecular basis of haemophilia, Dr High has developed novel approaches to correcting haemophilia with gene therapy in studies.

H. Ham - Louis R. Wasserman Lecture Award The Society presented the Thomas H. Ham - Louis R. Wasserman Lecture Award to Dr Clara Camaschella, of the Università Vita-Salute San Raffaele in Italy in recognition of major contributions related to the understanding of iron homeostasis and its clinical importance.

OCTOBER 2014 www.thalassaemia.org.cy

TIFMAGAZINE

Congratulations!

24

OCTOBER 2014 www.thalassaemia.org.cy

25

Each day is a passage to all we have been through and a passage to that which is yet to come!

By Sohaib Azam

Whenever I go on Thalassaemia awareness talk shows, I introduce myself like this: I am Sohaib Azam and I am a Thalassaemic. I am 31 years old, and I have lived most of my life in the Middle East, and for the last 17 years in Dubai. I am an MBA graduate, and I have been working as a Project Manager at a leading nutrition company.

of my greatest achievements during university was winning the competition for the best public speaker of all the country’s Universities. My academic excellence and extracurricular activities were these achievements.

Getting a job wasn’t any easier. Many companies asked me how many times I would fall sick and how many days off I would need for my treatment. They asked these questions to see if I would be able to cope with the work role that I would be hired for. However, nowadays many people know about thalassaemia and employers recognize that it is not a problem provided patients manage their health appropriately.

Sometimes, it is difficult to work long hours due to low haemoglobin levels, especially when it is time to get a transfusion. But it is important to keep faith strong and not to give up.

My life started as a normal child who was born to parents who were both doctors. When I turned almost two, a sudden atomic bomb dropped onto our head which was the great news of my diagnosis with BetaThalassaemia Major. Of course it was catastrophic; my parents did not know how they would ensure to get me blood transfusions for the rest of my life! This question was in their minds for a long time. However, my parents And now for the toughest stage of life: Getting had enough faith and strength to get me through this. married!

So here I am today because of the hardships that they went through to raise me and because they stood by This is a very crucial part of any thalassaemic my side. patient’s life. I must admit, it is very hard for parents who never had thalassaemia in their family, to agree We all go through ups and downs in life, no life is for child to marry someone with this disease. But perfect. Growing up, my parents made me go through all theretheir are people who understand enough to know that it the hardships that a normal child would go through. isn’t a challen ge if one partner is genetically clean.

It was difficult at the beginning as a lot of time and effort had to be invested in me by my parents. I still In my case, I met a girl through a common remember that my mother used to stay with me at the acquaint ance and then we became good friends. Later hospital when there was no day care transfusion. What on, we decided together. For our marriage proposal, is more, she couldn’t spend much quality time with my I did not invitetomybewife to a formal restaurant. Instead, only sister who is just a year older than me. My father I had invited my father and also had to babysit and take the day off from work to with me and stay with memother in law to have lunch take care of my sister while I stayed in the hospital for care centre, to see how the at the Thalassaemia day transfusions. My mother used to sleep on the hard floor know what is going on. This treatment is so that they way, they felt better about with her cloak as her bed sheet. making the decision to give their daughter to me. My optimism towards life has always helped me through When I went to secondary school, I was among the difficult times. That’s why thalassaemia patients top ten students of the school. My parents’ efforts paid shouldn’t lose hope. off and my very own thirst to learn proved to this world that I wasn’t any less of a normal person. However, I moment, I am happily married. Recently, I often had difficulty in maintaining attendance when it wentAtfortheholiday Switzerland where me and my wife was time for my transfusions, but, with God’s blessings, were offered 50%todiscount I managed to pass each grade every year with flying tickets as I tend to look at a train station for the younger than what my age is. colours and climbed up the ladder of success through As we all know, most thalass aemics look younger than my will power and determination. their actual age and this is an added bonus to me!

As I grew up, I went to University, where I graduated never ask God: “WHY ME?” I always thank God majoring in Information Technology. Then, I subsequently thatI He “CHOSE ME!” completed an MBA course. Of course, it was a lot of hard work and combined with the problems of Thalassaemia Thalassaemia became the reason for my strength, it was quite difficult. University life was also hectic optimism having to take Desferal (Deferoxamine) injections. One the world!, and gave me the chance to tell my story to

I feel blessed for having Thalass aemia!

OCTOBER 2014 www.thalassaemia.org.cy

TIFMAGAZINE

Patient’s Story

26

Other News Non-Transfusion Dependent Thalassaemias (NTDT) Voices: Together we stand up for them!

Exciting News! It’s our pleasure to share with you the toolkit for NonTransfusion Dependent Thalassaemias (NTDT) Voices,a project which was launched during the 13th International Conference on Thalassaemia and the Haemoglobinopathies that took place in October 2013, through which TIF aims to inform, educate and raise awareness about non-transfusion dependent thalassaemias, the clinical significance of which has been underrecognised by the medical/research and health policy-making communities alike.

We truly hope that the range of tools that have been designed to meet the patients’ and the community’s needs, will inspire, move, educate and raise awareness about NTDTs under the motto “ Together we stand up for them!”

Visit our website for more information at www.thalassaemia.org.cy/news-archive.shtml

Pioneering new project: the THALAMOSS Project TheTHALAMOSS Project (THALAssaemia MOdular Stratification System for personalized therapy of beta-thalassaemia) is another ambitious project in which TIF is involved. This project is cofounded by the European Union’s 7th Framework Programme for Research, Technological Development and Demonstration. THALAMOSS is aimed at the development of universal sets of markers and techniques for the stratification of β-thalassaemia patients into treatment subgroups.

In this context, TIF participated in the 3rd THALAMOSS General Assembly Meeting which had been organized between 25-27 June 2014 in Nicosia, Cyprus. The aim of the meeting was to discuss clinical complications in β-thalassaemia and sickle cell disease, to update on the collection of patient data, as well as to monitor the progress and planning of the THALAMOSS project .

The THALAMOSS tools and technologies are anticipated to facilitate identification of novel

OCTOBER 2014 www.thalassaemia.org.cy

diagnostic tests, drugs and treatments specific to patient subgroups and guide conventional and novel therapeutic approaches for β- thalassaemia, including personalised medical treatments.

Screening and Prevention Programmes for Haemoglobinopathies Assessment (SPHerA) We are excited to inform you of the proposal entitled “Screening and Prevention Programmes for Haemoglobinopathies Assessment (SPHerA)”.

This project has made it to Phase II of the EU’s Horizon 2020 Funding Scheme, under the Research and Innovation Actions. The consortium for this project is comprised of partners from 15 different countries in Europe and abroad and TIF is participating as a Work Package Leader.

SPHerA Project Objectives The main objective of the proposed project is to assess the effectiveness of the existing screening and prevention programmes for haemoglobinopathies in all European countries.

More specifically, this project aims to: • Assess and compare existing screening and prevention programmes to produce a full Health Technology Assessment (HTA) report, investigating available evidence on the effectiveness and cost effectiveness of the assessed programmes, and including the evaluation of relevant health outcomes, quality of life (QoL), and their ethical considerations.

• Monitor the current and future burden of haemoglobinopathies in Europe and neighbouring non-EU affected countries which are also impacted by migration, in order to support and implement the most appropriate EU-wide and national public health policies.

• Provide a roadmap towards the alignment of surveyed programmes with comparable socioeconomic parameters and the minimisation of the combined haemoglobinopathy-related health burden in EU. This way, the best prevention strategies will lead to the reduction of the impact of haemoglobinopathies and additional inappropriate costs for the European Health Systems will be avoided.

27

The Registry is the first step of a major project to ensure that all Australians with a haemoglobinopathy continue to receive the best possible care. Further details about this project will follow in our next TIF Magazine.

Prospects for aligning action through the e-ENERCA Project We are pleased to inform you that TIF has participated in the ENERCA Executive Committee Meeting, represented by TIF medical advisor, Dr Michael Angastiniotis as well as Prof Christos Schizas of the Department of Computer Science of the University of Cyprus. The ENERCA Executive Committee Meeting was held in Barcelona, Spain, on 30 April 2014 with the purpose of discussing 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.

At this stage of the e-ENERCA project and after TIF’s participation in the ENERCA Executive Committee Meeting, TIF has already presented its first deliverable concerning the legal and ethical requirements of the European Community pertaining to electronic registries for patients and the implications for sharing such information between centres and countries. Currently the data is in the hands of computer programmers of the University of Cyprus who are working on the project. This registry will not only include thalassaemia but also sickle cell disease and Rare Anaemias.

TIF continues to promote the e-ENERCA project, since the ultimate aim is to achieve through electronic means, access to the same level of services across Europe, independently from the country of practice and the origin of the patient. The main objective of TIF’s involvement is the development of a European Registry for Rare Anaemias including thalassaemia.

Work in progress: Mobile Application programme Thal Health Web App The countdown is underway for submitting a project proposal for the creation of TIF’s mobile application program with the aim of greatly aiding and improving the lives of people with thalassaemia.

This is a clever application that will be designed specifically for the thalassaemia community with the depth of knowledge required to add true value to the community. The Thal Health Web App will help to connect the members of the thalassaemia community to one another and to those that care for them. The application will be designed with the active input from the thalassaemia community to ensure value, ease of use and applicability.

The envisioned program is expected to be delivered by the end of 2015 via mobile app, and will reach millions of people around the world living with thalassaemia.

OCTOBER 2014 www.thalassaemia.org.cy

TIFMAGAZINE

To Australia and beyond...

TIF has initiated a new endeavour, a motivational new project called the Australian Blood Project. Australia has a significant number of thalassaemia-affected people, which is increasing as migration patterns change. However, the exact number of affected people is unknown as well as the severity of each case.

Little is known about their long-term complications and outcomes. Although different centres currently manage the same disease, they do not follow a common protocol, and there is a need to determine which strategies work best for patients.

In this context, we are delighted to share with you that TIF has been invited to join a grant application which is to be submitted to the National Health and Medical Research Council (NHMRC) of Australia, with the aim of creating a national haemoglobinopathy electronic registry in this country.

The invitation was sent by a group of haematologists led by the Head of the Transfusion Research Unit at the Department of Epidemiology and Preventive Medicine (DEPM) in Australia, Dr Erica Wood.

The aims of the national Haemoglobinopathies electronic registry are to: • Provide an important framework for future research to improve patient care • Be used to help to plan health care services for the future • Bring together a network of Australian health care professionals with a special interest in haemoglobinopathies, for sharing ideas.

28

TIF’s next big mission: establishing Reference Centres The project on the reference centres is on the way, having identified and compiled considerable information from treating centres across the world which is to be placed in a database and classified so that the project will be tailored to the needs of each centre within and across countries.

More specifically, for this project a questionnaire has been sent and distributed to approximately 400 treatment centres with questions pertaining to the services that they provide to patients. The concept is to use this information to create a database and to classify centres so that we can identify the strengths and the needs of each centre across the world.

A vision turned into action: MSc e-Course programme in Thalassaemia and Sickle Cell Anaemia TIF is thrilled to announce that it is in the process of establishing an MSc e-course programme in Thalassaemia and Sickle Cell Anaemia through the University of Nicosia, aiming to offer a postgraduate academic course to health professionals in the field of Haemoglobinopathies. More details about this programme will be announced in future TIF Magazines.

Ferriscan Project TIF in collaboration with a Diagnostic Centre in Cyprus supports the monitoring of iron overload with Ferriscan technology for a certain number of thalassaemia patients in Cyprus as per the guidelines of the project. More specifically, TIF is financially supporting this examination for 40 patients as prescribed by their treating physicians.

The first patients have already been referred to the collaborating MRI Centre in Nicosia. We anticipate the completion of this initiative shortly.

Renzo Galanello Fellowship Programme, with the support of Novartis We are pleased to inform you that following a thorough assessment process, the Selection Committee selected two candidates out of 18 applications from 11 countries, received for the Renzo Galanello Fellowship Programme: one from Bangladesh and one from Nigeria.

OCTOBER 2014 www.thalassaemia.org.cy

Even though all applications met the academic criteria for the fellowship, the selection has been made according to the need of education in each country.

Priority, therefore, only for the year 2014, has been given to applicants from countries that are lacking specialists in the field. The training course of these medical experts has already begun in September 2014.

The use of generic iron chelation drugs in patients with Hb Disorders - A first study of evaluation of their case” Given the increasing trend of the use of generic drugs in thalassaemia treatment,TIF has embarked on a new project to address the rate of use of generic drugs in the iron chelation treatment of thalassaemia and to see how they are used and authorised in different countries as well as how their safety of effectiveness is measured. 10 EU and 10 countries outside the EU in the Middle East and Asia will be included and the drugs to be investigated in their generic forms are Desferrioxamine, Deferiprone, and Deferasirox. In the same study, we aim to obtain information on: • the health care system in the context of reimbursement policies • the number of patients receiving generic iron chelation • the strengthening of patients’/ parents’ education on the benefits but most importantly on the risks and side effects of generic drugs, as well as the need to know, to report, and to share knowledge and experience • the existence of and adherence to national pharmacovigilance programmes The study will be divided into three phases: I, II and III and will include, amongst others, United Kingdom, Italy, Cyprus, Bulgaria, Greece, Iran, Lebanon, United Arab Emirates , Thailand, India, and Pakistan.

The enquiry, on which TIF is embarking, aims to bring to light the issues of efficacy, safety and tolerability of existing generics while first establishing which generics are circulating internationally, to what extent they are used compared to brand names and to what extent, if at all, reporting or studies on adverse events, reactions or side effects potentially or likely related to the use of generic drugs have to take place. Very important in such an investigation is the opinion of the patients who are the main users and the most concerned on the effects of these drugs on their wellbeing.

29

The TIF video sharing platform, designed with a primary focus of connecting thalassaemia patients across the world, constitutes one of TIF’s most successful projects. The aim of this project was to initiate a two way interaction between TIF and patients on a global Youtube platform, allowing patients to submit their own videos on a revamped TIF YouTube page and to provide them with an incentive for uploading videos through a contest which will build up to World Thalassaemia Day with the publication of the winning videos! We are delighted to have encouraged patients, parents and their friends and family to take a step forward with us!

We are overjoyed to share with you that we have received a great number of inspiring videos for TIF’s video challenge that went beyond our anticipations!

The deadline for the contest has ended on the 25th of October and the selection procedure of the participants that have entered the video sharing platform competition organised by TIF is still in process. TIF aims to continue this effort in Europe in 2015.

So stay tuned as the winning entries will be selected, announced, and shared soon with you on our social media and in the next issue of our TIF magazine!

We would like to thank all of our contestants for their participation. We were delighted to have received such motivational and touching videos!

All the entries of all the contestants will be displayed during the 4th Pan-European Conference on Haemoglobinopathies and Rare Anaemias in Athens (07-09 November 2014).

To view the videos visit the following link: www.thalassaemia.org.cy/video-sharing-platform.shtml

OCTOBER 2014 www.thalassaemia.org.cy

TIFMAGAZINE

Revolutionary new project: TIF video sharing platform

30

Obituaries Mr Muhammad Imran It is with utmost grief that we inform you of the sad demise of our beloved former TIF Board Member, Mr. Muhammad Imran, and Executive Committee Member of the Thalassaemia Society of Pakistan.

Born in 1984, he has worked at the Thalassaemia Day Care Centre in Sir Ganga Ram Hospital in Lahore, Pakistan for many years. He had been a great advocate for the cause of thalassaemia and worked towards motivating patients and parents to play an active role in combating their disease.

He participated as a volunteer in awareness and fundraising walks for thalassaemia patients in Pakistan, setting the record for being the first thalassaemia patient to complete many of these walks. In 2006, he was elected as a TIF Board Member.

He was the adopted son of Dr Yasmin Raashid, Secretary General of the Thalassaemia Federation of Pakistan, a loving husband, and a proud father of a young daughter. We have lost an outstanding human being who was dear to all of us.

We pray for God to give his family strength and courage to bear this irreparable loss. May his soul rest in peace.

OCTOBER 2014 www.thalassaemia.org.cy

31

EVENT

VENUE

01-03 October

17 European Health Forum Gastein (EHFG) 2014 -Electing Health: “The Europe we want!

Interactive Workshop for the Cyprus National Health Plan International Conference for Rare Diseases and Orphan Drugs (ICORD): “Societal value of Prevention, Diagnosis and Treatment of Rare Diseases” 8th Congress of Asia Pacific Society on Thrombosis and Hemostasis (APSTH) and National Scientific Conference of Haematology and Blood Transfusion Viral Hepatitis Congress 2014 TIF Workshop in China European Organisation for Rare Diseases (EURORDIS) - Council of National Alliances (CAN) Meeting TIF Workshop and Official Delegation Visit in Iran TIF Workshop in Morocco European Network for Rare and Congenital Anaemias (ENERCA) Executive Meeting

Bad Hofgastein, Austria

04 October 07-09 October

08-11 October

09-11 October 11-12 October 13-14 October

15-17 October 20-21 October 22 October

th

November Capacity Building Workshop of the Pancyprian

Thalassaemia Association 06-07 November 10th European Healthcare Fraud and Corruption Conference 07-09 November 4th Pan-European Conference on Haemoglobinopathies

and Rare Anaemias 20-23 November Conference on Thalassaemia and Haemoglobinopathies

of the Thalassaemia Federation of Turkey 22-26 November TIF Workshop in India

OCTOBER

Nicosia, Cyprus Ede, Netherlands

Hanoi, Vietnam

Frankfurt, Germany Guilin, China Paris, France Tehran, Iran Rabat, Morocco Barcelona, Spain

Nicosia, Cyprus Athens, Greece Athens, Greece Çeşme-Izmir (Smyrna), Turkey

Bhopal-Madhya Pradesh, India

OCTOBER 2014 www.thalassaemia.org.cy

NOVEMBER

TIFMAGAZINE

DATE

32

Medical Corner New hope, new advances: towards the total cure...

Brief update of bluebird bio’s clinical progress We are pleased to introduce bluebird bio to the global Thalassaemia International Federation network, and to provide this brief update of our clinical progress and activity to-date. bluebird bio is a clinical-stage biotechnology company committed to developing potentially transformative gene therapies for several severe diseases, including β-thalassaemia and sickle cell disease.

The gene therapy process involves using lentiviral vectors to insert genetic material into patients’ own hematopoietic stem cells ex vivo, then re-introducing the genetically modified cells to patients via a standard autologous transplantation.

Initial efficacy and safety data from the first two subjects with β-thalassaemia major treated in the HGB-205 study were presented at the European Hematology Association’s 19th Annual Congress on June 14, 2014. The data were presented by lead Principal Investigator Dr. Marina Cavazzana, Research Director at the Centre for Clinical Research in Biotherapy, Necker Hospital, Paris. Some highlights are provided below, as well as a graphic illustration of key data: • The data illustrate that early and high production of βA-T87Q globin has resulted in rapid transfusionindependence at near-normal Hb levels in both patients, similar to what might be expected from Promising, early, clinical proof-of-concept a successful allogeneic transplant. results for this approach were demonstrated in one • Subject 1 was producing 6.6 g/dL of βA-T87Q patient with β-thalassaemia major and reported -globin at 4.5 months’ post-treatment, while

by Cavazzano, Leboulch, et al in Nature in 2010.

Subject 2 was producing 4.2 g/dL of βA-T87Q That patient remains transfusion-free more than 5 -globin at 2 months. years post-treatment. No toxicities related to the • Neither patient experienced any gene-therapydrug product have been reported. related adverse events. bluebird bio’s current LentiGlobin® BB305 • These preliminary data provide proof of principle Drug Product candidate builds on that pioneering that early transfusion independence can be work. The amino acid substitution (βA-T87Q) was achieved with gene therapy in patients with preserved in the beta-globin gene from the β-thalassaemia major with β0/ βE genotype. earlier study that confers important anti-sickling • The new lentiviral vector and improved properties to help address sickle cell disease. This manufacturing process produce superior substitution also allows to accurately measure transduction efficiency as compared with the the amount of therapeutic globin being produced. earlier vector used.

However, now an improved version of the original lentiviral vector and manufacturing process are also used to increase the efficiency of gene transfer to stem cells.

Three clinical studies have been supported to explore the efficacy and safety of the LentiGlobin® BB305 Drug Product in patients with β-thalassaemia major and severe sickle cell disease. The studies are described in the table below. More detailed information about each study is available at the TIF website at www.thalassaemia.org.cy and at For more information, we encourage contacting bluebird www.ClinicalTrials.gov. bio at clinicaltrials@bluebirdbio.com or at patient. advocacy@bluebirdbio.com

OCTOBER 2014 www.thalassaemia.org.cy

33 New Drugs are now the leading treatment for Hepatitis C

GT1 30%; GT2 and GT3 56.6% and GT4 29.8%.

The percentage of responders improved significantly by the end of the 1990’s, with the discovery and use of Ribavirin and the ‘upgraded’ form of α-interferon, known as pegylated α-interferon (PEG-IFN), which caused the percentage of responders to increase to 45%, 87%, 77% and 56% for GT 1, 2, 3 and 4, respectively.

However, the first true revolution as far as the clinical management of Hepatitis C, genotype 1 which is considered to be the most resistant and by extrapolation the most difficult genotype to cure -, came in 2011 with the discovery and licensing of two new drugs, namely of the Boceprevir (BOC) and of the Telaprevir (TVR) - first Generation Directacting Antiviral [DAA]. Their use increased the percentage of responders of patients with GT1 of HC to 63%-79%, using either of them (BOC or TVR) in combination with Ribavirin and pegylated interferon in a treble combination therapeutic scheme (BOC+RB+PegIFN).

diagram 1

diagram 2

Nevertheless, many and significant side effects and other medical complications were recorded using the above-mentioned schemes, which in conjunction with the high costs of these drugs, rendered the inclusion and use of these drugs (BOC and TVR) in therapeutic protocols extremely restricted. Moreover, until today, there is a great number of countries that did not approve the licensing of these drugs, and others where special criteria have been put in place for their use (BOC and TVR ).

To the great relief of the medical and the patients’ communities alike, the advent, immediately after 2011,of two new drugs in 2014, namely that of Sofosbuvir Protease Inhibitor (SBV, January 2014) and Simeprevir Protease Inhibitor (SPV, May 2014), concomitantly earmarked the second revolution in the therapy of HCV - see diagram 1.

Using the former in combination with the already existing Ribavirin (with or without the use of pegylated α-interferon) the percentage of responders with genotypes GT1,2,3 and 4 reached to 90%, 93% - 97%, 93% and 96%, respectively. Using the latter (SPV) in combination with ribavirin (with or without the use of pegylated α-interferon), the percentage of responders for genotype 1 reached 79-80%, while for genotype 4 reached 88%.

Moreover, when the two new drugs (SBV and SPV) are used in combination, it was shown that the use of interferon (with all its numerous and well-known undesirable side effects) could be excluded from the therapeutic protocols.

This new protocol has tremendously increased the percentage of responders of genotype 1 (1b & 1a) to nearly 100%, showing an extremely improved safety profile, that is with exceptionally minimised side effects.

In addition, it is worth-mentioning that a series other new drugs, namely BMS325, Asunaprevir, Ledipasvir, ABT450/ r+Ombitasvir+Dasabuvir, Faldaprevir, Daclatasvir, whose approval and licensing by the relevant organisations such as the European Medicine Agency (EMA), and/ or the Food and Drug Administration (FDA), are in the pipeline, are now entering Phase III of their respective Clinical trials.

These new drugs are promising to have high percentages of response and cure, as well as the mildest side effects’ profile. In light of these facts, it stands to reason that the relevant guidelines will be continuously updated, at regular intervals, in order to include their use in therapeutic protocols, once their approval and licensing are granted (Diagram 2).

Global Alert and Response: Ebola Virus Disease

TIFMAGAZINE

The last two decades have been earmarked by rigorous research and the discovery and development of new drugs for the management of Hepatitis C (HC). In the beginning, the percentage of responders to α -interferon, the first and only drug at the doctors’ disposal for many years, was relatively low for all the four (4) main and most frequent genotypes (GT) of Hepatitis C (GT1, GT2, GT3, GT4), as shown below:

WHO has issued a roadmap to guide and coordinate the international response to the outbreak of the Ebola virus disease. The roadmap aims to stop ongoing Ebola transmission worldwide within 6-9 months while rapidly managing the consequences of any further international spread. It also recognises the need to address, in parallel, the outbreak’s broader socioeconomic impact.

Ebola virus disease is a severe, often fatal illness and one of the world’s most virulent diseases.

The infection is transmitted by direct contact with the blood, body fluids and tissues of infected animals or people. During an outbreak, people at higher risk of infection are health workers, family members and others in close contact with sick people and anyone who has died from Ebola. People who are ill with the disease need to receive expert care in appropriate facilities.

Ebola outbreaks can devastate families and communities, but the infection can be controlled through the use of recommended protective measures. WHO is coordinating a global response to the latest outbreaks in Africa.

To protect yourself, your family, and your community from the Ebola Virus Disease transmission, immediately report to the nearest health facility if you develop symptoms indicative of Ebola Virus Disease, including high fever, body aches, joint pain, vomiting, diarrhoea, or haemorrhaging. Isolation and professional clinical treatment increase a person’s chance of survival.

To read more about WHO’s road maps and the latest news about the Ebola virus, visit the following link: www.who.int/csr/disease/ebola/en/

OCTOBER 2014 www.thalassaemia.org.cy

35 TIFMAGAZINE

OCTOBER 2014 www.thalassaemia.org.cy

Season’s Greetings

We are approaching the end of 2014, a year full of challenges and rich in achievements! We hope this year-end will be an opportunity for each of us to take good resolutions for 2015 and beyond.

We would like to wish you and your families, happy end-of-year celebrations, and a new year full of health, happiness and personal and professional achievements!