TIF Magazine - issue 66 by Thalassaemia International Federation (TIF)

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TIFâ&#x20AC;&#x2122;s International Conference 2016 14th International Conference on Thalassaemia & Haemoglobinopathies & 16th TIF International Conference for Parents & Patients

Bids are now open for hosting TIFâ&#x20AC;&#x2122;s International conference For more information, visit our website: www.thalassaemia.org.cy MAY 2015 www.thalassaemia.org.cy

Dear Reader, Welcome to issue 66 of TIF Magazine, the first issue of 2015. Already, this year has been landmarked by many exciting activities for TIF, and as it unravels, a new chapter slowly unfolds whilst we embrace our journey and set out for new endeavours! During this time of new opportunities, we look forward to sharing challenges, concerns, problems, and accomplishments, with you, as we hope to shape the future of our new chapter together! This issue of TIF Magazine is dedicated to a wonderful friend, supporter and visionary of our global thalassaemia family, Mr Ioannis Levidiotis, a great figure in the field of thalassaemia in Greece, but also globally, who sadly passed away on 22 December 2014. Mr Levidiotis was a prominent figure who translated TIF’s magazine to Greek for distribution to the Greek-speaking thalassaemia patients and other interested readers in both Greece and Cyprus. He was also a man with immense courage and a fighting spirit, who stood proudly throughout the years in the face of challenges, advocating for the safeguarding of the rights of patients with thalassaemia mainly at the national level in Greece, but serving as an example and role model on a global basis.This issue is a tribute to him, to pay deep and sincere appreciation for his invaluable support throughout the years, contributing greatly to TIF’s activities and to the accomplishment of its mission. Our magazine also proudly brings to you the great news of TIF’s receiving the “Dr Lee Jong-Wook Memorial Prize for Public Health”, granted by the World Health Organisation (W.H.O.). This prize is awarded to people or institutions, governmental or nongovernmental organizations, which have made an outstanding contribution to public health. Such is the stature of the Dr Lee Jong-Wook Memorial Prize for Public Health that we feel honoured, humbled and deeply moved that TIF’s work has been recognised and found worthy of this high distinction. The prize reaffirms our conviction that with hard work, courage and

determination our dreams are within our reach and that no challenge is big enough to deter us from reaching our goal: the provision of quality healthcare to patients all around the world. However, with this prize comes the great responsibility to ceaselessly continue and strengthen our work and services, always placing the patient’s needs in the centre of our attention. Strengthening our collaboration networks, accelerating our efforts to provide all patients with appropriate, quality healthcare and developing effective prevention strategies in collaboration with national health authorities and international dignitaries, still remain key priorities to achieving our mission. The past few months have seen the organisation by TIF of an abundance of delegation visits in many countries including India, China, Egypt, Vietnam, Algeria and Morocco, as well as the participation in and support of meetings and conferences in many parts of the world including Switzerland, Germany, United Kingdom and Spain. The special focus of this issue is the organisation of the 2nd Pan Asian Conference on Haemoglobinopathies, between 25 and 27 September 2015, in Hanoi, Vietnam, which is part of the key regional activities of TIF. Organised in collaboration with the Vietnamese Thalassaemia Association (ViTA) and the National Institute of Hematology & Blood Transfusion (NIHBT), this event will be even grander than its predecessor, providing an excellent forum for discussion and interaction. You can find more information about this event on Tif’s website, on page 19 and soon on our new conference website. With the ‘8th of May’ 2015 theme “Enhancing Partnership Towards Patient-Centred Health Systems: Good Health Adds Life to Years”, we join hands to react to the changing landscape and create real value for patients, promoting their rights for better health and quality of life. Patients and national, regional and international official services and health institutions, each separately and all collectively, have a veryimportant role to play: to support, collaborate and act in solidarity, because the driving forces of any

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Foreword from TIF’s President and Executive Director

Panos Englezos President

Dr. Androulla Eleftheriou

state are its healthy and productive citizens. Within the following pages you will read about TIF’s exciting plans for this year’s International Thalassaemia Day, which include the provision of an ‘8th of May’ toolkit, available to our Members to use for their activities on this Day. TIF has also scheduled a number of awareness-raising activities such as a Musical Evening and Gala Dinner, a photography competition, a press conference, and many more. The major deliverables and outcomes of the 4th Pan-European Conference on Haemoglobinopathies and Rare Anaemias, which was held from 7 to 9 November 2014 in Athens, Greece, are also accounted for in this issue. These, range from the delivery of TIF’s Consensus Papers to EU officials to the launching of our new publication, the 3rd edition of the Guidelines for the Clinical Management of Transfusion Dependent Thalassaemia (TDT), and two other translated versions of existing books. The scientific but also the patients’ programme covered an amazing range of topics which, indeed, has truly made this Conference an unforgettable learning experience! Many projects in 2015 are successfully ending or entering a new phase. As part of our Educational Programme, TIF is delighted to announce that it is currently in the process of establishing an MSc course programme in Inherited Haemoglobin Disorders - Thalassaemia and Sickle Cell Syndromes (Blended) - in collaboration with the University of Nicosia, Cyprus. Its aim is to offer a postgraduate academic course to health professionals in the field of haemoglobinopathies. Our “TIF Renzo Galanello Fellowship”,a programme established in collaboration with the Whittington Hospital in London, UK, and with the support of Novartis Pharma, is also in the

process of recruiting new candidates for the second year of its existence. TIF has also entered into the digital age through compelling projects, such as the development of a patients’ mobile application, ThaliMe, and the revolutionary Video Challenge platform. TIF views the use of mobile technology imperative to patient empowerment and outreach, but also a critical component of the overall thalassaemia care. Our new application, ThaliMe, has already been launched in the Apple store and is available for downloading. A second application is in the process of being developed which will provide a digital bookshelf for TIF’s Publications. Furthermore, our Video Challenge Competition has culminated with the selection of the three winning submissions, which we proudly announce in this issue of our magazine (page 31). Another aspect of TIF’s Educational Programme is its extensive range of educational materials, which include the publication of books and other printed material, the majority of which have so far been translated into up to 20 different languages. You will find information on all these and many other developments in this issue of our magazine. We are very proud of the immense progress that is being made on so many fronts, and even more so because this progress is the result of cooperation on every level with the national thalassaemia associations, governments, medical practitioners and the industry. However, our driving force is the strength, determination and courage of our patients who inspire, guide and motivate us each day as we move on to address numerous challenges and bridge many gaps around the world!

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Panos Englezos

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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 Ramli Mohd Yunus Robert (Bob) Ficarra Saeed Jafaar Al-Awadhi Gabriel Theophanous

Foreword TIF’s Activities Around The World International Collaborations Strengthened Collaboration with the WHO: key to moulding the future of haemoglobin disorders… A Tribute to Mr Ioannis Levidiotis TIF Projects 2nd Pan-Asian Conference on Haemoglobinopathies 4th Pan-European Conference on Haemoglobinopathies and Rare Anaemias TIF Publications International Thalassaemia Day, 8 May 2015! International News Patient’s Story TIF’s e-Applications Winners of our Video Challenge Competition Other News TIF Renzo Galanello Fellowship Calendar & Upcoming International Events Obituaries Special focus on TIF’s MSc in Inherited Haemoglobin Disorders Medical Corner

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Technical specifications for photographs 1. 2.

Photographs submitted for TIF Magazine should be in JPEG format (although.PDF and.TIFF are also accepted). Photos should be “print quality”. This means: • The largest size possible. Please do NOT reduce the images! Send them either one by one via email, or alternatively on a CD. • High-resolution (at least 300 dpi). Please ensure that when transferring photos from a digital camera, a high resolution is selected. (How this is done depends on the camera model.) 3. All photos should have captions - a short text explaining what is happening in the photo and the names of the people in the photo. 4. It is the responsibility of the person submitting photos to ensure that any person(s) featured in them has given their permission to publish the photo. Disclaimer: Reproduction of material published in TIF Magazine for educational purposes is encouraged, provided it is accompanied by the following attribution “…according to TIF magazine, the official newsletter of the Thalassaemia International Federation”.The contents of any scientific article or presentation of any material by manufacturers does not imply the expression of any opinion on the part of Thalassaemia International Federation. The mention of specific companies or products does not imply that they are endorsed or recommended by TIF in preference to others. The contents express the opinions of the authors who alone are responsible for the views expressed. TIF does not accept any legal responsibility for their contents.

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TIF’s Activities Around The World South East Asian Region (SEAR) Promising times ahead for India! A project with great expectations! The journey to transforming India into a well-structured and vibrant country with regard to its infrastructure for thalassaemia has been long and productive! Forming a part of this eventful journey, a delegation visit was organised on 27 November 2014 in the Indian states of Gujarat and Uttar Pradesh, with a view to address the problem of haemoglobin disorders in a holistic manner, while remaining in anticipation of responses regarding the Charters of Priorities for the States of Delhi, Punjab, Haryana, Chandigarh, Uttar Pradesh, Maharashtra and Gujarat, and for the Ministry of Health and Welfare of the Central Government of India, which have already been officially dispatched by TIF. In Gujarat, the TIF delegation met with the top ranks of India’s Rotary Clubs to discuss a plan of collaboration in a programme to raise awareness about haemoglobin disorders across India. Furthermore, TIF’s delegation met with medical specialists in Ahmadabad, Gujarat, where the latest scientific developments were discussed. In Uttar Pradesh, the delegation, accompanied by local associations and professionals, met with State Ministry officials to promote services for both patient care and prevention. Local associations, health professionals and health officials participated in a workshop aimed towards identifying the weaknesses in the state services and to developing a state policy. This visit provided an excellent opportunity for TIF’s representatives to strengthen collaborations with all relevant stakeholders in the country and opened discussions to accelerate progress in this country with regard to thalassaemia

Dr Androulla Eleftheriou, TIF Executive Director, during the delegation visit in Morocco (28-30 November 2014)

Eastern Mediterranean Region (EMR) Morocco’s continued fight for better care Infrastructures for Thalassaemia in Morocco are starting to take shape! As a follow-up of last year’s high level delegation visit to Morocco, headed by Mr Panos Englezos, TIF President, and Her Highness Sheikha Sheikha Bint Seif Al-Nahyan, an esteemed Board Member of TIF, another official delegation visit was conducted from 28 to 30 November 2014. Seeking to ascertain the latest developments regarding the status of the diagnosis, prevention and clinical management of thalassaemia in the country, MAY 2015 www.thalassaemia.org.cy

TIF’s Delegation (comprised of DrAndroulla Eleftheriou, TIF Executive Director, Mr Angelo Loris Brunetta, TIF Board Member, Dr Matheos Demetriades, TIF Senior Countries Officer, and Dr Essam Dohair, Director of the TIF Regional Collaborating Office in Abu Dhabi) participated in the 1st Thalassaemia Management Workshop for key stakeholders in Rabat. The Workshop was organised by the Ministry of Health of Morocco and members of the Expert Haemoglobinopathy Forum of the country. Attended by 39 medical specialists and patient/ parent representatives from across the country, the Workshop covered topics relating to the National Programme for Thalassaemia in Morocco, the actual situation and the challenges in thalassaemia prevention and management, as well as the establishment of Reference Centres in the country. In the context of this Workshop, Dr Androulla Eleftheriou (TIF Executive Director), outlined TIF’s engagement in Morocco, drawing comparisons to experiences from other countries. In the course of the visit, theTIF Delegation had the opportunity to converse with representatives from the Ministry of Health of Morocco, the Director of the WHO Country Representative in that country, and very importantly, with patients and parents. In the course of this visit the regional collaborating office of TIF donated 20 iron chelation pumps to a newly-established patients’ association, the “Association Marocaine de Thalassémie et Drépanocytose”. This was an important step towards instigating changes with a view of improving the quality and accesibility of healthcare services to patients with haemoglobin disorders in this country. In addition, TIF reiterated its commitment to supporting the strengthening of the infrastructure of the newly-established patients’ association. Egypt coming into the light! Egypt is a country where tremendous work has been achieved with regards to thalassaemia, albeit there are still many gaps that need to be bridged and many challenges that need to be addressed. There is still a need for the implementation of a national program

program, as well as public awareness programs on thalassaemia. African Region (AFR) Algeria moving forward! Over the past few years, significant positive changes were made in Algeria which have been pivotal to the country’s general progress in the field of haemoglobinopathies. In continuation of the work accomplished so far in Algeria, Prof Dimitris Loukopoulos (TIF’s Coordinator for the Maghreb Region), participated in the faculty of speakers in a Workshop on Sickle Cell Anaemia, which was organised by the Military Hospital Ain Naadja in Algiers, on 29 October 2014. In the context of this workshop, Prof Loukopoulos also had the opportunity to meet with officials from the Ministry of Health of Algeria and other stakeholders, including the President of the Algerian Haematology Association and other key medical professionals who are members of the Expert Algerian Group on Haemoglobinopathies (designated by the Ministry of Health of Algeria).This targets to discuss the agenda of the forthcoming Ministry of Health’s 1st official meeting of this group, in the presence of TIF, scheduled for 2015. This meeting falls within the agreed framework of the Joint Plan of Action accompanying the Memorandum of Understanding between TIF and the Algerian Ministry of Health (signed in September 2013). It will be a strong first step towards ensuring the materialisation of the jointly agreed road map, but more importantly towards monitoring the implementation of improved services for patients in Algeria. Western Pacific Region (WPR) China’s seamless rise is becoming a reality! The years 2013 and 2014 have been pivotal years for thalassaemia in China as it enters a new phase! TIF has played a vital role in the recognition, development and integration of prevention programs for thalassaemia at a national level. TIF is now moving towards exerting continuous efforts for supporting the holistic care of thalassaemia patients in this country.In this framework,TIF was officially invited in Nanning of the Guangxi province in China, from 11 to 12 October 2014 to participate in the 2nd International Conference for the Prevention of Birth Defects and Thalassaemia after thalassaemia prevention was officially integrated by the Central Government into the relevant WHO programme. MAY 2015 www.thalassaemia.org.cy

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to prevent this disease and ensure its proper holistic care across the country. In this context, TIF was recently invited by the Egypt Thalassaemia Association (TIF’s voting member since 1993) and by the medical specialists of the country to provide its expertise and knowledge for developing, shaping, improving and strengthening existing services for thalassaemia in Egypt. TIF, represented by Dr Androulla Eleftheriou, TIF Executive Director, and Dr. Michael Angastiniotis, TIF Medical Advisor, conducted a delegation visit to this country from 18 to 20 February 2015. As part of this visit, many meetings were organized with state health officials. The delegates met with the Minister of Health and Population of Egypt, His Excellency Dr Adel Hassan Al Adawi, and had the opportunity to discuss the development and implementation of a national prevention and control program for thalassaemia, as well as policies by the Egyptian government that will improve the care for patients with thalassaemia in the country. Distinguished Egyptian scientists were also present at this meeting, including Prof Amal ElBeshlawy (TIF’s regional medical advisor) and Prof Mona Hamdy, both professors at the Department of Paediatric Haematology of the Cairo University Hospita,l and Dr Naglaa Shaheen, consultant genetic paediatric haematologist and spokeswoman for the Egyptian Health Insurance Organization. After the extensive discussions that followed, it was agreed that TIF would draft a memorandum of understanding, supported by a plan of activities, in collaboration with the Egyptian experts, which is intended to be reviewed and considered for adoption at the national level by the Egyptian government. TIF delegates also had the opportunity to participate in a patients’/parents’ workshop held on 19 February 2015 in Alexandria, through presentations and interactive discussion. This workshop was the highlight of a very motivational journey aimed at reviewing the current situation in patient care, the national prevention

The minister of Health and Population of Egypt, Dr Adel Hassan Al Adawι, with TIF Executive Director, Dr Androulla Eleftheriou, TIF Medical Advisor, Dr Michael Angastiniotis, and distinguished medical professionals in Egypt

This Conference was organised by the National Health and Family Planning Commission of the People’s Republic of China, with the objective of encouraging all the provinces of the country that have a significant prevalence in thalassaemia (mainly in the Southern part) to adopt effective policies for the control of these disorders as well as other birth defects.The conference adopted a declaration, known as the Nanning Declaration, which seeks to promote the implementation of prevention measures as early as possible. TIF was represented by Dr Michael Angastiniotis (TIF Medical Advisor), and Prof Suthat Fucharoen of the Thalassaemia Research Centre, Mahidol University, Thailand. The presence of high officials of the national and provincial health authorities as well as around 150 health professionals and regulators from institutions of all affected provinces, underscored the importance of this Conference. The officials included, amongst others, the Vice-Chairman of the 11th National People’s Congress Standing Committee, academics, the Honorary President of the China Woman and Child Health Research Association, the Vice Minister of the National Health and Family Planning Commission of the People’s Government of the Guangxi Zhuang Autonomous Region of the People’s Republic of China, as well as the Commissioner of the State Administration of Traditional Chinese Medicine and the Vice-Chairman of the country. With the opportunity of this visit, TIF also met with Mr Liu Zhengchen, Founder and Chairman of the New Sunshine Charity Foundation (NSCF), a patient-founded haematology organization in China, to discuss further avenues of collaboration in order to empower patient groups and provide thalassaemia patients with a united and officially recognised voice. Deepening Mutually Beneficial Ties with Vietnam... The outlook brightens for haemoglobin disorders in Vietnam! Following TIF’s previous delegation visits and activities in the country,TIF has completed a delegation visit to Hanoi, Vietnam, between 13-16 October 2014. This enabled TIF to meet with representatives of the Ministry of Health, and more specifically, with the Vietnamese Vice Minister of Health, Prof Nguyen Viet Tien, and the President of the Health Insurance System of the Ministry of Health of Vietnam.

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The aim of this meeting revolved around the efforts or the implementation of a National Health Programme for Thalassaemia in the country as well as the organization of TIF’s 2nd Pan-Asian Conference on Haemoglobinopathies and Rare Anaemias, which is to take place in Vietnam between 26 and 27 September 2015.This significant educational event is co-organised by TIF, the Vietnamese Thalassaemia Association (ViTA) and the National Institute of Hematology & Blood Transfusion (NIHBT). The future for Vietnam is looking brighter and we anticipate that hosting this 2nd Regional Conference for South East Asia and the Western Pacific will provide momentum for raising awareness about haemoglobin disorders, not only in the country but also very importantly across these two regions! European Region (EUR) Exciting new chapter unravels for Cyprus!

Making our voice heard about the Cyprus National Health Plan An important highlight of TIF’s activities in 2014 has been the workshop on the Cyprus National Health Plan, which took place at and under the auspices of the University of Nicosia, in Cyprus, on 4 October 2014. This workshop was organised by the Cyprus Alliance for Rare Disorders (C.A.R.D.), of which TIF is a founding member, with the collaboration of the Pancyprian Federation of Patients’ Associations and Friends and the participation of the Cyprus Medical Association and the Cyprus Confederation of Organizations of the Disabled (CCOD). The main objectives of this workshop were to enlighten patients with rare and chronic disorders about the upcoming changes in the national health system in Cyprus, and to examine its provisions with regard to rare diseases including haemoglobinopathies and their holistic care. Present at, and participating in this workshop were all important stakeholders relating to this

The workshop was marked by the participation of distinguished medical professionals as well as patients in discussions which revolved around health policy decision-making and the implementation of the new National Health Plan in Cyprus.

Attending the 5th Cyprus Conference for Thalassaemia! TIF participated in the 5th Cyprus Conference for Thalassaemia, organised by the Thalassaemia Clinics in Cyprus in collaboration with the Cyprus Institute of Neurology and Genetics (CING), on 13 December 2014, in Larnaca, Cyprus. TIF, represented by Dr Androulla Eleftheriou (TIF Executive Director), and Dr Michael Angastiniotis (TIF Medical Advisor), actively participated in the Conference. This Conference focused on various aspects of the management of thalassaemia, such as hepatitis C in thalassaemia patients, new methods for treating Hepatitis C, blood transfusion and iron chelation , and new approaches to treatment. The Pancyprian Federation of Associations of Patients and Friends (POSPF) gives a voice to patients TIF has embraced and supported an initiative of vital importance by attending a workshop on the topic of patients’ participation in health policies and decision-making. The workshop, titled “Developing a Policy Plan (Declaration) for Patient Involvement in Health Policy Making” was organized by the Pancyprian Federation of Patients’ Associations and Friends (POSPF) and took place on 16 January 2015 in Nicosia, Cyprus. On behalf of TIF, Dr Androulla Eleftheriou (TIF Executive Director), played an active role in this workshop.

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field, such as representatives of the Cyprus Ministry of Health, of the Ministry’s Pharmaceutical Services, and members of the National Committee for Rare Diseases. The programme of this workshop was informative, featuring many key speakers and covering many aspects of this matter. Among the topics covered were health and social care for rare diseases,the Cyprus National Strategy for implementing the National Health Plan, the role of Medical Schools in the education of health professionals, and the rights of patients and the disabled in the National Health Plan.

International Collaborations TIF participated in and contributed to the European Liver Patients’ Association (ELPA) Policy Meeting in Amsterdam! The 2014 November ELPA Policy Meeting was organised in Amsterdam and was the first attended by TIF. These Policy Meetings are organised on a regular basis by ELPA and provide patient representatives, members of ELPA, with comprehensive information on what has been achieved until this day, what had been the weaknesses and possible causes of delays (if any) and what are the objectives of its plan of activities for the next year (in this meeting for 2015). Significant achievements and concerns were explored and the attendants had ample time for productive interaction. The main objectives of ELPA’s work focus on increasing awareness on prevention of liver disease, promoting screening activities, increasing treatment rates with the latest medication, and reducing stigmatisation of liver patients. National activities were also discussed including the support provided to members with regards to capacity building strategy, training, fundraising, and the development of a white economic paper in order to support that its work in proving the cost effectiveness of preventing and treating patients with viral hepatitis There was considerable effort on behalf of ELPA and its members to bring the liver topic before official decision making bodies including the members of the European Parliament, the European Commission, the EU presidencies (Latvia and Luxembourg), the European Centre for Disease Prevention and Control (ECDC) and different stakeholders, groups and non governmental organisations including TIF. The launching of important papers including a macro paper and a white economic paper also formed part of the discussion. These papers focused on the economic part of the strategies and activities of ELPA. More information will follow in our next magazine. The 2015 activities included the contribution and participation of ELPA in the 2015 International Liver Congress of the European Association for the Study of the Liver (EASL) , in the World Health Organisation’s (WHO) national planning group, in new projects of the Centre of Disease Analysis (CDA) led by Homie Razavi, in the American Pacific Association for the Study of Liver (APASL) meeting, in the World Hepatitis Alliance’s (WHA) global PAG meeting and

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in the International Symposium on Hepatitis Care for Substance Users (INHSU) which offers an update for the hepatitis C virus and provides information on the guidelines for people using injected drugs. Fighting Viral Hepatitis in Resource-Limited Member States Institutional and non-institutional stakeholders joined together in a discussion of paramount importance, titled “Fighting Viral Hepatitis in Resource-Limited Member States”, held on 4 December 2014 at the European Parliament in Brussels. The meeting sought to understand the disease burden associated with viral hepatitis in resource-limited Member States, taking into account the current experiences and solutions of addressing viral hepatitis in these Member States, and to call for specific policy actions to improve access and care for the benefit of patients living with viral hepatitis. TIF participated actively in this event, represented by its Executive Director, Dr Androulla Eleftheriou. Hepatitis B (HB\/) and C (HCV) are viral infections which create a significant burden to healthcare systems due to their high morbidity, mortality and costs of care and treatment. According to the World Health Organization (WHO), one third of the world’s population has been infected with HB\/ and more than 350 million suffer from chronic infection, including many patients with transfusion-dependent thalassaemia, the percentage of who are affected with HBV or HCV in some countries reaches 85%. This situation is creating a significant pressure on healthcare systems, particularly in Member States where healthcare resources are limited. Priorities established through an Advocacy Meeting in Zurich Patient representatives of national thalassaemia associations from 10 countries of the Middle East and South East Asian region participated in a twoday Patient Advocacy Meeting organised by Novartis in Zurich, on 13 - 14 December 2014. The Meeting aimed to identify perceptions about the role of patient groups in addressing the needs of patients with both transfusion dependent

Joint activity with TIF’s EU funded project, ENERCA, and the European School of Hematology (ESH) Health Professionals from around the world, active in the field of haemoglobinopathies, came together in Barcelona, Spain, to attend the Training course on haemoglobin disorders, organized by the European School of Hematology (ESH) and the European Network for Rare and Congenital Anaemias (ENERCA), held on “Collaboration between TIF 23-24 January 2015. Dr and the European Medicines Michael Angastiniotis Agency (EMA) opens up new opportunities! (TIF Medical Advisor) participated in the workshop with a lecture on prevention programmes for thalassaemia and sickle cell disease, focusing on case study countries and on the role of patient-parent associations. This training course forms part of an ENERCA initiative intended to promote interaction and ample discussion between scientists and clinicians working in this specific area of health. International leaders constituted the faculty and provided the current and future perspectives of the many exciting developments in the field. Results from both basic and clinical research were presented in the different sessions of the programme.

A number of key topics were presented and discussed, including epidemiology, screening and prevention of haemoglobinopathies, new clinical and biological aspects of thalassaemia, new and future trends in this field, the laboratory diagnosis of thalassaemia syndromes and the complications and treatment of abnormal haemoglobins. Collaboration between TIF and the European Medicines Agency (EMA) opens up new opportunities! The European Medicines Agency (EMA), the official European drug regulatory body, has embarked on a new and exciting project, aiming to speed up access of drugs to patients. In this context, Dr Androulla Eleftheriou (TIF Executive Director), virtually attended a meeting on this new Adaptive Licensing Pilot Project, on 7 January 2015 that regards to Gene Therapy drugs. Adaptive licensing revolves around the early authorisation of a medicine in a restricted patient population, collecting evidence in this group and then adapting the marketing authorisation to allow a broader patient population to access the medicine. The meeting brought together multiple stakeholders, including patients, who contributed to the discussion on the proposed design and timing of the clinical trials. Furthermore, TIF participated in an EMA Training Session for Patients’ and Consumers’ Organisations, on 25 November 2014, in London, UK. TIF was represented by Miss Liana Prastiti (TIF Communications Officer). The Training Session included an overview of the Agency, its centralised procedures, how patients are involved in scientific advice, the Scientific Advisory Group (SAG) meetings, the review of information to patients, and pharmacovigilance. Through this workshop, the EMA reiterates its commitment to engage in dialogue with European patients and consumers.This interaction allows patients and representatives of organisations to engage with the Agency on issues that affect them and to share their real-life experiences. This contributes to the quality of the decision-making process by bringing to light the real-life implications of regulatory decisions. A more thorough description of the patients’ role and participation in EMA will be published in our next magazine.

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and non-transfusion dependent thalassaemias. TIF was represented by Ms Lily Cannon (European Affairs Senior Officer). The vibrant and lively discussion highlighted common areas of concern and the importance of involvement of the thalassaemia patient community in safeguarding fundamental patient rights. TIF, as the international voice of thalassaemia patients, remains committed to fulfilling its vision for equal access to quality healthcare for all patients with thalassaemia through the empowerment of patients & parents and the sensitisation of national and regional health authorities. Importantly, however, empowerment can only occur if the knowledge of patients on the disease reaches adequate levels. Hence, an important pillar of TIF’s work is transforming patients into expert stakeholders in global efforts.

EMA Training Session for Patients’ and Consumers’ Organisations, on 25 November 2014, in London

Strengthened Collaboration with the WHO: key to moulding the future of haemoglobin disorders…

Attaining the nine global NCD targets: a shared responsibility! An initiative of the International Alliance of Patients’ Organisations (IAPO) We are overjoyed to share with you that TIF has virtually attended the launch of the second Launch of the WHO Global Status Report on NCDs (2014), second WHO titled “Attaining the Nine Global NCD Targets: a Global Status Shared Responsibility”, on 19 January 2015. Report on NCDs The first WHO Global Status Report on (2014), titled “Attaining the NCDs (2010) was launched at the 1st Global Nine Global NCD Ministerial Conference on Healthy Lifestyles and Targets: a Shared Non Communicable Diseases’ (NCD) Control in Responsibility”, on 19 January 2015 Moscow, on 28-29 April 2011. The report provided the foundation for advocacy, policy development and global action, based on scientific knowledge, available evidence, and a review of international experience, framed around the six objectives of the WHO Global NCD Action Plan 2008-2013. The second Report focuses around the nine global targets for NCDs to be attained by 2025. It provides guidance on how Ministries of Health can set national NCD targets and lead the development and implementation of policies and interventions to attain them. The meeting was effective in conveying the key messages of the report. The WHO DirectorGeneral, Dr Margaret Chan, Ambassadors from Permanent Missions in Geneva, as well as panelists from the NCD Alliance, the International Olympic Committee, the World Economic Forum,

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the University of Lausanne, and the United Nations Development Programme (UNDP), who participated as speakers during this launch event. Working together for the Compilation of Scientific papers We are delighted to announce that two of the four scientific papers prepared by TIF’s Scientific Medical Panel in collaboration with the WHO Regional Directors and the WHO Directors of the Non Communicable Disease (NCD) Units, have finally been completed! The completed scientific papers were compiled in collaboration with the Regional Office for the Eastern Mediterranean (EMRO) and the Regional Office for the Western Pacific (WPRO) of the WHO. The remaining scientific papers, that of the Regional Office for Europe (EURO) and the one of the Regional Office for South-East Asia (SEARO) will be finalised by the end of 2015. This is a TIF initiative that stems from the need to review and analyse the situation of the countries of each WHO Region and aims to map the current status of the control of haemoglobin disorders, and in particular thalassaemia, in terms of their prevention and clinical management within and across all countries of each WHO region. Already, the EMRO paper has been delivered to the respective Regional Office and the WPRO paper is still open to consultation by regional and national experts. The papers are expected to be submitted to WHO by the end of May 2015.

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A Tribute to Mr Ioannis Levidiotis, our valued friend, our exceptional collaborator As previously announced, this issue is dedicated to Mr. Ioannis Levidiotis, a great friend and supporter of TIF, a man of principles, values and vision. The world thalassaemia family mourns the loss of a great man, a man who always put the needs of others above his own, a father who fought for decades to create a better world for his own children, and for all children with thalassaemia in Greece that he considered his own. He was a pioneer in the field of thalassaemia in Greece, and gave his heart and soul to ensure a better future for those in pain, despair, agony, fear and uncertainty. With his immense courage and exceptional fighting spirit he proudly struggled through the years against the many and multiple health and social challenges, advocating for the rights of patients with thalassaemia in Greece. As a father of a thalassaemia patient, Mr Ioannis Levidiotis started his journey 35 years ago. In 1986, together with other patients and parents he established the Pan-Hellenic Voluntary Movement for Thalassaemia “KINISIS for Volunteers”, with the vision to unite the forces and strengthen the voices of all those affected by thalassaemia in Greece. He was a member of the Board of this Association until 2004, when he was elected Chairman of the Association’s Board, a position held until his death. The quarterly magazine “KINISIS for Volunteers”, was an initiative of this visionary. His vast experience and exemplary competence in the publishing field, the great power of his pen but above all his persistence, patience and undivided commitment were those elements that contributed to the success of his work. Ioannis Levidiotis has also pioneered and contributed decisively in the creation of the Greek Thalassaemia Federation in 1991 and was a member of its Board of Directors for many years. For TIF, he is and will always be remembered as a great friend, supporter, and a very valued collaborator. He undertook the translation in Greek of a variety of publications, including the TIF Magazine, as well as the distribution of the latter in Greece since 2009. He believed deeply in TIF’s role and impact in the thalassaemia community globally and in the close collaboration between Greece and TIF. In more recent years he undertook and coordinated the translation of important publications, including the TIF Magazine, as well as its distribution since 2009. He believed deeply in TIF’s role and impact in the thalassaemia community globally, and in the close collaboration between Greece and

MAY 2015 www.thalassaemia.org.cy

Ioannis is to be remembered as a man of punctuality, voluntarism, and passion. On this difficult, challenging, life-long journey, Ioannis most probably neglected himself. His large heart broke on 22nd December 2014, a few days before Christmas. Ioannis Levidiotis was for all of us an extraordinary personality and an exceptionally wonderful and gifted person, and we are truly fortunate and very privileged to have met him and worked with him. All of us who had the honour to meet him will always keep in our hearts and memories, his tremendous and invaluable contribution to the Greek thalassaemia family, which will constitute the leading light for many others to follow. We are confident that Thanos, his son, will continue his fathers’ work, both because he has inherited the competence and because he is equally devoted and knows deep in his heart that continuing Ioannis’ work will be the best tribute to his father. TIF, in the memory of Ioannis Levidiotis, will proceed to establish a new Sponsorship Programme in his name, for the participation of patients to TIF’s international conferences: the “Ioannis Levidiotis Sponsorship Programme”.

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TIF. In more recent years he undertook and coordinated the translation of important publications, including the United Kingdom’s Standards of Care for the Clinical Management of Adults and Children with Sickle Cell Disease. His absolute commitment to patients, which was a whole life’s work, led him in the last few years to focus attention and serious thoughts on supporting patients with sickle cell disease as well.

TIF Projects The e-ENERCA project reaching new heights! At this stage of the e-ENERCA project, we are pleased to inform you that TIF has participated in the ENERCA Executive Committee Meeting, represented by Dr Michael Angastiniotis (TIF Medical Advisor).This Meeting was held in Barcelona, Spain, on 22 October 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. The main objective of TIF’s involvement is the development of a European Registry for Rare Anaemias, including thalassaemia. TIF will continue 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 country of origin of the patient. The importance of the use of generic iron chelation drugs in patients with Hb Disorders This project was designed in collaboration with international experts with the aim of bringing to the light the potential issues of efficacy, safety and tolerability of generic drugs used by patients with thalassaemia, while identifying which generics are circulating internationally, to what extent they are used compared to brand names and to what extent, if at all, adverse reactions or side effects are reported or studied. In phase I of the project, the questionnaires through which data will be gathered will be reviewed by the advisory group of TIF and their finalised version will be diffused to different national regulatory authorities, treating medical specialists and industry in different countries of Europe, the Middle East and South East Asia to be completed to the fullest possible extent. In Phase II, a different type of questionnaires will be directed to patients in order to obtain their perspective as well on the use of generic drugs. The objective of the whole project is to assess whether truly generic iron chelating drugs are used which pass through proper quality tests for their safety and effectiveness. Safeguarding the rights of patients with Thalassaemia and Viral Hepatitis: the position of TIF TIF, representing and safeguarding the rights of

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patients with haemoglobin disorders across the world and defending their right to quality health care, has decided to react to the lack of recognition of the importance of providing antiviral treatment to thalassaemia patients infected with Hepatitis, through giving a particular focus to viral Hepatitis C in relation to thalassaemia. In this context, a manifesto was compiled, titled “Viral Hepatitis C in Thalassaemia”. This paper, which due to many advances regarding therapeutic protocols for Hepatitis C has been revised and updated since its first announcement during the 4th Pan-European Conference on Haemoglobinopathies and Rare Anaemias (7-9 November 2014, Athens, Greece), is now ready to be officially delivered to official related stakeholders including the WHO, representatives of the European Commission, the European Parliament, the Ministers of Health initially in Europe and afterwards across the rest of the world, the Regional Director of the International Organisation for Migration (IMO) and - importantly - to every patient organisation and to the national health authorities in the respective organisation’s country. We are delighted to announce to you that this document has now been officially endorsed by the European Association for the Study of the Liver (EASL), the European Organisation for Rare Diseases (EURORDIS), and the European Liver Patients’ Association (ELPA). TIF seeks to boost the Magnetic Resonance Imaging (MRI) Project TIF seeks to boost the Magnetic Resonance Imaging (MRI) Project, which mainly revolves around the compilation of informational material on the Magnetic Resonance Imaging (MRI) technology and is targeted towards three types of audience: patients/ parents, medical specialists (mainly haematologists and paediatricians treating thalassaemia), and radiologists. 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 13th TIF International Congress that took place there. TIF’s expert team at its Headquarters, led by Dr Androulla Eleftheriou (TIF Executive Director), is now in the process of finalising this informational material. More information will be available in our next magazine and on TIF’s website.

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Promising an exciting blend of educational, inspirational and motivational moments at the 2nd Pan-Asian Conference on Haemoglobinopathies

Objectives The objectives of the 2nd Pan-Asian Conference as of every other TIF Regional Confere nce, are:

A. For patients • To educate, empower and create co re groups of patients in each country to support and We are delighted to announ strengthen ce the their role in monitoring their own dise organisation of the 2nd Pan-Asian Con ase; ference on • To develop the skills and capacities of patients for Haemoglobinopathies which will take place on strengthening their advocacy role 26 and 27 September 2015 in Hanoi, for achieving Vietnam. We pro duc tive part icip atio n in dec warmly invite you to be part of this ision-making at exciting event the national and regional level; that aims towards fostering a forum of interaction • To develop a robust infrastructure for national for patients and health professionals, while bringing pati ents ’ associations, making them trul together hundreds of participants y from a wide sup por tive to their patients and their families at range of disciplines from around the world. the national level. Substantial medical achievements in improving the status of patients with haemog lobinopathies B. For Health Professiona ls were achieved in the past decade in the Asian • To develop a network of col laborators from each Region but there are still many mile s to cover and country, ensuring a more tailored sup many gaps to fill. Determination, unit port by TIF y, and above all at the national level; a united voice are urgently needed. • To develop a core group of exp ert medical The 1st Pan-Asian Conference on Haemoglospecialists across disciplines relevan binopathies was organised in 2012 t to the and gathered multidisciplinary care of haemoglobi together more than 200 health pro nop athies, fessionals and aiming to support the pillars of TIF’s scientists involved in the field of educational haemoglobiprogramme, which are: nopathies, as well as patients and their parents, - the preparation, translation and dist from all over the region, for a tota ribution of l of over 450 publications and educational materia participants. l; - the scientific programmes in educati onal events; The organisation of the 2nd Pan - Asian - the development of or contrib Conference on Haemoglobinop ution to athies will academic courses led or suppor ted provide an even greater opportu by TIF. nity to help catalyze changes bringing together and bridging C. For Policy Makers perspectives from various disciplines with the aim • To provide a forum of interaction for policy makers to influence governments, organiza tions, agencies to support and recognise the health priorities of and institutions around the world to meet the this disease based on case examples , cost effectichallenges of improving the health of those affected veness and the disease burden in man y countries. by haemoglobinopathies. Organised in collaboration The exceptional ambience and charm of HaNoi is with the Vietnamese Thalassaem ia Association bound to leave an everlasting memory in the minds (ViTA) and the National Institute of Hematology of all those who will attend the Conference. & Blood Transfusion (NIHBT), this promises to We look forward to welcoming you at the 2nd be unforgettable in every way, providi ng a unique Pan-Asian Conference on Haemog lobinopathies in forum for discussion and interaction between all HaNoi,Vietnam, on 26 and 27 Sep tember 2015. relative stakeholders. More information is available on our website! Register early! Get cheaper prices!

MAY 2015 www.thalassaemia.org.cy

Special Highlights of the 4th Pan-European Conference on Haemoglobinopathies and Rare Anaemias 7-9 November 2014, Athens, Greece

The Conference Venue: Titania Hotel, Athens, Greece

Delivery of the consensus papers

Ms Petia Nenova of the Directorate General for Health, European Commission & Consumers

Dr Eleni Theocharous, Member of the European Parliament

The 4th Pan-European Conference on Haemoglobinopathies and Rare Anaemias, organized by the Thalassaemia International Federation (TIF) and the Greek Thalassaemia Federation (EOTHA) has been completed with immense success, between 7-9 November 2014, in Athens, Greece. The Conference was placed under the patronage of Mr Tonio Borg, former European Commissioner for Health & Consumers (2011-2014), the Hellenic Society of Haematology and the Greek Ministry of Health. It was also and very importantly observed by the European Commission (Ms Petia Nenova of the Directorate General for Health & Consumers), the European Parliament (Dr Eleni Theocharous, Member of the European Parliament), and the European Haematology Association, (Prof. Francesco Rodeghiero), who delivered keynote speeches in the programme. The 4th Pan-European Conference included significant and official announcements launching and delivery of: 1. The 3rd edition of the Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT). 2. The United Kingdom’s “Standards for the Clinical Care of Sickle Cell Disease in Adults” (Greek). 3. The “Emergency Management of Thalassaemia” and the “Guide for the Haemoglobinopathy Nurse” (Greek). This Conference also concluded with two consensus papers, the “Access of Patients with Thalassaemia infected with Viral Hepatitis C to effective treatment or Cure” and “The Future of Haemoglobin Disorders and Rare Anaemias in Europe: Observations, Key Findings, and Recommendations”. These were officially announced to the representatives of the European Commission, the European Parliament, the Regional Director of the International Organisation for Migration (IMO), and the Minister of Health of Greece, and will be delivered when finalised and updated in May 2015 to the Ministry of Health of each European Member State and other official decision-making bodies. To read these papers, please visit our website at http://www.thalassaemia.org. cy/news-archive.shtml Conference Programme The extremely interesting and informative programme offered a unique platform of exchanging and sharing ideas and has touched all aspects of best practices, research, but also concerns, successes and expectations. Each topic was covered by national, regional or international experts in this field (the proceedings book of the conference is finalised and can be found on our website at http://www.pagepressjournals.org/index.php/thal/issue/view/285 The scientific programme, rich and multifaceted, has focused on issues relating to prevention, iron chelation, methods of treating complications, the development of criteria for haemoglobinopathies’ reference centres and their accreditation, as well as on the impact of the economic crisis in the health sector. Over 40 world-renowned experts who specialise in the field of haemoglobinopathies participated in the faculty of this conference. Furthermore, the patients’ & parents’ programme focused on developing skills for both patients and patients’ associations in Europe via interactive discussions. The organisation of the conference in Greece was an excellent opportunity, highlighting the remarkable achievements of the country’s patient/parent community throughout the last decades and the struggle to preserve patients’ rights in a background of economic austerity. To view the Conference’s programmes, please visit the following links: Scientific Programme: http://www.tif2014.org/programme/scientific-programme Patients’/Parents’ Programme: http://www.tif2014.org/programme/patientsparents-programme

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Participation The Conference was attended by 630 participants, 324 Healthcare professionals and 267 patients and parents, from 42 countries, including 27 of the European Region. An opportunity to highlight TIF’s ongoing electronic projects… • During this Conference an official presentation was completed for TIF’s Mobile Application,ThaliMe. Participants were offered a download link to test the application as well as a live demonstration during the conference. Please find more information on page 30. • TIF’s video sharing platform, designed with a primary focus to connect thalassaemia patients across the world, constitutes one of TIF’s most successful projects. This project aimed to initiate a two-way interaction between TIF and patients on a global video platform, allowing patients to submit their own videos on a revamped TIF YouTube page! We are overjoyed to share with you that we have received a great number of inspiring videos for TIF’s video challenge. All the entries of all the contestants were displayed during this Conference at TIF’s dedicated Exhibition Booth. To view the videos, please visit the following link: www.thalassaemia.org.cy/ video-sharingplatform.html

During the scientific programme of the conference

Special focus to TIF’s MSc Course programme in Thalassaemia and Sickle Cell Anaemia! Preliminary information on the MSc Course programme on Thalassaemia and Sickle Cell Anaemia was provided during the Conference. The Course is currently being established through the University of Nicosia (Cyprus) in collaboration with TIF. Once officially launched, it will offer a postgraduate academic course on haemoglobinopathies to health professionals. More details about this programme on page 37. Results of a questionnaire on participants’ impressions with regard to the conference Health professionals (shades of red), Patients/ Parents (shades of blue)

Publications distributed at TIF’s dedicated booth

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Creation and strengthening of partnerships! The 4th Pan-European Conference provided an excellent opportunity for the development of new collaborations and the strengthening of existing partnerships! TIF President, Mr Panos Englezos, TIF Executive Director, Dr Androulla Eleftheriou and TIF Medical Advisor, Dr Michael Angastiniotis, met with numerous country delegations and other interested stakeholders to discuss issues of mutual interest and avenues of collaboration. Of notable importance was the private meeting of Her Highness Sheikha Sheikha Bint Seif Al Nahyan, an esteemed Board Member of TIF, with Mr Panos Englezos, TIF President, and Dr Androulla Eleftheriou, TIF Executive Director. Her Highness was accompanied by Dr. Mahmoud Taleb (Executive Director of His Highness’ Sheikh Sultan Bin Khalifa Al Nahyan Humanitarian & Scientific Foundation - SBKF), Dr. Essam Dohair (Director of the RCOTIF) and Dr. Mohammed Naveed (Director - Planning and Operation, SBKF). In addition, the TIF Board of Directors’ Meeting has been effectuated with great success at the venue of the Conference on 9 November 2014. During this meeting a number of key topics were presented and discussed, including aligning joint efforts and identifying and planning future action.

Testimonial of Dr Charalambos Bakirtzis, Director of the Anastasios G. Leventis Foundation “I take the opportunity to warmly thank you for inviting me to attend the 4th Pan-European Conference on Haemoglobinopathies and Rare Anaemias in Athens, and to chair the first session of the “Patients’ and Parents’” Programme. The experience was sensational. The sense of a genuine and tenacious desire for life overwhelmed the conference hall leading me to the decision to no longer use the term “disease” to refer to thalassaemia, or “patients” to refer to thalassaemia patients, and I urge TIF to do the same, being confident that this will help you towards the realisation of your mission. Moreover, the term thalassaemia, which is composed by the Greek terms thalassa (sea) and aema (blood), refers to the joy of the blue sea, which is reflected in the bright blue sky, and the lively, red blood flowing in the souls of those with thalassaemia. My feelings go beyond the desire to congratulate you (TIF) as you have created a global family, whose members look up to you with confidence, faith, and love. It is an honour that the headquarters of TIF are in Cyprus is, making our small island the centre of a highly delicate and fragile world.” Charalambos Bakirtzis Director of the Anastasios G. Leventis Foundation * Established in May 1979, the A. G. Leventis Foundation is the vision of the Cypriot entrepreneur Anastasios G. Leventis (1902-1978), which focuses on society, education and culture. Its activities range from the reconstruction of a large number of classical monuments and the enrichment and presentation of Cypriot antiquities’ collections in museums around the world, to the systematic granting of scholarships for the development of projects in Southern Europe, Africa and world-wide.

MAY 2015 www.thalassaemia.org.cy

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 and medical professionals. Incredibly well-researched and assembled, TIF’s widely-read publications are renowned in the medical community and distributed freeof-charge. They are a great source of insightful content and features relevant to their 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 the world to over 60 countries. In 2015 alone, requests for TIF publications totalled over 9,000!

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TIF Publications

OUT NOW!!! New Publications • The Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT), 3rd edition have been released on the NLM Bookshelf’s public site. The book’s contents are accessible by search query. Publications Translated in 2014 (in Greek) • Standards for the Clinical Care of Sickle Cell Disease in Adults (2008) • A Guide for the Haemoglobinopathy Nurse (2013) • Emergency Management of Thalassaemia (2012) COMING SOON Translations of Publication upcoming in 2015 • All About Thalassaemia Cartoon Booklet (2010) in Bahasa Indonesian • Sickle Cell Disease (2008) in Arabic • Emergency Management of Thalassaemia (2012) in French, Arabic and Turkish • A Guide for the Haemoglobinopathy Nurse (2013) in Burmese and Farsi • Guidelines for the Clinical Management of Transfusion Dependent Thalassaemia (3rd ed.) (2014) in Chinese, French, Arabic, Turkish and Greek Translations of Videos • “All About Thalassaemia” Cartoon Animation (2013) is being translated into Bahasa Indonesian, Malaysian, Chinese, Hindi, Urdu and Turkish You can find the following publications (and their translations) οn TIF’s website in e-form, at international libraries (such as PubMed) and on TIF’s Digital Library application (soon to be released) 1. Guidelines for the Management of Transfusion Dependent Thalassaemia, 3rd Edition (2014) - Cappellini MD, Cohen A, Porter J, Taher A, Viprakasit V 2. Prevention of Thalassaemias and other Haemoglobin Disorders, Vol 1, 2nd Edition (2013) - Old J, Galanello R, Eleftheriou A, TraegerSynodinou J, Petrou M, Angastiniotis M 3. Guidelines for the Clinical Management of Non-Transfusion Dependent Thalassaemias (2013) - Cappellini M D, Taher A, Musallam K 4. A Guide for the Haemoglobinopathy Nurse (2013) - Aimiuwu E, Thomas A, Roheemun N, Khairallah T, Nacouzi A N, Georgiou A, Papadopoulou C 5. Emergency Management of Thalassaemia (2012) Porter J, Taher A, Mufarij A, Gavalas M 6. Prevention of Thalassaemias and Other Haemoglobin Disorders, Vol. 2: Laboratory Protocols (2012) - Old J, Harteveld C L, TraegerSynodinos J, Petrou M, Angastiniotis M, Galanello R 7. All About Thalassaemia (2010) - Eleftheriou A 8. Sickle Cell Disease (2008) - Inati-Khoriaty A 9. Guidelines for the Clinical Management of Thalassaemia, 2nd Edition Revised (2008) Cappellini M D, Cohen A, Eleftheriou A, Piga A, Porter J, Taher A 10. About Thalassaemia (2007) Eleftheriou A 11. Beta-Thalassaemia, Alpha-Thalassaemia and Sickle Cell Disease Educational Community booklet (2014) - Eleftheriou A, Angastiniotis M 12. Patients’ Rights (2007) Eleftheriou A 13. A Guide to Establishing a Non-Profit Patient Support Organisation (2007) Eleftheriou A MAY 2015 www.thalassaemia.org.cy

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

Fuelling the spirit of the International Thalassaemia Day, 8 May 2015! Many exciting activities are in store! Like every year, the International Thalassaemia Day this year has a new theme, aiming to achieve its mission in enhancing lives and exceeding expectations for patients living with thalassaemia across the world. The 2015 theme: ”Enhancing Partnerships Towards Patient-Centred Health Systems: Good Health Adds Life to Years!” With an evolving landscape and overwhelming expectations, the transformation of healthcare systems to meet the needs of this changing environment is becoming of increased importance. Integrating better healthcare systems ensures that the right patient gets to the right facility at the right time to get the right care. Quality healthcare is critical to transforming the health of communities across the world and to reducing healthcare costs. TIF is involved in a variety of initiatives and collaborations to improve healthcare quality. Through collaborations with other healthcare entities, both locally, regionally and internationally, TIF seeks to strengthen the ability of healthcare systems to provide quick, effective, Activities for the 8th of May 2015 th

8 of May Toolkit 8th of May Website 8th of May International Photography contest Video challenge contest winner announcement

coordinated and efficient access to care. TIF’s current efforts to improve collaborations, share best practices and establish consistent protocols throughout healthcare systems across the world have resulted in significant improvements. Because patient safety is in our hearts and activities at the front line, TIF opts for a care delivery model that provides patients with the appropriate level of care in each phase of their condition and is designed to meet the needs of every patient in need of medical intervention. Through quality, improved access, and operational excellence, healthcare systems will be able to respond to the historic realignments in industry and the economy. 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. As the days approach for the International Thalassaemia Day, honoured every year on the 8th May,TIF has been working for many months now in preparing for the many activities leading up to the 8th of May week. Below is a list of the activities and events that are scheduled to take place this year:

Events for the 8th of May week Date Event Time 28 April 2015 Press Conference Formal Musical 10:00 (to be confirmed) Evening and Gala Dinner 6 May 2015 “Precious Rubies” in the context of 19:15 the 3rd consecutive convening of the Ambassadors Circle, to be held under the auspices of H.E. the President of the Republic of Cyprus 8 May 2015 Blood Donation dedicated to 10:00 International Thalassaemia Day 2015

Venue Journalist House, Nicosia Presidential Palace, Nicosia

Ammochostos Gate, Nicosia

Our activities explained in further detail… Spread the message through our 8th of May toolkit and videos! Our website and toolkit for the International Thalassaemia Day have already been launched and are available at the following link: thalassaemia. wix.com/thalassaemiaday2015 TIF offers its annual campaign communication material to help build awareness for thalassaemia. Share it to show your support for our 2015 campaign! Materials available include: • The official International Thalassaemia Day poster • The official International Thalassaemia Day logo • The official International Thalassaemia Day video • An email banner MAY 2015 www.thalassaemia.org.cy

Deadline The deadline for this year’s competition has ended on Tuesday 31 March 2015.

Subject The subject of this year’s photography contest was based on this year’s theme for the International Thalassaemia Day 2015, which is “Enhancing Partnerships Towards Patient-Centred Health Systems: Good Health Adds Life to Years!” The prizes granted by Thalassaemia International Federation for this competition are the following: The first winner $500 The second winner $300 The third winner $100 “Precious Rubies”: Formal Musical Evening and Gala Dinner for the 3rd Convening of the Ambassadors’ Circle We are in the privileged position to inform you about TIF’s Musical Evening and Gala dinner titled Precious Rubies”. This event will take place in the context of the International Thalassaemia Day, on the 6th of May 2015, at the Presidential Palace in Nicosia, Cyprus. The Gala Dinner will be held under the auspices and with the presence of His Excellency, the President of the Republic of Cyprus, Mr Nicos Anastasiades, and of the First Lady, Mrs Andri Anastasiadou. We have named this event “Precious Rubies” to underscore the preciousness of blood and in

particular of the red blood cells within it, which have the same shape and colour as the precious stones rubies.The aim of this event is for TIF to maintain and strengthen a productive network of collaboration with the Ambassadors of its member countries. We consider that the knowledge and influence of the Ambassadors through their diplomatic role is extremely valuable towards achieving our mission: to minimize the pain and agony of the patients and their families with these previously fatal haematological diseases in every ‘affected’ country. Our guests will include ministers of the Republic of Cyprus, ambassadors and heads of diplomatic missions that are based in Cyprus, state officials, and other local and international dignitaries. The agenda of the event will include a 50-minute delightful musical concert of classical repertoir, followed by a special dinner for all our distinguished guests. We encourage all of you to use the name “Precious Rubies” to invite official stakeholders to our own event, to hear your voice, your concerns, your worries and your gratitude where appropriate! Blood donation drives dedicated to the International Thalassaemia Day! The Cyprus Blood Centre in collaboration with the Nicosia Municipality has decided to devote the blood drives held on the 8th of May to the Thalassaemia International Federation and to the International Thalassaemia Day.

Video Challenge Winner Announcement Our Video Challenge Competition winners will be announced in our social media and communication material! We are delighted to have encouraged patients and their parents, friends and family, to take a step forward with us! More information on page 31.

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TIF’s Photography Contest for the International Thalassaemia Day 2015! For a second consecutive year, we are proud to announce our International Photography Contest 2015, which has been launched with the aim of creating awareness about thalassaemia in the context of the International Thalassaemia Day.

International News

The fight continues and strengthens: The voice becomes louder- the impact becomes greater. Are we there? Not yet.

Marathon Event

South East Asia Region (SEAR) India On 11 September 2014, a visit was completed by the High Commissioner of the Republic of Cyprus in India, Ms. Maria Michael, to the Thalassaemia Unit of Sir Ganga Ram Hospital, New Delhi, where she met with thalassaemic patients and their parents. She was welcomed there by Dr Nirmal Kumar, Head of the Thalassemia Unit, Mrs Shobha Tuli, Vice-President of TIF and Secretary of Thalassemics India, and Mr Deepak Dhingra, Joint Treasurer of Thalassemics India. The High Commissioner interacted with each one of the thalassaemia patients while they were undergoing their blood transfusions. Dr. Nirmal Kumar gave a brief presentation about the facilities available at the Unit. The High Commissioner was also welcomed at the hospital by Dr. V. K. Khanna, Head of the Preeti Tuli Thalassemia Unit, Mr. Deepak Chopra, President, and Executive Members. A meeting at Sir Ganga Ram Hospital was also arranged with the doctors of the hospital, during which the available thalassaemia services in India were discussed. A number of suggestions were given by the High Commissioner to improve these services in the country. The Blood Patients’ Protection Council observed Patients’ Solidarity Day On 6 December 2014, the Blood Patients’ Protection Council (BPPC), an organization for blood disorder patients like Thalassemia, Sickle Cell Anemia, Hemophilia, and Leukemia, observed the Patients’ Solidarity Day by organising a marathon for patients and parents with blood disorders in front of the Medical College Hospital in Calicut, Kerala, India. The marathon was organised to honour the memory of patients who have passed away and to pay a special respect to the eminent human rights’ activist and former Supreme Court Chief of Justice of India, V.R. Krishnayyar, who passed away on 5 December 2014 and who always supported the Blood Patients’ Protection Council’s activities. The marathon was held under the slogan “Equal Access to Quality Health Care, Patient-Centred Treatment and Free Treatment to All the Patients”. Among others, the BPPC’s General Convener, Dr Kareem Karassery, led the marathon. After

MAY 2015 www.thalassaemia.org.cy

the marathon, an event which was open to the public was held at the Medical College, which was inaugurated by Dr Karassery. The Blood Patients’ Protection Council (BPPC) had previously organized on 25 October 2014 another Marathon for blood disorder patients as a part of a Global Action Programme The Blood Patients’ Protection Council (BPPC) in Kerala. This Marathon was held at S. K. Pottakkad Square in Calicut.The renowned writer and gynaecologist, Dr. Khadeeja Mumthas, inaugurated the Marathon. After the completion of this event, the BPPC organized a discussion session at Shikshad Sadan, Calicut, on the topic of the plights and rights of fatal-disease-affected patients, based on Article 25 of the UN Human Rights Declaration (1948). Dr Kareem Karassery, General Convener of the Blood Patients’ Protection Council, inaugurated the discussion. The Council also prepared a consensus paper which was presented to the Government of India, demanding that it takes urgent steps towards adopting policies which respect patients’ rights and provide completely free treatment to all patients. Pan-American Region (PAHO) Cooley’s Anemia Foundation (CAF) meets with Government Agencies! The Cooley’s Anemia Advocacy Forum (CAAF) is a project aimed at helping thalassaemia patients and their families learn how to make their voices heard on Federal issues of importance to the thalassaemia community in the USA. On 24 September 2014, the Cooley’s Anemia Foundation hosted a government-wide stakeholder meeting at the main campus of the National Institutes of Health (NIH) in Bethesda, Maryland. The objectives of the seven-hour long meeting were to understand the major thalassaemia-related initiatives in the Federal Government and to identify ways in which the Foundation and the Federal Government can work more closely together for the benefit of patients and their families. Government officials and official health

Egyptian Health Insurance Organization (HIO) and under the patronage of Prof. Dr. Ali Hegazy, Chairman of the Board of HIO. The conference addressed blood disorders, giving an emphasis on thalassaemia as well as on the milestones achieved in terms of patient support, including the announcement of a decision to provide oral iron chelating medication for thalassaemia patients of all ages in Egypt.

United Arab Emirates Under the patronage of H.H. Sheikha Sheikha Bint Seif Al Nahyan, distinguished Board Member of TIF and Chairperson of the Emirates Thalassaemia Society, the Emirates Thalassemia Society organized a fun filled, colourful, grand celebration for thalassaemia patients and their families from the whole of the United Arab Emirates (UAE) on the occasion of the 43rd United Arab Emirates’ National Day, celebrated on 23 November 2014. A large number of performing artists and famous Emirati celebrities, including Goodwill Ambassador Badria Ahmed, Emirati actress Fatima Jasim, famous sports and media presenter Hessa Ryasi, Emirati Model Rafeeaa Al Hajisi, and Emirati Presenter Ali Jasmi, gave a bright tone to the celebrations with their presence. The celebration included numerous musical performances by the students of the Al Aweer Primary School, the Rawda Al Baraa Kindergarten and the Dubai International School, all depicting Bowling for a Cure! On 16 November 2014, the Suffolk chapter of the rich heritage and traditions of the UAE. Other Cooley’s Anemia Foundation (CAF) held a “Bowling activities that kept the patients and their families for a Cure” event at Bowlmor Lanes in Melville, entertained were the magic show, the henna stall, the arts and crafts corner, and fun games for both New York. Organized by Sarah and Mike Connolly, parents children and adults. Under the leadership of the President of the UAE, of Zayna, an adorable little girl with thalassaemia, this was a marvellous way to have fun while raising His Highness Sheikh Khalifa Bin Zayed Al Nahyan, funds that help CAF maintain its important medical research, patient services and education/awareness programs. Eastern Mediterranean Region (EMR) Egypt The International Pediatric Hematology Conference of the Health Insurance Organization was held on 16 October 2014 in Cairo. It was the first such conference to be held unde the umbrella of the

MAY 2015 www.thalassaemia.org.cy

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authorities each presented information related to their ongoing thalassaemia-related projects, while the Food and Drug Administration (FDA) was able to offer general observation and comments on the issues that were discussed. The most interesting part of the day was the open discussion in the afternoon. The conversation revolved around some of the major themes that emerged throughout the day, including current management strategies (e.g. transfusion/chelation and stem cell transplant), evolving management strategies (e.g. matched unrelated transplants, gene therapy) and experimental management strategies (e.g. novel chelators, gene editing). Further more, this year, CAF is organising the “Celebrating Self Empowerment” event from 26 to 28 June 2015, at the Wyndham Hamilton Park Hotel and Conference Center. During this event, CAF will ask patients to submit a photo and enter CAF’s story-telling contest. All the submissions will be published in CAF’s Conference newsletter. In addition, CAF will select one of the submissions received by 6 June 2015 and will ask the winner to share his/her story at the Conference. Registrations are now open for this event! Deadline is on 7 June 2015. For complete information, please go to www.bitly.com/PFC2015.

His Highness Sheikh Mohammed Bin Rashid Al Maktoum, Vice-President, Prime Minister and Ruler of Dubai, His Highness Sheikh Mohammed Bin Zayed, Crown Prince of Abu Dhabi and Deputy Supreme Commander of the Armed Forces, and Their Highnesses, the Supreme Council Members and rulers of the Emirates, it is worth mentioning that the UAE is one of the pioneers in providing excellent support services for the thalassaemia patients throughout the Emirates. We hope that these efforts will flourish with even more success within the spirit of a healthier and happier future for UAE thalassaemia patients. Local Conference: “Challenges in the Management of Thalassaemia Major in the United Arab Emirates (UAE)”, November 15, 2014 Renowned experts in the field of haemoglobinopathies addressed the latest innovation advances and challenges regarding thalassaemia major in UAE during an event titled “Challenges in the Management of Thalassaemia Major in UAE”, which was held at Jumeirah Emirates Towers, Dubai, UAE, on 15 November 2014.

the thalassaemic Palestinian refugees in Syria. Many of them have been displaced from their homes to temporary camps inside Syria and in neighbouring Lebanon. Furthermore, the TPFS has carried out important work in Lebanon by visiting Palestinian Camps there to investigate the situation of thalassaemia patients in that country and the quality of medical services provided to them, with the view to support and improve the conditions in the area in as many ways as possible. Several meetings took place with many national and international health organizations. Among these organizations was the Lebanese Chronic Care Centre, which already has several thalassaemia patients in its care. In this context, an agreement was signed to initiate the development of a registry of all Palestinian thalassaemia patients in Lebanon. A second agreement of partnership and cooperation was signed with the Lebanese Voluntary Work Centre to improve the medical services provided for the Palestinian thalassaemia patients in Lebanon.

European Region (EUR) Azerbaijan As a result of the joint efforts of TIF and involved stakeholders in the country, important progress has been made in Azerbaijan. According to information from the Central Blood Bank, about 1400 patients with thalassaemia Palestine We are proud to share some very important news are registered there and are supplied with from the Thalassaemia Patients’ Friends’ Society donor blood by this Blood Bank. There are also (TPFS). The TPFS has carried out many activities divisions of the Blood Bank in 7 regional Centres aimed to support thalassaemia patients throughout of the Republic of Azerbaijan. Patients receive Palestine including the Gaza Strip. These activities main treatment in the Republican Centre of covered many aspects: health, social, education, Thalassaemia in Baku. More than 800 patients receive their regular blood transfusions at this awareness and blood donation campaigns. More specifically, the TPFS has delivered to Centre. Since May 2014, transplantations of bone the Palestinian Ministry of Health a shipment from the Emirates Red Crescent, which contained marrow are performed in the Republican Centre iron chelation medicaments, other medicines and of Thalassaemia. 9 children and adolescents were supplies, such as filters, blood bags and other items. successfully operated in 2014. The Government of Part of these items have been offered to patients Azerbaijan meets all costs of the operations. Congratulations to our members in Azerbaijan in the Gaza Strip. TPFS has also joined its voice with the Palestinian for all the work accomplished. We are confident Red Crescent to donate medical supplies or money that things will move forward in a fast pace in that to the Red Crescent in Syria, in order to tend to country.

MAY 2015 www.thalassaemia.org.cy

TIF exerts an effort to present these stories in an as authentic form as possible. .

My name is Namitha and I am from Bangalore, India. Last year, in August 2013, I nearly lost my life as I I want to share my story to empower and enable others suffered from liver failure following a 3-week viral flu. to express themselves. I am now a PhD student working I remained in a hepatic coma for 4 days but came out of on disability and psychology at the National Institute it alive and kicking! I had a lot of complications at this of Advanced Studies, Bangalore, India. I have come very stage, and I needed 5 units of blood transfusion. My liver far from where I began, having been born with beta- functions had sky-rocketed and I was living on IV fluids. Somehow I fought back and came home in September. thalassaemia. I was four years old when I was first diagnosed with I knew I had to stay alive to complete my PhD. beta-thalassaemia. I was falling sick frequently and my With support from my family, friends, colleagues and heart beat was very slow. Finally, I was diagnosed with professors I bounced back to full living. At this stage thalassaemia by haematologists, who had put together a I realized that I should justify my life on Earth by line of treatment for me with monthly blood transfusions. joining the crusade against genetic disorders. Finally, I had undergone splenectomy in Switzerland when I was I no longer felt angry about letting people know that 5 years old. I had to continuously take antibiotics for a I had thalassaemia. I joined hands with others with year after that, as the splenectomy affected my immune genetic disorders as we all share dysfunctional genes. I interact with people who have Hemophilia and other system. genetic issues. I feel a strong kinship with them and I Despite the fact that I had thalassaemia and my life hope for treatment and cure for all genetic disorders. revolved around visits to hospitals and doctors, I had a fairly happy childhood. Although I couldn’t join in all From the time I was diagnosed with thalassaemia activities at school or at play, I managed to participate until now, my family kept up the search for a cure. My grandfather, a Chemistry professor, wrote to a number in many games and fun activities. of doctors and researchers in the US, including the Sometimes I was very angry when I couldn’t play like controversial Dr Martin Cline. the other children but soon I discovered other things to do. I was good at public speaking so I participated in Hope dances like a star above the horizon. Gene debates and speech competitions. I completed my school therapy for thalassaemia is in the first phase trial. The retroviral-mediated globin gene transfer has been successfully and joined college. successful in inducing a fully functional globin gene I took up a Master’s Degree in English. thereby, cutting out the need for blood transfusions. Thalassaemia in adults has its own share of I am thankful to Dr Michel Sadelain and other doctors complications and I went through a severe crisis due to and researchers who continue on this disease. an endocrine disorder. In 2006 I first realized that my life I am hopeful of being cured inresearch my lifetime along with was going to be very different from others. Until then I my other friends. I long for the day when gene somehow thought that thalassaemia would not affect becomes a reality. If there is something that Itherapy me and that I could be like others. I finally realized the like to tell people with thalassaemia or with anywould other truth that thalassaemia is a condition that has to be genetic disorders, it is this: accepted. I got diabetes due to transfusion hemosiderosis Do not be defined and in 2007. controlled by whatever My endocrinologist has been very supportive and disorder you have. extremely good with my endocrine care. He encouraged your limitations butKnow me to get out of my limitations and do other things. know that limitationsalso are I was working with Target Corp (US retail chains) in there to be defied! Go out Bangalore during this time and I started taking interest and do whatever you in taking on activities that would benefit the larger in life and whateverlikeis community. I won the Best in Community Award in 2007 possible for you to do. for the activities I undertook. Don’t be controlled by In 2010 I began my PhD programme. I am now in my your genes final year and I have done very well so far. I did field work with people with disabilities (physical and sensory) and found that we share common narratives, narratives of struggle, pain, faith, and hope. MAY 2015 www.thalassaemia.org.cy

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Patient’s Story

TIF’s e-Applications

A first glance at TIF’s new digital library App! TIF has undertaken an exciting new project revolving around the development of a mobile application for iOS and Android devices. The move into the mobile space attempts to create a more “immersive” experience for users, providing them with a powerful platform containing all of TIF’s publications and informing them regarding all of TIF’s events and news. This application enters the digital age in a very clever and compelling way, offering users a simple way to have TIF’s publications available on all of their devices at any place and any time. It will consist of a digital bookshelf and it will enable the users to select a publication, download and read it, providing some stunningly-looking interactive features that harness the touch controls of mobile phones. The application will also allow users to be informed regarding the latest news and events that TIF has planned through push notifications, alerts, and a digital calendar with all upcoming and past events. More information will soon be available! TIF finally launches ThaliMe, its ground-breaking New App! It is with immense excitement that we share with you that the ThaliMe app, a unique exciting new mobile application that promises to aid and improve the lives of people living with thalassaemia, is now available for downloading at the Apple Store! It has already been officially presented during the 4th PanEuropean Conference on Haemoglobinopathies and Rare Anaemias, held on 7-9 November 2014 in Athens, Greece.

MAY 2015 www.thalassaemia.org.cy

ThaliMe is a much needed innovative mobile program, developed and deployed to greatly aid and improve the lives of people living with thalassaemia, ensuring value, ease of use and applicability. TIF views this innovative approach to patient care and the use of mobile technology for patient empowerment and outreach as a critical component of the overall thalassaemia care. ThaliMe gives people living with thalassaemia, their families and caregivers, a private mobile support network and a set of tools to simplify daily management and monitor overall health. ThaliMe is easy to use, personalized and provides users with helpful tools to manage everything from medication reminders to appointment scheduling, from mood and mobility levels to transfusion dates and access to the latest research. It provides an easy to use, simple health tracking and information management functionality that eases the daily challenges of this disease’s management. Among its many features are data visualization tools that translate health tracking into visual format, thus providing an easy and motivating way to chart personal health. Medication and appointment reminders are also available to encourage adherence and timely care. TheThaliMe also helps to connect the worldwide thalassaemia community members to one another and to those that care for them. Through ThaliMe, patients are linked to private, peer-to-peer and peer-to-caregiver support networks to reduce isolation and improve the patients’ sense of support. Patients dealing with similar difficulties will be able to bond with each other, as well as manage the daily tasks associated with caring for themselves, such as medication tracking and appointment reminders. Download our app at the Apple store now!

We are thrilled to present our Video Challenge Competition winners! We are so excited to have received so many original submissions that blew us away! Each video highlights the story behind great individuals affected by thalassaemia, whether patients or parents, and the incredible struggles they have endured to achieve the greatness they exemplify today. All the stories were so inspiring and unique in their own way that it was nearly impossible to choose an absolute favourite. We are delighted to have encouraged patients and their parents, friends and family to take a step forward with us! Choosing the winners was a long and difficult road. The stakes could not have been higher. The contest’s winners have been determined using a voting system implemented within the video challenge platform allowing voters to award points to their favourite submissions. The submissions awarded the most points by voters were declared winners and are presented below:

Winner of the First Prize - 500 USD Name: Marsha De Salvatore • Title: Comedy Therapy Description: The inspiring story of a patient and a stand-up comedian who talks about the importance of laughter in dealing with the hardships of this disease.

Winner of the Second Prize - 300 USD Name: Josephine Bila • Title: Thalassaemia or Not, We Are All Born Magnificent Description: Josephine Bila is an author, speaker, and life coach who shares her story about finding inner strength, courage and power to live as fully as possible in the hopes of inspiring others to think positively about their life.

Winner of the Third Prize - 200 USD Name: Shaheen Essani Rao • Title: Zaid and His Signatures Description: This is a video of a thalassaemia major patient who passed away on 2 October 2013. This video comprises clips of different T.V. shows in which he was invited to participate as a motivational speaker. We would like to thank each and every one of you for your participation. We could not have done it without your help! We believe many patients will benefit from your stories and your inspirational words. It is so important to spread this message to all patients of the Thalassaemia community. We truly believe that your stories have served as the stepping stone for many patients to open up and tell their own story. We appreciate each and every one of you who decided to submit videos for this competition. Hear their voices on our website! To view the submitted videos, please visit the following link: www.thalassaemia.org.cy/video-sharing-platform.shtml MAY 2015 www.thalassaemia.org.cy

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TIF video sharing platform: Announcing the Winners of our Video Challenge Competition!

Other News The ENERCA White Book - Recommendations for Centres of Expertise in Rare Anaemias out NOW! The ENERCA White Book is an extended position paper, developed as a deliverable of the ENERCA 3 project. It intends to contribute to the creation of a European Reference Network in Rare Anaemias (ERN-RA) by providing recommendations and, in particular, the definition of the criteria that Centres of Expertise (CoE), local centres (LC) and their interrelations have to fulfil as healthcare providers. It has been nourished by all the activities that have been performed over the past ten years within the ENERCA framework. The White Book is addressed to authorities in charge of identifying CoE, as an essential requirement of the promotion of the ERN. It is also addressed to the patients, as a way to empower their community in this process. One particular characteristic of the White Book is the integration of the three main aspects of a Centre of Expertise: a) ethical and legal frameworks to ensure the non-discrimination and nonstigmatisation of rare disease patients across Europe, within their sphere of competencies; b) clinical and laboratory frameworks for defining technical and quality criteria including scope, general and disease specific elements currently defined as technical and professional standards for the diagnosis, treatment and follow-up of patients with rare anaemias; and c) the expectations patients have of CoE. Conceived as a working tool directed to a broad range of stakeholders, the White book has been designed and structured to be comprehensible even to non-technical and /or non-professional audiences. The results of the CATGLOBIN newborn screening for sickle cell disease pilot study in Catalonia have led to the imminent implementation of universal neonatal screening for haemoglobinopathies CATGLOBIN, a Catalan network for the diagnosis and monitoring of haemoglobinopathies and thalassaemia, is a project funded by Marató TV3 and integrated by a multidisciplinary team of 56 health professionals belonging to 15 hospitals distributed all over the region of Catalonia. Their main objective is to ensure a correct diagnosis, prevention and clinical management of sickle cell anaemia, thalassaemia syndromes and other haemoglobinopathies associated with chronic anaemia. Since the beginning of the network in 2009, the MAY 2015 www.thalassaemia.org.cy

project offers to partner institutions the possibility of carrying out the genetic study of patients through the web platform project (www.catglobin.cat). Taking into account that mortality associated with sickle cell syndrome decreases from 8% to 1% through detection in the neonatal period and the start of appropriate preventive measures, in addition with the fact that Catalonia presents the highest flow of immigration potentially carrier of sickle cell trait in Spain, CATGLOBIN in collaboration with the Public Health Agency of Catalonia, launched a universal newborn screening for sickle cell disease pilot study in March 2013 for the participating centres. The program came to an end in March 2014 with more than 26,000 newborns screened (40% of the total population born) allowing the prevalence estimation for sickle cell disease in 1 per 3909 newborns, and 1 per 144 in case of sickle cell trait. The implementation of systematic and universal neonatal screening for haemoglobinopathies in the early detection official program of Catalonia has been imminent and will start in the beginning of 2015. Report is available in Public Health Agency of Catalonia website. e-HEMATimage, a specific program devoted to Continuing Education in Hematology e-MEDICINimage is a continuous training initiative for Laboratory Medicine professionals. It is promoted by the FCBM Association (Formation Continue en Biologie et Médecine) and co-funded by the European Commission. One of its Programs, “e-HEMATimage”, is devoted to Continuing Education in Haematology. Available in 9 languages (in alphabetical order: Chinese, Dutch, English, French, German, Italian, Polish, Portuguese and Spanish), it produces 10 teaching dossiers per year (from real patients). The final objective of the initiative is Improving the knowledge and expertise in the fields of Haematology, Medical Mycology and Parasitology, for all biomedical laboratories, whatever their infrastructure and technical systems in place. It is designed for all professionals (doctors, scientists, technicians) involved in carrying out and analysing the basic haematological, mycological or parasitological tests that enable an initial diagnosis to be made. We encourage you to visit e-MEDICINimage and find new information sources at www.enerca. org/activities-news/news/98/e-medicinimage-acontinuous-training-initiative-for-laboratory-medicineprofessionals

General information TIF, in the context of its Educational Programme, has launched a new initiative since 2013: a fellowship program, the “TIF - Renzo Galanello Fellowship”, in honour of the late Professor Renzo Galanello, a pioneer in the field of thalassaemia. This programme has been established in collaboration with the Whittington Hospital in London, UK, with the support of Novartis Pharma. We are pleased to announce that for a second consecutive year, this programme is offered to physicians who are specialists in the field of haematology, paediatrics or internal medicine. The training programme has been developed by Dr Farrukh Shah, Consultant Haematologist and an international expert in the field, and is held under her supervision. Dr Shah has ensured the participation of many other international experts in the faculty, comprising the multidisciplinary group essential for the management of such a multi-organ challenging disease. The selection of the candidates is the sole responsibility of the Selection Committee set by Dr Shah and is based on agreed criteria. Fellowship Programme Duration: The fellowship is of a 2-4 month duration (depending on the level of knowledge and needs) for one or two physicians per year. Venue/Training Centre: Whittington Hospital NHS Trust, Magdala Avenue, N19 5NF, London, UK. Starting Period: The course begins each September. Applications: Begin on 1st of January and close on the 20th of April every year. Successful candidates will be announced on the 8th of May every year. Funding: i. A stipend of $2000 USD/month for each successful candidate that intends to cover the successful candidate’s accommodation, transportation and other living expenses. Any expenses above this amount will be the responsibility of the successful candidate. ii. A fee of up to $500 USD for each candidate to the training centre for its administrative needs. iii.In addition to the above, the Fellowship will cover each successful candidate’s airfare (economy class) to and from his/her country.

How to apply The Application Form is available on TIF’s website The Application Form should be completed and returned to the Thalassaemia International Federation by email, at thalassaemia@cytanet. com.cy or tif@thalassaemia.org.cy, or by fax at +357 22 314552, with the subject line “TIF-Renzo Galanello Fellowship”. In the event you cannot use email or fax, a postal address is denoted on the Application Form. Candidates interested in applying can find the conditions and the application forms on our website at www.thalassaemia.org.cy/educational-programme/ renzo-galanello.shtml Testimonials of the first two fellows that have finalised the first round of the fellowship programme “With this training, offered by TIF, I will be able to develop a Centre of Excellence for Thalassaemia patients in Chittagong. I further hope that additional training and support will help us develop in human resources in order to give the utmost care to our patients. During my tenure, I had the opportunity to obtain experience, knowledge, skill and information about the management of Thalassaemia, which I can implement in my country, that is, Chittagong, Bangladesh.” Prof Mahmood Ahmed Chowdhury (Arzu) MBBS, DCH, FCPS (Paed), FRCP (Edin)

“I wish to sincerely thank you and the entire management of TIF for seeing me worthy for the TIF - Renzo Gallanello Fellowship under the able tutelage of Dr Farrukh Shah at Whittington Hospital. I must say that I had a great time observing and interacting with managing staff on subjects pertaining to haemoglobin disorders. There is no doubt that my sojourn at the Whittington Hospital and observership under Dr Shah will go a long way in improving the management and outcomes of our patients with thalassaemia and sickle cell disease.” Prof Hafsat Rufai Ahmad MBBS (ABU) FWAC Paed, MPH (MMU), Senior Lecturer/Consultant Paediatrician

MAY 2015 www.thalassaemia.org.cy

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TIF Renzo Galanello Fellowship, with the support of Novartis Pharma

MAY 2015 www.thalassaemia.org.cy

EVENT

VENUE

18-20 April

Conference: ‘The Laboratory Medicine: Digging for More Treasures’, organised by the Egyptian Society of Laboratory Science

Cairo, Egypt

22-26 April

International Liver Congress (ILC) 2015

Vienna, Austria

2-3 May

Meeting of the Board of Directors of the Thalassaemia International Federation

London, UK

5-7 May

8th of May Conference

Tanta and Cairo, Egypt

6 May

Formal Musical Evening and Gala Dinner “Precious Rubies” Nicosia, Cyprus

8 May

International Thalassaemia Day

8-9 May

2015 Pan-Hellenic Informative Event for Thalassaemia, organized by the Greek Thalassaemia Association (EOTHA) and the Thalassaemia Association of Ioannina, to be held under the auspices of TIF

Kalamata, Greece

18-23 May

68th World Health Assembly - Official Ceremony of WHO Prizes

Geneva, Switzerland

28 May

6th Annual General Meeting of the European Myasthenia Gravis Association (EuMGA)

Madrid, Spain

28 May

European Organisation for Rare Diseases (EURORDIS) Council of National Alliances (CNA) Meeting

Madrid, Spain

29-30 May

European Organisation for Rare Diseases (EURORDIS) Membership Meeting

Madrid, Spain

11-14 June

20th Congress of the European Hematology Association (EHA)

Vienna, Austria

27 June-01 July

25th Regional Congress of the International Society of Blood Transfusion (ISBT)

London, UK

26-27 Sept

TIF’s 2nd Pan-Asian Conference on Haemoglobinopathies

Hanoi, Vietnam

• T IF on ’s 2 nd P Ha Haem an-A noi , Vi oglo sian etn • T am bino Conf IF’s pat ere Ven Inte hie nce s ue rna & D tion ate al C to b onf e c eren onf ce, irm ed MAY 2015 www.thalassaemia.org.cy

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Obituaries of two exceptional personalities in the Haematology medical ield Professor Hermann Heimpel We would like to inform you of the sad passing of a distinguished researcher, teacher and patient advocate of international respect and recognition, Prof. Hermann Heimpel. Professor Heimpel collaborated with the European Network for Congenital and Rare Anaemias (ENERCA) for the last 10 years and decisively shaped the University Hospital of Ulm, Germany, as regards research, teaching and administration. On 7 October 2014, the former Medical Director of the 3rd Department of Internal Medicine (1969-1996) and former ViceRector of Ulm University,died at the age of 84. As a doctor, a highly respected researcher and an academic teacher, he was closely connected to the University’s Medical Center well beyond retirement. He was greatly appreciated among his colleagues for his scientific reputation and human qualities. Just last May (2014), the internationally renowned hematologist and oncologist was honored by Winfried Kretschmann, the Prime Minister of the State of Baden-Württemberg, with the State Order of Merit, in particular for his contribution for improved care for cancer patients in Baden-Württemberg and for establishing the Ulm Cancer Center and the State Advisory Council for Oncology. It must be mentioned that during the last ten years Prof. Heimpel had been deeply involved in the ENERCA project where he participated as an outstanding partner and a dedicated leader for the creation of the European Congenital Dyserythropoietic Anaemia (CDA) Registry. Professor Heimpel was an extraordinary personality. He shaped haematology and oncology in Germany in the past decades unlike anybody else. He was not only an exceptional doctor, curious scientist, dedicated teacher and academic visionary, but also an especially wonderful person. He was all this until his last days for all who knew him, for the employees of his hospital and for his former and present students. May the memories help his family find peace.

Professor Phaedon Fessas The international medical community as well as the global thalassaemia family across the world, mourn the death of the professor and pioneer in the filed of thalassaemia but also of haematology in general, Prof Phaedon Fessas. Professor Phaedon Fessas was born in Athens, Greece, in 1922. In 1957 he moved to the “Alexandra” hospital where, for twelve years and under primitive conditions, he pursued his research projects along with the clinical care of patients with thalassaemia and other responsibilities. This activity was subsequently transferred to Laikon hospital where Fessas has served as Professor of Medicine and Head from 1969 to 1988. During these twenty years he made his clinic an example in teaching and training of young doctors, especially in clinical haematology. The list of his original contributions is exceptionally long and includes the identification of various variant haemoglobin and thalassaemia types along with their richly informative interactions, the observation of the “fast” fraction in newborns with α-thalassaemia (later named Hb Barts), the description of the Greek type of hereditary persistence of fetal haemoglobin, the δβ-thalassaemias, Hb Pylos and others. Among these, his main contribution has been the establishment of the concept that thalassaemia is the result of unbalanced globin chain synthesis, supported by the demonstration that the inclusion bodies within the erythroid cells of thalassaemia patients represent unbound unstable α-chains which precipitate intracellularly causing premature cellular death. Moreover, Fessas has been instrumental in setting up a program for the prevention of thalassaemia across Greece and was involved in the moral and ethical issues arising from the progress of medical knowledge. Prof Fessas has been a competent physician, productive investigator and a good teacher and mentor. His scientific work has won international recognition and led to various distinctions and honors including the Gold Cross of the Order of Phoenix by the Greek Government and the Bronze Medal of the Academy of Athens. In 2013, he received the prestigious Sheikh Sultan Bin Khalifa Al Nahyan International Thalassemia Award for excellence and pioneer work in the field of haemoglobinopathies. Unfortunately, at such moments words are inadequate to describe the greatness of his soul and the magnitude of his contribution. On behalf of the global family of thalassaemia we would like to express our most sincere condolences to his family.

MAY 2015 www.thalassaemia.org.cy

TIF is delighted to announce that it is currently in the process of establishing an MSc course in Inherited Haemoglobin Disorders - Thalassaemia and Sickle Cell Syndromes (Blended mode), in collaboration with the University of Nicosia, Cyprus, and aims to offer a postgraduate academic course to health professionals in the field of haemoglobinopathies. To our knowledge, this is a unique programme with no other similar in Cyprus or abroad. The University of Nicosia (UNic) is the largest private 2 university in Cyprus and it is an independent, co-educational, equal-opportunity institution of higher education. International in philosophy, the University provides a multicultural learning environment, promoting friendship, cooperation and understanding. The University pursues excellence in education through research and high teaching 3. standards, in a continually improving academic environment. OBJECTIVES OF THE PROGRAMME The overall aim of the programme is to increase access of quality health care to patients with haemoglobin disorders (thalassaemia and sickle cell syndromes) and to support the development of effective control strategies globally through prevention and treatment, through strengthening the education, training and skills of medical specialists. The specific objectives of the programme are: 1. To develop an educational programme at the postgraduate level of Master of Science to cover the holistic care of haemoglobin disorders, addressed to medical specialists globally; 2. To utilize the knowledge, experience and expertise of a diverse range of medical and scientific disciplines from the different parts of the world, particularly Europe, where successful control programmes have been established and where wide research programmes have been conducted, for developing the programme curriculum; 3. To bring the control of haemoglobin disorders, including the components of prevention and treatment, to the forefront of the agendas of health authorities at the national, regional and international levels, including official bodies such as the World Health Organisation (WHO) and the European Union (EU), and to support their integration into established official programmes including programmes for non-communicable diseases, prevention of birth defects, or rare diseases; 4. To bring to the forefront of the agendas of professional health bodies, industry and research, the needs to allocate more resources and focus on further improvement in the care and total cure of haemoglobinopathies; 5. The components of prevention and management of these diseases will constitute an example for building up and constructing control programmes for other genetic diseases.

LEARNING OUTCOMES Upon successful completion of this programme, the graduates should be able to: 1. Demonstrate in-depth knowledge on how to treat patients with haemoglobin disorders with confidence; 2 Fully describe and utilize the knowledge on the genetics and pathophysiology as well as the natural history of these disorders in order to achieve the best possible outcomes; 3. Confidently interpret laboratory, radiological and other results both for the purposes of accurate diagnosis but also for the follow up of patients under treatment; 4 Evaluate the impact of the inherited haemoglobin disorders on the economical and public health aspects, understand their social repercussions and promote effective control programmes, including effective prevention and appropriate clinical management; 5 Work collaborate closely with and guide policy makers on developing effective control programmes; work and collaborate with official bodies, including WHO and the EU, to promote the implementation by national health authorities of the specific resolutions adopted by the World Health Assembly (WHA) in 2006 and the EU Recommendation of 2011 and/ or to promote the integration of these resolutions into the established programmes of the national health authorities for non-communicable diseases, prevention of birth defects or rare diseases.

CAREER PROSPECTS There has been a long-term request from many countries around the world for the need for the education of medical specialists in the field of haemoglobinopathies to allow the promotion of these disorders on their national health agendas and on the guidance of policy makers at the national level to develop appropriate programmes.Therefore, graduates of this programme would have employment opportunities in universities, specialized health centres, government health offices, in national associations and in international offices that promote worldwide health (such as the WHO or international associations). ACCESS TO FURTHER STUDIES We expect that a significant percentage of medical specialists who will acquire the MSc degree will have the interest, the motivation and the medical prerequisites to continue to further research and PhD. More details about this programme at http://www.thalassaemia.org.cy/ educational-programme/e-msccourse.shtml

MAY 2015 www.thalassaemia.org.cy

TIFMAGAZINE

Special focus on TIFâ&#x20AC;&#x2122;s MSc in Inherited Haemoglobin Disorders: Thalassaemia and Sickle Cell Syndromes (Blended)

Medical Corner

New developments in thalassaemia care...

The contents of the medical corner are compiled by the TIF Medical advisor, Dr Michael Angastiniotis. An effort is exerted to include each time all important documented scientific news as they emerge from public research studies or clinical trials. We are open to receive any scientific news you are aware of which have not reached us! Please contact us! Tell us what you want to know more about, what you would like to see published or sent to you privately! Patient care so far has been following a routine of several decades involving blood transfusion, iron chelation, monitoring for complications and managing complications.The only way to ‘cure’ is stem cell transplantation which can benefit a minority of patients. At last, now there are visible indications that this routine may change in the coming few years. Research has brought us nearer to both cure and to other new approaches, already in clinical trials, which may drastically change both prognosis and quality of life. This text summarises some of these developments:

Balancing the globin chains The pathology of beta thalassaemia is based on the fact that beta globin chains are not produced or are severely reduced and so alpha globin chains are left in excess with the result that they cause apoptosis (cell destruction) in young red cells in the bone marrow. In order to correct or minimize the globin chain imbalance in thalassaemia, there are several approaches: Increasing Fetal Haemoglobin Hydroxyurea, which has found an important role in sickle cell disease management, is disappointing in thalassaemia. A group of drugs that affect the expression of the γ-globin gene have been the focus of research for many years.These are the Histone De-Acetylase Inhibitors (HDAC) which include butyrates, decitabine, and many others. In clinical practice these substances seem to have a place in non-transfusion dependent thalassaemia (NTDT) but have not, as monotherapies, a major impact on transfusion dependent thalassaemia (TDT).

Blood transfusion New sources of blood: Stem cell generated blood: Human haematopoietic stem cells, derived from various sources such as peripheral adult or cord blood or human embryonic stem cells, can actually be expanded in culture, using cytokines and other factors such as dexamethasone and oestradiol, in order to produce mature red cells on an ‘industrial’ scale. This will help to meet the challenge of donor blood shortages especially of rare blood groups. These ‘young’ red cells have additional advantages: Factors regulating red cell maturation they are rich in fetal haemoglobin and are viable for a longer Janus Kinase Inhibitors: Janus Kinase (JAK2) is a gene that period in circulation and so will increase transfusion interval. regulates the production and maturation of blood cells. Inhibitors of JAK2 might reduce ineffective erythropoiesis and marrow Artificial blood: A new product called Sanguinate, has hyperplasia which are seen in β-thalassaemia. This hypothesis has been developed in the USA by a company called Prolong been confirmed in studies with mice, and preliminary findings Pharmaceuticals. This product has already completed phase 1 suggest that the combined use of a JAK2 inhibitor and blood trials with no serious side effects. Phase 2 studies of efficacy transfusion is superior to either treatment alone in ameliorating in sickle cell disease are ongoing and beta thalassaemia is one ineffective erythropoiesis. of the target diseases. Hepcidin: Hepcidin is well known as the main regulator of iron metabolism but it also has a role in red cell production through the Blood safety: Pathogen inactivation: trials in thalassaemia of S-303, a Erythropoietin/JAK2 pathway. Hepcidin plays a role in regulating product which can inactivate a broad spectrum of pathogens erythropoiesis and ineffective erythropoiesis has a role in hepcidin in donor blood, are ongoing in several centres. Treatment in regulation, so the two are closely interconnected. Hepcidin, the blood bank with S-303 not only is effective in ‘cleaning’ in experiments involving mice, has been shown to reduce iron the blood but also preserves red cell quality. S-303 is an overload but also improve erythropoiesis. Activin receptor ligands:Activins are proteins which belong to a alkylating agent which binds nucleic acids (DNA & RNA) present in all nucleated cells in blood, including contaminants family of factors that regulate red celldevelopment and maturation. such as viruses and bacteria, without destroying red cells This group of factors are collectively known as Transforming Growth Factor-β (TGFβ) and include growth differentiation which are enucleated. factors. Two substances from this family, Sotatercept (ACE 011) and Luspatercept (ACE-536) regulate late-stage erythrocyte (red Iron chelation: Two new iron chelators are in the pipeline: blood cell) precursor cell differentiation and maturation. This HP 602 (formerly FBS 0701) by Shire Pharmaceuticals, which mechanism of action is distinct from that of erythropoietin (EPO), was in phase II trials until March 2014, was placed on clinical which stimulates the proliferation of early-stage erythrocyte hold as Shire was evaluating nonclinical (in rats) toxicology precursor cells. More detail is provided in the next article. findings. Sideris, another company, has a clinical candidate, SP-420, which is designed to be a “best in class oral iron Gene therapy chelator”. Recruiting for phase I trial started in October. Both Following the first reported success of gene therapy some these compounds are desferrithiocin based and can be given years ago in a patient with HbE/ beta thalassaemia (who is still transfusionfree and without any adverse consequences), gene orally on a once daily regime. therapy has made an impressive entry in human trials. Two trials MAY 2015 www.thalassaemia.org.cy are now ongoing and early results are encouraging.