PRESIDENT’S ADDRESS
“... Our focus is ever on knowledge, because knowledge is power. This is also the motto of this year’s World Thalassaemia Day...”
TIF President Panos Englezos Dear friends, Welcome to issue 58 of TIF Magazine, the last one of 2010. Another year has passed, filled with activities and achievements. As always, our purpose is to keep you informed and updated on all topics touching on thalassaemia, providing you with news and announcements about medical and technological advancements, educational events and publications. Our strength lies in unity, and the goal of our magazine is to strengthen the ties of the international thalassaemia community by sharing stories and knowledge. Our focus is ever on knowledge, because knowledge is power. This is also the motto of this year’s World Thalassaemia Day, and it holds true for each and every patient across the world. The theme of knowledge runs through most of 2010’s conferences and publications. Empowering patients through transparency of information on treatment and drugs is a welcome and long-awaited turn in European and international health policies. At long last, the active participation of patients in the decisions affecting their lives has become the centre of international attention. For TIF this is significant news, since for us patients have always been the driving force behind our efforts and hopes. In this issue, you will find a variety of articles focusing on transparency of information. In fact, the magazine itself is a reflection of this theme. Its purpose is to keep you informed through articles on medical advancements and breakthroughs, reviews and news on thalassaemia from every corner of the world, and policies and directives issued by every major health organisation. Like previous years, 2010 was an extremely busy, but also rewarding year for TIF. It has seen many important educational events across the world, including workshops, conferences and delegation visits in countries such as Morocco, Algeria, Malaysia, and Russia, with more visits and workshops coming up in the final months of 2010
to China and Thailand, and support provided to other countries such as Egypt, Pakistan, Cambodia, Afghanistan, Romania, Bulgaria, Saudi Arabia, Trinidad and Venezuela. Both in Europe and elsewhere, we have worked with our network of partners on monitoring and developing health-related policies, especially on chronic and rare conditions. We have expanded our collaboration with European and international health organisations, such as the World Health Organisation (WHO) and European Public Health Alliance (EPHA), we have met with the new European Health Commissioner to promote haemoglobinopathy issues, and have taken an active role in the establishment of the first national alliance of patients with rare diseases in Cyprus (CARD) of which TIF is also a member. Our preparations for the 12th International Conference on Thalassaemia and Hemoglobinopathies and the 14th TIF International Conference for Patients and Parents in May 2011 in Antalya, Turkey, are ongoing and nearing finalisation. You can find out more about the conference and the venue, as well as the updated programme, in the centrefold pages. We hope to see many of our readers there. I would like to extend my sincere gratitude to our collaborators – medical advisors, patients, and friends – and to all our members around the world: You are the reason the Federation exists, and in 2011 we will drive ourselves even harder in our mission to ensure a good life and access to quality health care for every patient with thalassaemia in the world. As always, keep visiting the TIF website to learn about our the latest news and events. On behalf of the TIF Board of Directors and staff, I would like to end by extending the season’s greetings and wishing you all a peaceful and happy new year 2011.
Panos Englezos TIF President
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CHIEF EDITOR’S ADDRESS
“...Our goal is always to place you, the patient, in centre stage for every activity that we undertake....”
Chief Editor Dr Androulla Eleftheriou A warm welcome to all our readers. As you thumb through the pages of the current issue of TIF Magazine, you may notice that it is more attractive and interesting than ever. In our ongoing efforts to create a streamlined magazine that is easy on the eyes but also highly informative, we have created this news-packed edition, knowing that every year it reaches more patients worldwide and empowers them with knowledge on their condition. As we continue to adapt to your needs and wishes, we hope for and greatly appreciate your feedback and reactions. This year has been busy and exciting as we strove to reach higher goals than ever. In this issue you can read about TIF’s activities in different countries, ongoing projects and collaborations with health organisations, and upcoming events and plans of action for 2011 and beyond. The programme of TIF’s active involvement in delegation visits, workshops and meetings was rich and multifaceted, ranging from medical conferences to patients/parents’ organisations workshops, meetings with health officials on the European and international levels, to conventions of executive committees of health organisations and press conferences. Every act undertaken by TIF aims at improving the life of thalassaemia patients around the world. In the framework of our internationally recognised educational programme, TIF organises several conferences and workshops each year at local, national and international levels. TIF is currently finalising the programme and organisational details of its largest and longest-established international event, the 12th International Conference on Thalassaemia and Hemoglobinopathies and the 14th TIF International Conference for Patients and Parents, which is expected to attract hundreds of delegates from all corners of the world. Another aspect of TIF’s educational programme consists in its
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extensive range of educational materials, which include the TIF Magazine, but also books and leaflets which have so far been translated into up to 20 different languages. A new publication, due to come out by the end of this year or beginning of the next, is TIF’s delightful cartoon for young children about thalassaemia – because not only adults need to be knowledgeable about and aware of their condition. As you can see, our goal is always to place you, the patient, in centre stage for every activity that we undertake. In addition to new publications, several new translations of TIF’s publications have been completed and can be downloaded from our website and/or ordered in hard copy from the TIF office. My sincere thanks to those dedicated volunteers who have undertaken to translate our publications – we could not have done it without you. This issue of our magazine brings the sad news of the loss of a wonderful friend, supporter and visionary of our global thalassaemia family – Prof Calogero Vullo, a great figure in the field of medical research in thalassaemia, who passed away on June 22, 2010. This issue of TIF Magazine is dedicated to his memory. I look forward to working with you, and for you, in the coming year, and I hope that you will continue to work with us, giving us your feedback and suggestions. Wishing you a happy and productive new year 2011.
Dr Androulla Eleftheriou Executive Director
EDITORIAL TEAM
EDITORIAL TEAM Chief Editor Androulla Eleftheriou, PhD Editor Chrystalla Thoma, PhD Contributors Dr Michael Angastiniotis Shobha Tuli Aaron Cheng Dr PM Kutty Dr. Waleed Bin Azhar Ijaz Ali Founder Lt. Gen(R) Fahim Ahmad Khan Kashif Mulla Dr Shaimaa Kider Al-Jalad Altaee Thair I. Khalel Noor Alam Maria Liza Naranjo Tanushree Chatterjee Ayesha Mehmood Prof Vincenso de Sanctis Afshan Shirkavand Kareem Karassery Shakeela Bashir Giuliana Ferrari
TIF MAGAZINE
Thalassaemia International Federation PO Box 28807, 2083 Nicosia, Cyprus Tel: +357 22319129 / 22319134 Fax: +357 22314552 E-mail: thalassaemia@cytanet.com.cy www.thalassaemia.org.cy Designed by Team Up Creations Ltd Printed in Nicosia
TIF BOARD MEMBERS Panos Englezos - President (Cyprus) Shobha Tuli - Vice President (India) George Constantinou - Secretary (non country affiliate) Riyad Elbard - Treasurer (Canada) Katrina Demetriou - Assistant Treasurer (UK) Anton Skafi - Assistant Secretary (Palestine) Fatemeh Hashemi - Board Member (Iran) Robert Ficarra - Board Member (USA) Mouna Haraoui - Board Member (Lebanon) Christina Stephanidou - Board Member (Greece) Ramli Mohd Yunus - Board Member (Malaysia) Dawn Adler - Board Member (non country affiliate) Michael Michael - Board Member (UK) Musa Zenelaj - Board Member (Albania) Nailya Guliyeva - Board Member (Azerbaijan) Gargi Pahuja - Board Member (USA) Loizos Pericleous - Board Member (Cyprus) Manal Safwat Shoukry - Board Member (Egypt) 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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November 2010 6_
Board Members 6_
TIF Board of Directors
10_ TIF Projects 10_
Projects Updates
12_ Around the World 12_
World Thalassaemia Day
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Association Reports
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The World
27_ 12th International Conference on Thalassaemia and Hemoglobinopathies 28_
Scientific Programme
36_ Feature Articles
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Patients’ Stories
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Obituary of Prof Vullo
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TABLE OF CONTENTS 33_
Distinctions to four health professionals
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Every crisis comes with an opportunity
36_ International / European Organisations Corner 36_
WHO
NEWSLETTERS 39_
WHO
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IAPO
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EPHA
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EMA
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EURODIS
45_ Upcoming Events 45_
Events Calendar
46_ Medical Focus 46_
Gene Therapy
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Iron Chelation
48_ Important Information for Patients 48_
Viral Hepatitis
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New Device Announced
51_ Registration / Accommodation Form for the 12th International Conference on Thalassaemia and Hemoglobinopathies
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BOARD MEMBERS CORNER
“These are individuals who through their work and commitment have made a difference in the lives of patients with thalassaemia”
TIF Board of Directors Currently, the Board of Directors of TIF numbers 18 members from 12 countries. These are individuals who through their work and commitment have made a difference in the lives of patients with thalassaemia not only in their countries but also through their participation in TIF’s activities across the world. In every issue, we will highlight three or four members with a brief biography and their contribution to the national and international thalassaemia cause. In this issue, we feature Panos Englezos (President), Shobha Tuli (Vice President), George Constantinou (Secretary), and Fatemeh Hashemi (Member).
Federation, and since then he has served as TIF’s chairman/President. Not content to stop there, Mr Englezos has travelled extensively promoting TIF’s objectives and mission, establishing thalassaemia associations, and promoting improved medical care, international research and prevention programmes. He chairs and actively participates in all TIF’s conferences.
Panos Englezos, President Panos Englezos is a graduate of London University in Economics and accounting, and Chairman and Chief Executive of a group of companies which he founded in 1961. In 1969, he promoted the establishment of the Cyprus Thalassaemia Association and was elected its first president, a post which he held until June 1995. Since then he has been voted and nominated President Emeritus (honorary) of the Cyprus Thalassaemia Association.
In 1986, he promoted the establishment of the Thalassaemia International Federation. His involvement was decisive for the foundation of the
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For his pioneer role in the field, Mr Englezos has received several awards, such as the District Governor’s Appreciation by Lions International Gold Medal in 1982, the Greek Haematological Society and Greek Thalassaemia Association’s Silver medal “Kilikas” in 1993, the King Abdulazis University Award in 1993, the Gold Medal of the Cyprus Thalassaemia Association in 1996, and the Rotary Paul Harris Fellow for outstanding contribution in 1997, among others. One of the most successful businessmen in Cyprus, with a history of numerous awards in his list of successes, Mr Englezos has held important public positions, but has also had invaluable contributions in public institutions, NGOs, and charity organisations. These include the chair of the Cyprus Telecommunications Authority, the chair of the
BOARD MEMBERS CORNER Cyprus Publishers Association, the chair of Cyprus Industry Society, the chair of the Cyprus Tourism Organisation, and the presidency of the Rotary Club Nicosia. Mr Englezos is a member of a number of national committees and advisory boards at the level of the Ministry of Health. Of particular note here is the fact that Mr Englezos was a founding member and member of the Board of Directors for two terms of the Cyprus Institute of Neurology and Genetics, an internationally recognised academic research institute. He has also been the first president and founding member of the Pan European Blood Alliance, and a member of the Board of the European Patients Forum (2008-2010). Mr Englezos lost a son with thalassaemia on 8 May 1993. Despite his grief, he never let up on his intense efforts to ensure that other parents around the world are spared the pain of losing a child. In the memory of Mr Panos’ son and of all thalassaemia patients who have left us, that date has since been established as World Thalassaemia Day and is commemorated every year in many countries around the world.
unlimited patience and sincere commitment patients across India. Mrs Tuli has served TIF from many officer posts, including that of the President, and she is currently holding the position of Vice President on TIF’s Board. In 1999, in the course of the International Conference on Thalassaemia in Bangkok-Thailand, she received the ‘George Englezos award’ from TIF for her extraordinary services to thalassaemia patients in India and abroad. Mrs Tuli has travelled extensively on behalf of TIF and has represented TIF with utmost commitment and devotion for many years. Her vast experience on the needs and challenges regarding Hb disorders in low resource countries is invaluable and contributes significantly to the work of TIF.
George Constantinou, Secretary
Shobha Tuli, Vice President Shobha Tuli is the founder member of Thalassaemics India, established in 1987 in New Delhi. She served as Junior Secretary of Thalassaemics India until 1992. From 1993 onwards, Mrs Tuli served as Secretary of the Board of Directors of Thalassaemics India. In 1993 she supported the establishment of a thalassaemia unit at Sir Ganga Ram Hospital, in New Delhi, known as the ‘Preeti Tuli Thalassaemia Unit’, after her late daughter Preeti. Thalassaemics India received the ‘Best Social Organisation Award’ in 1998 from the Ministry of Health, while Mrs Tuli served as its secretary. Mrs Tuli has been member of the Board of Directors of TIF since 1996 representing Thalassaemics India, and later the Federation of Indian Thalassaemia Associations. Since 2000, she has been President of the Federation of Indian Thalassaemia Associations.
Her contribution in bringing thalassaemia awareness and management to the forefront at the national level is unique and she has travelled as a volunteer extensively, assisting, supporting and guiding with
George Constantinou obtained a degree in Hotel and Tourism studies in London and has had a long and successful professional career. He is currently holding a managerial post in Olympic Holidays. His contribution, passion, and undivided commitment to thalassaemia is invaluable. Since the early years, he has been involved with the Cyprus Thalassaemia Association, where he served as a member on its Board for many years, including the post of Secretary and President. He has also been among the founding members of the Thalassaemia International Federation to which he has been ever an active member and officer of the Board. He is also currently the secretary of TIF and of the UK Thalassaemia Society.
Always with the same commitment, interest, and energy, Mr Constantinou has helped TIF in countless ways. He will stop at nothing to achieve what he thinks is right and fair for patients around the world. Mr Constantinou has been involved in numerous delegations visits in various parts of the world for the promotion of haemoglobin disorders. He is an exemplary expert patient who fights with commitment and undivided devotion for the improvement of not only the treatment but the quality of life and the securing of the rights of patients for a better life. His presentations, statements, position papers, and active involvement in the activities of TIF have had a great impact. He has shown us all that a knowledgeable patient can make indeed a difference. TIF MAGAZINE_NOVEMBER 2010_www.thalassaemia.org.cy
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BOARD MEMBERS CORNER Mr Constantinou is the coordinator of a significant, recently initiated project of TIF, the Expert Patients programme, through which his immeasurable knowledge and experience on the topic can be utilised to educate, motivate and empower patients in every corner of the world to understand that knowledge is the power of patients. Mr Constantinou fights to bring hope to patients everywhere.
Fatemeh Hashemi, Board Member Fatemeh Hashemi holds an BS in Biology and is currently pursuing a PhD in International Affairs at Warsaw University. Mrs Hashemi has been the President of the Charity Foundation for Special Disease since its establishment in 1995, and has held several important posts, including Secretary General for Women Solidarity Association since 1993, Chairman for the Board of Directors of Special Medical
Centre since 2004, President of Moslem Women NGOs Communication network since 1996, and Member of the Board of Founders & Trustees of Tasnim School since 2004.
As the President of Charity Foundation for Special Disease, including thalassaemia, Mrs Hashemi has taken great efforts to promote scientific and social awareness of thalassaemia among patients and in the community, in order to draw the attention of government authorities and private sectors and to establish projects for prevention of the disease and the betterment of therapeutic and social conditions of patients. Mrs Hashemi has been a member of the Board of TIF since 1999, with an invaluable contribution in its work and mission. She represented TIF in numerous high level visits and has hosted a TIF Board Meeting in Iran. A pioneer and a visionary, she initiated the first education and awareness campaign in her country – a highly affected country as regards thalassaemia, both in the region and in the world. At the national level, Mrs Hashemi has contributed tremendously to the development and implementation of an effective prevention programme – a programme which to date is considered a model for other countries with similar risks and magnitude of problem, as well as similar religious, cultural and social constraints.
Join the Thalassaemia Family – Become a TIF Member! Is your thalassaemia association already a member of TIF? If not, it should be! TIF’s Members are part of a global family of patients, parents and medical professionals, and can enjoy many useful benefits.
Please take a moment to consider becoming a member. Each and every contribution is an invaluable expression of support for the Federation, and allows us to continue our work for the benefit of patients with thalassaemia across the world.
All members receive TIF’s publications free of charge and can participate andcontribute to its activities, particularly its internationally recognised educational programme. After one year, General Members are eligible to become Voting Members and contribute to TIF’s global policy.
For more details about the benefits of being a General or Associate Member, contact TIF at:
Non-thalassaemia related patients and other organisations, as well as health professionals and other individuals interested in supporting the cause of thalassaemia can become Associate Members.
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Thalassaemia International Federation PO Box 28807, 2083 Nicosia, Cyprus Tel: +357 22 319 129 Fax: +357 22 314 552 or by email at: thalassaemia@cytanet.com.cy
TIF PROJECTS All About Thalassaemia Thalassaemia Cartoon for Young Children TIF’s new delightful cartoon for young children (ages 310) about thalassaemia is currently in the pipeline. This book belongs to the range of educational publications of the Federation. It was written with a lot of expertise on the topic and much love for the children with thalassaemia – children who need to understand their condition, accept it, and draw strength from this knowledge to build a better future for themselves. Page after page of simple explanations, especially designed to educate children and remove their fear of the treatment, is accompanied by and illustrated with colourful, attractive characters, including Harry the red cell who is the perfect narrator for children, with his down-to-earth storytelling. Sophisticated in its simplicity and with every detail thought out with a young audience in mind, this cartoon is certain to appeal to children and help them understand their condition. The cartoon was created for and is dedicated to all children in the world born with thalassaemia – to their awe-inspiring will to survive, their ceaseless fight against the disease, and their dreams of a long, healthy, and happy life. May all their dreams come true!
TIF Epidemiology Project TIF’s Epidemiology Project is a survey for Haemoglobin disorders and their treatment globally. The project aims to identify and update information on carrier rates of Hb disorders, and calculate the affected births suffering from beta-thalassaemia expected to occur every year based on birth rate, population size, carrier rate and absence of prevention policies. Furthermore, the project aims to review and identify the healthcare infrastructure and services for chronic disease patients and more specifically for Hb disorders (including reimbursement models used at the moment) in each country. This project is nearing completion, and TIF is preparing a publication on the results with the aim of submitting it to relevant journals and periodicals over the next months.
Expert Patients Programme Part of the ongoing “Reach the Patient” project, the Expert Patients Programme aims to extend quality information to patients with thalassaemia across the world on their disease, its medical care and other forms of care – the core objective being to empower patients to take meaningful and timely decisions concerning their healthcare and quality of life. Objectives and methodology of materialisation were closely discussed in the course of the 2nd Paneuropean Conference in Berlin 2010 March. The first official meeting of the Expert Patients Programme is due to take place in London in November 2010, where these initial activities and plan will be put forward.
DOMAINE DOMAINE was a public health project aiming to increase and maintain a sufficient supply of safe blood across Europe through the promotion of regular 100% voluntary nonremunerated blood donations, facilitate cooperation between blood establishments and patient-driven organisations, and build a consensus on good donor management practices across Europe. TIF has led Work Package 6 on “testing of the manual”. This manual’s purpose is to help harmonise blood donation practices and management. The manual has now been completed and published, and it is being distributed to all participating blood establishments who have responded to our questionnaire. A training programme for the application of the manual is currently being drafted. Our hope is that the European Commission and the European Council will promote this manual and so help set a quality practice standard in the whole of Europe in the area of blood donation.
Please visit the DOMAINE website for further details: www.domaine-europe.eu
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TIF PROJECTS TSEC Thalassaemia Specific Electronic Communications TIF has just completed a project funded through the EEA Financial Mechanism/Norwegian Financial Mechanism 2004–2009 Fund for Non-Governmental Organisations - the “Thalassaemia Specific Electronic Communications” (TSEC). The project introduces electronic patient records specifically adapted to thalassaemia based on already existing software called JAnaemia. The project has also set up two new tools, building on the existing software but introducing innovative features to enhance clinical support and to improve patient care: these new modules are a serious infections register, and an adverse drug reactions record. TIF is the main coordinator of the grant, together with partners from North Cyprus and Norway. The software will first be piloted in Cyprus and refined further based on the outcomes of the pilot phase. It has already been installed in three treatment centres, and data transfer has started. The project ended with a final press conference on 27 September 2010 for the announcement of the results to all interested parties. A close follow up of the practicability, the user-friendliness level, and other strengths and weaknesses of the software will be reported every three months in the course of the next year. Final objective is for TIF to disseminate worldwide a software for electronic registry of patients with thalassaemia.
3rd European Symposium on Rare Anaemias An ENERCA – ALHETA – TIF collaboration ENERCA is organising the 3rd European Symposium on Rare Anaemias in Madrid, Spain, aiming to stimulate interest and inform colleagues on the subject of congenital and rare anaemias. At the heart of ENERCA policies, like those of TIF and the Spanish group ALHETA, are always the patients. In this respect, and specifically in the context of the 3rd European Symposium on Rare Anaemias, the three organisations have collaborated in setting up the first Spanish Symposium of Thalassaemia patients and professionals in Madrid, co-organised with ENERCA and integrated in the framework of the 3rd European Symposium on Rare Anaemias. In the Symposium, an interactive workshop has been scheduled with patient panellists and doctors, focusing not only on clinical management but also on prevention and social action for thalassaemia and haemoglobinopathies.
ENERCA: The European Network for Rare and Congenital Anaemias is a project funded by the European Commission with the main objective of the establishment of a European Reference Network (ERN) of Expert Centres (EC) in Rare Anaemias (RA).
Contribute to TIF Magazine! Are we missing important news? Do you have something to share with the thalassaemia community? Share it with us!
Thalassaemia International Federation PO Box 28807, 2083 Nicosia, Cyprus Email: thalassaemia@cytanet.com.cy Please note that although we endeavour to publish all contributions, this may not always be possible due to space constraints.
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AROUND THE WORLD
World Thalassaemia Day 8 of May World Thalassaemia Day is observed each year by the global thalassaemia community to commemorate and mourn the death of thousands of patients due to lack of medical prevention and treatment, but also to celebrate the life-saving power of knowledge and the need to raise awareness everywhere in the world.
Thalassaemia associations around the world organised varied events again this year to observe World Thalassaemia Day and mark the achievements of patients, parents and experts in their fight with this chronic condition. This year the theme of the day was twofold: encouraging patients’ knowledge about their condition, because knowledge is power and an informed patient has control over his/her choices, and sharing the knowledge through patient involvement in policy making, collaboration with other organizations
and individuals, creating associations and making a difference not only at the national but also at the international level. TIF received many reports from member associations recounting their activities on this important day. As the previous issue of TIF Magazine came out in May, and all texts need to be processed about a month before printing, we were unable to include the 8 of May events before now. We are very pleased to give you a full report in this issue.
Thalassaemia Day events held in Malabar India Contributor: Dr PM Kutty/President of THAS
THAS Society and the Blood Patients’ Protection Council (BPPC) observed International Thalassaemia Day in a superb, patient-centred programme of activities. The programme included a free serumferittin test, Hepatitis B vaccination, presentation of gifts, and distribution of a new pamphlet about the prevention program of thalassaemia, started last year by BPPC and THAS. A radio program was aired on the previous day to alert the public about the upcoming activities and to inform them about thalassaemia, its prevention and treatment possibilities. The effort paid off,
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and the turnout was high, making for an exciting atmosphere. Nearly two hundred patients and their parents participated in the events, which included various
AROUND THE WORLD presentations and games for the entertainment of the children. All patients received gifts as tokens of appreciation for their daily struggles in living with thalassaemia.
patients, adults and children, attended the programme, during which entertainment was put out by thalassemic children and professional performers.
We of TIF were very impressed with this report and wish to congratulate both THAS Society and the Blood Patients’ Protection Council on their dedication.
Chief guest Sh P.R. Sawhney, Mayor of Delhi, praised the children, urged them to believe in the future, and promised all possible help for the awareness programme of Thalassemics India. Mrs Shobha Tuli, Secretary of Thalassemics India and Vice President of TIF, presented the annual report enumerating achievements and goals, while Mr Deepak Chopra, President of Thalassemics India, introduced four adult thalassemics as patient representatives on the Board of the association, and expressed confidence that they will keep up the good work. Prizes and gifts as tokens of appreciation were given to all participants.
Awareness-raising campaign in Delhi India Contributor: Shobha Tuli/President of TI
Congratulations to the association for this full week which reflects their hard work throughout the year!
Thalassaemia Day in Karachi Pakistan Contributor: Dr. Waleed Bin Azhar/Fatimid Foundation
Thalassemics India have set themselves the mission of achieving zero thalassaemia births in Delhi and quality treatment for all patients. From the second to the eighth of May 2010, the association held a week-long intensive awareness campaign in the context of International Thalassaemia Day. As part of the campaign, Thalassemics India, Sir Ganga Ram Hospital and Apollo Hospital joined efforts in ensuring visitors to the two hospitals received information on the topic. Blood donation camps for patients were set up by IRCS at SGRH, and Keane International, Noida & Gurgoan, where 150 units of blood were collected in total. Poster, slogan and essay writing competitions on blood donation and thalassaemia awareness were held for students of Delhi schools. Furthermore, a painting competition for thalassaemia patients was held at Sir Ganga Ram Hospital, and a variety programme organised by Thalassemics India was held at Sri Sathya Sai International Centre. Over 600 thalassaemia
Fatimid Foundation commemorated International Thalassemia Day with an entirely patient-oriented programme, advertised a day earlier in the newspapers. Altogether thirty-two patients participated in different competitions and presentations. The chief guest, Mr. Pir Mazhar ul Haq, Senior Minister for Education and Literacy, awarded certificates and gifts to all participants. The Chairman of the Fatimid Foundation and President of the Thalassaemia Federation of Pakistan, Gen. (R) Moinuddin Haider, also attended the event. Large numbers of patients and their parents attended the programme, and the children were entertained with face painting, but also with puppet and conjuring trick shows.The events continued in the following day with a oneday, thalassaemia minor screening programme, blood sample collection, and thalassaemia minor screening tests organized by the Fatimid Foundation.
Fatimid Foundation’s tireless dedication to patients’ welfare is admirable and an example to us all.
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AROUND THE WORLD With pomp and show for community awareness in Peshawar Pakistan Contributor: Ijaz Ali Founder/Chairman of Hamza Foundation
Hamza Foundation observed International Thalassaemia Day with great enthusiasm, and even organised a walk in front of Peshawar Press Club. Reflecting on the theme of informing and empowering patients, the organisation also organised a seminar to discuss thalassaemia, prevention and treatment options. The message reached people from different walks of life, with patients and parents, the management of Hamza Foundation, doctors, students of different universities, colleges and schools, religious personalities, writers and poets and many other people participating in the walk and seminar. The idea behind the event was to draw attention to the meaning of this day and in this manner spread awareness about thalassaemia in the community - a very original and successful concept. The chief guest Mr. Syed Kamran Shah, Project Director of Tanzeem Lissail-eWalmahroom (Chief Minister’s Welfare Project), promised the Chairman of Hamza Foundation support and cooperation for the noble cause of aiding thalassaemia patients achieve a quality life. With the purpose of raising awareness about thalassaemia, special supplements were published in the newspapers, and messages from different celebrities were aired on several television channels.
A magic show was organised by the Hamza Foundation for the benefit of thalassemic children and their parents. At the end of the day, thalassemic children gave a beautiful presentation and the guests left in high spirits.
Chief guest was Mr Zamurad Khan, Managing Director of Pakistan Bait-ul-Mal. He praised the society’s activities and kindly offered to become Honorary Member of its Executive Committee. He announced five welfare grants and the offer of six infusion pumps for patients. He was presented with a Memorable Shield of the society, also awarded to Dr Khalif Bile Mohamud and other important medical and social workers and participants.
Lt. Gen. Fatim Ahmed Khan reported on the activities of the society over the past year, during which hundreds of patients received regular blood transfusion and chelation treatment, given for free to patients with low income. At the end of the function, young thalassaemia patients delivered fascinating speeches on their resolve to fight the disease and grow up as useful members of the community.
They also recited verses and gave presentations, displaying their full enthusiasm for life, rekindled through the support of the Thalassaemia Welfare Society. This is indeed a cause for celebration!
Young patients enthusiastic about life in Rawalpindi Pakistan
Karachi commemorates International Thalassaemia Day Pakistan
Contributor: Lt. Gen(R) Fahim Ahmad Khan/President
Contributor: Kashif Mulla Hon. Joint Secretary/Treasurer of KITCC
Thalassaemia Welfare Society invited over 250 patients and parents, as well as the local elite, the medical community and social workers to commemorate this year’s International Thalassaemia Day.
The Kashif Iqbal Thalassaemia Care Centre organised a rich programme of events in commemoration of International Thalassaemia Day. Mr Kashif Mulla, Hon. Joint Secretary/Treasurer of the centre, inaugurated the day with
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AROUND THE WORLD At the close of this full day, the humanitarian association AlEthar put a smile on all thalassemic children’s faces with gifts.
Gala for Thalassaemia Day in Mosul Iraq Contributor: Altaee Thair I. Khalel / Ninava Thalassaemia Society Chairman
the message addressed to Pakistan’s thalassaemia patients on behalf of the President of TIF, Mr Panos Englezos. The Centre conducted free public screening tests (Hb Electrophoresus) to check for thalassaemia traits. Afterwards, Thalassemic children put on stage a lovely performance, during which they danced, sang and recited poems. The guests were delighted with the children’s talent and their infectious enthusiasm.
At the end of the function, achievement rewards were presented to thalassaemia patients to let them know that on this day we celebrate their extraordinary courage in their fight against this debilitating disease.
Solemn celebration in Bagdad Iraq
Iraq’s Ninava Thalassaemia Society gala for International Thalassaemia Day was attended by the Health Official in Mosul, the Thalassaemia centre staff, doctors, and many patients and parents. The occasion was seized for the announcement of an array of good news for thalassaemia patients, among them the acquisition of a PCR unit in the Ibn al Atheer Hospital in Mosul, the initiation of a project to build a Blood disease unit in the same Hospital which would also comprise a Bone Marrow Transplant Centre, a lab, a Thalassaemia Centre and a Leukaemia Centre (expected to be ready in the next two years), and a prevention campaign starting this year under the auspices of the Ninava Thalassaemia Society.
We congratulate Ninava Thalassaemia Society on their hard work and great results!
Contributor: Dr Shaimaa Kider Al-Jalad
The Iraqi Thalassaemia Association & Thalassaemia Centre observed International Thalassaemia Day in Ibin Al-Balabi hospital in Baghdad, in the presence of thalassaemia patients and their parents, doctors, nurses, members of the Iraqi Thalassaemia Association and other humanitarian associations. Dr Shaima K.Al-Jalad gave an informative lecture about the situation with thalassaemia in Iraq, its management, and how it has developed since the opening of the first Thalassaemia centre in the Ibn-Al-Baladi Hospital in Baghdad in 1989. Dr Razeqa Al-Ebady gave a very instructive and engaging presentation about the importance of nutrition for thalassaemia patients which was received with great interest.
‘No More Thalassaemia’ from Dhaka Bangladesh Article adapted from Star Campus News
“No More Thalassaemia”. With this dynamic slogan as a starting point, the students of the Faculty of Business Studies of the University of Dhaka organised a well-planned awareness programme. The programme was supported by Thalassaemia Support Initiative (TSI), a voice for thalassaemia awareness, support and care. Multimedia presentations about the disease were conducted in all the classes of Business Studies Faculty during the whole month of April to raise awareness in the students about the disease, its cause, its consequences and ways of prevention.
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AROUND THE WORLD TSI’s admirable vision is to stabilise the number of thalassaemia patients in Bangladesh and help provide quality treatment for all.
Patients empowered through knowledge China Contributor: Tanushree Chatterjee President, Fight Thalassaemia Working Group, Southern Medical University, P.R. China
Fostering patient camaraderie and education Philippines
Fight Thalassaemia Working Group observed International Thalassaemia Day with events organised around the theme of empowering patients through knowledge. The Group organised a collage competition with the topic “Empower Thalassemics–Spread the information” with the participation of both Chinese and International students. The turnout was high, the atmosphere electric, and the day was concluded with the presentation of gifts for everyone present.
Tanushree Chatterjee, Programme Coordinator and the Group, addressed the guests and advocated patient
Contributor: Maria Liza Naranjo / Balikatang Thalassaemia Foundation
Balikatang Thalassaemia (Ba-Tha) commemorated International Thalassaemia Day with a bingo raffle and fun activities on the hospital grounds of Dr Fe del Mundo Medical Centre. The dual goal of this event, the fifth such local celebration, was to foster friendship bonds among patients belonging to the thalassaemia community and volunteers, as well as to educate them further on the subject matter. This goal reflects the theme of this year’s events: empowering patients through knowledge. The event was preceded by a variety of cultural activities coorganised with Balikatang Thalassaemia, including a concert, which aimed at raising awareness in the community.
This year marks another important step in Balikatang Thalassaemia’s fight against thalassaemia in Philippines, and we of TIF wish to express our fullest support in their efforts.
knowledge and awareness with emphasis on testing and prevention of thalassaemia.
Greece honours doctors in their fight with thalassaemia Greece From Kinisi Ethelonton Magazine for thalassaemia and voluntary blood donation
“The 8 May has been established as World Thalassaemia Day not only to remind us of the existence of the disease, but also to urge scientists and the society to take measures for prevention, treatment and cure of the disease,” stated Mr Vasilis Demos, President of the Greek Thalassaemia Federation (EOTHA).
This year, on this significant day, Greece honoured the following doctors for their dedication and commitment to thalassaemia and Sickle Cell Disease: Prof Phaidon Fessas, Prof George Stamatogianopoulos, Dr Christos Kattamis, Dr Dimitris Loukopoulos, Dr Aphodite Loutradi, Dr Dina Politi, Dr Soso Frangatou, Dr Eleni Berdousi, Dr Vasilis Ladis, and Dr Markesia Karayiorga.
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AROUND THE WORLD Thalassaemia Association Reports Impressive report of activities from Iranian Thalassaemia Society By Afshan Shirkavand
The Iranian Thalassaemia Society is a non governmental organisation acting at the national level for the improvement of public and patients’ knowledge about thalassaemia. Their goal is to raise awareness on the optimal management of thalassaemia, and to provide equal access to health facilities and quality of life for all patients in Iran. The Society organised a number of important events over the last year, including a Congress of Nursing Care of Thalassaemia in a joint action with the Iranian ministry of health and medical education on 7-8 February 2008, a Seminar of Medical Care in Thalassaemia on 15-16 January
2009 in Tehran, and an Iranian Thalassaemia Summer School on 23-24 September 2009 in Namak Abrood. Such programs are evidence of the dedication of the Iranian Thalassaemia Society in raising awareness and ensuring that the voice of thalassaemia patients is heard at the regional and also national level. Congratulations to the Society for their hard work throughout 2008-2009, and we are certain we shall receive another such impressive report for 2010! Visit the Iranian Thalassaemia Society website for more information: www.iranits-ngo.com
First Lady of Indonesia becomes an “Icon of thalassaemia” Indonesia Thalassaemia Foundation reports a wealth of activities for 2010 Contributed by Indonesia Thalassaemia Foundation The year 2010 began with a full programme for the Indonesia Thalassaemia Foundation. In January, they organised a press conference in Jakarta on social integration of patients and prevention programs in Indonesia and the establishment of Thalassaemia Celebrity Volunteers. February proved a significant month, in which the Foundation met with the Minister of Health of Indonesia and the minister of women empowerment and child protection to discuss the current status of thalassaemia patients, the government’s commitment to help them, and the declaration of 2010 as the inauguration year for social integration and prevention programs of thalassaemia nationwide. Also in February, the Foundation met with the First Lady of Indonesia and discussed the organisation of the Thalassaemia Foundation and Parent Association, which already has 20 branches in Indonesia, and the issue of thalassaemia. The First Lady stated that she is highly aware of the problems thalassaemia patients endure, and is willing to become an “Icon of Thalassaemia”. During that same month, a delegation from TIF, comprising TIF’s medical advisor and a member of the Board, visited Indonesia and met with the Foundation to investigate problems faced by
thalassaemia patients. In April, the Foundation signed collaboration accords with Indonesia's largest private laboratory in order to work together on thalassaemia prevention programs.
Mrs. Watty Ruswandi with the First Lady Of Indonesia in Merdeka Palace Photo: Ruswandi
To commemorate World Thalassaemia Day, on 8 May 2010 the Foundation conducted interviews on national TV with the First Lady of Indonesia and the Minister of Health to raise awareness about the topic, and organised a talk show about thalassaemia together with the Center Information for
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AROUND THE WORLD Student Association (CIMSA) in Jakarta. The Foundation also organised a Bike Tour for Thalassaemia in which participated 4.300 bikers, with the presence of the Minister for Welfare, the Minister of Health, and the Governor of Jakarta, in the framework of World Thalassaemia Day. Motto of the day was “Be a friend of Thalassaemia and check your blood before marriage”. A screening of blood donors was also
organised for that day together with CIMSA. In May, the Foundation celebrated 23 years from its establishment and inaugurated a new branch in Central Java, as well as a new branch secretariat office. Throughout this first half of 2010, the Foundation collaborated closely with the Patients/Parents Thalassaemia Association and worked hard to disseminate knowledge about thalassaemia across Indonesia.
A long journey for a noble cause Patients with blood disorders in India march for their rights By Kareem Karassery, General Convener, Blood Patients Protection Council Malabar
They travelled three thousand km by train, taking three days and two nights to reach New Delhi, capital of India. Most of them were Thalassaemia patients and parents, while others had other blood disorders, like Sickle cell anaemia, Haemophilia, Applastic anaemia and Leukaemia. Many couldn’t speak or understand English or even Hindi, the national language of the country. They marched in front of the Indian Parliament and formed a road blockade to demand better treatment and life for saving drugs for blood disorder patients. The march and demonstration on 29th Oct 2009 was an event organised by Blood Patients Protection Council (BPPC) under the leadership of Kareem Karassery, the General convener of BPPC. Blood Patients Protection Council is an organisation for patients with acute blood disorders like Thalassaemia,
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Haemophilia, Leukaemia, and Sickle cell anaemia. The patients’ parents have been fighting for better health care, life saving drugs, and vital rights of patients for the past fifteen years. It is in this spirit of struggle for patients’ essential rights that this event was organised.
Union Health Minister stating that he was taking the matter under consideration, and another letter, sent by the secretary of All India Congress Committee (AICC), which mentioned that the President of the ruling party had forwarded the application to the Health Minister.
The patients handed over a ten-point demand letter, including the setting up of antenatal screening facilities at
The result is a breakthrough in the health field of Kerala. Ms. P. K. Sreemathi, health minister of Kerala, declared that all blood disorder patients who have not yet reached maturity will receive free treatment regardless of income. The Central Health Minister informed the local MP that significant funds have now been allocated to the development of the oncology department MCH Kozhikkode with immediate effect.
“It is in this spirit of struggle patients’ essential rights that this event was organised” Calicut Medical College Hospital on which depend thousands of people for treatment. The ruling party of India promised to bring the matter before the president of the Indian National Congress Committee. When the patients and parents returned home, they found a letter from the
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With such impressive results, the long journey to New Delhi was definitely all worth it.
AROUND THE WORLD Malaysia: Penang Thalassaemia Society Data provided by Pulau Penang Thalassaemia Society
2010 has been a very productive year for Penang Thalassaemia Society, member of TIF.
participants to socialise amongst themselves, since camaderie has proven to be a sturdy pillar of support for everyone affected in one way or another, as patient, parent or carer, by thalassaemia.
In May 2010 the Society organised the 14th Thalassaemia Camp in conjunction with World Thalassaemia Day for thalassaemia patients, parents and carers, followed by the 15th Thalassaemia Camp in October 2010. The latter was attended and supported by a member of the TIF Board. The aim of these camps is to provide psychological support in thalassaemia management for both affected families and carers, as well as to urge
targeted at doctors, nurses and laboratory staff involved with thalassaemia.The Society is also planning their 1st Workshop in Basic Nursing Care for Thalassaemia and the 1st Counselling Workshop in Thalassaemia in Malaysia which aims at training personnel and helping young adults with thalassaemia cope with social pressures. We believe these are excellent initiatives targeting different aspects and issues of thalassaemia. Well done to Penang Thalassaemia Society for their achievements and goals.
Also in October, the Society organised the 5th Thalassaemia Workshop for Doctors, Nurses and Health Personnels. The workshop was
MSc COURSE IN HAEMOGLOBINOPATHY Make a difference in your professional life and in the lives of people affected by haemoglobin disorders! A new e-Msc course launched in 2009 by University College London (UCL) and TIF. UCL is one of the most prestigious universities in the world and one of the few to have the necessary infrastructure and expertise to teach through e-learning. This course represents a unique opportunity for health professionals to specialise in haemoglobinopathies online with minimum disruption to professional and personal lives. For further information, please visit www.instituteforwomenshealth.ucl.ac.uk or www.thalassaemia.org.cy/msc.html or contact TIF or UCL:
It is designed to meet the needs of a wide range of medical professionals, including : • medical graduates interested in haemoglobinopathy (general physicians, specialists such as paediatricians, haematologists, clinical geneticists, obstetricians/ gynaecologists, behavioural scientists)
• other healthcare professionals interested in haemoglobinopathy (counsellors, clinical psychologists, nurse specialists and midwives) SUPPORTED BY:
• science graduates interested in medical research related to haemoglobinopathy and genetics UNIVERSITY COLLEGE LONDON “MSc in Haemoglobinopathy” 88-96 Chenies Mews London WC1E 6HX, United Kingdom Tel: +44 (0)20 7679 6060 Fax: +44 (0)20 7380 9984 Email: Sc_haemoglobinopathy@ucl.ac.uk
THALASSAEMIA INTERNATIONAL FEDERATION “MSc in Haemoglobinopathy” 31 Ifigenias Str., 2007 Strovolos, Cyprus Tel: +357 22 319 129 Fax: +357 22 314 552 Email: thalassaemia@cytanet.com.cy
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Global Financial Crisis Challenging times in 2010 for Rare Disease patients in Europe Adapted from Rarediseaseblogs - International opinion on rare diseases and orphan drugs
Times of austerity always affect those who are most vulnerable. For this reason, 2010 could become a difficult year for patients with Rare Diseases. Already a number of disappointing news confirm this fear: Recently we learned that the directive on cross boarder health care will not bring the results Rare Disease patients had hoped for. On the contrary, there is absolutely no mention of rare disease patients in the final version of the proposal. In addition we start seeing examples of access to reimbursement of new drugs being delayed or denied at national level. For instance, a recently authorised orphan drug, Kuvan, developed for PKU (Phenylketonuria) patients, will not be automatically reimbursed. This drug will only be reimbursed if the local authorities of the regions decide it. This is a major threat to PKU patients who have difficulty complying with the normal very restrictive diet. In addition, the process leading to this decision violates the hard-won principles of patient involvement. The way many countries have designed their procedure for reimbursement decision implies that it is up to the drug producing company
to document the need for the drug. Therefore, patients have to rely solely on drug company representatives’ competence and commitment to provide this information, whereas those most affected, the patients and their organisations, are not consulted and have no right to contribute separate information or challenge the decision of the committee. The decision on Kuvan has now been brought to the Swedish court. But what will be the situation while waiting for a decision? At its meeting on July 2, the board of directors of Eurordis (the European Organisation for Rare Diseases) discussed to what extent governments will cut back on spending for orphan drugs. The board decided to monitor the development closely and to invite rare disease national alliances to collaborate in a survey with the purpose of identifying changes in access to reimbursement of orphan drugs. In the current political context, it is necessary that the new EU Committee of Experts on Rare Diseases soon becomes operational. Therefore it was seen with some concern by the board of Eurordis that the first meeting of this important new committee, initially foreseen for May of this year, has been postponed to 1st December 2010.
For more information visit:
www.rarediseaseblogs.net
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AROUND THE WORLD The World EUROPE President and Executive Director of TIF meet with new EU Health Commissioner John Dalli in Brussels TIF pushes to make thalassaemia a priority on the European health agenda Many of the health policies being developed and promoted prevention and treatment strategies, but also on the current at the European level are relevant for the promotion of status of epidemiology, health services, and control chronic and rare diseases globally. They form the basis on strategies in Europe. In the course of the meeting, it was which other countries can tailor their own national policies stressed that the health care system in most European to meet their own needs. Therefore, a way to promote the countries is of high quality, and therefore with small yet global voice of thalassaemia patients is by advocating their essential alterations and with coordination of the existing needs to the EU Health Commissioner. The post of the EU health services, control of thalassaemia and other Commissioner Designate for Health and Consumer Policy haemoglobin disorders like sickle cell disease can be was held until the beginning of this year by Androulla considerably improved. Vassiliou. In February this “it was stressed that the health care year a new Commissioner Commissioner Dalli expressed system in most European countries was named, Mr John Dalli. his sincere understanding is of high quality ” and interest in the above TIF’s President Mr Panos issues, and made some Englezos and TIF’s Executive Director Dr Androulla important commitments to the President of TIF, Mr Englezos, Eleftheriou met with the new Commissioner in Brussels, sending a very optimistic message to all patients with Belgium, on 5th October to discuss thalassaemia and TIF’s thalassaemia both in Europe and the world. activities in Europe. Attending was also the Adviser and former Vice President of FIODS-IFBDO (International Concluding the meeting, on behalf of the TIF Board of Federation of Blood Donors Organisations) and current Vice Directors, Mr Panos Englezos invited Commissioner Dalli to President of the Malta Blood Donors Association (MBDA), attend and address the opening ceremony of the 12th Philip M Chircop. International Conference on Thalassaemia and the Haemoglobinopathies & the 14th TIF International The Commissioner was updated on the pathophysiology and Conference for Patients and Parents on 11th – 14th of May clinical development of thalassaemia, as well as on 2010, which will take place in Antalya, Turkey.
Who is Mr John Dalli John Dalli has served as a Cabinet Minister in the Maltese Government since 1987, and as Parliamentary Secretary for Industry (1987-1990), Minister of Economic Affairs (199092), Minister of Finance (1992-1996, 1998-2003) Minister of Finance and Economic Affairs and Minister of Foreign Affairs and Investment Promotion (2004). Between March 2008 and February 2010, John Dalli
served as Minister for Social Policy of Malta.
In February 2010 Mr Dalli was appointed as European Commissioner responsible for Health and Consumer Policy. At his European Parliament hearing on 14 January 2010, he stated that the underlying theme of his work was "Patients first. Consumers first".
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AROUND THE WORLD European Health Commissioner sets out his agenda Adapted from HSCNews Bulletin June 2010
6 May 2010 The European Commissioner for Health and Consumer Policy John Dalli gave the opening speech at Active Citizenship Network (ACN)’s 4th European Patients’ Rights Day Conference in Brussels, Belgium, outlining his priorities for health. Mr Dalli stated that one of the underlying priorities of his mandate is to put patients and consumers first. Patient Safety, he emphasised, is among the main areas scheduled to receive attention from the Commission. Cross Border Healthcare was another subject of interest. “Only a few patients are aware of the right to be reimbursed for healthcare received in another EU Member State, and only a few can afford to exercise this right. I hope that Member States will soon give their green light to the European Commission’s proposal on patients’ rights in cross-border healthcare, which
will enable patients all over Europe to access safe and good quality treatment across borders – and be reimbursed for it,” he said. The Commission is also due to improve the current “highlyunsatisfactory situation” concerning
balance.” Mr Dalli concluded by adding Data Protection Legislation to the list of Commission Commission’s
concerns: “The Data Protection
“Only a few patients are aware of the right to be reimbursed for healthcare received in another EU Member State ” Information For Patients, Mr Dalli said. “Patients have very different access, depending on the language they speak, and on whether able to use the Internet. In addition, they are often confronted with uncontrolled information, or even with advertising on the Internet from non-European sources. I am determined to improve this, and am determined to work with stakeholders, Member States, and the European Parliament to find the right
Directive, which gives patients the right of protection from unauthorised access to medical data, is currently in the early stages of review to meet the evolving challenges to this very sensitive and serious issue.”
TIF’s educational books available in new languages For downloadable PDF versions of all available languages please visit TIF’s website: www.thalassaemia.org.cy Or contact TIF, your national thalassaemia association or your country’s TIF Board Member to order a hard copy.
They are absolutely free for patients and parents! 22_
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AROUND THE WORLD Cyprus Alliance For Rare Disorders – A TIF Initiative For the first time in Cyprus, patients with rare disorders unite their energies and voices to establish the Cyprus Alliance for Rare Disorders. Owing to the close collaboration and initiative of Cypriot organisations which represent patients with rare disorders and after almost two years of intensive actions and deliberations, the Cyprus Alliance for Rare Disorders was founded and registered according to the Cyprus Company Law in June 2010. Members are Muscular Dystrophy Association Cyprus (MDA Cyprus), Adult Congenital Heart Defects Association Cyprus (ΑCHDAC), Pancyprian Antianaemia Association (PAA), Cyprus Myasthenia Gravis Association, Cyprus Primary Association and Friends – Immunodeficiency, Cyprus Society for the protection of spastic and handicapped children (ΕLΕPAP), Parents Society of the centre for the protection of spastic and handicapped children, and Thalassaemia International Federation (TIF).
concerning the latest developments in medicine and research, and to raise awareness regarding rare disorders in the Cypriot society. The Cyprus Alliance for Rare Disorders has already applied and been accepted as associate member of EURORDIS, the European Organisation for Rare Diseases. The founding of the Alliance was announced at a Press conference on 23 September 2010, in the presence of the Minister of Health of Cyprus, and medical experts and patients.
Principal goals of the Alliance are to promote health and other services to patients with rare disorders, to strengthen the patients’ and their families’ voice, and to support and ensure progress of the national rare disorders programme recently announced by the Cyprus Health Ministry. Ultimate goal is the betterment of health and quality of life for every patient with rare disorders, to provide support and continuous education to the patients and their families
Cyprus: Inner Wheel Club of Nicosia makes significant donation to TIF In May 2010, TIF’s President Panos Englezos accompanied by Dr Angastiniotis, TIF’s Medical Advisor, were invited to a meeting of Inner Wheel Club Nicosia, branch of an international humanitarian organisation closely linked to Rotary Club. The President of Inner Wheel Club of Nicosia, Mrs Louca, complimented TIF on its efforts and hard work in its mission of ensuring equal access to quality health care for every patient with thalassaemia across the world. Mrs Louca, on behalf of Inner Wheel Nicosia, presented TIF’s representatives with the sum of five thousand Euros in support of TIF’s activities.
On behalf of the Board of Directors of TIF, President Mr Englezos extended our deepest and sincere gratitude to the Board members of Inner Wheel.
Inner Wheel Club Nicosia have always shown genuine interest and warmest support on a number of occasions over the years to assist TIF toward achieving its mission. We believe that the efforts of humanitarian organisations like Inner Wheel contribute significantly to the work of non governmental organisations (NGOs) and national public organisations worldwide. TIF MAGAZINE_NOVEMBER 2010_www.thalassaemia.org.cy
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AROUND THE WORLD Russian Federation: Contact initiated in the field of thalassaemia The Russian Federation is a relatively closed country regarding data on haemoglobin disorders, including thalassaemia. TIF has been investigating ways to support and assist the national health authorities in the Russian Federation in their efforts to control haemoglobinopathies in the country.
TIF’s goal is to draft a joint plan of action to promote the status of haemoglobin disorders in the country, under the guidance of the local and regional WHO offices.
This year’s WHO-EURO (the 60th Session of the WHO regional committee for Europe) took place in Moscow on 13-16 September 2010. TIF participated and contributed to the proceedings by addressing participants with a 3-minute intervention statement. Taking advantage of the opportune venue, TIF’s representatives at the Session set out to compile data on thalassaemia and to contact relevant experts with the aim of opening a line of communication with the Russian Federation health authorities.
AFRICA Maghreb Region Morocco: TIF delegation visit and workshop On 29-30 May 2010, a TIF delegation visited Morocco and met with the Director of Epidemiology of the Ministry of Health. TIF expressed the willingness and commitment of the Board to collaborate with National Health Authorities in Morocco, in order to improve services for thalassaemia patients and to develop and implement an effective control programme. The delegation also visited the National Blood Bank and the Regional Transfusion Centre, the WHO office in Morocco and the WHO representative, and the Secretary General of the Ministry of Health for an exchange of views and discussion on collaboration. TIF paid a visit to the Children’s hospital director and the new haematology-
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oncology paediatric day care Centre which also serves as a thalassaemia centre, and fulfils many of the criteria to act as a Reference centre. A workshop was organised at the Conference Centre, Hay Riyad, Rabat, for health professionals as well as patients and their families. TIF proposed a three-year Joint Plan of Activities to be undertaken with the Ministry of Health of the Kingdom of Morocco as the permanent health provider, with the active involvement of the patient support association MATHED. In this framework, TIF recommended the creation and establishment of a Scientific Expert Panel or National
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Advisory Group and of a national patient registry, the adoption of a national, government - controlled prevention programme, including premarital screening and genetic counselling, creation and establishment of at least two thalassaemia reference centres (one in Rabat and one in Casablanca), and provision of free multidisciplinary care according to each patient’s needs. TIF also recommended promotion of education for both health professionals and patients through participation in international, regional and local workshops, and promotion and strengthening of the activities of the existing thalassaemia association, as well as the establishment of new associations.
AROUND THE WORLD Algeria: TIF delegation visit and workshop During this first delegation visit in Algeria, TIF held a press conference on 8 May 2010, in the course of which the issue of thalassaemia and sickle cell anaemia as a global public health problem, and its socioeconomic repercussions at the national and international level were extensively discussed. At the press conference, it was stressed that with the new medical and scientific advances in the fields of prevention and clinical management for these disorders, particularly iron
chelation, thalassaemia and sickle cell anaemia are both preventable and treatable, especially when effective control programmes are implemented. TIF visited the hospital where patients with haemoglobin disorders are treated, and a celebratory event commemorating the 8 May International Thalassaemia Day was held there with the participation of more than 250 patients and parents. TIF’s President and Executive Director
met with officials from the Ministry of Health and discussed the ways in which the Federation could offer its services through the appropriate ministerial channels and in the most effective way.
An educational workshop was organised devoted to updating and addressing issues of concern from haematologists, paediatricians and other health specialists, as well as patients/parents organisations.
Closed Maghreb Meeting in Cyprus As part of TIF’s educational activities for this year and as follow up on the delegation visits in Algeria and Morocco recently, the Federation organised a Closed Meeting in Cyprus on 1-2 October 2010, with medical specialists and patients and their families, to establish the Maghreb Network and to discuss a Joint Plan of Action for the Region and individually per country as tailored to its strengths, needs and weaknesses. Invited to speak at the conference were international experts on thalassaemia, eminent haematologists of the region, and association representatives. These included Dr Demetrios Loukopoulos (University of Athens, Greece), Dr Demetrios Farmakis (General Hospital, Athens), Dr Dora Bachir (Henri Mondon Hospital, France), Loris Brunetta (President of Associazione Ligure Thalassemici, Italy), Mohammed Merdas (President of Emirates Thalassaeamia Society, United Arabic Emirates) and Loizos Pericleous (Pancyprian Anti-anaemia Association (PAA). In addition, members of TIF’s Expert Patients Group also participated and contributed. In a letter to TIF after the conclusion of the meeting, Dr Loukopoulos stated the following:
“The Meeting in Nicosia was successful and I trust that it will prove very valuable and effective in the near future. Please accept my congratulations for this initiative. On my side, I confirm that I will do my best to contribute to the implementation of the overall plan. Prevention and Care of the hemoglobinopathies in the Maghreb should take priority in the goals of TIF.”
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AROUND THE WORLD Eastern mediterranean Egypt: HPLC instrument donated In the framework of supporting Egypt’s efforts to combat thalassaemia, TIF has mediated for the donation of an HPLC instrument (BIO-RAD variant). The instrument was donated by a UK-based company, and will be installed at the Cairo University Paediatric Clinic with the purpose of initiating a population screening programme for the identification of carriers with haemoglobin disorders. The Thalassaemia International Federation is currently acting as an intermediary between the two ends.
Middle East United Arab Emirates: Strengthening ties with Emirates thalassaemia Society TIF delegation visits Dubai for the occasion of the Middle East Thalassaemia Forum in February 2010
Asia Great news for Cambodian thalassaemia patients with TIF’s support Over the past years, TIF has supported the foundation of a thalassaemia association in Cambodia, mediated for the donation of HPLC equipment, made financial donations, and translated About Thalassaemia and thalassaemia booklets into Khmer, which were then distributed to patients/parents and health professionals across Cambodia. In 2007 and 2008 TIF organised two delegation visits and supported a workshop in Cambodia. Frequent meetings and a long correspondence between TIF and the Department of Preventative Medicine of the Ministry of Health of Cambodia resulted in promoting awareness, intervention and motivation. In this framework, 2009 marked a great success for thalassaemia patients in Cambodia: Cambodian Health authorities took the initiative of forming the Thalassaemia Working Group in a first coordinated effort to control haemoglobinopathies in the country.
Contributed by Shakeela Bashir, Emirates Thalassaemia Society
Dubai has always constituted a focal point for great advancements in the control of haemoglobin disorders. The TIF delegation travelled to Dubai in February 2010 in a move to consolidate relations with the medical community of the UAE, and visited the Thalassaemia Centre in Dubai which has all the credentials to be transformed into a reference centre in the region for patients with such disorders. Our collaboration with the Emirates Thalassaemia Society continues to grow and we are very pleased with the progress we see taking place concerning thalassaemia issues and patients’ rights in the United Arab Emirates.
Although TIF was unable to have a physical presence in Cambodia this year 2010, TIF’s guidance and frequent correspondence has borne more fruits. With our constant feedback and encouragement, and based on our book Guidelines for the Clinical Management of Thalassaemia, the Thalassaemia Working Group succeeded in drafting the document ‘’Guidelines for Clinical Care of Children with Thalassaemia’’, in collaboration with the Cambodian Ministry of Health. They plan to distribute these Guidelines widely among the involved parties to raise awareness both in the medical community and the public. TIF is also offering support for the organisation of the workshop during which the drafted guidelines will be discussed.
Send us your news If you have important news you want to share with the world thalassaemia community, let us know! 26_
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FEATURE ARTICLES Patients’ stories A message to the world By Aaron Cheng, Thalassaemia Patient My life began, well, when I was born, as lives tend to do, and for a while I lived normally, a chubby little tyke who rolled around on the floor. However, when I was only a few months old, a five-syllable word crudely entered my life and took control of it.
Thalassaemia. This seemingly Martian term isn’t as alien as it may appear. In fact, this term describes a blood disease that is carried by over sixty million people in the world. Thalassaemia, in simple terms, is a genetic mutation that affects the blood cells so that they cannot carry oxygen. While red blood cells should be plump and red, my cells are shrivelled and useless. I was diagnosed with Thalassaemia Beta Major and my life changed.
My infancy was the most difficult part of my life. Doctors were unable to find suitable veins in my tiny arms, so they stabbed my feet with the needles. At school, I was often ostracized because
“Within thalassaemia I found a hidden jewel: the treasure of dedication and passion” of the frequency of my doctor appointments. And when I realised that the blood that was being pumped into my body was from other living people, I felt like a vampire – not as shiny and awesome as Edward Cullen, but a vampire nonetheless. It was during my elementary school days when I resolved to repay all of my blood donors for their generosity and all of my doctors for the work they put into me.
Therefore through middle school I immersed myself into the world of academia, determined to make an impact on the medical community, to ensure that everybody with Thalassaemia would be able to lead happy and productive lives. Within thalassaemia I found a hidden jewel: the treasure of dedication and passion. The darker my circumstances, the brighter its light will shine. It is because of thalassaemia that the motivation to succeed runs through mytveins. It is because of thalassaemia that I have learned to endure pain. And it is because of thalassaemia that I am able to lead a productive life today.
Empowering From Heaven: A Pakistani’s Legacy to Strengthen the Bonds of Family By Ayesha Mehmood / FAiTh – Fight Against Thalassaemia FAiTh, a group dedicated to supporting Pakistani people with thalassaemia and their families, was founded by the late Salman Mehmood. Salman was a thalassaemic himself, who was able to leverage his illness for the benefit of society. He demonstrated that far from leaving him impaired, thalassaemia did not prevent him from leading a full life – something many of us are not able to accomplish. This was his strategy: he showed to people that potential is never hindered, only harnessed, and that you were never a victim unless you chose to be. Salman established a blood donor system in Pakistan, and organised blood donation camps and awareness campaigns. Furthermore, he made accessible a corpus of research, news, and trial information to the Pakistani population living with thalassaemia. After Salman passed away, the group he created kept up his work and went on expanding its activities and outreach, including the establishment of a free thalassaemia screening facility in Karachi, in collaboration with Hospital Supply Corporation and Usman Memorial Hospital, and screening camps in association with Pakistan’s Fatimid Foundation. A milestone that Salman set was a
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legislative Bill for mandatory testing of thalassaemia, Hepatitis, HIV, and other blood-related diseases. The Bill was passed recently. Following in the footsteps of Salman, FAiTh continues to raise awareness about the illness and motivates people to not only support thalassaemic patients, but also encourage them to get involved in raising awareness and enhance their quality of life. For more information, please visit the FAiTh website at www.thalassemia.com.pk
FEATURE ARTICLES The impact of Iron Chelation on the quality of life - A patient’s story From Thalassaemia Australia Inc. Newsletter Spring 2010 – Anonymous It was just the beginning of many more needle site and sleep disturbance, but and when you see what iron overload years of arguments, begging and as an adolescent when all my friends can do to your body, how it affects the rebellion. What kid wants to learn how were beginning to dress up and go out heart, liver, pancreas; as it has affected to inject themselves and wear a needle at night, I would be self conscious of so many of my friends and the pain in all night, five nights a week? What the unsightly lumps and bruises on my their parents eyes as they advise you to parent wants to inject their look after yourself, you know child and argue; or give in what the priority should be, “No one seems to understand, brothers, when they know that this is you understand how sisters, parents, other family, friends and the only way their child important it is to chelate but peers don’t really know” could survive? It hurts, you oh, how easy it is to stray. can’t get comfortable, you’ve got a arms, legs and tummy and refused to pump which pushes the medication wear tight clothing. I also had to I’ve done my best to lead a ‘normal’ through the syringe strapped to your choose the evening I went out and plan life. Finished school, continued my side and as the medication is injected what nights I would wear the pump. education and am now a successful under the skin it becomes extremely medical clinician and still at times cheat itchy. All things you learn to live with as On top of all of this I had my parents when it comes to wearing my pump. I the years roll into decades. constantly questioning me, when I educate others on the importance of chose not to wear my pump, I had to wearing the pump and still I am not No one seems to understand, brothers, justify the reason I wouldn’t and see always the most compliant. Fitting in sisters, parents, other family, friends the guilt in their face when an school, university, work and a social and peers don’t really know. No one argument broke out, worse still when life, meanwhile taking care of your that is, except for other patients with they let me get away with it. health is a challenge but such is the Thalassaemia and Sickle Cell disease. cycle of life or my life at least. In order Over the years the bond developed When L1 or otherwise known as to maintain a quality of life I know I with other patients grows stronger, Deferiprone came out as a trial I must sustain good health and to do only they are able to convince you to jumped at the chance to give up the that I must comply. comply, and through their experiences pump. When the trial was over it was you learn to cope with all the issues back to the pump, well by that stage I A message to all patients who find it that impact your life because of had become accustomed to sleeping hard, you are not alone. To all those iron chelation therapy. without it and the leisure of late nights, who helped me, thank you. To all the I had a social life to maintain. friends we’ve lost along the way, we As I grew older the issues of having to miss you! chelate became a little more To most it won’t make sense that these complicated than just the pain at the things take priority. Well they don’t,
TIF Wants to Hear Your Story! If you have a story that touches on some of the issues faced by thalassaemia patients in their lives, send it to TIF.
Or if you know of an inspirational or influential individual in the thalassaemia community, whom you think TIF should interview, let us know about it. We always welcome suggestions and will contact you if your story is selected for publication.
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FEATURE ARTICLES Obituary Global Thalassaemia Family mourns the loss of a great friend, supporter and visionary: Professor Calogero Vullo Contributed by Prof Vincenso de Sanctis, Santa Anna Hospital, Ferrara, Italy
A great figure in the field of medical research in thalassaemia, Professor Calogero Vullo, passed away on June 22, 2010. Prof. Vullo was born in Crotone on March 24, 1927, and obtained his degree in Medicine and Surgery in 1949 from the Universita degli Studi di Sassari. After specialising in Paediatrics at the Universita degli Studi di Pisa, he
get married, have children, and are well integrated in society. He understood the fundamental role associations could play in the fight against thalassaemia. Today these associations are present in many countries in which thalassaemia represents a medical and social problem. Here are his teachings: honesty and professional correctness, respect for deontological and ethical aspects, commitment to work, help for the infirm and for the patients of chronic diseases.
worked as assistant in Clinical Paediatrics for many years, and became a lecturer in this same topic. He
‘’We will miss him enormously. We will go on, conscious that we have had the good and director of the Thalassaemia Centre of Ferrara fortune to have lived alongside an (Centro della Talassemia di Ferrara). From 1958 to extraordinary Doctor and Man.”
was Hospital director at Cesena from 1962 to 1997
1959 he was researcher at the Walter Reed Army
Institute of Research in Washington and contracted Professor of Bioethics at the Institute of Philosophy of the Universita di Ferrara. Prof. Vullo was founder and first President of the Italian Society of Medicine for Adolescents (Società Italiana di Medicina dell'Adolescenza) and held the position of coDirector of Perspectives in Paediatrics since its foundation in 1996. Prof. Vullo won the “George P. Englezos Award” of the Thalassaemia International Federation in 1997, and the “Doctor of Italy Carlo Urbani 2009” (“Il Medico d’ Italia Carlo Urbani 2009”) of the Order of Doctors and Dentists of the Province of Ferrara for his successes in the cure and prevention of thalassaemia. Prof Vincenso de Sanctis, a friend, former pupil and collaborator of the deceased, wrote to TIF: “I would like to recall some aspects of his professional commitment. In the past, the fate of children with thalassaemia sooner or later was death. This had convinced family members of patients and also many members of the medical profession that thalassaemia was an incurable disease. Thanks to Prof Vullo’s commitment, research was sustained in various fields, leading to improvement of survival and quality of life, and psycho-social integration of thalassaemic patients. He has contributed to the introduction in society of an individual who can now lead an active and useful life. Today, many patients reach adulthood,
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FEATURE ARTICLES Distinctions to four health professionals for their exceptional contribution to the field of medicine and/or in society Sir David Weatherall,leading international expert on thalassaemia, receives prestigious 2010 Lasker Award Adapted from September issue of Cooley’s Anemia website (http://www.thalassemia.org) The Albert and Mary Lasker Foundation honors four visionaries “whose insight and perseverance have led to dramatic advances that will prevent disease and prolong life.” One of these four recipients of the award, Sir David Weatherall is Regius Professor of Medicine Emeritus and retired Honorary Director of the Weatherall Institute of Molecular Medicine at the University of Oxford. He has received the 2010 Lasker~Koshland Special Achievement Award in Medical Research for five decades of dedicated work in
biomedical sciences and groundbreaking discoveries in genetic diseases of the blood, as well as for his leading role in improving clinical health care of thalassaemic children around the world. Sir Weatherall has been an inspiration to generations of young physicians and researchers and a bright figure in our search for knowledge about thalassaemia, its description and treatment. He started exploring the genetic signature of thalassaemia in the 1950s, and provided one of the first medical descriptions of gene
deletion causing a human disease. He applied his findings to patient treatment and developed diagnostic tests and therapies for thalassaemia patients. Result of his vision is a robust network of collaborators in developing countries for the management and study of the disease and the Oxford University’s Institute of Molecular Medicine, which he founded and which was renamed in his honour in 2000.
Lebanese Professor of Medicine and specialist in haemoglobinopathies,
Dr Ali Taher, recognised for outstanding contribution to the field Dr Ali Taher, Lebanese professor of medicine with a special interest in haemoglobinopathies and important collaborator of TIF, received recently the Medical Alumni Chapter Gold Award, presented at a reception and dinner of the annual Middle East Medical Assembly (MEMA) in April 2010 in Beirut. The Medical Alumni Chapter, headed by Dr George Abi Saad, chose Dr Ali Taher for his achievements as a medical practitioner and his stimulating research in the field of medicine.
scientific articles, as well as two books and three book chapters, and has co-authored the globally distributed Guidelines for the Clinical Management of Thalassaemia of the TIF. We of TIF are extremely proud and honoured to collaborate with such a distinguished academic and professional. Congratulations, Dr Ali Taher, and we hope that you can be with us for many years to come, with the same strength, kindness, and unique devotion to the cause of thalassaemia!
Dr Taher has been consultant haematologist of the Chronic Care Centre in Lebanon since 1993. Through the Centre, he was able to form Thought Leaders Groups, which include various centres and investigators from the Middle East. Through these groups, he and his colleagues independently initiated clinical trials which contributed significantly to medical knowledge and patient care specialised in the fields of thalassaemia, sickle cell disease, thrombosis, and hemostasis. Dr Taher has published hundreds of important
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FEATURE ARTICLES Lt. Gen. Fatim Ahmed Khan receives award The colleagues, friends and well wishers of Lt. Gen. Fatim Ahmed Khan met on 17 April 2010 to celebrate his 20 years of dedication to the thalassaemia cause. Lt. Gen. Fatim Ahmed Khan is a pioneer in establishing the first thalassaemia patients society in Pakistan in 1990 and through his efforts, and the awareness campaign he initiated various such associations with treatment facilities were founded in all major towns of the country, as well as the Thalassaemia Federation of Pakistan where he was elected as Honorary Chairman.
Lt. Gen. Fatim Ahmed Khan was member of the Board of Directors of TIF for four years (2002-2004). We are honoured to have collaborated with such a dedicated and outstanding person as Lt. Gen. Fatim Ahmed Khan, and we look forward to the continuation of this partnership.
The treatment centre he helped found has now its own fivestorey building in Tipu Road and caters for full treatment and care of over five hundred patients. Facilities exist for blood donation, screening and chelation therapy both by injections and orally. During this past year, it has used over six thousand pints of blood, about nine thousand Desferal injections, and oral chelators which were in their majority free for the patients. A prevention programme with an advisory centre for premarital testing and expectant mothers is also in place. A quarterly newsletter is issued and distributed for free to raise awareness in patients and the general public.
Prof Dr Mahmood Ahmed Chowdhury receives recognition of his dedicated work We are happy to share with you the news that Prof Dr Mahmood Ahmed Chowdhury, Chief Physician of the Thalassaemia Welfare Centre in Bangladesh, fellow (FCPS) of the Bangladesh College of Physicians and surgeons, and renowned child specialist in Chittagong, Bangladesh, has been awarded a fellowship (FRCP) from the Royal College of Physicians, Edinburgh, UK. Our warmest and most sincere congratulations.
INTERESTED IN JOINING THE REGIONAL (EUROPEAN, ASIAN, AMERICAN, ME, GLOBAL) NETWORK OF PATIENTS’ ORGANISATIONS AND MEDICAL SPECIALISTS IN HAEMOGLOBIN DISORDERS? BENEFITS FROM BEING PART OF THE NETWORK INCLUDE: Priority to attend meetings and conferences • Priority in sponsorship programmes • Reduced registration fees •
• Authorship/contribution to educational material • Being part of the faculty in educational events • Participation in high-level meetings of TIF
To find out how to join, visit our website: www.thalassaemia.org.cy
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FEATURE ARTICLES Every crisis comes with an opportunity Contributed by Shobha Tuli, Vice President of TIF and President of Thalassemics India ‘A Problem can give us a chance to grow’ says Marty Varnadoe Dow. Crisis is painful, frightening, and difficult. None of us consciously invite crisis into our lives. We do not look forward to the next life-threatening event coming our way. The news of a crisis brings feeling of sadness, fear, and regret. No one wants to face death, financial loss, or a broken marriage. We do not wish these events on others.
So, how is it that I can tell you that every crisis contains a seed of good? I have had the opportunity in my life to see many people as they navigated the troubled waters of a crisis. As a thalassaemia parent, I have felt and experienced pain and helplessness while going through the unsuccessful BMT of my daughter, Preeti. As a friend, I have had the privilege to hear the secret descriptions of pain and despair felt by my classmate who lost both her parents in an accident. As Secretary of Thalassemics India, while counselling thalassaemia parents, I have observed the suffering which a family experiences while fighting against thalassaemia. The other day, I stood next to a hospital bed for an hour while the mother cried and expressed her helplessness on seeing her son slipping away from her hands. I see the expressions of pain and
agony when parents open their hearts and share with me the problems that they face while treating their children. As a person, I have felt my own grief and fear as I faced the crisis of death in my family. If this was all I had seen in these difficult situations, I would not call crisis an adventure; however, I have been blessed with seeing another side to trauma. Out of the ashes of despair, I have observed the power of love heal the wounds of a troubled family. I have seen the news of loss bring people together in an closeness they had never dreamed possible. I have seen people around me help one another to overcome their problems. I have seen people reach down deep inside themselves to find a superhuman strength to overcome a physical disability. I have seen despair turn people toward the power of God
something new to develop from the ruins of our way of life. As people reevaluate the meaning of life during their crisis, they often discover their life mission, which then gives them a greater sense of direction and purpose. Sometimes people in crisis will find an inner strength they never knew existed. Most people find a deeper connection to God. I have heard about people who have accepted the challenge and transformed their lives for the betterment of others. These people are not special. They are ordinary human beings. If they can do it, so can you. In a way, it is an opportunity given to you to prove your inner strength and abilities. Being a thalassaemia parent, one should look at thalassaemia not only as an inherited condition, but also as a challenge. It is up to you to prove that
“I have seen people reach down deep inside themselves to find a superhuman strength.” within them. I have watched seemingly helpless people become spiritual giants as they exercised the power of prayer to overcome an impossible situation. Crisis is a two-edged sword. It brings the destruction of life as we have known it. It challenges our beliefs. It pushes our ability to cope. But crisis always brings opportunity for
how you can best fight thalassaemia, in spite of all the odds in your way. Who wants to be defeated? No one. But one can only win if one learns to accept His wishes and move forward in life, realising that perhaps thalassaemia – or any crisis – is an opportunity to do something extraordinary in life.
Interested in joining TIF’s Global Specialist Nurses Network? Please write to us and complete the relevant form to send you more information. TIF MAGAZINE_NOVEMBER 2010_www.thalassaemia.org.cy
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INTERNATIONAL / EUROPEAN ORGANISATIONS CORNER World Health Organisation (WHO) Proudly announcing a closer and more productive collaboration with the WHO Dr Victor Boulyjenkov has been consultant of the Human Genetics (CHP/CPM programme) and Coordinator of the Human Genetics Programme of the WHO. He is now retired and currently residing in Geneva. TIF is greatly honoured to have secured Dr Boulyjenkov’s collaboration after his recent retirement. He now is the official focal person between TIF and WHO Headquarters. His knowledge and vast experience on issues relevant to TIF’s mission and objectives are of immense benefit to TIF and by extension to all patients with thalassaemia in the world.
Essential to keep WHO’s Human Genetics Programme alive! After Dr Boulyjenkov retired from his post as Coordinator of the Human Genetics Programme of the WHO, he was replaced by Dr Patricia Aguilar Martinez. Dr Martinez Director of the Laboratory of Hematology, Montpellier University Hospital (France), is now Medical Officer of the CHP/CPM Human Genetics programme of the WHO in Geneva for the ensuing period of six months. Dr Aguilar Martinez has immense experience and expertise in the field of Rare Diseases, including Haemoglobin disorders, and we are confident that, through her, our collaboration with the WHO will continue to expand and become more productive.
JOINT PLAN OF ACTIVITIES Following a visit in May 2010 in Geneva with assistant Director General of WHO, Dr Alaa Alwan, and Coordinator of Chronic Diseases Prevention and Management programme of the WHO (which forms part of the Non Communicable Diseases Programme), Dr Shanthi Mendis, a joint plan of activities 2011-2013 was formulated. The plan was based on (i) the resolutions of the 118th Executive Board in 2006 and the 59th WHA in May 2006, (ii) the outcome of the WHO-TIF Joint Experts meeting on haemoglobin disorders which took place in November 2007 in Cyprus, and (iii) on the objectives set in 2008 by the Non Communicable Diseases Programme of the WHO. Work to finalise this plan is ongoing.
WHO REGIONAL MEETINGS TIF attends WHO regional committee for Europe in Moscow In recent years TIF has exerted immense efforts in bringing haemoglobin diseases to the forefront, supported through its collaboration with the WHO and other health and disease-related organisations. In this context, TIF participated in the 60th Session of the WHO regional committee for Europe which took place in Moscow on 13-
16 September 2010. The session provided TIF with an excellent opportunity to place the haemoglobin diseases firmly on the health agenda of the World Health Organisation and press for an initiative at the international level for better standards and access to health care for every patient with haemoglobin disorder, including thalassaemia, across the world. TIF’s statement, which discusses the management of thalassaemia, was delivered by Dr Victor Boulyjenkov.
WHO – World Health Assembly Resolutions Before the World Health Organisation (WHO) adopts a resolution, a proposal goes through the Executive Board and World Health Assembly (WHA) for discussion and voting. The resolutions, validated in the course of this meeting (the WHA being the one of highest impact), constitute important steps to raising worldwide awareness and application of policies/recommendations at the national and international level. The two resolutions, EB 118R1 and WHA59R20 on thalassaemia and Sickle Cell Disease respectively adopted in
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INTERNATIONAL / EUROPEAN ORGANISATIONS CORNER May 2006, marked international recognition of Hb disorders as a major public health issue for the first time, and in addition their tremendous socio-economic repercussions. These resolutions encourage all “affected” member-states to proceed with addressing the issue of control of these diseases.
Current World Health Assembly Resolutions New Progress - The WHA adopts new resolutions tangent on Hb disorders The 63rd World Health Assembly (WHA), which took place in May this year 2010, adopted strategies to further promote non-communicable diseases and birth defects, a category which also includes genetic disorders, and by extension thalassaemia and Sickle Cell Disease (WHA63.17). In addition, during these proceedings, two other resolutions directly connected and relevant to the management of these disorders were adopted: the resolution relating to Blood Availability and Safety (WHA63.12), and the resolution concerning Viral Hepatitis (WHA63.18) – see below.
Member States “… to implement and/or improve epidemiological surveillance systems and to strengthen laboratory capacity, where necessary, in order to generate reliable Information for guiding prevention and control measures…” FOR EUROPEANS and residents of other countries where a Rare Diseases programme exists or was initiated: In addition to WHO, other organisations and agencies focus on haemoglobin disorders, for example the European Commission through directives, regulations, programmes, research studies and position papers which are relevant to Rare Diseases. Take advantage. Read about them! Ask TIF and we will provide all the information! Visit the websites of the WHO Regional Offices for more information!
WHO Regional Sessions
Excerpts from the resolutions
On 13-16 September 2010 in Moscow, as mentioned in detail above, took place the most recent regional meeting of the WHO – the WHO-EURO, the 60th Session of the WHO regional committee for Europe. WHO Regional Office for Europe (EURO): www.euro.who.int
WHA63.12 “Availability, safety and quality of blood products”
TIF has also participated in other recent regional WHO sessions with three- minute statements.
The Sixty-third World Health Assembly, having considered the report on availability, safety and quality of blood products (A63/20) urges Member States “… to take all the necessary steps to establish, implement and support nationally-coordinated, efficiently-managed and sustainable blood and plasma programmes according to the availability of resources, with the aim of achieving self-sufficiency, unless special circumstances preclude it;.”
WHA63.18 “Viral Hepatitis – I” The Sixty-third World Health Assembly has considered the report on viral hepatitis (A63/15) and taken into account the fact that some 2000 million people have been infected by Hepatitis B virus and that about 350 million people live with a chronic form of the disease. Furthermore, hepatitis C is still not preventable by vaccination and around 80% of hepatitis C virus infections become chronic infections. The World Health Assembly has expressed concern at the lack of progress in the prevention and control of viral hepatitis in developing countries, in particular in sub-Saharan Africa, due to the lack of access to affordable, appropriate treatment and care, as well as an integrated approach to the prevention and control measures of the diseases, and insists on the need to reduce liver cancer mortality rates, given that viral hepatitides are responsible for 78% of cases of primary liver cancer. The World Health Assembly therefore urges
On 27 September-1 October 2010 in Washington, during the 62nd Session of the Regional Committee for the Americas of the World Health Association (PAHO), TIF Board Member Mr Bob Ficarra, a founding member of TIF and past president of TIF’s Board with vast expertise and knowledge in the field, presented the topic of “Management of Haemoglobin Disorders: Chronic multi-organ diseases – A public Health Issue”. WHO Regional Office for the Americas (PAHO): www.paho.org On 3- 6 October 2010 in Cairo, Egypt, during the 57th Session of the Regional Committee For The Eastern Mediterranean of the WHO (EMRO), a three minute intervention statement on behalf of TIF with the topic “Control of Haemoglobinopathies – Prevention and management” was distributed to all present. WHO Regional Office for Eastern Mediterranean (EMRO): www.emro.who.int On 18-23 January 2010 in Geneva, Switzerland, TIF participated in the Executive Committee of the WHO (EB126) with two items on the agenda, statement 4.16 on the “Availability, safety, and quality of blood products”, and statement 4.7 “Birth Defects”, both presented by Dr Boulyjenkov on behalf of TIF.
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INTERNATIONAL / EUROPEAN ORGANISATIONS CORNER On 7-10 September 2009 in Bangkok, Thailand, TIF participated in the 63rd Session of the Regional Committee for South-East Asia of the WHO (SEARO) with a statement (Agenda item 5) on “Control of Haemoglobinopathies – Prevention and Management” presented on behalf of TIF by Dr Prosanto Chowdhury. WHO Regional Office for South East Asia (SEARO): www.searo.who.int On 21-25 September 2009 in Hong Kong, China, TIF participated in the 60th Session of the WHO Regional Committee for the Western Pacific (WPRO) with a statement (Agenda item 7) on “Control of Haemoglobinopathies – Prevention and management” presented by Dr Chi Kong Li on behalf of TIF. WHO Regional Office for Western Pacific (WPRO): www.wpro.who.int
We encourage everyone to join our efforts in the promotion and implementation of all the above mentioned resolutions. TIF strongly encourages all its members to take actions by: 1. familiarising themselves with the Resolutions (past and
current), and learning what they propose 2. promoting them and discussing them with the National Health Authorities 3. arranging meetings and writing letters for their promotion
Find the resolutions on the WHO website: http://apps.who.int/gb/ebwha/pdf_files/EB118/B118_R1en.pdf They are also available on TIF’s website by clicking on the following links! www.thalassaemia.org.cy/pdf/WHO_Res_Thal.pdf www.thalassaemia.org.cy/pdf/WHO_Res_Sickle_Cell.pdf WHO is the directing and coordinating authority for health within the United Nations system. It is responsible for providing leadership on global health matters, shaping the health research agenda, setting norms and standards, articulating evidence-based policy options, providing technical support to countries and monitoring and assessing health trends. For more information visit: www.who.int/en
Submitting photos to TIF Magazine We are always pleased to receive your contributions and especially photos. However, sometimes photos are too small or very low-resolution and we cannot use them. To make sure your photos are print-quality, please follow these guidelines when submitting photos: 1. Photographs intended for publication in TIF Magazine should be in JPEG or TIF format 2. To be “of printable quality” means: • High-resolution (ideally 300 dpi or more). Please select a high resolution on your camera. (How this is done depends on the camera model. You can ask a camera shop for advice.) • Send the original image file (even if large). Please do not re-size the photos. • Send photos separately by email, via YouSendIt or on a CD. Please do not insert photographs into word or PDF documents.
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3. All photos should have captions – i.e. a short text explaining what is happening the photo and who the people featured in the photo are. 4. It is the responsibility of the person submitting photos to ensure that any person(s) featured in them have given their permission to publish the photo. Please make sure any patients, parents or other private persons featured in your photos have agreed to their publication. Because of the technical nature of some of these guidelines, we recommend that any person entrusted with taking photos at official events checks with a professional (e.g. a photographer or a camera shop assistant) how to adjust their camera’s settings. Thank you for following these guidelines!
INTERNATIONAL / EUROPEAN ORGANISATIONS CORNER Newsletters of International Health Organisations WHO Newsletter WORLD HEALTH ORGANISATION
Safe Surgery Saves lives – make this your new slogan! The goal of the Safe Surgery Saves Lives Challenge is to improve the safety of surgical care around the world by ensuring adherence to proven standards of care in all countries.
From WHO – PATIENT SAFETY newsletter August 2010 The WHO Surgical Safety Checklist has improved compliance with standards and decreased complications from surgery in eight pilot hospitals where is was evaluated. In sites that ranged from small district hospitals to large medical centres in diverse geographical settings, the use of the 19-item checklist was demonstrated to reduce the complications and mortality associated with a variety of surgical procedures by greater than 30 percent. The checklist has been designed to be simple to use and applicable in many settings. It is currently in active use in operating rooms around the world.
A Global Health Challenge Urgent need to improve access to medical care for patients in rural and remote areas. Adapted and compiled from WHO Bulletin May 2010 Half the population of the world lives in rural areas – but are served by less than a quarter of all doctors and less than a third of all nurses in the world. Providing access to medical care for people in remote areas constitutes a global challenge. Heroes still exist, giving up everything to live and work in rural areas, helping people, even at the risk of their own health. Unfortunately, such heroes are a rare commodity these days. Mostly, the trend is a reverse one: health workers migrate from the countryside to the cities, and from poor countries to richer ones. Extraordinary individuals still care for poor patients, but governments should not rely on their goodwill for the welfare of their citizens. Governments should assist these individuals, and find ways to retain them by making their life and their work easier, otherwise, the problem of shortage in health professionals in rural areas will stagnate. The Kampala Declaration (2008) sought to address this issue: “to assure adequate incentives and an enabling and safe environment for effective retention and equitable distribution of the health workforce”.
Did you know? 3,865 hospitals representing 122 countries have registered as Safe Surgery Saves lives participating hospitals. Is your hospital registered? 25 countries have mobilised resources to implement the WHO Surgical Safety Checklist at a national level. Has your country implemented the checklist? Visit the website for the checklist, available in English, Arabic, Chinese, French, Russian and Spanish, as well as for more information and for frequently asked questions about Implementation of the checklist: http://www.who.int/patientsafety/safesurgery/en
The World Health Organisation (WHO)’s response has been to establish a programme of work to “increase access to health workers in remote and rural areas through improved retention” by means of three strategic pillars: (i) building the evidence with the long efforts of experts, (ii) developing policy recommendations from the evidence, and (iii) supporting countries in implementing the recommendations and evaluating their impact. Obviously, there is no easy answer or simple solution to the problem. Maldistribution is a major issue, as the absolute number of workers might not be the crux of the problem, but the type of workers and their job location may be. Here we note that this problem is commonly shared in all countries of the world. Interventions in education, regulation, financial incentives and personal and professional support can resolve different aspects of the problem. Specifically, the “pipeline-topractice” approach to education (study targeted to specific career choices) seems promising, especially if medical schools adopt a social accountability framework to make students more responsible to communities. Compulsory service is often used but rarely evaluated. Contracting systems are explored by a few countries like Senegal, while others, like Norway, focus on professional development to reduce feelings of isolation. Effective retention strategies must meet both the population’s needs and the expectations of health workers. Monitoring and evaluation should be integrated in the design and implementation of such strategies, as well as context parameters and external factors.
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INTERNATIONAL / EUROPEAN ORGANISATIONS CORNER IAPO Newsletter INTERNATIONAL ALLIANCE OF PATIENTS ORGANISATIONS
IAPO Responds to a European Medicines Agency (EMA) Consultation on Third Country Clinical Trials
Adapted and compiled from IAPO Newsletter October 2010 IAPO, the International Alliance of Patients Organisations, has emphasised the urgent need to strengthen the informed consent process and to improve the framework for ensuring access to drugs for clinical trial participants after such trials are completed. These were key recommendations in IAPO’s consultation response to the European Medicines Agency (EMA)’s ‘consultation on ethical and good clinical practice aspects of clinical trials of medicinal products conducted in third countries and submitted in marketing authorisation applications’. The EMA stressed that if clinical trials are conducted outside the European Union, they must be required to meet internationally agreed ethical and data quality standards.
IAPO’s Recommendations: IAPO strongly supports the development of appropriate regulations and effective enforcement of these regulations to clinical trials and marketing authorisation applications, but also the appropriate consideration of their impact, especially on access to safe, quality medicines. All strategies about clinical trials should be inline with the principles of patient-centred healthcare, considering the impact of those strategies on the patient – in terms of access to safe, quality, and appropriate treatments and information. Only with the involvement of patients and patients’ organizations in these strategies can they be truly patient-centred. Patients have an obvious and central role in clinical trials. A fundamental premise of patient-centred healthcare is that patients and patient groups are involved in all stages of healthcare decision-making, including the setting of international policy. This is an essential policy for IAPO whose
main purpose is to represent patients’ rights and be their voice. IAPO states that there is a moral imperative for the involvement of patients in clinical trial design and regulation, as it is patients that take on the personal risks associated with participation in a clinical trial. In addition, IAPO recommends the strengthening and development of good practice guidelines on the communication of information to the potential participants in research, in order to better describe the research and the implications of being involved. These guidelines should be developed with the input of patients’ organisations and community groups, as well as other experts in ethics and clinical trials. Ethical considerations which IAPO considers important: a. Full involvement of patients in ethics committees b. Strengthening the process of obtaining informed consent c. Ensuring that patients are fully informed about the implications of being involved in a clinical trial d. Strengthening protection for vulnerable patients e. Ensuring access to treatments for patients after completion of the clinical trial. IAPO’s full response is available online and can be downloaded on IAPO’s website: www.patientsorganizations.org
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INTERNATIONAL / EUROPEAN ORGANISATIONS CORNER Newsletters from European Health Organisations TIF focuses particular attention on European health policies, because they often form the basis for international policies which can secure the efficiency, safety and access to treatment for all patients with thalassaemia across the world. TIF also concentrates on promoting good, productive and transparent conduct with relevant stakeholders and collaborators, topics highlighted in these European policies as well.
EPHA Newsletter EUROPEAN PUBLIC HEALTH ALLIANCE
implemented without compromising the easy availability of reasonably priced medicines at short order.
Counterfeit Medicines - legislative update
Adapted from EPHA newsletter May 2010
Concerns of EPHA:
The European Parliament and Council are currently reviewing a proposal for a Directive and a Regulation on Falsified (counterfeit) medicines.
EPHA supports the majority of measures set out in the proposed Directive. We therefore limit our comments to those areas where we have concerns.
On 1 September 2009, Günter Verheugen, Vice President of the Commission and Commissioner for Enterprise and Industry, presented the ‘Pharmaceutical Package’ to the ENVI. The Package was presented along with several other Commission initiatives that the European Parliament will have to discuss in the coming term such as the banning of incandescent light bulbs and a review of carbon leakage.
1. Need for a clear definition of falsified medicines. First and foremost, EPHA strongly calls for the insertion of a clear and internationally agreed definition for “counterfeit medicines”, and inclusion of reference to the World Health Organisation’s definition of counterfeit medicinal products : “А counterfeit medicine is one which is deliberately and fraudulently mislabelled with respect to identity and/or source. Counterfeiting can apply to both branded and generic products and counterfeit products may include products with the correct ingredients or with the wrong ingredients, without active ingredients, with insufficient active ingredients or with fake packaging.” 2. Need to ensure that costs of improved safety measures are not solely borne by patients and thus contribute to increasing health inequalities. EPHA is particularly concerned that increased costs could have repercussions on patients’ access to medicines. The costs implied by the new safety measures need to be spread throughout the whole supply chain, in order to avoid creating new barriers to the affordability of medicines and therefore increasing already rising health inequalities in Europe. 3. Need to ensure that the measures do not hinder competition or impose barriers to trade for third countries. Any issues regarding false labeling as to identity or source (counterfeiting), should not be confused with compliance to Good Manufacturing Practice (GMP) or Good Distribution Practice (GDP). This would unduly widen the scope of counterfeit regulation to the extent it would hinder competition and impair access to medicines for consumers. Furthermore, patent infringements should be explicitly excluded from the definition and the regulation. 4. Authentification of medicines. EPHA supports in principle the use of pharmacy level authentication of medicines. The addition of a safety feature in the form of a bar code or RFID tag, together with the mass serialization of individual medicine packs, offers, in the view of EPHA, a strong line of attack against the counterfeiters.
Mr Verheugen has said in the past that the trade in counterfeit medicines is "rampant" and that the problems are more widespread and serious than solely the issues of security around internet pharmacies, as some have suggested. The Commissioner said that he wanted to instill confidence in pharmaceutical products and that patients need to feel safe in the knowledge that the products they are taking are safe and genuine. In order to achieve this he said that the traceability and safety monitoring must be improved and that the proposals on counterfeiting in the Package aim to achieve this. On 8 December 2009, EPHA released its position on counterfeit medicines as a contribution to the debate in the European Parliament and Council on the Falsified Medicines Directive from the Pharmaceutical Package. EPHA welcomes the Commission’s legislative initiative aimed at combating counterfeit medicine, and more particularly for the following reasons : 1. The risk of penetration of counterfeits into the legal supply chain is growing. 2. Death and injury, untreated conditions, and a loss of confidence in the supply exemplify the disastrous consequences of the increased number of counterfeits on the market. Precaution and immediate action are therefore necessary. 3. Many measures aimed at increasing security in the supply chain can easily be achieved. Furthermore, they can be
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INTERNATIONAL / EUROPEAN ORGANISATIONS CORNER Risks of internet pharmacy Unfortunately, the world wide web is the main distribution channel for counterfeit medical products. Although in most Member States internet provision of prescription - only medicines is already illegal, EPHA suggests the following measures to address illegal internet sales: a. Stronger cooperation between the EU and individual Member States, involving the stakeholders acting in this field, including the public health community ; b. A ban on sponsored advertising of illegal pharmacies online; c. The creation of certification systems for authorised online pharmacies in those countries where online sales are legal; d. Special initiatives to make consumers aware of the risks they run when purchasing online medicines from unknown sources on the internet; e. Educating consumers and patients about the dangers of counterfeit medicines and the hazards of buying prescription - only medicines from unregulated sources.
The European Public Health Alliance (EPHA) is a network of non-governmental and other non-profit organisations working in the field of public health in Europe. EPHA’s mission is to promote and protect the health of all people living in Europe and to advocate for greater participation of citizens in health-related policy making at the European level. EPHA acts as an advocate (a channel of communication to voice concerns arising from the public health sector) and as a platform that provides a rallying point for active public health operators in Europe. Among its core values are equity, accessibility and quality of health and healthcare. Currently, TIF participates through its executive director Dr Androulla Eleftheriou in EPHA’s Executive Board. Some of the health-related policies focused on and promoted by EPHA which are relevant to thalassaemia include cross border health, counterfeit medicines, clinical trials, information to patients, patients rights policy, and patients safety.
EMA Newsletter
EUROPEAN MEDICINES AGENCY
FOCUS ON PATIENTS’ NEEDS AND TRANSPARENCY The European Medicines Agency adopts new policies on handling the conflicts of interests and on access to documents at its 68th meeting
Adapted from EMA Newsletter - Press release on 8 October 2010
Agency policy on handling conflicts of interests EMA insists on a transparent process to ensure that scientific committee members and experts participating in the Agency’s activities have no interests in the pharmaceutical industry which could affect their impartiality in the Agency’s activities, including meeting attendance, involvement in the scientific assessment and guidance development, as well as participation in inspections. The new policy answers demands for the Agency to strengthen its handling of conflicts of interests, whilst ensuring that the best scientific expertise is involved in the assessment process. This is a complex topic with no single bullet solution, but the Agency is taking drastic measures to ensure the focus of EMA remains on patients and not financial interests.
EMA’s board endorsed a new policy that defines how the EMA deals with written requests for access to documents related to medicines for human and veterinary use – originated, received or held by the Agency. The policy also sets out how the Agency will proactively disclose such documents in the future. This policy is expected to ensure wider access to EMA documents for the public. Although some documents or part of them may have to be redacted prior to disclosure, to protect commercially confidential information and personal data, this is a huge step in facilitating access to information for patients. The policy is now being finalised and will be published in the first week of November 2010, and will come into force at the time of publication.
The European Medicines Agency (EMA) is the agency for the regulation of medicinal products which are distributed in the European Union. EMA is composed of four units: Human Medicines Development and Evaluation (activities related to medicines for human use), Patient Health protection Agency policy on access to documents (pharmacovigilance and risk management), the Veterinary Information to patients in a topic close at heart for EMA. Medicines Unit, and the Information and Communications Knowledge empowers patients, and lack of information Feeling inspired? Technology unit. endangers them, therefore EMA exerts great efforts to your ideas forand8th 2010 activities with TIF! ensure transparencyShare of information on drugs drugMay Visit the website for more information: www.ema.europa.eu policies.
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INTERNATIONAL / EUROPEAN ORGANISATIONS CORNER EURORDIS newsletter EUROPEAN ORGANISATION FOR RARE DISEASES
Eurordis: Celebrating 10 Years of the Orphan Drug Regulation in Europe
Adapted from EURORDIS newsletter June 2010
“Though this [regulation] is not sufficient to address the huge medical needs rare disease patients are facing, we now know that the drivers for rare disease therapies are the coexistence of patient groups for the disease, of a patient registry and of a European network of researchers or clinicians,” adds Yann Le Cam, EURORDIS’ Chief Executive Officer and former COMP Vice Chair. The incentives built into the Regulation have boosted drug development and helped accelerate the time to approval of market application. The Regulation has also benefited the biotech industry, small and medium-sized companies, and sponsors with an orphan drug designation. However, there is still room for improvement.
A medicinal product is designated as an orphan drug if it is intended for the diagnosis, prevention or treatment of a lifethreatening or chronically debilitating rare condition, defined according to the European definition as affecting one in two thousand persons. The European Orphan Drug Regulation, in force since 2000, sets up the criteria for orphan designation in the EU and describes the incentives (e.g. 10-year market exclusivity, protocol assistance, access to the Centralised Procedure for Marketing Authorisation) to encourage the research, development and marketing of medicines to treat, and prevent or diagnose rare diseases. “Overall the Regulation can be considered a success. It has led to medicinal products coming to market for rare conditions previously untreatable, transforming the lives of the sick and dependent, as well as improving quality of life and survival”, says Lesley Greene, a former EURORDIS President and current member of the Committee for Orphan Medicinal Products (COMP). Since its adoption, more than 1,000 applications for orphan drug status have been reviewed and the number of new applications is rising every year. In 10 years, 728 have been designated as ‘orphan drugs’ and 60 have received marketing authorisation, benefitting around 2.5-2.6 million patients.
“Companies could be oriented to areas where there is lack of treatment in order to focus their research efforts,” argues Fabrizia Bignami, Therapeutic Development Director at EURORDIS. “There is also a crying need for more basic research into diseases with no current investigation and no drug treatment on the horizon. For this reason the collection of data on off-label use of approved orphan drugs and other medicines would be useful”. Yann Le Cam explains that every year medicines are withdrawn from the market, when many of those have a not-yet proven “off-label” use for orphan conditions. “We need to find ways to identify systematically these drugs and to study their off-label uses, so that, if results are positive, a more favourable environment can be created to keep the product on the market,” he argues. Another significant challenge remains to translate market authorisations into real availability for patients in every Member State. Most countries with smaller populations suffer from longer delays in availability of orphan drugs, and in some countries with high GDP, there are only a small number of drugs genuinely available. EURORDIS together with industry and academic leaders have been pushing for harmonising and speeding up pricing and reimbursement decisions in all Member States, as well as for the prompt creation of a Working Party at the European Medicines Agency (EMA) for the scientific assessment of Clinical Added Value Assessment activities.
Keep TIF informed of your events We would request all our member associations to notify us of any important or major events as early as possible, so that we can take them into consideration when planning TIF’s annual activities.
For support of educational events, or for co-organising an event, TIF requires a notice and invitation 6–12 months in advance to consider the proposal within its projected budget. The Federation should also be included in the organising committee of the event for closest possible collaboration.
Thank you! We look forward to hearing about your upcoming activities in 2012! TIF MAGAZINE_NOVEMBER 2010_www.thalassaemia.org.cy
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UPCOMING EVENTS EVENTS CALENDAR EVENT
VENUE
DATE
DIA Orphan Drugs conference "Future Direction for Orphan Drugs in Europe" Information: www.eurordis.org/rare-disease-news-events
Paris, France
3 November 2010
DIA 2nd Health Technology Assessment (HTA) Conference Information: www.eurordis.org/rare-disease-news-events
Paris, France
4-5 November 2010
Conference (by Flemish Agency for Care and Health): Reducing Health inequalities from a regional perspective – what works and what not? Information: www.epha.org
Genk, Belgium
8-9 November 2010
The power of patients – patients’ role in shaping the healthcare system of the future Information: www.careum-congress.ch
Gottlieb Duttweiler Institute, Rüschlikon / Zürich
11-12 November 2010
1st AMM-AMS-HKAM Tripartite Congress/ 44th Malaysia-Singapore Congress of Medicine “Benefits and Risks of Recent Medical Advances” Information: www.hkam.org.hk/tripartite
Hong Kong SAR, China
12-14 November 2010
4th European ANME Symposium (about complementary and alternative medicine (CAM)) Information: www.epha.org
Vienna, Austria
13-14 November 2010
4th Session of the Conference of the Parties to the WHO FCTC (COP4) Information: http://apps.who.int/gb/fctc/E/E_cop4.htm
Punta del Este, Uruguay
15-20 November 2010
2nd Workshop on Patients’ Engagement in Health Technology Assessment (HTA) Information: www.epposi.org
Brussels, Belgium
17 November 2010
3rd European Spanish Symposium on Rare Anaemias & 1st Spanish Thalassaemia Meeting for Patients & Health Professionals Information: http://www.enerca.org
Madrid, Spain
19-20 November 2010
11th Workshop on Partnering for Rare Disease Therapy Development Information: www.epposi.org
Prague, Czech Republic
29-30 November 2010
2nd Workshop on Chronic Conditions – A European Strategy for Chronic Conditions Information: www.epposi.org
Brussels, Belgium
9 December 2010
TIF International Conference Information: www.tif2011.org
Antalya
11-15 May 2011
64th World Health Assembly Information: www.wha.org
Geneva-Switzerland
16-24 May 2011
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MEDICAL FOCUS Gene Therapy Gene therapy for beta-thalassemia: results from the first trial By Giuliana Ferrari, San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) Milan, Italy.
Gene therapy of genetic diseases affecting the hematopoietic system is based on the transplantation of a patient’s own bone marrow cells that have been corrected by inserting a new copy of a normal gene. In the case of severe thalassaemia, transplantation of autologous hematopoietic cells, genetically corrected to express normal level of haemoglobin, would provide the advantage over the conventional transplantation to cure virtually all patients, without donor limitation and reducing the risk of rejection or graft-versus-host disease associated with allogeneic transplantation. In order to drive a functional ß-globin gene in the cells, we need a shuttle, named the vector, which is derived by a modified virus no longer dangerous. Recently, new vectors were developed which are able to transfer with high efficiency genes in hematopoietic stem cells. The procedure of gene therapy implies the following steps: - development and production of the vector - harvest of hematopoietic stem cells from the patient - gene transfer of ß-globin gene by the vector into the cells - treatment of the patient to reduce the erythroid mass and to allow engraftment of the transplanted cells in the bone marrow - transplantation of genetically corrected cells to the patient Several investigators have been intensively working in the field of gene therapy for thalassaemia in U.S.A (Michel Sadelain, Philippe Leboulch, Punam Malik, Derek Persons) and in Italy (Giuliana Ferrari) and the results demonstrate feasibility and efficacy in preclinical models. Now, the results from one patient treated in France by Philippe Leboulch and collaborators in the first clinical trial are available and show potential and limitation of gene therapy (M.CavazzanaCalvo et al., Nature 467, September 16th 2010). In 2007, an 18-year-old male patient affected by HbE/ßthalassaemia was transplanted with his own genetically modified bone marrow cells. In this type of patients one mutated allele is able to produce an Hb variant, called HbE, but the compound heterozygous state (ßE/ß0) leads to thalassaemia major. A functional ß-globin gene was inserted in the patient’s cells using as shuttle a modified virus derived by HIV (lentiviral vector). The vector is able to enter in the cell DNA and deliver the new globin gene in between the other genes of the cell. Before administering his genetically corrected cells, the patient was treated with chemotherapy to make space for the incoming transplanted cells. After 3
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years, the patient is transfusion independent, with a level of 9-10g/dl Hb. The genetically modified cells represent only a fraction of the total bone marrow cells (11%) but they are able to give rise to healthy blood cells, producing 1/3 of the total haemoglobin (3.7g/dl). Concomitantly, the discontinuation of transfusion led to the rising of HbE and HbF, which contribute to the 2/3 of total Hb, raising the level to 9-10g/dl. This is an encouraging result proving the efficacy of gene therapy in patients requiring a limited contribution from vector-derived Hb, like in the case of ßE/ß0-thalassemia. On the other hand, this result was obtained in the presence of a small amount of cells repopulating the patient’s bone marrow after chemotherapy. Among them, one is predominant, carrying specific vector integration in a gene called HMG2A. The predominance of this clone can be due to the effect of vector integration in that particular gene and/or to the poor content of repopulating cells in the transplant, favouring the expansion of cells endowed with the best survival potential. The monitoring of the patient’s state will give the final answer in terms of safety. At the same time, the preclinical evaluation of gene therapy with a lentiviral vector for ß-globin, called GLOBE, was conducted in Italy (by the group of Giuliana Ferrari) on a large number of patients’ samples (Roselle et al., EMBO Molded 2, 315, 2010). The results of this extensive work can be summarised in the following points: a) the gene transfer procedure does not adversely impact the functional capacity of bone marrow cells; b) the GLOBE vector is able to correct ß-globin deficiency at low copy number of integrations; c) the vector integrates everywhere in the DNA, without favouring dangerous genes. Therefore, utilising the right preclinical models we can make a good prediction of safety, although the final answer will come only from treating patients. Currently, the Italian group is completing all the tests required for clinical application of the vector in gene therapy. Finally, we can foresee that in the near future new trials for thalassaemia will start and from the upcoming results we will learn the best way to treat thalassaemia through gene therapy.
MEDICAL FOCUS Iron Chelation
Iron Chelation: monitoring FBS0701 Compiled from Cooley’s Anaemia Newsletter September 2010. With comments from Dr Michael Angastiniotis, Medical Advisor of TIF TIF is closely monitoring progress in the comprehensive development programme of the desazadesferriothiocin chelator (code name FBS0701). This is conducted by a company called FerroKin Biosciences and clinical trials have already commenced. Phase I trials have been completed, including three clinical trials on the safety, pharmacokinetics, tolerability and iron clearing activity of FBS0701, one in healthy volunteers and two in patients with iron overload resulting from transfusion for the management of both hereditary and acquired chronic anaemias, including thalassaemia and sickle cell disease. “We are excited about the potential of FBS0701 in treating iron overloaded patients,” says Hugh Young Riendhoff, Jr, founder and chief executive officers of FerroKin Biosciences. “Data from this important study will help us further establish
the relative safety and efficacy of FBS0701.” Phase II open-label, 24-week trials are currently ongoing with Dr Ellis Neufeld in charge, aiming at evaluating two dose levels of FBS0701 administered orally. “There remains a significant clinical need for an iron chelator that is safe and well tolerated by all patients,” says Dr Neufeld, Professor of Paediatrics at Children’s Hospital Boston, who has been invited to the 12th International Conference on Thalassaemia and Hemoglobinopathies to give us an update on the topic.
About FBS0701 FBS0701 is an orally available iron chelator currently in development for the treatment of transfusional iron overload. FBS0701 has received Orphan Drug Status from both the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The desazadesferriothiocin group of drugs were known to be efficient in removing iron for many years. Earlier compounds however were found to be toxic and never reached human trials. FBS0701 is a new compound belonging to the same family, which seems to provide the benefits without the toxicity. The clinical trials, we hope, will confirm this.
Working together to remove excess iron: Desferrioxamine and Deferiprone By Dr Michael Angastiniotis, Medical Advisor of TIF The combination of the two chelation agents, Desferrioxamine (known to most patients by the original commercial name Desferal) with Deferiprone, has been known for some time to be effective in protecting from heart complications. There are several published studies on the topic. The combination is usually prescribed for patients with accumulation of iron in the heart (measured by T2* >20ms on cardiac magnetic resonance) or liver (when the iron concentration is more than 15mg/g dry weight measured by liver biopsy or MRI).
trial conducted in Corinth and Athens, Greece. In this trial, the authors, (Farmaki K et al. British Journal of Haematology, 2010, 148: 466-75) have looked not only at the effects on the heart but also on endocrine function. They observed 52 thalassaemia patients who were transitioned from desferrioxamine alone to the combination regime because of high iron overload. They were observed for 7 years, and apart from reduced ferritin levels, heart and liver iron (measured by MRI), the following observations on endocrine functions were also made:
In Cyprus for example, improved survival in thalassaemia major was attributed to switching patients to this combination (see the article in Haematologica 2009, 94: 1777-8), which led to a sevenfold improved survival. Likewise in Greece, it was found that the risk for a de novo cardiac event was 23 times less common with combination, compared to Desferrioxamine used alone, and the odds for cardiac death were also much reduced (see the article in the European Journal of Haematology, 2010, 85: 335-44). Good results are also reported in patients with established heart disease in the USA (Blood Cells Mol Dis, 2010, 45:136-9).
39 patients had abnormal sugar metabolism and 44% normalised after the combination treatment. • 18 had hypothyroidism and 10 (55.5%) were able to stop thyroxin treatment. • 14 males had hypogonadism and seven of these were able to stop treatment with testosterone • 19 females had hypogonadism and 6 (31.6%) were able to become pregnant.
There are several other publications demonstrating similar results. Impressive are the results obtained from a clinical
All this makes the choice of combination treatment attractive. However it is still used for patients with evidence of heavy iron load because of increased toxicity compared to using one drug alone.
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IMPORTANT INFORMATION FOR PATIENTS Viral Hepatitis “This is Hepatitis…’’ Do you also have a story to tell?
Adapted and compiled from the World Hepatitis Alliance website Hepatitis means inflammation of the liver and its severe, chronic forms are caused by viral infection. Hepatitis B and C are two such viruses and one in twelve people worldwide live with them. Hepatitis constitutes a particularly common problem for thalassaemia patients, who are often infected with hepatitis B or C through the transfusion of untested blood, a problem current blood safety campaigns seek to address and redress. Working to improve awareness, prevention and treatment of hepatitis, a non-governmental organisation that represents approximately 280 hepatitis B and C patient groups from around the world was formed: The World Hepatitis Alliance. As a coalition of advocacy groups, the World Hepatitis Alliance is a global voice for the 500 million people worldwide living with chronic viral hepatitis B or C.
The most common ways to become infected are: • Blood transfusions and blood products using unscreened blood (in most countries, but not all, blood has been screened since about 1990) • Medical or dental interventions without adequate sterilisation of equipment • Mother to infant during childbirth • Sharing equipment for injecting drugs • Sharing straws, notes etc. for snorting cocaine • Sharing razors, toothbrushes or other household articles • Tattooing and body piercing if done using unsterilised equipment
In 2008, the Alliance launched an ongoing campaign, in the framework of which an annual World Hepatitis Day was declared – the 19th May. World Hepatitis Day aims to raise awareness on chronic hepatitis and raise the political support for the disease to levels seen in HIV/AIDS, tuberculosis and malaria. The long-term objective of the World Hepatitis Day campaign is prevention and improvement of health and quality of life for people living with chronic hepatitis.
If you think you could have been at risk from either hepatitis B or C, it is important to get tested!
This year’s World Hepatitis Day theme is “This is Hepatitis…”, and the idea is to tell the global human story of viral hepatitis through the personal stories of individual patients.
In the case of hepatitis B, infection can also occur through having unprotected sex with an infected person.
Remember: Getting vaccinated is the best way of preventing hepatitis B infection. There is currently no vaccine for hepatitis C. 2. TREATMENT Hepatitis B Treatment
1. Prevention
A. Treatment according to type of hepatitis B: a. Acute hepatitis B: Nine out of ten new infections go away on their own, with or without treatment. Antiviral drugs may only be necessary and helpful in rare cases, if the acute infection causes very aggressive liver inflammation. b. Chronic (long-lasting) hepatitis B: consult with your doctor about your situation. Treatment does not usually cure you of hepatitis B, but it can turn an ‘aggressive’ hepatitis B infection into a mild infection. This can stop the liver from being damaged. If the infection is mild, it might be better to monitor it and wait until later for treatment. You can treat chronic hepatitis B with peg-interferon or with pills, which are called nucleoside or nucleotide analogues (see below).
The hepatitis B virus (HBV) is spread between people through contact with the blood or other body fluids (i.e. saliva, semen and viginal fluid) of an infected person. The hepatitis C virus (HCV) is spread through direct contact with infected blood. Very rarely it may be passed on through other body fluids.
B. Drugs: a. Peg-interferon alfa comes in the form of injection, and stimulates the immune system against the virus. Treatment takes about 24 to 48 weeks, and while not all hepatitis B patients respond well to interferon, certain types of hepatitis
Responsibility for tackling the disease rests with everyone, individuals and organisations. However, it is only on the national level that a real difference can be effectuated and policy changes implemented to improve awareness, prevention, diagnosis, care, support and access to treatment. Governments need to work with patients groups, healthcare professionals and non-governmental organisations to formulate a comprehensive response to the public health challenge of viral hepatitis.
Prevention & Treatment
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IMPORTANT INFORMATION FOR PATIENTS B infection do. Interferon treatment should not be used if you already have cirrhosis of the liver. b. Nucleoside and nucleotide analogues come in pills. They stop the virus from replicating. The pills have very few side effects, and even patients with cirrhosis can take them. However, patients need to take their pills every day, for several years and sometimes a lifetime. If the virus becomes resistant to one type of pill, it might stop working, and another, different drug will need to be added to their treatment to get the virus back under control. Do not forget to take your pills, even if you feel well. If you miss many doses or stop treatment too early, the disease might become worse than it was before. Hepatitis C Treatment Pegylated interferon alfa and ribavirin cures approximately half of all hepatitis C patients. A patient is considered to be cured if there is no virus in the blood six months after the
end of treatment. The duration of treatment is different from patient to patient. You usually need 24 to 48 weeks of treatment, but in rare cases, 72 weeks may be recommended. There are several subtypes of the hepatitis C virus, which are called genotypes. Patients infected with genotypes 1, 4, 5 and 6 are more difficult to cure than those infected with genotypes 2 and 3. There are a number of new hepatitis C drugs currently in development. The first generation of these new drugs will likely be used in combination with pegylated interferon alfa and ribavirin. For more information, an international patients’ blog where everyone can post their own story, and materials on prevention, diagnosis, protection and treatment of hepatitis, please visit the World Hepatitis Alliance website at: www.worldhepatitisalliance.org
Check out the WHA resolution voted recently on Viral Hepatitis! You can read the article in this issue of TIF Magazine.
Show me your veins! Innovative device helps avoid needless pain
Adapted from Lucius, newsletter of LUCY, January-April 2010 / AccuVein Website VHave you ever experienced the trauma of having a health provider search desperately for a vein in your arm? Are you a nurse who wants to spare the pain of drawing blood for patients whose veins are hard to find?
Most patients don’t think needles are any fun. But perhaps this device can lessen the stress of blood transfusions, blood drawing and blood donation. What’s more, seeing a “map” of your veins is surely an unforgettable moment. For more information visit: www.accuvein.com
A group of local engineers might have an answer for you. They have designed a portable vein-locating device that can be held above a patient's arm, allowing the nurse or doctor to see the where the best veins are located. "It's designed primarily for people who may have difficulty giving blood," says Paul T. Gross, Project Manager for Development Engineering at Benchmark in Rochester. The locator device (brand-named "AccuVein" or "AV300") has two infrared lights. When it is passed over a patient's arm, the veins appear as if on X-ray, making the arm glow. One light detects the vessels and the second produces a picture of the veins directly on the skin. To use, you simply point the device at an area of the skin and click to display the peripheral veins beneath. Since the device was designed to be non-contact, no sterilization between uses are needed. It runs on a rechargeable battery, so it doesn’t need to be plugged in to a socket.
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