TIF Magazine - issue 62

TIFMAGAZINE MARCH 2013

Issue 62

Published by the Thalassaemia International Federation

www.thalassaemia.org.cy

“ 2013 International Thalassaemia Congress” 20-23 October 2013 Abu Dhabi, UAE

“ International Thalassaemia Day - 08 May”

‘’ The right to quality healthcare for every patient with thalassaemia, major and beyond’’

08MAY THA LAS SAE MIA INT ERN ATIO NAL F E D E R AT I O N

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IN PARTNERSHIP WITH

Dear Friends, Dear Readers, Welcome to this special edition of the TIF magazine.

A major new award programme for significant contributions in the field of thalassaemia and other haemoglobinopathies has been established by H.H. Dr. Sheikh Sultan Bin Khalifa Al Nahyan, through His Highness’s Humanitarian & Scientific Development Foundation, in partnership with the Thalassaemia International Federation (TIF).

We are proud to take the lead in establishing such a prestigious prize in an endeavour to strengthen efforts to educate the patients and the public about these genetic disorders, as well as to provide further motivation and encouragement to all those working in research and the medical/clinical fields to improve the health and the quality of life of patients with thalassaemia.

The award’s aim is to bring thalassaemia and other haemoglobinopathies to the forefront of the political and health agendas globally. The award will be given every two years and include both national and international categories with monetary and non-monetary prizes. Winning individuals and/or organizations will be announced during the biennial international conferences organized by TIF, followed by an award ceremony in Abu Dhabi.

INTERNATIONAL AWARD CATEGORIES include: - Sultan Bin Khalifa International Award for life-long scientific contribution in the field of Thalassaemia/Haemoglobinopathies - USD 20,000 - Sultan Bin Khalifa International Award for Innovative Medical Research - USD 20,000 - Sultan Bin Khalifa International Award for Clinical Centre of Excellence - USD 20,000 - Sultan Bin Khalifa International Award for Thalassaemia Society of Excellence - USD 20,000 - Sultan Bin Khalifa International Scholarship for Translational Research in Thalassaemia USD 60,000 - Sultan Bin Khalifa International Leadership in Thalassaemia Award - Non-Monetary Information available at: Sultan Bin Khalifa International Thalassaemia Award - www.sita.ae Thalassaemia International Federation - www.thalassaemia.org.cy

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This issue, Number 62, aims to provide information on important upcoming activities and initiatives of the Federation for 2013 - namely, (i) The 13th International Conference on Thalassaemia and Haemoglobinopathies and the 15th International TIF Conference for Patients and Parents, which is to be held in Abu Dhabi from the 20-23 October, and (ii) The ‘Sultan Bin Khalifa International Thalassaemia Award’ - a unique activity through which innovative medical and translational research, excellence in the work and services of clinical centres and Thalassaemia Societies, and leadership in the field will be rewarded.

Abu Dhabi, the capital of the United Arab Emirates (UAE), is rapidly developing into a vibrant, modern city while at the same time offering visitors its rich cultural heritage, which, coupled with the unique hospitality of the hosts, guarantees an unforgettable experience. Added to this, the programme compiled by the Higher Scientific Committee - comprised of renowned international experts who are authorities in their field - promises an extremely high scientific level and a unique forum for developing collaborations and partnerships, within and between the medical and the patients/parents communities.

Augmenting the scientific conference, rich patient/ parent programmes - one for thalassaemia and one for sickle cell anaemia - will offer the patient community the opportunity to discuss topics and themes of common interest and concern, while a number of ‘side’ meetings, such as the 2nd Pan-Middle East and WHO meetings, the expert patients programme and other satellite symposia, will indeed offer valuable and very exciting sources of information and topics for discussions for all.

Dear friends, these are events not to be missed by any directly or indirectly involved with these diseases.

Both of the above-mentioned activities constitute a significant step forward in strengthening education, awareness, networking, empowerment and motivation of the medical, research, scientific and patients/ parents communities - vital ingredients for promoting

and achieving TIF’s mission and vision of developing and implementing national control programmes for haemoglobin disorders, thalassaemia and sickle cell anaemia; an effort that is essential to supporting the establishment of ‘equal access’ of patients to quality medical and other care - not only in the East Mediterranean Region (EMR), but also across the world.

The TIF Board is confident that both activities will bring about a new era, and lift the work of TIF to an upgraded phase, enabling it to better and more effectively support the cause of combating these disorders and their widely challenging needs and associated pain, emotional agony, fear and uncertainty for the future.

This special magazine issue will also include a brief version of other important news, including the ‘Executive Summary’ of the 3rd Pan-European Conference on Haemoglobinopathies and Rare Anaemias that took place in October 2012 in Cyprus, the 2013 ‘8th of May’ theme and message, and a summary report of the delegation visits and/ or workshops already finalised, upcoming shortly or on-going, together with those planned for 2013.

My message to each and every one who reads this issue is: • Join us in our activities for better health and quality of life for our patients across the world; education and knowledge lead to strong patients’ voices, which is of pivotal importance for improvements; • Join us in our ‘8th of May’ activities and promote this year’s theme (http://www.thalassaemia.org.cy); • Join us in Abu Dhabi for TIF’s biggest and most important biennial educational happening in October 2013 (http://www.thalassaemia.org.cy); www.tif2013.org; • Take advantage, use and do not miss the opportunity to apply for the unique ‘Sultan Bin Khalifa International Thalassaemia Awards’; • Join us for better days and a brighter future for our patients, wherever they may reside!

Panos Englezos President Thalassaemia International Federation

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TIFMAGAZINE

Foreword from TIF’s President

“Join us in our activities for better health and quality of life for our patients…”

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Contents Chief Editor: Dr. Androulla Eleftheriou TIF Editorial Team: Mikaela Panayiotou Lily Cannon Design Editing and Layout: • Design for Life Ltd • Wordcraft Printing: Cassoulides Masterprinters

Foreword from TIF’s President

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TIF Activities Around The World

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Upcoming Activities

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Country News

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TIF Publications

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International Thalassaemia Day - 08MAY

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Upcoming International Events

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World Health Organization (WHO)

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Other News

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Patients’ Stories

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TIF Members Activity

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Calendar of Events

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Executive Summary of the 3rd Pan-European Conference on Haemoglobinopathies and Rare Anaemias

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ISSN:1450-3093 TIF BOARD MEMBERS Panos Englezos President Shobha Tuli Vice President Loisos Pericleous Secretary Riyad Elbard Treasurer Her Highness Sheikha Sheikha Bint Seif Al-Nahyan Anton Skafi Christina Stephanidou Duru Malyali Fatemeh Hashemi George Constantinou Ivan Dimitrov Ivanov Loris Angelo Brunetta Michael Michael Mouna Haraoui Nailya Guliyeva Ramli Mohd Yunus Robert (Bob) Ficarra Saeed Jafaar Al-Awadhi Cover Photo by: Noor Alam

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

2. Photos should be “print quality”. This means: • The largest size possible. Please do NOT reduce the images! Send them either one by one via email, or alternatively on a CD. • High-resolution (300 dpi). Please ensure that when transferring photos from a digital camera, a high resolution is selected.

(How this is done depends on the camera model.)

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

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

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

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INDIA TIF President, Mr. Panos Englezos and TIF Executive Director, Dr. Androulla Eleftheriou represented the Federation in an official delegation visit to India in the context of the 5th International Conference on Thalassaemia (supported by TIF) that was organized

by Thalassaemics India and held in New Delhi on 2426 November 2012. TIF representatives were received

Dr. Androulla Eleftheriou

on the prevention and management of thalassaemia and met with the Director General of the Cyprus Ministry of Health and the Head of the Molecular Genetics Department at the Cyprus Institute of Neurology & Genetics. This visit culminated with a meeting at TIF Headquarters, where a Joint Plan of Action and a Memorandum of Understanding was presented to the delegates as a framework of future collaboration with TIF.

PEOPLE’S REPUBLIC OF CHINA An official delegation from the Central Government of The People’s Republic of China visited Cyprus on the 29-30 of November 2012, following the success

GERMANY Nicolas De Nittis, an empowered patient from Germany visited TIF Headquarters on the 17 January 2013 to discuss the current status of haemoglobinopathies in Germany and the difficulties that patients face on a daily basis, despite the existence of high quality services in haematology and the health system of Germany by extension. A number of problems have been discussed and joint suggestions and solutions on how to address the situation and move forward have been documented. First on the list is the creation of a strong, dedicated patients/parents network across Germany in an effort to unite patients in the country, followed by awareness and educational activities to sensitize the medical community on haemoglobinopathies. In addition, following information regarding the unequal access to various specialized services of the healthcare system, TIF has contacted the Federal Ministry of Health in Germany concerning the routine monitoring of thalassaemia patients using MRI T2* technology.

Highlighting the importance of the effective and accurate monitoring of iron load in the heart of patients with transfusion dependent thalassaemia, TIF has advocated that it is essential to provide all patients, irrespective of geographical location, access to this technology and its use free of charge.

of the 1st Pan-South China Thalassaemia Workshop that was organized the previous month in Nanning, Guangxi province and other previous activities in 2004 and 2007. The official delegation exchanged expertise

GREECE During an official visit on 20 January 2013, on the occasion of the Greek Thalassaemia Federation General Assembly,TIF President, Mr Panos Englezos and TIF Executive Director,Dr. Androulla Eleftheriou met with patients and parents from across Greece.

Congratulations were given to the new Board on behalf of TIF and warm wishes for a successful term. In addition the TIF President discussed further issues of strengthening collaboration and the possibility of organizing the 4th Pan European Conference on Haemoglobinopathies and Rare Anaemias in Athens.

and greatly facilitated throughout their stay by TIF’s Vice-President Mrs. Shobha Tuli and met with members of the central and state government of India, industry representatives and patients associations from across India. TIF’s future actions in India and the region have been discussed in an official meeting with representatives of the WHO office for the South East Asian Region.

TIF is in close collaboration with His Excellency the Ambassador of India in Cyprus Mr. Ashok Kumar and is planning to follow up the progress in the country with a series of delegation visits/workshops throughout the year - originally planned for April, June and September 2013.

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In this visit and upon receiving an official invitation from the Panhellenic Association for the Protection of Thalassaemia Patients and its President Mr. Kostas Papageorgiou, a formal meeting was held which introduced to TIF a new initiative regarding the creation of a network of thalassaemia patient-doctors.

This has been a remarkable experience and a unique network on a global basis and TIF was exceptionally impressed. TIF strongly committed to support the active involvement of the members of this network (a dynamic list) in as many ways as possible through a framework of collaboration.

In addition, the distribution of the soon-to-befinalized translated guidelines on the management of sickle cell disease, were discussed during a meeting with ‘KINISI’ Patient Association and its President Mr. Yiannis Levidiotis who kindly proposed to undertake the distribution across Greece. In the context of its collaboration with TIF, the Greek part of TIF’s magazine, and regular updates, will be translated into Greek by the ‘KINISI’ editorial team, who in addition will undertake its distribution. Overall this delegation visit has strengthened TIF’s collaboration with the patient associations in Greece.

KINGDOM OF BAHRAIN TIF representatives Dr. Michael Angastiniotis and Dr. Matthew Demetraides attended the International Conference on Sickle Cell Disease held in Bahrain recently.The conference was organized by the Ministry of Health in Bahrain in response to the high prevalence of the condition in Bahrain and the whole of the Arabian Peninsula, and was attended by about 220 health professionals from the peninsula, together with international experts in the field of sickle cell disease, haematology and genetics. Also present were patients and members of the local association. Conference speakers from over 11 countries presented topics covering an enormous range of aspects relevant to sickle cell disease. The sessions were followed by discussions and there was a final panel discussion session.

A ’meet the expert’ programme was organized by the Ministry of Health in collaboration with the Bahrain Society for Sickle Cell Disease. International experts formed the panel, and an open discussion took place. Many topics were discussed; the main one being pain management, with patients complaining that when they go to emergency departments in acute pain, they are regarded as addicts and not believed concerning the intensity of pain. This

opened a long discussion of the issue and on correct practices in pain control.

The TIF delegates had informal meetings with the Society which caters for the thalassaemia patients of Bahrain as well as the SCD patients. Delegates also met with medical professionals and were able to discuss a wide range of related issues.

MALDIVES With the highest β-thalassaemia carrier rate globally, the Maldives constitutes a priority for TIF. In this spirit, and following a request by the Maldivian Thalassaemia Society, an official TIF delegation visited from 10-13 February 2013, to see for themselves the situation in the island nation. The TIF delegation comprised Mr. George Constantinou (TIF Board Member) and Dr. Michael Angastiniotis (TIF Medical Advisor).

The purpose behind the visit was to gain a greater understanding of the current problems facing patients, parents, carers and the medical community relating to all areas identified by the Society, and to offer help, advice and guidance where requested.

The delegation met with representatives of the Society, patients and parents, the medical community and the industry. They were briefed on issues faced by patients as a result of changes in the national control programme over the period 2008-2010, and on concerns that patient treatment is not keeping up with current developments in patient care, especially since there is now a growing young adult population of patients throughout the country.

Visits to the National Centre for Thalassaemia and other Haemoglobin Disorders, the Indira Gandhi Memorial Hospital and the Society for Health Education, and meetings with specialist medical professionals, helped the delegation to more fully understand the problems faced, and they were able to outline a series of strategies that would help the Society and the Government in assisting in the treatment and combating of thalassaemia in the country.

The visit culminated with an official meeting with the Maldivian Minister of Health who gave generously of his time, and covered many issues that were brought up during time spent with all the interested parties - including the national control programme, and the implementation of legislative decisions ratified last year for the development of services and protection of patients’ rights. The delegation said that TIF could prepare a report about the visit which would be well received by the Minister.

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TIFMAGAZINE

TIF Activities Around The World

Delegation vist to Maldives

Mr. George Constantinou

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Delegation vist to Yemen

YEMEN The first TIF investigatory visit took place in February 2013, and our representative, Mr. Stavros Melides was hosted by the Chairman of the Yemen Society for Thalassaemia and Genetic Blood Disorders. On the first day, there was an opportunity for doctors, parents, patients, medical staff and managers in Sanaa to get together and discuss the situation in the country in general. Our representative was shown around the existing treatment facility, and a new building under construction, where improved treatment will be made available.

Day two was split into three parts. The first was a medical and social presentation by Dr. Essam Dohair, from the regional collaborating office in Abu Dhabi, followed by an intense discussion between medical professionals, parents and managers from various thalassaemia/sickle cell centres.The discussion focused on the current thalassaemia situation in Yemen and how to address the future. It was apparent that there was a great desire to learn more about the global situation, and problems faced by patients - and those treating them - in other countries, and how other countries’ treatment models could be applied to the situation in Yemen.

The creation of a national federation of patients was discussed in order to provide a voice on behalf of patients. This was approved, and the intention is to embrace as many regions of Yemen as possible. It is felt that much support from outside Yemen is needed in order to establish this federation in an efficient and effective way.

It became apparent during the visit that the situation in Yemen is critical for the well-being of patients, and for those treating, caring for and supporting them.

And that although there is already an over-reliance on foreign aid, the inequity in access to treatment needs special attention from the global community.

CYPRUS From 6-8 March 2013 the Thalassaemia International Federation, in collaboration with the Ministry of Health of Cyprus - and as part of its educational programme - organized a three-day workshop covering ‘Emergency Management of Thalassaemia and Sickle Cell Disease’, held in Nicosia, Limassol and Larnaca.

The aim of the workshop was to educate and inform health professionals inAccident and Emergency, and Emergency, and Pathology Departments of Cyprus Hospitals on the treatment of thalassaemia and sickle cell anaemia. The workshop consisted of lectures and presentations of patient cases.

AMBASSADORS’ CIRCLE 1st official working meeting - 20 March 2013 Among the grandest projects that TIF has undertaken for 2013, as a means of further promoting the development and implementation of national control programmes for the prevention and clinical management of thalassaemia worldwide, is the Ambassadors’ Circle.

The objective of this prestigious group is to share ideas and knowledge on haematological genetic diseases which, in addition to being a significant public health burden, carry immense social and economic repercussions. TIF aims to highlight the fact that although considerable work has already been achieved in the direction of their control, there are still many gaps and services that need to be promoted worldwide.

TIF is committed to supporting national authorities for further progress and, with the support of members of the Ambassadors’ Circle, to promote protocols for national strategies for securing the provision of quality healthcare for every patient. To this end, a first official working meeting has been scheduled for 20 March 2013.

INDUSTRY CIRCLE Another promising project that began in 2013 is the creation of an Industry Circle for the Thalassaemia International Federation. This is in an effort to strengthen TIF’s collaboration with industries in the field of haemoglobin disorders by creating a substantial collaboration with them, offering them certain benefits. More information available on request.

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ALGERIA, April 2013 TIF is organizing an official delegation and three educational workshops in the cities of El-Tarf, Skikda and Annaba in Algeria during 12-17 April 2013.

This follows TIF’s three previous delegation visits and activities in the country. The workshops aim to educate medical specialists on the management of thalassaemia, and to sensitize local health authorities to take decisions on the control (prevention and management) of thalassaemia. For the patients and parents, the workshops will focus on educating them on thalassaemia, as well as on capacity building and how they can use their associations to promote awareness throughout the country and contribute to any decisions to be taken in collaboration with the national health authorities and the medical community.

AFGHANISTAN, May 2013 A first official exploratory delegation visit, organized

by TIF, to Afghanistan will take place in May 2013.

This is after receiving numerous cries for help from individual patients and other non-governmental organizations in the country. The aim of the visit is to investigate the current situation of thalassaemia in the country, and to establish contacts with the medical community and the Ministry of Health, as well as patient & parent associations.

TIF has one association member in Kabul: the Life-Saving Organization for Afghanistan. Through communication with them, we are aware of the existence of Khateez Thalassaemia Foundation in Jalalabad. Recently, a report/request was received by TIF from an English-speaking patient in Afghanistan, and this is the basis for the visit.

UPCOMING DELEGATION VISITS Greece

29-30 March, Delegation visit

Algeria

12-17 April, Delegation visit & workshop

India

20-22 April, Delegation visit & workshop

Morocco

May, Delegation visit

Afghanistan

May, Delegation visit

Russian Federation 7-9 June, IV Interregional Conference

of National Society of Paediatric

Haematologists and Oncologists India

July, Delegation visit & workshop

Philippines

8-11 September, 43rd Philippine Society of Haematology and Blood Transfusion Annual Convention

Oman

September, Delegation visit

Kuwait

September, Delegation visit

Vietnam

October, Delegation visit

Tunisia

December, Delegation visit

China

December, Delegation visit & workshop

** These Delegation Visits & Workshops are subject to change according to local circumstances

MOROCCO, May 2013 After three or four previous visits to Morocco, an official high-level follow-up visit by a TIF delegation is planned sometime in May 2013. This is to ensure the effective implementation of the national thalassaemia policy that was recently put forward, and also in accordance with the officially co-signed joint plan of action between TIF and the then (2011) Minister of Health. The delegation will visit in collaboration with the TIF Regional Collaborating Office in Abu Dhabi.

Her Highness Sheikha Sheikha Bint Saif Al Nahyan will lead this high-level official delegation, which is organised in cooperation with the TIF’s Regional Collaborating Office in Abu Dhabi.

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TIFMAGAZINE

Upcoming Activities

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Briefings from around the world

ALBANIA In October 2012 the Minister of Health, Mr. Tavo, issued an order to hospitals in Tirana and Lushnja where patients with thalassaemia can be treated.

This requested the hospitals to make sure that the protocol for treatment is carefully followed, and that patients can also have treatment during the afternoons (thalassaemia wards only open Monday to Friday from 8am to 2pm). This is very important because many patients work or study during the day, and have difficulties in obtaining treatment during the usual opening hours.

The moves were seen as a giant leap forward for Albania in the treatment process. However, there is much disappointment over the non-implementation of the order issued - we are informed this is caused by staff shortages, and we hope the situation will improve quickly.

Treatment is also difficult on many occasions due to the lack of blood supplies. This is because the blood-donor programme is voluntary, and not enough people come forward to donate.

TIF Publications LUXEMBOURG Haemoglobinopathies in Luxembourg (1978-2012) Prior to 1978 endemic haemoglobinopathy was almost totally unknown in Luxembourg. Dr. Paul Groff, Head of the Haematology Division at the National Health Laboratory, together with a small dedicated team, began haemoglobin diagnostic work. 35+ years later the division still operates from the well-equipped Hb laboratory that was established in the very early days.

In 1993, Dr. Groff founded the Association Etudier et Combattre les Maladies de l’Hémoglobine (ECMH) to provide advice to patients and families regarding the risks of this serious disease, and ECMH became aligned to TIF. The challenge comes now and for the future, as Dr. Groff has retired and to date no replacement has been appointed. Also the bioengineer and geneticist are planning to retire this year.We do not know what the future will hold.

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UPCOMING TIF PUBLICATIONS • Community Awareness Booklets on α-thalassaemia, β-thalassaemia & Sickle Cell Disease (Greek).

(Eleftheriou A) - expected to be printed in March 2013 • Prevention of Thalassaemias and other Haemoglobin Disorders,Vol 1, 2nd Edition (Old J, Galanello R, Eleftheriou A, Traeger-Synodinou J, Petrou M, Angastiniotis M) - expected to be printed in April 2013 • Guidelines for the Clinical Management of Non-Transfusion Dependent Thalassaemias (Cappellini M D, Taher A, Musallam K) - expected to be printed in April 2013 • Sickle Cell Disease: Guidelines for the Clinical Management of Adults and Children (Greek). Translated

by Farmakis D - expected to be printed in June 2013 • Sickle Cell Disease: A booklet for parents, patients and the community, 2nd Edition (Inati-Khoriaty A) - expected to be printed in September 2013 • Guidelines for the Clinical Management of Transfusion Dependent Thalassaemias, 3rd Edition (Cappellini M D, Cohen A, Eleftheriou A, Piga A, Porter J, Taher A) - expected to be printed in September 2013 LIST OF PUBLICATIONS NEW! A Guide for the Haemoglobinopathy Nurse (2013) - Aimiuwu E, Thomas A, Roheemun N, Khairallah T, Nacouzi A N, Georgiou A, Papadopoulou C

GERMANY TIF has received encouraging news from the medical community that five university hospitals in Germany have been given an official national mandate from the German Society of Paediatric Oncology and Haematology (GPOH) to organize a sickle cell disease programme in Germany, and that a thalassaemia programme will follow soon. We hope that this will be a first step towards improving the situation of haemoglobinology patients in Germany.

BULGARIA TIF’s position paper ‘Chronic Hepatitis C in Transfusion Dependent Thalassaemia’, and subsequent meetings with key public health officials in Bulgaria have gone a long way to support the hard work of the Thalassaemics Organization in Bulgaria (TOB), together with the medical community, in their efforts to secure better treatment and conditions for thalassaemia patients infected with hepatitis viruses.

These combined efforts have resulted in the development by the Bulgarian Ministry of Health of a specific treatment protocol for the treatment of chronic hepatitis C for transfusion-dependent patients, including thalassaemia patients.

Congratulations and we hope this constructive action will bring many positive results!

TIF Publications cater to the needs of readers of all ages and educational backgrounds, and provide concise upto-date information on every aspect of thalassaemia - from prevention to clinical management.TIF’s publications have been translated into numerous languages in order to cover the needs of the medical, scientific, patients and parents communities and the general community.

TIFMAGAZINE

Country News

NEW! Emergency Management of Thalassaemia (2012) - Porter J, Taher A, Mufarij A, Gavalas M NEW! Prevention of Thalassaemias and Other Haemoglobin Disorders, Vol. 2: Laboratory Protocols (2012) - Old J, Harteveld C L, Traeger-Synodinos J, Petrou M, Angastiniotis M, Galanello R

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

http://juliekornblum.wordpress.com/

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

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

Hard copies and CD-ROM or DVD versions can be ordered directly from TIF and are distributed free of charge. Place your order at thalassaemia@cytanet.org.cy The translation of TIF’s educational publications into various languages continues in 2013.

All translated publications are or will become available on our website.

Check with us to get updated on the latest translations!

Please visit our website at http://www.thalassaemia.org.cy/publications.htm

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Dear Friends, Dear Collaborators,

May 8th, a day devoted to the rights of patients to quality healthcare

The decision to dedicate one day of the year to spreading awareness and knowledge about aspects of the prevention and management of thalassaemia, derived from the need to unite and strengthen the patients’ voice worldwide: an essential pre-requisite to achieving our mission aimed at the promotion of equal access to quality healthcare.

Every year on the 8 May since 1994, we bring to our thoughts, and pay respect and recognition to all those anonymous patients, parents and families who have fought for a better future for patients with this disease and are unfortunately no longer with us, but indeed have left to the world an immense and invaluable history of altruism, passion and commitment and have paved the way for all of us to continue.

Every year, TIF proposes a different theme and encourages member associations and officials to focus on it with activities and actions.

This year’s Thalassaemia Day is devoted once again to the rights of patients to quality healthcare.

However, we extend this year’s focus on patients beyond those with β-thalassaemia major.

More specifically, this year’s theme is: ‘’The right to quality healthcare for every patient with thalassaemia, major and beyond.’’

The Thalassaemia International Federation calls on the global thalassaemia family - all the patients’ associations, members and non-members, the global health-professional community, national and international health authorities and organizations - to unite their voices and raise awareness of thalassaemia and other haemoglobin disorders and their control.

It is essential and very timely to extend and

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disseminate the knowledge and experience that has been accumulated over the years in many parts of the world, not only for β-thalassaemia major but also for other non-transfusion dependent or infrequently transfused forms of thalassaemia. The latter are forms of haemoglobin disorders which for many decades remained under-estimated and under-recognized with regards to their clinical significance. The physical history and medical needs of patients with forms of thalassaemia such as β-thalassaemia intermedia, HbE/ β-thalassaemia and HbH disease (α-thalassaemia) were indeed very poorly understood and consequently inappropriately addressed clinically. High rates of morbidity and very poor quality of life have been noted and reported for decades amongst these patients.

Considerably more knowledge exists today, which is scientifically based, and data is available to guide the medical community and the national health authorities to MOVE FORWARD and develop and implement prevention and management protocols tailored to the needs of these patients.

Today this patient community is “fortunate” because, mainly as a result of the focus and research on β-thalassaemia major through the years, considerable knowledge, expertise and experience are on the table for scientists and researchers to build on and so extend support to patients with these other forms of thalassaemia.

We therefore encourage first and above all, you, the global patient community, on this day and through the next year, to become acquainted with this new information and to spread awareness - aiming to sensitise every related stakeholder and partner in this fight, including the medical community, the policy makers and the industry.

TIF prepared a position paper in 2012 (position paper 5.4) on this topic which is available on our website,

www.thalassaemia.org.cy and we will shortly provide you with a proposed campaign kit containing patientoriented information. The kit will also include a video with patients’ stories,logo,banner and other documents to facilitate your activities in your own countries.

We propose a workshop and/or a press conference in 2013 to take place in every country, to allow you to communicate effectively this year’s message.

Dear friends, under such economic global austerities, it is our responsibility and duty as patients organizations, while continuing the fight for adequately managing β-thalassaemia major, to focus on improving the health and lives of our friends with these other forms of haemoglobin disorders. Unless we ourselves become knowledgeable and true ambassadors of recent advances, these conditions will continue to remain under-recognised and under-estimated.

I would like to end this short introduction with a few of our most challenging messages:

TIFMAGAZINE

International Thalassaemia Day - 08MAY ‘’Unity is our strength’’ ‘’Knowledge is our power’’ ‘’Nothing for the patients, without the patients’’ ‘’Equal access to quality healthcare for every patient’’ Best regards, Panos Englezos President Thalassaemia International Federation

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“…we aim to provide highquality scientific and educational content that we have come to expect from TIF members from around the world…”

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Upcoming International Events Invitation from Her Highness Sheikha Sheikha Bint Saif Al Nahyan It gives me immense pleasure to invite you all to attend the TIF 2013 World Congress, which combines both the “13th International Conference on Thalassaemia & the Haemoglobinopathies” and the “15th TIF International Conference for Patients and Parents”, to be held between 20-23 October 2013, at the Abu Dhabi National Exhibition Centre, United Arab Emirates.

I would like to extend a particularly warm welcome to all the participants, delegates, patients and their families, as we aim to provide high-quality scientific and educational content that we have come to expect from TIF members from around the world together with a unique social and cultural programme that truly reflects the Arabian hospitality of the UAE.

We have chosen to hold the conference in one of the most modern exhibition centres in the world - the Abu Dhabi National Exhibition Centre (ADNEC), to ensure that all participants experience a stimulating educational environment.

Preparations are well underway to ensure that you, and your accompanying guests, experience the very best of Abu Dhabi, whereby you will be able to see, hear and taste the unique Arabian culture and indeed warm hospitality.

I look forward to seeing you all in Abu Dhabi.

Her Highness Sheikha Sheikha Bint Saif Al Nahyan TIF 2013 Congress Chairperson www.tif2013.org

Invitation from Mr Panos Englezos I am delighted to cordially extend an invitation to you to attend the 13th International Conference on Thalassaemia and the Haemoglobinopathies, and the 15th International Conference for Patients and Parents, which will take place in Abu Dhabi, United Arab Emirates in October 2013.

The global thalassaemia family is extremely honoured and privileged to have this conference held under the auspices of Her Highness, Sheikha Sheikha Bint Seif Al Nahyan, President of the United Arab Emirates Thalassaemia Society. The great history of Her Highness’s magnanimous humanitarian work and her personal commitment and wholehearted devotion in alleviating the pain and suffering of patients with chronic diseases such as thalassaemia and sickle cell disease guarantees the success of this educational event, which is the biggest and most significant activity in the context of TIF’s educational programme.

The Middle East is a region where significant progress has been achieved over the decades with regards to the management of the haemoglobin disorders. Now, with the family of H.H. Sheikh Sultan Bin Khalifa Al Nahyan as an ally,we are confident that together we will have an even greater impact in enforcing equal access and excellence in the treatment of patients with haemoglobinopathies, not only in the region, but globally.

In recent years, considerable and important scientific and medical advances have shed light on many aspects of the prevention and management of the haemoglobin disorders beyond sickle cell disease and β-thalassaemia.And importantly, nowadays, strong networks and research programmes have developed globally in almost every region of the world, bringing medical specialists, patients, their families, and public health officials together to share knowledge and experiences.

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This conference will be very special, in that for the first time the Sultan Bin Khalifa International Thalassaemia Awards will be presented to those individuals, researchers, centres and patients, who have excelled in their work and contribution in the field.

Abu Dhabi is a place of magnificent beauty, offering an intriguing blend of the old and new, through its unique heritage and culture and many modern attractions. I am confident that our collaborators, the H.H. Sheikh Sultan Bin Khalifa Al Nahyan Humanitarian and Scientific Foundation and our local and regional collaborators, will exert every effort to facilitate the stay of each participant, putting together a conference to remember both in terms of scientific offerings and of social activities.

Along with our hosts, I take the honour to invite you to experience the exquisite hospitality of Abu Dhabi, and to join us in this world-renowned educational event.

I look forward to seeing you all in Abu Dhabi in October 2013.

13TH INTERNATIONAL CONFERENCE ON THALASSAEMIA AND HAEMOGLOBINOPATHIES 20-23 OCTOBER 2013 ABU DHABI, UAE

SCIENTIFIC PROGRAMME (PRELIMINARY)

On behalf of the Board of Directors Panos Englezos President Thalassaemia International Federation TIF2013 Congress Co-Chairperson www.tif2013.org

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SCIENTIFIC PROGRAMME

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PATIENTS & PARENTS PROGRAMME

15th TIF INTERNATIONAL CONFERENCE FOR PATIENTS AND PARENTS 20-23 October 2013 - Abu Dhabi National Exhibition Centre

PATIENTS & PARENTS PROGRAMME (PRELIMINARY)

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Thalassaemia Patient Advocacy Workshop at the 13th International Thalassaemia Conference on Thalassaemia & Haemoglobinopathies and the 15th International Conference for Patients & Parents - Abu Dhabi, 21 October 2013

Your opportunity to reconnect, update and identify - so as to strengthen the patient voice

Background Despite the leadership and positioning of TIF as an international patient organization dedicated to providing education to patients and caregivers as well as advocating for the right to quality healthcare for all thalassaemia patients, many patients and families are still struggling. Several thalassaemia organizations have been making local or regional efforts to not only create awareness, but work closely with the scientific community to reduce current discrepancies.

Today, there are more than 100 thalassaemia organizations pursuing the same or similar objectives: to improve thalassaemia patient outcomes and provide quality of life to their members.This huge goal cannot be achieved in isolation. Taking into account the incremental challenges in the healthcare systems and the attention to the patient voice from the decision makers, the 13th Thalassaemia Conference represents a great opportunity to gather thalassaemia patient group leaders from around the world to share and learn from each other about the current situation and the future of this disease.

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Objectives 1. Reconnect the thalassaemia patient advocacy organizations and update the key challenges the organizations are facing in order to help them achieve their goals 2. Get a scientific update about critical new trends in thalassemia population 3. Identify areas for international collaboration in the future to strengthen the patient voice.

Methodology Participants might answer in advance an online questionnaire to identify the key challenges in the advocacy role, as well as the strengths and success stories to be analysed and presented, in order to provide context for a productive and collaborative workshop.

A moderator will help to drive the discussion and facilitate the “team work” during the workshop.

Participants might identify a solution for one or more of the issues discussed earlier. They might leave the session with a feeling of belonging to a network of independent organizations seeking similar goals and open to exploring areas of cooperation

Call for Abstracts The scientific committee welcomes the submission of abstracts for oral and poster presentations.The deadline for submission is June 2013 For more information go to http://www.tif2013.org/call_for_abstracts.html REGISTER NOW! Registration Fees

Physicians Patients, Parents and Accompanying Person Nurses, Technicians, Allied Healthcare Medical Students Residents

Early-Bird Deadline 15 July 2013 USD 550 USD 150 USD 350 USD 250 USD 300

Pre-Registration Deadline 10 October 2013 USD 650 USD 200 USD 450 USD 300 USD 350

Onsite Fee After 10 October 2013 USD 750 USD 200 USD 500 USD 350 USD 400

For registration details and conditions go to http://www.tif2013.org/registration.html

Accommodation As a thriving, modern metropolis, Abu Dhabi offers an endless number of award-winning hotels. A variety of 5, 4, and 3 star official congress hotels is available to suit all tastes, requirements and budgets. For hotel bookings go to http://www.tif2013.org/accommodation.html

TIF’s Sponsorship Programme for Patients with Haemoglobinopathies (Thalassaemia and Sickle Cell Disease) 15th TIF INTERNATIONAL CONFERENCE FOR PATIENTS AND PARENTS 20-23 October 2013 - Abu Dhabi National Exhibition Centre As for every international conference and other educational events, the Thalassaemia International Federation (TIF) will operate a Patients Sponsorship Programme to enable as many patients as possible to attend the 15th TIF International Conference for Patients & Parents which will take place in Abu Dhabi on the 20-23 October 2013.

This year’s sponsorship programme will award a sum of money to each successful applicant.

Different levels of sponsorship will be allocated to successful participants according to their country of origin and residence, based on the World Health Organization (WHO) country division: • WHO Eastern Mediterranean Region : 600USD per successful applicant

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• WHO European and African Region: 800USD per successful applicant • WHO South East Asian, Western Pacific and Pan American Region: 1000USD per successful applicant.

Succesful applicants will be notified via email as soon as the decisions are taken and not later than 30th of June 2013.

If you wish to be considered for a patient sponsorship, please visit TIF’s website at www.thalassaemia.org.cy for more information about the criteria for succesful applicants and for downloading the application form.

Sponsorship Applications Deadline: 15 May 2013 Late applications will not be given priority.

Submissions at: thalassaemia-mp@thalassaemia.org.cy

Photos are taken from the following link http://visitabudhabi.ae/

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Register now, and don’t forget to book your accommodation!

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World Health Organization (WHO)

WHO and TIF together making a difference

Regional Consultation Meeting in South-East Asia Region On 25-27 February, in New Delhi, India, the WHO Regional Office for South-East Asia organized a Regional Consultation Meeting to develop a Regional Strategic Action Plan with Indicators and Targets for Prevention and Control of Non-Communicable Diseases in the South-East Asia Region.

TIF Medical Advisor, Dr. Michael Angastiniotis, actively participated with a 3-minute statement at this important meeting.This initiative is a landmark for the SEARO region and constitutes a reward for TIF’s long-term efforts in the development of an action plan to reduce the public health burden of NCDs. Its specific objectives were to: 1. develop a Regional Strategic Action Plan (20132020) for the prevention and control of noncommunicable diseases in the South-East Asia Region; 2. discuss the role of different sectors in prevention and control of non-communicable diseases; and 3. develop a regional monitoring framework, indicators and targets for prevention and control of noncommunicable diseases taking into account the global voluntary targets.

The session was attended by representatives from 119 Member States, one regional economic integration organization, one intergovernmental organization and 17 nongovernmental organizations.

Statement from the Thalassaemia International Federation: Mr. Chairman, Ladies and Gentlemen It is a gratifying sign of the progress achieved over the years in the health profile of many nations that non-communicable diseases have come to be recognised as a health priority, in view of the heavy burden of infectious diseases and malnutrition still prevalent but gradually taking second place. This change also comes at a time when health systems are being reviewed in many nations, and new ideas are being introduced to address the economic and other consequences of the changing disease pattern and the aging population.

In all WHO regions the issue of non-communicable diseases has become synonymous with the four major categories of cardiovascular diseases, diabetes, cancer and chronic respiratory diseases. The Thalassaemia International Federation represents a

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group of chronic, hereditary disorders affecting the haemoglobin molecule, which are the commonest of a much wider group of chronic disorders, each disorder rare in itself, but which as a whole affect a very large proportion of the population and have very serious social consequences. These rare and/ or chronic disorders are mentioned in the SEAR documents and deliberations on non-communicable diseases under the general title of birth defects and are given a secondary role.

Our intervention aims to emphasise the need for a closer look at these disorders and to consider a separate approach in planning health services from the strategies and plans under discussion in this Consultation.

Concerning the haemoglobin disorders it must be noted that they are highly prevalent in the countries of SEAR, with the exception of the Democratic Republic of Korea. From the epidemiological database of our organization, we note that at least 40376 new cases are added each year suffering from beta thalassaemia syndromes (1.12/1000 live births) and 20108 cases suffering from sickle cell syndromes (0.56/1000 live births) a total of 1.68/1000 live births.These are added to the many thousands of patients already alive with these syndromes and who expect a long and good quality of life by the provision of adequate treatment, which must be lifelong and is beyond the reach of individual families. There are well-documented strategies which can address both the prevention and the management of these disorders, but which are not included in the health planning of most countries of the region. This despite WHO resolutions WHA 59.20 and EB118.R1 on Haemoglobin Disorders as well as WHA63.17 on Birth Defects. A few countries, such as Sri Lanka, Thailand and the Maldives, have taken significant initiatives.

Birth defects are in general underestimated.

If we consider that there are more than 5000 rare diseases listed, most of which are congenital and chronic, and which according to western sources affect 6-8% of the population, 6% even if each disorder is rare, then we can expect over 100 million people in SEAR to be affected. This is not a negligible health burden when considering the chronicity and complexity of care that they require.

It is the position of our organization that the haemoglobin disorders as well as the many other chronic and hereditary disorders require special attention and separate policies from the four major non-communicable

diseases. In the WHO agenda for non-communicable diseases we propose that a major section is initiated for these diseases, in order to establish strategies and policies suitable for the countries of each WHO region.

Thank you.

WHO Global Forum for Blood Safety, 6-8 May 2013, Sao Paulo, Brazil TIF will be represented by Mr. Riyad Elbard, Treasurer and long-term member of the Board of Directors of the Thalassaemia International Federation, a patient himself and with a special interest in blood safety.

The WHO Blood Transfusion Safety Programme, in collaboration with WHO PAHO/AMRO, WHO/ PAHO Country Office in Brazil, and with the collaboration and generous support of the Brazilian Government, will convene the WHO Global Forum for Blood Safety (GFBS) in Sao Paulo, Brazil on 4-6 March 2013. WHO has established this global forum as a mechanism to foster collaboration, and enhance communication and information exchange among key international experts, institutions, organizations and other stakeholders working on blood safety issues.

The forum meets once in two years to deliberate on a specific topic relevant to global blood safety concerns, in order to make structured observations and identify priorities for action at national and international levels.Among others, the key themes for this forum were Blood System Reforms for Universal Access; Quality Management; and Risk Assessment and Management for Blood Safety.

Response by TIF to the proposal for the inclusion of Whole Blood and Red Blood Cells on the WHO Essential Medicines Lists (EML) The Thalassaemia International Federation (TIF) is a non-profit organization dedicated to improving quality of life and life expectancy for patients with thalassaemia and other haemoglobinopathes. TIF is comprised now of 91 member associations from 47 countries and has been working in official relations with WHO since 1996.

Recognizing the public health burden of these diseases, theWorld HealthAssembly (WHA) has formally adopted the following specific resolutions in May 2006: EB118.R1 on thalassaemia and other haemoglobinopathies, and WHA59.20 on sickle cell anaemias.

WORLD BLOOD DONOR DAY - 14 JUNE 2013 theme: “Every blood donation is a gift of life” The focus for this year’s campaign - the 10th anniversary of World Blood Donor Day - is blood donation as a gift that saves lives. WHO encourages all countries to highlight stories from people whose lives have been saved through blood donation, as a way of motivating regular blood donors to continue giving blood and people in good health who have never given blood, particularly young people, to begin doing so.

The objectives of this year’s campaign are to: • thank blood donors for their life-saving donations; • promote voluntary unpaid blood donation; and • convince ministries of health to commit to achieving self-sufficiency in safe blood and blood products based on 100% voluntary unpaid blood donation.

The host country for World Blood Donor Day 2013 is France.

To get updated on this year’s activities and future events, visit the website: http://www.who.int/campaigns/world-blood-donor-day/2013/en/index.html

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Blood transfusion therapy is the cornerstone and the essential first arm of the medical management of patients with β-thalassaemia major, and is also required to ameliorate certain medical complications for patients with other major haemoglobinopathies such as sickle cell disease and patients with milder forms of haemoglobin disorders.

Our global patient community constitutes perhaps the largest numbers of transfused and lifelong users of whole blood and/or red blood cells (RBCs). In this context, our interest in the safety, efficacy, cost-effectiveness and adequacy of blood is immense and the quality of whole blood and red blood cells constitutes a major focus of our activities.

This particularly applies within the context of supporting the development and implementation of national programmes for quality transfusion services and the acquisition of blood through voluntary nonremunerated blood donation (VNRBD) practices as recommended in the WHA resolution of 1975 (WHA28.72 - Utilization and Supply of Human Blood and Blood Products); the EU legislation (Directive 2002/98/EC) and in AABB recommendations. In addition, TIF has for many years focused on the implementation of other key resolutions, directly or indirectly related to blood and blood safety. These resolutions include: WHA56.30 Global Health-Sector Strategy for HIV/AIDS(2003); WHA58.13 Blood Safety: Proposal to Establish a World Blood Donor Day (2005); WHA63.12 Availability, Safety, and Quality of Blood Products (2010);The Melbourne Declaration on 100% VNRBD (June 2009); and WHA63.18 Viral Hepatitis (2010) - all of which focus among other things on the relationship of VNRBD and safety of blood and quality of transfusion practices, and on the rationale, that blood is not a medicine and cannot be bought. TIF also provided a statement to the WHOEB126 (January 2010) on the agenda item ‘Availability, Safety, and Quality of Blood Products’, highlighting the need for safe blood systems based on VNRBD as essential public health functions of governments.

As TIF we do not feel very confident and comfortable - and indeed, have not clearly and fully understood - the underlying reason behind the proposal for inclusion of Whole Blood and Red Blood Cells on the WHO EML. Particularly, because according to the above resolutions - it was advocated

and agreed that blood, as a substance of human origin, is excluded from becoming a commodity. And moreover, we believe that the ‘essential’ nature of blood has been a recognized issue since the early days and thus requires no further underlining. Therefore, the potential negative effects that this proposal may have on national efforts for promoting transfusion medicine, blood systems and VNRBD practices are issues of grave concern to us.

The lessons learned from the HIV and hepatitis epidemics that have occurred through the years, and of course the possibility of new infections occurring in the future, have put - and will continue to put - the safety of blood and blood products for transfusion into jeopardy and constitute a constant threat to our patient community, particularly if Member States choose, as a result of the inclusion of blood in the EML, to become more flexible in their sources of Whole Blood and RBCs. The experience of plasma factors that have, for some time, been included in the EML with increasing commercial involvement and paid plasma donation, adds greatly to our concerns.

It is therefore, important to underscore, at this point, that the empowerment and education of voluntary non-remunerated blood donors, and the community at large, to make regular blood donations of their own free will, without payment, has been a TIF activity that greatly enhances its awareness campaigns for thalassaemia prevention and treatment.

The nature of human blood and its description as a gift of life or as a precious act of love have been used

by TIF to encourage and promote voluntary nonremunerated practices for many years. A number of our Federation position papers - and certainly many of our educational activities - focus on the clear relationship of voluntary non-remunerated donation to the safety of blood and the importance of nationally coordinated transfusion services with a national blood policy, plans, legislation and regulation, as a means of securing and guaranteeing the safety and adequacy of blood - and consequently, the quality of treatment for our patients. A significant number of our member national associations globally are actively involved in blood donation campaigns, and the development of a voluntary culture occupies a central role in these. It will thus be very difficult for TIF to ‘suddenly’ support such drastic changes.

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e-ENERCA TIF is pleased to inform you that e-ENERCA new e-Health Services for the European Reference Network on Rare Anaemias - has been accepted for funding from the European Commission’s Executive Agency for Health and Consumers under the call for proposals in the 2012 second programme of community action in the field of health (2008-2013).

The general objective of the e-ENERCA project is to assure the access of professionals and patients to the same level of services, independently from the country of practice and the origin of the patient. For this, several e-health information and communication technology tools will be developed, aiming to comprehensively collect clinical and epidemiological data across Europe and to share knowledge and expertise among Member States, with specific measures foreseen to include the new EU Member States.

TIF is the leader of the Work Package on European Epidemiological Surveillance for Major Rare Anaemias, which aims to develop a centralised electronic registry service for patients with haemoglobin disorders.

the FDA’s accelerated approval programme, giving patients earlier access to promising new drugs intended to treat serious or life-threatening illnesses while the company conducts additional studies to confirm the drug’s clinical benefit. Exjade was approved based on clinical data showing it can reduce LIC to less than 5 mg/g dry weight, a surrogate endpoint that is judged reasonably likely to predict a clinical benefit to patients.

For more information: http://www.fda.gov/NewsEvents/Newsroom/ PressAnnouncements/ucm336478.htm THALASSAEMIA CLINICAL TRIALS In an effort to make services more accessible, Ithanet has developed a web-page dedicated to clinical trials concerning thalassaemia that are currently underway around the world. The page is searchable, and can be accessed at the following website http://www.ithanet.eu/index.php/clinical-trials

FDA

THALAMOSS ITALY

FDA approves Exjade to remove excess iron in patients with genetic blood disorder The U.S. FDA has further approved use of Exjade (deferasirox) for patients 10 years and over who have chronic iron overload resulting from the genetic blood disorder non-transfusion-dependent thalassaemia (NTDT). NTDT is a milder form of thalassaemia that does not require frequent red blood cell transfusions.

However, some patients are still at risk for iron overload that can lead to damage to vital organs.

The FDA is also authorizing FerriScan as an imaging companion diagnostic for Exjade therapy in NTDT patients. The agency previously cleared FerriScan for measuring liver iron concentration (LIC), but its use in Exjade clinical studies to select patients for therapy, and to manage therapy, defined its role as an imaging companion diagnostic necessary for Exjade’s safe and effective use. FerriScan measures LIC noninvasively using magnetic resonance imaging.

Exjade should be used in patients with NTDT who have an LIC of at least 5 milligrams of iron per gram of dry liver tissue weight.

Exjade’s new indication is being approved under

The first meeting of THALAMOSS (THALAssaemia MOdular Stratification System) was held in January at Ferrara, Italy. THALAMOSS is a research project funded by an EU research and development programme and brings together 13 leading European and US research institutions, and European and Israeli SMEs. The ITHANET Portal partners the project. At the meeting - entitled ‘From Basic Research to Novel Applications on the Road of Personalized Treatment of Thalassaemia’ - it was announced that the project aims to develop novel methods for associating variation in genomic data with phenotypic variation.THALAMOSS will combine cutting-edge computing technology with optimized algorithms to mine unique datasets provided for biologically and medically relevant patterns that can be reliably associated with specific patient groups, treatment response and disease progression. In addition to stratification of patient samples for their molecular properties, it will also analyse responsiveness to advanced therapeutic approaches to classify patient samples, analysing this responsiveness both as a consequence of molecular properties and as a determinant of the success of novel therapies.

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Patients’ Stories Report on the 3rd Pan-European Conference on Haemoglobinopathies & Rare Anaemias 24-26 October 2012 Grand Resort Hotel - Limassol, Cyprus Theme and mission statement: Knowledge is our power - Unity is our strength.

Aim: Bringing people together from many countries, joining together to share knowledge and educate.

Purpose: Understanding the current role of thalassaemia in the various medical fields, the socio economics of the condition and the importance of education.

“A positively overwhelming experience. I am privileged to have been elected to go…”

In conference afterglow, let me reflect on an experience that has made a mark on my life.

I cannot say it was easy to leave the family behind, and l questioned whether this was the right decision for me and my family.

After all the farewells were made, l stepped onto the plane with my heart in my mouth, equally excited and apprehensive, about the conference and the people l would meet and the stories that would be shared, their insights and ultimately, the knowledge to be gained at a conference about conditions that have completely re-prioritised our existence.

At the Grand Resort Hotel - the conference venue - I was handed my delegate pass. It read patient/parent, and it dawned on me what my presence represented not just a parent of a child with this condition, but all parents at home with children with these conditions.

I promised myself to meet as many people l could, talk to as many people time would allow, absorb as much as my head and heart could take.

I was eager to experience the opening ceremony.

There were many people waiting to enter, and many were obviously regular attendees. I began reading passes; there were diverse groups - medical, scientific, officials, the Sheik, patients and parents.

I entered the next day looking with new eyes; I saw a collection of people eager to get started - some to present, others to listen. Progressing through the schedule l felt reassurance, knowing that this conference meant something to people: “The Quest, a forum for everyone, for exchanging of knowledge and experience to create a greater understanding for all.” I also felt reassurance knowing that conferences like this exist; that there are passionate people

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putting these events together to assist those touched by these conditions; doctors and specialists dedicated to the various conditions, research scientists, seeking cures - no matter how ‘Sci-Fi’ some of them sounded.

There are also philanthropists and members of various Thalassaemia Societies addressing the socio-economic issues. They all come together to further the cause; prepared to look outside the box to understand, to further education about, and treatments of, these conditions. It warmed my heart to be present, to witness all this activity.

It gave me hope that we are progressing and that key issues are shifting towards better quality of life for our patients. We fully understand that these professionals are furthering their personal career goals, are forging their status in the industry, but on the flip side of this, who are we to complain if the end results are positive outcomes for the treatment of thalassaemia and like conditions?

Day 2 of the patient/parent conference was mostly about conditions associated to thalassaemia. Some sessions piqued my interest; a patient recounting her life with thalassaemia - focusing on how she has overcome obstacles and turned them into triumphs and now dedicates her time to the Cyprus Thalassaemia Society and is giving back.

A controversial topic was “Are rare anaemias a disability?” This had many talking during the break, some saying that they did not believe this should be a session topic as they did not want to steer these conditions down the disability path. The presentation was delivered, and surprisingly was answered satisfactorily: that the conditions themselves are not a disability, rather a disability only comes about if a patient has serious complications from associated medical issues.

The highlight for me was the session “Challenges in personal relationships” presented by Photis Eliade, delivered from a personal perspective; Photis shared his account of the important relationships throughout his life: with his parents, siblings, spouse

and children. His presentation was informative and engaging, and he reinforced the message that the condition should not affect relationships, which are what you make them. I would have appreciated more of these types of sessions, and wished that they could have been longer to really explore the different relationships. Examining patient psychology at different stages of life - infant, early childhood, teenage, early adulthood, etc. - would have also been of interest, as would a focus on physical and emotional wellness, with tips and recommendations for managing the conditions.

One notable factor was Thalassaemia Australia’s presence at this conference and its position within the international thalassaemia community. Many knew and recognised our society - people from the Thalassemia International Federation, the scientific community, and from other societies. This was pleasing, especially as we are quite removed from the European community and had made the effort of representation at this event. They recognised our regional work, our staff and their contributions to patients and the thalassaemia community. One conversation l had with an important member of TIF stood out, as they mentioned that they keep up to with Thalassaemia Australia through our website, acknowledging that social media was the new tool in reaching patients and educating people about the condition.

I met many people from other nations and heard many stories; some that made me feel like running out of the room screaming, others that left me speechless.

I heard of how the European crisis is affecting the cost in some countries for the treatment of blood; how in one country, the cross-match process is not undertaken - hence numerous accounts of reactions to the blood provided; how some doctors are not knowledgeable about the conditions; of the distances many travel to receive their treatments.These stories confirmed my appreciation of our health system in Australia.

A common theme among Thalassaemia Societies was membership levels compared to numbers of patients. Many societies have low membership levels and difficulties retaining members. Often, patients have either become complacent as treatments have advanced, or expect others to participate in the charity/fundraiser activity, rather than themselves.

Participation in organized fundraising events is often low, and committees struggle to raise funds in the

current economic environment.

My conference really began during a break in the Friday schedule, when l had a powerful discussion with another parent who allowed me to ask her many probing questions; she listened to me, to my concerns, fears and joys. She was open and shared her stories and experiences over the years of raising her daughter with a genetic blood condition. She generously gave her time to befriend me and be my guide though this conference experience.

We covered many topics - various available treatments, medications, other related side-effects and issues. We discussed raising a child with these conditions: how it alters relationships, affects siblings; how it feels to be the parent; effective parenting tools. One topic merged into another; conversations flowed, introductions were made, more stories told and tears were shed. I cannot thank her enough for graciously giving her time; for opening up and taking me under her wing, introducing me to the greater family - the Conference family of patients and parents.

And I thank everyone else, for the stories shared and for the friendships shared and created.

The most important information l can bring back to deliver, is that Australia has a world-class medical service provider. Our patients have quality care - in treatments received, quality of blood and medications - and we have very high standards in care and treatment of these genetic blood disorders 3rd Pan-European Conference in comparison to other countries. Australia’s Medical on HaemogloTherapy Units are centres of excellence, and by binopathies & having patience and working together we can deliver Rare Anaemias a stronger service-focused solution, with information and education as the power for the future.

This was a positively overwhelming experience. I am privileged to have been elected to go, and grateful to have had the opportunity to attend.

Cheers Helen Kosmarikas Parent & Thalassaemia Australia Inc. Committee Member 2012/2013

TIF WANTS TO HEAR YOUR STORY! If you have a story that touches on some of the issues faced by thalassaemia patients and parents send it to TIF at thalassaemia@cytanet.com.cy

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award ceremony, for all the winners and participants

UNITED ARAB EMIRATES This year, under the patronage of Her Highness Sheikha Sheikha Bint Seif Al Nahyan, President of the Emirates Thalassaemia Society, an awards ceremony was held on 10 January 2013 for all the winners and participants of the 2012 competition ‘What is Thalassaemia?’ who were honoured with trophies and certificates personally by Her Highness.

The 2013 campaign was also launched, and this year the topics for awareness are: 1. ‘Together to limit the spread of Thalassaemia’ 2. ‘A family without Thalassaemia’ 3. ‘Test to be safe. Say NO to Thalassaemia’ The Emirates Thalassaemia Society intends to run this campaign annually, and with even more zeal from the students and the educational institutions throughout the UAE. The Emirates Thalassaemia Society assumes the responsibility to target our whole nation, and secure a future free from Thalassaemia.

‘What is Thalassaemia?’ is an educational competition where students from primary, to higherlevel schools, universities, colleges & institutes undertake projects related to thalassaemia. The competition has been running successfully for five consecutive years, and is very carefully categorized into three levels, where each level targets the capabilities of students according to their ages.

It not only educates and encourages our next generation to understand the benefits of premarital tests, and donating blood, but also endows us with the freshest, most fascinating, and most innovative concepts to run our awareness campaigns.

PALESTINE ‘Community Training and Awareness Project’ sponsorship and cooperation with the PalTel Group Under the auspices of Dr. Leila Ghannamthe, Governor of Ramallah & Bireh District, at a celebration on 5 May 2012 as part of the Thalassemia International Day celebrations, the signing of an agreement to sponsor the ‘Community Training and Awareness Project’ campaign workshops was undertaken between Dr. Bashar Karmi, TPFS President, and Mr. Raed Awwad, Manager of Community Services at the Palestine Telecommunications Company (PalTel Group).

PalTel Group took the lead through its social responsibility initiative to undertake this innovative endeavor in Palestine. The campaign is a 6-month project aimed at preventing new thalassaemia births by 2013. Tens of associations worked together to

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make the project a success, and help reach its targets.

Workshops were held to target health professionals, community health workers, medical students, as well as members of other NGOs working in the field.

A Media Conference was sponsored by the Ministry of Information advocating such projects with the objective of ‘Palestine free from new thalassaemia births in 2013’. The conference was hosted by: the Minister of Social Affairs Mrs. Majeda al Masri, President of TPFS Dr. Bashar Karmi, Member of the National Committee for Thalassaemia from the MOH Dr. Nazeh Abeb, the Religious Court representative, together with the Director of Planning at PalTel. Major activities of the project included: • Media Awareness: This included radio spots interviews, and newspaper articles • Blood Donations: Campaigns focusing on the importance of blood donations for both the donor and the recipient • Training and Educational Workshops: These were held in coordination with several NGOs. The culmination was the creation of a group of community educators to carry out awareness activities in their nearby schools.

PAKISTAN Shaikhupura is an industrial city and capital of Sheikhupura District in the province of Punjab, about 35 km northwest of Lahore in Pakistan. Here, there is a great need for health education in general, and because of the traditionally common practice of cousins marrying, the instances of thalassaemia are significant.

The Rifah Welfare Foundation will be holding a three-day campaign at Punjab College, Sheikhupura, aimed at educating young people about thalassaemia and the need for pre-marital screening and testing.

This will help spread the word that tests are vital if society is to control, and eliminate, thalassaemia from the community.

IRAN Charity Foundation for Special Diseases (CFFSD) Iran is one of the Middle Eastern countries with significant numbers of thalassaemia patients. These numbers have been gradually increasing over the past five years, and are now estimated at 19000.The increase is due to the cultural and ethnic infrastructure, and in many provinces incidents of new-born thalassaemics has not been controlled satisfactorily, owing in part to traditional interfamily marriages.

The Charity Foundation for Special Diseases, one of the pioneers in the medical, social and mental wellbeing of thalassaemia patients, has organized and conducted a series of workshops addressing different aspects of the thalassaemia problem, to help the public and private sector gain widespread understanding about the issues and the solutions. This series of workshops was attended by members of the health authority, directors, medical and nursing staff, and all related professions in the country.

A series of workshops, conducted in 2012, included: Management issues for thalassaemia prevention.

The goals were: to raise the overall awareness of the scientific and religious authorities; to carry out epidemiologic studies; and to estimate the costbenefit of screening programmes in order to prevent the birth of thalassaemia major babies.

Management issues in thalassaemia treatment.

The second workshop reviewed the general National Protocols on thalassaemia treatment, the issues surrounding bone marrow transplants (in regard

to different techniques and their effectiveness), the potential and limitations of oral medication, the efficacy of various medications and the possibility of their coverage by insurance companies.

The complications of thalassaemia treatment.

The main objectives were the recognition and proper management of endocrine, cardiologic, and oral health complications in thalassaemic patients,with the participation of various medical universities, social welfare offices, and heads of thalassaemic wards, and haematologists.

The management of nursing and care of thalassaemic patients.

Important issues regarding the special care during transfusion, self-protection, patient nutrition, and coordinated care in a clinical setting were discussed, and after highlighting the major concerns from the caregivers’ perspective, it was agreed to have an estimation of the minimum necessary workforce required to uphold the standards and to provide related supervision directly by the specialist.

CFFSD is organizing the 5th thalassaemia workshop, entitled ‘The management of legal, social and welfare issues of thalassaemic patients’, to be conducted with the participation of social services, the Ministry of Labour and Social Welfare, the Sport Medicine Federation, and the Ministry of Health and Medical Education. This workshop will address important and sophisticated subjects, such as the availability and challenges of higher education for thalassaemics, their integration into the workforce, their marriage obstacles, and important aspects related to their quality of life in general, and we will discuss how the government bodies, as well as public awareness, can come to new concepts and fashion new laws to improve that quality.

INDIA ‘Better Treatment on Thalassaemia’ workshop On 17 February 2013, the Thalassaemia Children Welfare Society in Ulhasnagar organized a workshop on ‘Better Treatment on Thalassaemia’.The workshop was conducted by Dr. Rashid Merchant and more than 250 child sufferers of thalassaemia major attended, together with their parents and 70 doctors from the area. This workshop was supported and managed by the Youth Thalassaemic Alliance.

Dr. Rashid Merchant, a paediatric specialist at Nanavati, Mumbai, explained in simple local (Hindi) language the requirements for better treatment, safe blood, proper chelation and regular health check-ups.

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Calendar of Events

Celebrating one year of a journey...

BANGLADESH Shyamal Chhaya celebrates one year of its journey On 3 March 2012 the first anniversary of the opening of the Shyamal Chhaya shelter was celebrated at the shelter premises in Dhaka, Bangladesh.

Accommodation for overnight stays is one of the challenges faced by thalassaemic patients and their families who come from rural areas of Bangladesh to the capital, Dhaka, for treatment. Shyamal Chhaya was established to help rural patients and their families by providing temporary accommodation in Dhaka.The tworoom facility provides minimum sleeping arrangements, a shared bathroom, and a shared kitchen.

The shelter was named after Shariful Islam Shyamal, an intelligent and poetic soul, who died of thalassaemia on February 26, 2008 at the young age of 19.

Shyamal Chhaya is the personal initiative of some friends: Kamrun Nahar Mukul, Noor Alam, Zubair Alam, Zaid Islam, and others who contribute to keeping it running.

DATE

EVENT

VENUE

12-13 March

Patients’ Advocacy on NTDTs

Zurich, Switzerland

16-17 March

Global Iron Summit

Copenhagen, Denmark

20 March

TIF Ambassadors’ Dinner

Nicosia, Cyprus

25-26 March

EUROPLAN Workshop

Rome, Italy

30 March

TIF Delegation to Greece

Athens, Greece

Get inspired by the events we are organizing in Cyprus!

04 April

WHO Dialogue of Strengthening Self-Care of Non-Communicable Diseases

Geneva, Switzerland

08 April

EHA Patient Organizations Meeting

EHA Offices, The Hague

RED DAY - This year we are running a ‘Red Day’ campaign at every school! We will be disseminating information to one of the most influential groups of our society - young people! On the day we will get school students to replace their usual uniforms with red clothes, and will organize educational activities aimed at awareness.

12-17 April

Delegation Visit and Workshops in Algeria

Skikda, Annaba & El-Tarf

18-19 April

EUROPLAN Meeting

Nicosia, Cyprus

19 April

EUPATI Annual Conference

Rome, Italy

20-22 April

TIF Delegation Visit to India

Mumbai, Maharashtra, India

21-26 April

Patient Advocacy Scholar Seminar, Harvard University, Harvard Faculty Club

Cambridge, Massachusetts, USA

23-24 April

IPFA/PEI 20th International Workshop on ‘Surveillance and Screening of Blood Borne Pathogens’

Crowne Plaza, Helsinki, Finland

24-28 April

EASL International Liver Congress

Amsterdam, Netherlands

6-8 May

WHO Global Forum for Blood Safety (GFBS)

Sao Paolo, Brazil

10 May

TIF 8th of May Press Conference

Nicosia, Cyprus

16 May

European Parliament event ‘Austerity and Access to Medicines in Europe’ organized by EPHA

Brussels, Belgium

20 May

IAPO Annual General Meeting

Geneva, Switzerland

20-28 May

66th World Health Assembly

WHO-HQ Geneva, Switzerland

23-25 May

BIT’s 2nd Annual International Symposium of Haematology Xi’an, China

29 May - 1 June

EURORDIS Membership Meeting

Dubrovnik, Croatia

2-5 June

23rd Regional Congress of ISBT in Europe

Amsterdam, Netherlands

5-7 June

TIF Board Meeting and Expert Patients Training Workshop London, UK

7-9 June

IV Interregional Conference of National Society of Paediatric Haematologists and Oncologists

Moscow, Russia

13-16 June

18th EHA Congress

Stockholm, Sweden

17-21 June

EURORDIS Summer School for Patient Advocates in Clinical Trials and Drug Delevelopment

Barcelona, Spain

18 June

World Orphan Drug Congress

Singapore

21-23 June

51st Hellenic Paediatric Conference

Alexandroupolis, Greece

08 May - International Thalassaemia Day

POSTAGE STAMP - TIF in Cyprus has designed a 34 cent postage stamp that can be purchased from us and used to officially mail domestically and internationally.

So every letter sent will promote our cause and message.

Why not take our idea and do the same in your country?

We can reach the world!

The anniversary celebration was attended by thalassaemic children, friends and well-wishers, the support team of the shelter, and by Prof. Wakar A Khan, Dr. Fahima and Prof. Bilkis Banu of the Dhaka Shishu Hospital Thalassaemia Centre.

Thalassaemia workshop in India

VIETNAM The Vietnam Thalassaemia Association (VITA) has published its Annual Report for 2012, together with its projected Action Plan for 2013.

Achieving substantial progress in a short time, VITA notes that the awareness of the wider public about thalassaemia is limited, however, an investigation into the current situation with regards to thalassaemia throughout the country is still underway. Notably, the President of the Association, Prof. Nguyen Anh Tri actively participated in the 63rd WHO WPRO Regional Meeting.

Plans for this year include the organization of workshops for patients & parents to develop knowledge, as well as the development of a multi-disciplinary service complete with experts on hepatitis and endocrinology.

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CAMPAIGN KIT -We have put together a special Campaign Kit, and we are promoting it worldwide via our website. It will contain lots of promo material, including the official ‘08 May’ logo, this year’s banner, a video and plenty of educational material related not only to β-thalassaemia, but to all non-transfusion or infrequent-transfusion forms of thalassaemia. Download the kit from the TIF site and use it in your country.

MEDIA CAMPAIGNS - We will hold a press conference, an awareness workshop, a blood donation session, and other awareness events that will communicate powerfully this year’s theme and our global mission. We suggest that you do something similar…. and please tell us all about it!

GET INSPIRED! - Follow us on our website and facebook page - you will find lots more ideas and information to help you make 08 May a Very Special Day. Our mission is to promote awareness and reach 08MAY every patient - even in the most distant corner of the world! Help us to Help you!

THALA SSAEM IA INTERN ATIONA L F E D E R AT I O N

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More than 750 participants from 17 of the 27 Member States of the EU as well as other surrounding countries of the Eastern Mediterranean Region and beyond gathered in Limassol to participate in the most successful regional conference on haemoglobinopathies and rare anaemias organized by the Thalassaemia International Federation to date.

The target of the 3rd Pan-European Conference was to address the needs for increasing education and updating of the medical and patient & parent communities, and - very importantly - to provide a unique forum for interaction,discussion and networking within and between the two communities.

the ancient times of Hippocrates and Galen (1108), extremely important archaeological medical findings, including medical equipment, surgical tools and herbal and mineral-related medicines have provided unequivocal proof.

On the first day of the Conference (Thursday 25th October) two programmes ran in parallel - the Scientific and the Patient & Parent - while on the second day (Friday 26th October), further to the continuation of the scientific programme, a number of other sessions ran in parallel covering a wide variety of topics that included sickle cell disease, rare anaemias, reference centres, laboratory aspects and

in overload disorders, the genetic and metabolic effects of iron metabolism in rare anaemias, and the monitoring of treatment of iron overload were the main topics covered in this session.

Cardiovascular disease, endocrine complications, growth, diabetes, fertility and bone disease constitute part of the multidisciplinary needs of these disorders and were comprehensively covered as overviews, while a focus on new advances was placed on blood safety and pathogen inactivation, liver disease, the diagnosis and monitoring and treatment of chronic hepatitis and on the cardiac and liver monitoring tools and technologies.

the delivery of most of the presentations. The programme focused on bone disease, pain, patient/ doctor relationship, disability, fertility in men and women, and liver disease. Focus was also placed on newly - emerging complications, including renal, and the session allowed for interactive, productive discussion.

Further to those directly related to medicine and science, other topics of importance and relevance to chronic rare diseases were discussed, and included patient-centred healthcare systems and effective services, and the development of better and more improved chronic-care models. Topics of constant

3rd PanEuropean Conference - Limassol, Cyprus

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Executive Summary of the 3rd Pan-European Conference on Haemoglobinopathies and Rare Anaemias

“The target… to address the needs for increasing education and updating of the medical and patient & parent communities…”

The Conference aimed primarily to cover prevention, management and curative approaches and research in the field of haemoglobinopathies and rare anaemias in Europe, but also to cover many other topics related to health systems, chronic disease models and European policies, directives and regulations related to rare diseases.

Ulrich Jäger and Demetris Michaelides - two academics, the former in the medical field and the latter in the field of archaeology - presented the two keynote speeches prior to the main programme. Professor Jäger guided us through the dramatic advances and breakthroughs that have occurred in recent years in the field of haematology, and in particular, in molecular diagnostics and targeted therapies, and hence the way to personalize medicine. Particular focus was given on the work of EHA towards addressing the need for further scientific programmes to be secured through funding on a national, European and international level and for the harmonization of standards and education of haematologists and the general medical profession throughout Europe in order to truly promote and achieve equal access of all Europeans to quality treatment in the field of haematology.

Professor Michaelides guided the audience through the historical documentation and evidence of medical practice in Cyprus, explaining that since

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the impact of migration policies on the control of haemoglobin disorders in Europe, to name a few of the most significant.

The scientific programme was ‘launched’ with Professor Dimitris Loukopoulos reviewing the history of the advances that have taken place through the years in the knowledge and the understanding of the pathophysiology and the natural history of β-thalassaemia, and that have led to the development of such treatment and monitoring interventions that truly converted a fatal paediatric disease into a chronic one. Erol Baysal and Marina Kleanthous covered the topics of prevention with an overview of the accumulated experience and know-how from successful programmes - with particular reference to new approaches and technical methods.

Prenatal diagnosis by CVS or AF, the case of iPS cells for transplantation in the perinatal period, PGD in diagnosis and as a therapeutic approach, and non-invasive prenatal diagnosis were amongst the key topics presented in this session. As in every educational event related to haemoglobin disorders - and more particularly, thalassaemia iron constituted a central theme, and this time extended to cover rare anaemias as well. The molecular and cellular basis of the toxicity of iron

Highlight Sessions Satellite symposium on mortality - The progress achieved and the demands behind it, as well as the remaining gaps and challenges

concern to patients with chronic diseases of multiorgan nature and multidisciplinary needs were also discussed, including adherence to treatment and patients as partners in medicine.

Non-transfusion dependent thalassaemias (NTDT) - The new focus of the medical community based on new information and evidence on how to improve treatment protocols of this group of haemoglobinopathies characterized by a wide genetic variability and long-term under-recognition, particularly in the area of management.

Conference Abstract Book The Abstracts from both the Poster Abstract Session of the Conference and the Scientific Programme can be found at http://www.thalassemiareports.org

Other important topics covered in the scientific sessions included the Sickle Cell Disease Session and the Rare Anaemias one, which covered diagnosis, epidemiology and treatment, and the many new advances achieved in these areas in recent years.

In the final part of the scientific session, other approaches to treatment, including induction of HbF, decrease of α-chains (improving the balance between α and β chains) and curative methods such as bone marrow transplantation and gene therapy were comprehensively discussed.

The Patients & Parents Session concentrated on topics of interest and concern, with their own participation both in the choice and also in

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