tif magazine - issue 61

2012 KEY EDucATIONAL AcTIvITY:

3rd Pan-European Conference onHaemoglobinopathies and Rare Anaemias24-26 OcTObEr 2012

“Patients rights-Revisited”NATIONAL THALASSAEMIA DAY

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 Highnes’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 field 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 organisations

will be announced during the biennial international conferences organised by

TIF, followed by an award ceremony in Abu Dhabi.

INTERNATIONAL AWARD CATERGORIES include:

- Grand International Award for life-long scientific contribution in the field of

Thalassaemia/Haemoglobinopathies.

- International Award for Innovative Medical Research

- International Award for Clinical Center of Excellence

- International Award for Thalassaemia Society of Excellence

- International Scholarship for Transnational Research in Thalassaemia

- International Leadership in Thalassaemia Award

Information available at:

Sultan Bin Khalifa International Thalassaemia Award – www.sita.ae

Thalassaemia International Federation – www.thalassaemia.org.cy;

[email protected]

IN PARTNERSHIP WITH

It gives me great pleasure tocommunicate with you again. The 61stissue of the magazine of our Federationpresents a great opportunity forsharing our news, our thoughts andsignificant information on those issuesthat matter most for the worldthalassaemia community.

It also gives us the opportunity tocelebrate the many advances andachievements that are happening, dayby day, through the relentless effort,unwavering commitment and sheerdetermination of all the members ofthis remarkable family of ours. So manypeople are working with dedication.Every single day they persevere andcarry on against all odds, always hopingand never giving up. I thank you allfrom the bottom of my heart.

I also thank the thalassaemiadoctors and the multidisciplinaryfaculty of specialists who do their bestfor each and every one of theirthalassaemia patients; the researchers,scientists and our stakeholders fromthe pharmaceutical industry who arededicated to bringing about a bettertomorrow for us; all the public healthprofessionals and influencers who, inconditions of scarce resources andausterity, are vehemently defendingthe territory that rightfully belongs tothe management of thalassaemia.I extend my profound gratitude to allour collaborators and colleaguesin the international and regionalorganisations and official fora for theircontinuous cooperation.

I particularly thank the people ofTIF, the Board of Directors, themanagement and the staff, who haveset high goals and are workingcollectively and painstakingly to attainthem.

Above all I want to thank everyperson with thalassaemia and theirfamilies for their fighting spirit andcourage, as well as our valuedmembers the thalassaemia patient

associations, for always defending therights of the patient with fervour andfor being the backbone of our commonstruggle.

More patient/parentorganisations than ever beforeare now active, in all continents.In Asia, the 1st Pan-Asian Conferencesuccessfully organised by TIF inBangkok, brought together patients,medical specialists and healthprofessionals from 20 South East Asiancountries, as well as SEARO, andculminated in a consensus declaration.As a result, Asia has embarked on a newpath and has started making collectiveand decisive strides which will securebetter recognition and management ofthe disease and better care forthousands of affected persons. Buildingon this momentum, the Indonesiangovernment announced new, inspiredpolicies. Meanwhile TIF organised asouth China conference in Nanningand is launching a strategic campaignin India and other Asian countries tostrengthen the local organisations andbuild the public and private sectorcapacities of specific regions tomanage thalassaemia and sickle celldisease.

In the Middle East, despite thedifficult conditions of recent months,TIF is stepping up its efforts toimplement ambitious plans, hoping togenerate a local response whichmirrors our ambition. We have been onseveral high level delegation visits toAlgiers, Morocco, Tunisia, the Kingdomof Saudi Arabia, the United ArabEmirates and for the first time, Oman.A programme of educational andcommunity awareness activities is inplace, as well as a series of importantmeetings with government andregional organisation officials.

In Europe and the Americas, ouractivities are focusing more on refiningand improving the quality of TIF’s

educational output and on theeffective implementation of disease-management policies, particularly innorthern countries and regions wherethe issue of thalassaemia is intertwinedwith issues of migrant populations andminorities. The 3rd Pan-EuropeanConference on Haemoglobinopathiesorganised by TIF in Limassol, Cyprus,under the auspices of the Cyprus EUPresidency, will prove a majormilestone. Another significantaccomplishment is the production ofseveral important new publicationsand other material which is at thedisposal of all our members,collaborators and friends.

You will find information on allthese developments in this issue of ourmagazine. We can be very proud of theimmense progress that is being madeon so many fronts; and even more sobecause this progress is the result of co-operation on every level, of close part-nerships with national thalassaemiaassociations, governments, medicalpractitioners and the pharmaceuticalindustry. But driving us all has been thestrength, determination and courage ofpatients. It is the patients who inspire,guide and motivate everyone in thefight against thalassaemia.

Panos EnglezosPresidentThalassaemia InternationalFederation

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SEPTEMBER 2012 www.thalassaemia.org.cy

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Foreword from TIF’s President

In the Middle East,despite the difficultconditions of recentmonths, TIF isstepping up itsefforts toimplementambitious plans.

http://www.thalassaemia.org.cy/

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EDITORIAL TEAM

Chief Editor:Dr Androulla Eleftheriou

Contributors:Mikaela Panayiotou

Michael AngastiniotisPhoebe Katsouris

Lily Cannon

Design Editing and Layout:Action Global Communications

Printing:Cassoulides Masterprinters

ISSN: 1450-3093

TIF BOARD MEMBERS

Panos EnglezosPresident

Shobha TuliVice President

Loisos PericleousSecretary

Riyad ElbardTreasurer

Her Highness Sheikha SheikhaBint Seif Al-Nahyan

George Constantinou

Saeed Jafaar Al-Awadhi

Loris Angelo Brunetta

Anton Skafi

Michael Michael

Robert (Bob) Ficarra

Fatemeh Hashemi

Mouna Haraoui

Ivan Dimitrov Ivanov

Duru Malyali

Ramli Mohd Yunus

Christina Stephanidou

Nailya Guliyeva

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 notimply 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.

Contents

n Foreword from TIF’s President . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .1

n Moving Forward: Introduction by Dr. Androulla Eleftheriou . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .3

n TIF Activities Around the World . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .4

n Medical/Health Professionals Network . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .6

n Publications . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .7

n Patients Rights Revisited / World Thalassaemia Day . . . . . . . . . . . . . . . . . . . . . . . . . . . .8

n Appeal to Free Palestinian Thalassaemia Patient . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .10

n Calendar of Events . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .12

n 3rd Pan-European Conference . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .13

n Pan-Asian Conference . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .14

n TIF & WHO . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .16

n Obituaries . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .18

n TIF Members’ Activities . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .19

n Member News and Country Advances . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .22

n Other News . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .23

n TIF speaks out for Iranian Patients . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .24

n Policy Update: Clinical Trials . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .26

n New Focus – Emerging Thalassaemia Syndromes . . . . . . . . . . . . . . . . . . . . . . . . . . . . .28

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.


Dear readers,

Welcome to the new upgradededition of TIF Magazine!I would like to take this opportunity togo back in time to two decades ago,when I first joined the Federation as avolunteer.

I was not at all prepared for whatwas ahead of me, or for the way myprofessional life would change sodrastically. For me, an incredible storyof passion, determination andcommitment began in 1993. Joiningthe global thalassaemia family andmeeting patients fighting on a dailybasis for their survival, the parents whostruggle to support their children, andso many others who place this struggleabove their personal lives and careers,has indeed changed my life.

As I look back on those days andcompare them with the situation as it istoday, it seems to me that I amwatching a silent black and whitemovie that is changing to a modernhigh-tech production. I am privilegedto have evidenced dramaticimprovements in almost every area ofour work: prevention; management;prognosis; diagnosis; cure; qualify oflife; patients’ and health professionals’education; recognition of patients’rights and many others.

I feel deeply honoured to havebeen given the opportunity to becomeinvolved in many of the activitiesthat led to those changes andimprovements through my work at TIF.Work, which I believe has added its ownsmall stone to the huge building blockof success and advances achieved inthe past two to three decades.

Certainly, through its years ofexistence, TIF has honoured its missionand paid deep respect to the rightsof patients for better health andquality of life. The two components ofTIF’s educational programme, theorganisation of events at the national,

regional and international level, thepreparation and distribution of the“Guidelines for the ClinicalManagement of Thalassaemia” andother educational material, as well asthe development of collaborations andpartnerships with the medicalcommunity and official health-bodies,have all been instrumental in theimprovement of consistency andappropriateness of care of this diseaseglobally. TIF has also played a leadingrole in the recognition of thalassaemiaand other haemoglobinopathies as apriority in the WHO and EU agendasthough the adoption of relevantresolutions and recommendations.

The impact of TIF’s work has beenrecognised by every relevantstakeholder in the field, including theWorld Health Organization (WHO),medical and patients’ communities,and other health organisations.

TIF continues to move forward toprotect and further promote the otherrights of patients for safe and effectivecare and active participation in thereform and/or development of issuesrelevant to their health and quality oflife - issues such as safe drugs, safeblood, research, clinical trials and manyothers.

But there is much more to be done. We need to move forward to

strengthen and support monitoring inareas that have been either under-recognised or not given sufficient focusdue to other priorities such as heartdisease. Liver disease, pain bonedisease and endocrine complicationsare just a few of the medicalcomplications that need our attention– considering that perhaps less than20% of the global patients’ populationhas access to appropriate iron loadmeasurements. Placing focus indeveloping, improving and adoptingtools for addressing these issues iscertainly another priority.

We need to work towards a better

understanding of other forms ofthalassaemias like Hbe/β- thalassaemia,β-thalassaemia intermedia HbH (theprevalence of which, may be evenhigher than that of β-thalassaemiamajor) in order to be able to offer moresupport to these patients.

We need to join hands withmedical specialists of differentdisciplines in order to develop andimprove a multidisciplinary approachto holistic care and, of course, add oursupport to all efforts towards themuch-awaited holistic cure.

Very importantly, we need to moveforward with ensuring thatknowledgeable patients all over theworld, fight more productively forimprovements in their care and qualityof life and that they become valuableand active partners in every reform ordecision taken that concerns theirhealth, at national, European andinternational levels.

Last but not least, we need to moveforward to solidify the many networksand collaborations we have managedto develop over the years, which arepatient-oriented or medical/scientificor industry-oriented. We must build onour strong infrastructure and our goodrelations with valuable collaboratorsand partners.

All of the above will be discussedand highlighted in the upcoming TIFmagazine issues.

We remain committed to keepingour patient community fully informedand involved in every step ofdevelopment and achievement in allareas relevant to their health andquality of life.

Enjoy reading this issue and pleasefeel welcome to communicate and/orsuggest whatever you feel we can do tomake it even better, more informativeand patient-oriented.

Dr Androulla EleftheriouExecutive Director

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TIF continues tomove forward toprotect and furtherpromote the otherrights of patientsfor safe andeffective care.

“Moving forward”


Thalassaemia centre in Nicosia Cyprus,Ministry of Health, visited Albaniawhere she had the opportunity to‘refresh’ TIF’s network with the medicalcommunity. More importantly, shehelped in further ‘bridging the gap’between the patients’ association andthe medical community. Dr Christoumet with Ministry of Health officialsand discussed issues relating tonational prevention, improvement ofmedical and multi-disciplinary care,and the issue of the MRI T2* technologyfor assessing the iron overload in theheart and liver - TIF’s main focus for thisyear for Albania. Dr Christou also metwith parents and patients, anddiscussed problems commonlyobserved during periods of ‘transitionof leadership’ in an organisation.

Bangladesh (March 2012)A delegation visited Dhaka andChittagong, with Dr Paul Telferparticipating on behalf of TIF.

Dr. Tefler reported a positivereception from the Minister of Health,but added that this needs to befollowed up promptly to ensure thatmomentum is not lost. The differentgroups are still not united and there aredisagreements particularly aboutwhether doctors should be involved inthe federation of thalassaemiaassociations and what the purposesand functions of this group should be.In Dr. Tefler’s view, this is probablydistracting from the main objective,which is to develop a national strategyfor prevention and treatment. For this,he adds, there needs to be an advisorygroup inclusive of all stakeholders andcommitment from the Ministry ofHealth in terms of recognising theproblem and appointing someonefrom within the Ministry to co-ordinatenational strategy and to work with theadvisory group.

Dr. Tefler plans to apply for fundingfor a large health partnership grant to

ALGERIA: (December 2011)As a follow-up to earlier activities inAlgeria, TIF Medical Advisor, Dr MichaelAngastiniotis, and Prof DemetrisLoukopoulos participated in adelegation visits / workshops. Duringthe visit, the Algerian medicalcommunity was updated on theappropriate management of patientswith thalassaemia and sickle cell disease,as well as on the latest advancesregarding prevention practices.

Algeria is a highly ‘affected’ countryin North Africa that is underdevelopedin many aspects of healthcare,including infrastructure for geneticdiseases such as haemoglobinopathies.TIF’s presence in this country isimportant and challenging but it hasnow established links with bothmedical and patients’ communities, aswell as with the national healthauthorities. Efforts are also being madeto establish a patients’ Federation –pending its registration as a legal entityin this country.

ISRAEL: (January 2012)Dr Michael Angastiniotis visited Israelas a follow-up to an earlier visit thattook place in October 2011 when TIF,together with Prof. Renzo Galanello,participated in the faculty of speakersfor a workshop. Stemming from theOctober visit, closer cooperationbetween the medical and patients’communities was established.

Although Israel faces neitherfinancial nor infrastructure issues, TIFbelieves that certain other aspects ofhealthcare provided to patients withthalassaemia, including the promotionof multi-disciplinary care andpsychosocial support, need to bestrengthened. Patients/parents and themedical community have been invitedto participate in the 3rd Pan-EuropeanConference.ALBANIA (January 2012)Dr Soteroulla Christou – Head of the

develop some of these thalassaemiathemes, initially based around theShishu Hospital, but outreaching toneighbouring towns and cities aroundDhaka, where there is nothing. He willtry to involve NGO's in the grantapplication, including TIF.

EGYPT (April 2012)Following TIF’s participation in theEMRO regional meeting in Jordan inJune 2011, where the EastMediterranean Regional ‘Task Force’,was formed, the Executive Director ofTIF, Dr Eleftheriou, was invited andaccepted to become a member of the‘Task Force’. Dr Eleftheriou activelycontributed in the reviewing of theEMRO Strategic Plan, the finalisation ofwhich took place in Sharm El-Sheik,Egypt (3-4 April 2012). This documentalso paves the way for the drafting ofthe ‘Guidelines for the clinicalmanagement of haemoglobinopathies’for the EMR countries.

TIF is also working towardsimproving the flow of informationbetween patients and all otherinvolved stakeholders, particularly theGovernment of Egypt. Dr Eleftheriouplans to meet with Ministry of Healthofficials to discuss ways by which thepercentage of the Government subsidy(towards covering drug and treatmentexpenses), should increase and/or tofind appropriate ways for patients to bereimbursed.

Bulgaria (April 2012)On behalf of TIF, ProfessorD. Loukopoulos attended theMultidisciplinary Workshop onThalassaemia for health professionals,in Varna, Bulgaria. Approximately 80-90people attended, including somephysicians dedicated to thalassaemiaand physicians taking care ofthalassaemia in minor centres. Therewere also specialists in other fields whoare involved in treating various

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TIF activities around the world


complications of this condition.The meeting included a number of

basic presentations includingoverviews of thalassaemia major(A. Kattamis), thalassaemia intermedia(D. Loukopoulos), endocrinologicalproblems (R. Chatterjee), heartproblems (A. Aessopos), liver problems(I. Kotsev) and presentations ofinteresting cases on which theparticipants had the opportunity tocomment. Long discussions took placeon the organization of “Centres ofThalassaemia”. Dr. Kaleva and othercolleagues supported the idea ofcreating at least three Centres acrossBulgaria (in Varna, Sofia and Plovdiv).TIF’s participation/contributionstrengthened these views. Commentsfrom TIF’s representative regarding theissue of prevention were also wellreceived. On the need for promotion ofmultidisciplinary care, the concernsregarding the absence of antiviraltreatment of HCV infected patientswith thalassaemia came to theforefront. TIF has already made plans tosupport patients in this area.

Indonesia (June 2012) TIF staff members, Dr MichaelAngastiniotis and Dr MatheosDemetriades, visited Jakarta as part of

the third TIF delegation visit toIndonesia. During their stay theyattended high level meetings with theGovernment and National HealthAuthorities to evaluate the statusfollowing the implementation of theinsurance scheme for thalassaemiapatients. They also visited the Vice-Director for Health and SocialDevelopment of ASEAN (Association ofSouth East Asian Nations) to discussfuture collaboration in the region. Ameeting with the Director of the WHOCountry Representative Office was alsoarranged to discuss the promotion offuture improvements in the quality ofhealth services to patients. During theirstay they also met with theThalassaemia Association of Indonesiaand with the medical communities andPresidents of the IndonesianHaematology and Paediatrics NationalAssociations.

The visit, gave TIF the opportunity

to mediate and bridge any difficultiesin communication between the variousstakeholders in the country, for thebenefit of the patients withthalassaemia; and more importantly tojointly decide with all stakeholderswhat the next steps forward for thenext 3 years will be.

Upcoming Delegation Visits

• Kingdom of Saudi Arabia andOman scheduled13-14 September 2012

• Hong Kong scheduled for22-23 September 2012

• China scheduled for28-29 September 2012

• Romania scheduled forOctober 2012 (tbc)

• Indonesia scheduled for 10-11 November 2012

• Morocco, Tunisia, Algeriascheduled in January 2013

• Russia – (tbc)

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cellence and the networking of treat-ment centres across Europe. The cul-mination of this 3rd phase is the pub-lication of White Book with all the rec-ommendations for centres of excellenceand networking between centres, in-cluding legal and ethical issues as wellas recommendations on laboratoryand clinical services. The project in-cluded educational activities and in-

The ENERCA (European Networkfor Rare and CongenitalAnaemias) projectsThis EU-DG Sanco funded project, cur-rently in its 3rd phase, is nearing com-pletion. Its main aim was to investigatethe services for rare anaemias in Europe,including thalassaemia and sickle celldisease, and to suggest improvementsthrough the creation of centres of ex-

formation for the public and for pro-fessionals involved with rare anaemiasthrough its website. Continuation ofthese activities through more advancedelectronic communications is plannedfor the 4th phase of the project.

The TIF Epidemiology projectTIF has created a global epidemiologydatabase for thalassaemia and sickle

update on ongoing projects in which TIF is involved

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first “School for Growth Disorders andEndocrine Complications in Thalas-saemia”. The goal of this educationalprogramme is to train paediatricians/physicians and haematologists to un-dertake the following:1. Acquire adequate knowledge about

growth and endocrine manifestations

International Network forEndocrine Complications inThalassaemiaAchieving optimum care for every pa-tient with haemoglobin disorders inorder for them to attain not only a longlife but also a good quality of life is awell-established goal for TIF. Becausewe are dealing with a multi-organpathology, which is encountered inthalassaemia, a coordinated multidisci-plinary team of specialists is required toachieve this goal.

Recognising this need TIF, has es-tablished different networks of collab-oration between health professionalsand medical specialists in the field ofthalassaemia, each having a specialfocus, i.e. on cardiology, liver, en-docrine, as well as a nurses’ network.

In this respect, TIF is pleased to in-form you that, on the 6-8 September2012 in Doha, Qatar, the InternationalNetwork for Endocrine Complicationsin Thalassaemia (I-CET) organised the

in patients with chronic haemolyticanaemias and improve their ability toselect and interpret data originatingfrom laboratory and radiological in-vestigations to solve these problems.

2. Help implement an internationallyacceptable set of standards on themanagement of the growth disor-ders and endocrine complications inthalassaemia.

3. Become competent and able to pro-vide assistance to children sufferingfrom endocrine disorders due to tha-lassaemia.

4. Encourage research in the field ofgrowth disorders and endocrinecomplications in thalassaemia.

Young endocrinologists who wishto pursue a career in the field ofhaemoglobinopathies or are in-terested in getting involved in themultidisciplinary treatment of pa-tients with thalassaemia, areurged to contact us for further in-formation.

The MRI projectMuch has been written in scientificjournals about the benefits of earlydetection and assessment of iron dep-osition in vital organs and the inter-vention through timely intensificationof iron chelation therapy, which canreverse even advanced heart and liverdisease. Survival studies have beenpublished that demonstrate a signifi-cant fall in cardiac-related deaths inthalassaemia through the non-invasiveuse of the MRI-T2*. Despite this, avail-ability of these services is extremelylimited. TIF investigation in 55 membercountries, revealed that only 19 (mostlyEurope) have the validated MRI T2*software and in some of these coun-tries access is limited to few patientssince the services are limited. The proj-ect aims not only to monitor the avail-

cell disease, which includes informa-tion on prevalence and frequency aswell as the services available in eachcountry. The main objective is to sug-gest and advocate improvements inclinical and other services throughknowledge of local conditions. Thedatabase draws its information frompublished data and from visits by TIFexperts. Suggestions and plans of ac-tion have already been developed forvarious locations using this informa-tion. Articles for medical journals arealso being prepared and one of these,concentrating on the situation in Eu-rope, is in the final stages of prepara-tion. The database is an ongoing proj-ect and is being continuously updatedas new information, such as changesin migration figures and health serv-ices, is gathered.

ability but also to promote availabilitythrough advocacy and through col-laboration with researchers who arepreparing cheaper and more afford-able technologies.

Electronic patient records -EPRThe importance of recording andstoring patient data electronicallywill benefit the patients directly bynot only including features such asdecision support but also by facili-tating research and networking.TIF has completed a pilot projectin Cyprus on the use of such anEPR and is expanding the projectwith new software to be made avail-able to any thalassaemia servicethat may wish to use it, free ofcharge. This will be available towardsthe end of 2013.

Medical/Health Professionals Networks


All our publications areavailable as PDF files on ourwebsite, completely free ofcharge.

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PublicationsA number of new and updated publi-cations, all of which are significant aseducational material in the context ofTIF’s educational and awareness effort,will be available from October 2012.

TIF’s publications cater to theneeds of readers of all ages,educational backgrounds andprofessional experience and provideconcise up-to-date information onevery aspect of thalassaemia, fromprevention to clinical management.TIF’s publications have beentranslated into numerous languagesin order to cover the needs of themedical/scientific, patients/ parentsand the general community.

NEW TIF PUBLICATIONSA short guide to addressing acute

complications in thalassaemia”– A valuable guide to health profes-sionals for addressing acute compli-cations in patients with thalassaemiain the emergency room. “Prevention of Thalassaemias andOther Haemoglobin DisordersVol. II” - The updated version of thesecond volume of this publication isnow available. The first volume iscurrently being updated and is sched-uled to be released next year. “White Book on the criteria for Refer-ence Centres and Networks for HbDisorders and Rare Anaemias – Avaluable tool for obtaining compre-hensive information on the existingcenters on Rare Anaemias in Europeand for presenting National Authori-ties the criteria to be included in their

respective national plans for estab-lishing reference centers andnetworks on rare anaemias. ThisWhite Book was finalised with thecontribution of TIF in the context ofthe ENERCA project in which TIF is awork-package leader.“Handbook for Haemoglobino pathyNurses” – An essential guide fornurses who are treating patients withthalassaemia and other haemoglobindisorders. This handbook is currentlybeing edited.“Guidelines for the Clinical Manage-ment of Thalassaemia Intermedia”– A necessary guide for healthprofessionals on the relativelyneglected field of clinical manage-ment of patients with thalassaemiaintermedia.

ORDER YOUR BOOKS!

Hard copies and CD-ROM or DVD versions can be ordered directly from TIF and aredistributed free of charge. Place your order at [email protected]

The translation of TIF’s educational publications into various languagescontinues in 2012.

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

free of charge

http://www.thalassaemia.org.cy/publications.htm


ones, in which patients have a role anda voice, and in which their opinion andcontribution would be respected andrecognised.

The establishment by WHO in 2004of the “World Alliance for PatientsSafety” was a global initiative and a keyapproach addressing issues such as themobilization and empowerment ofpatients – “Patients for Patients Safety”.It included the SPEAK UP campaign,most appropriately describing theresponsibilities of patients towardssecuring and promoting their rights:- Speak- Up if you have questions or

concerns: It’s your right to know.- Pay attention to the care you are

receiving.- Educate yourself about your

diagnosis and treatment.- Ask a trusted family member or a

friend to be your advocate.- Know what medication you take

and why.- Use a healthcare provider that

rigorously evaluates him/herselfagainst safety standards.

- Participate in all decisions aboutyour care and contribute to thedevelopment or reforming ofpolicies that affect your health andthe quality of your treatment.

It is obvious that knowledge is aprerequisite to exercising rights. TheWHO, the EU and other health bodiesacross the world have focusedconsiderable attention on safeguardingthe basic rights of patients but it wasnot until 2002 that these took (inEurope at least) the form of a Charter(EU Charter) on the basis of whichEuropean Countries were encouragedto build their legislation.

This Charter described 14 basicrights in total. The right to:• Preventative medicines• Access to health services needed

according to the disease withoutdiscrimination

“Knowledge is our power” was thetheme of one of TIF’s largest RegionalConferences in 2008 and is a concept onwhich the Federation has focusedconsiderable attention. As an interna-tional patients’ organisation, promotinginformation and ensuring that knowl-edge on the disease itself, (together withnew advances, results of clinical trials,concerns by experts and upcomingdevelopments in treatment) reachesevery patient, this undoubtedly formsan essential component of our mission.

It is equally important for NGOs tofocus on the education of patients onexisting, new and developingupcoming regulations, directives,recommendations, resolutions,declarations, on policies within andoutside the field of health that maydirectly or indirectly affect the patients’health and quality of life.

Patients’ rights stem from the UN’suniversal declaration in the 1940s onhuman rights, in the context of which aspecial article (Art.25) focusedspecifically on “The right of everyonefor a standard of living adequate for thehealth and wellbeing of himself and ofhis family, including food, clothing,housing and medical care…” Thisconstituted a critical first step inestablishing the concept of patients’rights and is reflected in the wordsmarking the founding of the WorldHealth Organisation (WHO) in 1948:“The enjoyment of the highestattainable standard of health is one ofthe fundamental rights, of everyhuman being, without distinction …”

Despite ongoing efforts and workin securing these rights, it was not untilthe 1960’s and 1970’s that Patients’Rights began to receive more seriousattention. In today’s world, officialhealth bodies, globally, are strivingmore than ever before to reduce healthinequalities. Today’s world is focusingon the reorientation of healthcaresystems towards more patient-centred

• Information• Consent• Free choice• Privacy and Confidentiality• Respect of patients’ time• Observance of quality standards• Safety• Innovation• Avoidance of unnecessary

suffering and pain• Personalised treatment• Complaints• Compensation

Many countries in Europe havealready transformed this Charter intonational laws, tailored to each one’sneeds and existing regulations onprevailing cultural and societal norms.Of course, many countries outsideEurope (mainly industrialised) havedeveloped their own Charters orregulations based on and encouragedby the work of WHO and its regionaloffices in the different parts of theworld. However, a commondevastating realization is the enormousgap existing in many of these countriesbetween the transposition into law andthe implementation and evaluation ofthe impact of these on patients’ qualityof life and active involvement indiscussions on health care services. Stillmore worrying is the realisation thatpatients in many more countries acrossthe world including manyindustrialised ones, have no definedrights, recognition and respect aspatients and certainly no room foractive involvement or participation inpolicy-making.

The great concern is that patientsthemselves are not aware, do not know,or are incompletely informed ormisinformed as to what exists or doesnot exist in their country.

In Europe for example, further tothe 14 basic rights described above, aplethora of other policies, directives,regulations, recommendations are also

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“Patients rights-revisited” World lassaemia Day


in place and many others are on theway or planned for the near or moredistant future. All aim at safeguardingthe rights of patients in many areas ofhealth, including: those in rare diseases,participation in clinical trials, protectionagainst counterfeit medicines,establishment of rights of patients forCross Border Health Care, for safe andadequate blood and blood products,protection of personal data and manyothers, which complement the rights ofpatients. Patients in Europe thereforeneed to become more knowledgeablein order to be in a position to utilisemore effectively what has been alreadygranted to them in order to becomeactively and truly involved in thereforming processes in their countriesthat affect their healthy ageing, quality

of life and free mobility across Europe.In other regions of the world, with

the exception of North America, whererights have also received considerableattention for some time now, patientsneed to fight to learn what exists intheir countries in term of patients’rights, charters or legislation and fightto put those to the forefront to supporttheir efforts for better health andquality of life.

Unfortunately, in many countries,patients have to fight from scratch fortheir rights, having the UN and WHOdeclarations as their solid basis and TIFas their closest ally.

TIF urges every patient to look andlearn about his/her rights, how theseare protected and respected in theircountry, to ensure that they are

continuously and accurately informedthrough their authorities, nationalpatient associations, TIF and accreditedsites on the internet.

Panos EnglezosPresidentThalassaemia International Federation

A special webpage will soon beuploaded on TIF’s website especiallyfor this theme.

You can also visit the WHO websitewhich includes a list of documentson human rights:http://www.who.int/genomics/public/patientrights/en/

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World alassemia DayCYPRUS

TIF PRESS CONFERENCEThe Thalassaemia InternationalFederation held a well attendedpress conference at the Hilton Hotelin Nicosia, Cyprus, on the 8th of Mayto mark international thalassaemiaday. This year’s theme “Patients’Rights revisited”, united the voices ofTIF, with those of the PancyprianAntianemia Association and theCyprus Alliance for Rare Disorders, inbringing to the forefront significantissues and the need to becontinuously vigilant.

The President of TIF, Panos,Englezos, called on government andparliament to move at a faster pace toensure the quality and safety of bloodand to implement the EU directives,particularly the European directive incross border health care which has tobe implemented by October 2013.

He pointed out that the laws andprocedures should be applied speedilyand always in an way that benefits thepatients. Moreover, Mr. Englezos,expressed the resolve of theThalassaemia International Federationto fight for the right of everythalassaemia patient for the care and

treatment that his or her physicianconsider as the best possible for theircondition.

A voluntary team was formed toassert the rights of all patients withthalassaemia, chronic and rare diseases.Support was pledged by the Presidentof the House of Representatives ofCyprus, Mr. Yiannakis Omerou, thechairman and members of theParliamentary Committee on health,the representatives of the Ministry ofHealth and Labour, the Ombudsmanand the First Lady of Cyprus Mrs. ElsiChristofia, who attended the pressconference.

This year’s theme“Patients’ Rightsrevisited”, unitedthe voices of TIF,with those of thePancyprianAntianemiaAssociation and theCyprus Alliance forRare Disorders, inbringing to theforefront significantissues and the needto be continuouslyvigilant.

Next year’s theme:“Beyond B-thalassaemiamajor”


mad began a strike, refusing to take hisrequired blood transfusions and medica-tions, and thereafter a hunger strike, tovoice the injustice for his arrest and on-going imprisonment. Mohammad’s fam-ily have not been allowed to see him.

The urgent case of MohammadSuleiman Arouri has been brought tothe attention of Thalassaemia Interna-tional Federation, and TIF has re-sponded with a call for action.

This patient’s survival depends onregular transfusions and on daily drugsto remove the excess iron from hisbody. Even if prison authorities are pro-viding blood transfusions, adequatehealth care for these patients, whichaims at survival and a good quality oflife requires a much more comprehen-sive approach. Mr Saleh is an adult pa-tient, married, with a child. At his age,he already has organ damage since fewpatients, however well treated, escapeit completely. His need for comprehen-sive, multidisciplinary care is an indis-putable right. Is the prison environmentconducive to such care? Failure to meethis needs will inevitably lead to poorquality of life and early death.

How much of a threat to nationalsecurity is a patient with a severecongenital anaemia?

However, any detainee has rightswhich must be respected. Detention forlong periods without trial is in itself aviolation of human rights. Sub-

Thalassaemia sufferer MohammadSuleiman Arouri is a Palestinian fromthe West Bank. A Social Sciencesgraduate of Bethlehem University,Mohammad is a married man andfather to a child he has never met.

On the 5th of April 2011, Moham-mad was arrested and sent to an Israeliprison under what is called an “admin-istrative detention order”. There are nocharges for keeping him imprisonedbeyond a claim of “security” issues, re-sulting from him having a relative whois a political detainee.

Mohammad was initially arrestedfor 6 months and was given a second 6months renewal without charge. Onthe 4th of April this year, his arrest wasrenewed for the third time for another6 months, still with no reason. Duringhis detention, his wife gave birth totheir first child – a child Mohammadhas yet to meet.

On the 5th of April 2012, Moham-

standard treatment of his seriousmedical condition is also a majorviolation of his rights.

The Board of Directors of theThalassaemia International Federation(TIF), which today represents andsafeguards the rights of hundreds ofthousands of patients with this diseasein more than 60 countries of the world:

DEMANDS FROMORGANISATIONS RELATED ANDPROMOTING SUCH RIGHTS TOTAKE IMMEDIATE ACTION ANDSUPPORT THIS PATIENT’S CASE.

We hope that organisations dealingwith human and patients’ rightsstrongly support this plea and protestto the government and militaryauthorities of the State of Israel on hisbehalf.

Sincerely yoursPanos EnglezosPRESIDENT THALASSAEMIA INTERNATIONALFEDERATION

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Appeal to free palestinian thalassaemiapatient held without charge

Mohammadwas initially

arrested for 6months and wasgiven a second 6months renewalwithout charge.

TIF Wants to Hear Your Story!

If you have a story that touches onsome of the issues faced bythalassaemia patients, send it to TIF at [email protected]

World Blood Donor Day - 14 June

2012 theme: “Every blood donor is a hero!”While recognizing the silent and unsung heroes who save lives every day through theirblood donations, the theme also strongly encourages more people all over the world todonate blood voluntarily and regularly.

To get updated on this years activities and future events, visit: http://www.who.int/worldblooddonorday

http://www.who.int/worldblooddonorday


calendar of EventsVENUE

Algiers, Algeriaand Rabat,MoroccoFrankfurt ,GermanyNicosia, Cyprus

Leiden, TheNetherlands

Doha, Qatar

Singapore

Rome, Italy

Malta

Jeddah , KSA

Muscat, Omman

Prague CzechRepublic

Brussels, Belgium

Hong Kong

Hanoi, Viet Nam

Harrogate,LondonBrussels,Belgium

Nanning,Guangxi ChinaGastein, Austria

Brussels,Belgium

Rome, Italy

Cairo, Egypt

Kiev, Ukraine

Limassol, Cyprus

Paris, France

Paris, France

Taipei, Taiwan

JakartaIndonesia

Brussels, Belgium

Brussels, Belgium

New Delhi, India

DATE

September 1-5

September 5

September 5 - 6

September 5-6

September 6-8

September 7 – 8

September 10 - 11

September 10-13

September 13

September 14

September 14-16

September 18

September 22 – 23

September 24- 28

September 26 - 28

September 27

September 28

October 3 – 6

October 9

October 8 – 9

October 17-18

October 19-21

October 24-26

October 29-30

October 30 - 31

November 3-4

November 8-10

November 12

November 20

November 24-25

EVENT

TIF Delegation Visits

1st European Patients’ Academy on Therapeutic Innovation (EUPATI) Workshop

Healthy Ageing Across the Lifecycle Conference

European Molecular Genetics Quality Network (EMQN) workshop on updating the2002 EMQN Best Practice Guidelines for Haemoglobinopathy Testing

Hamad Medical Corporation“School for Growth Disorders and Endocrine Complications in Thalassaemia” –Intensive course promoted by the International Endocrine Complications inThalassaemia Network (I-CET)

Pan-ASEAN Haematology Meeting

Inception Workshop on National Planning for Rare Diseases– EUROPLAN 2012-2015

62nd Session of the WHO Regional Committee for Europe

Sickle –Cell Disease Tour in Kingdom of Saudi Arabia

Sickle-Cell Disease Tour in Oman

EASL/AASLD Special Conference on Therapy of Hepatitis C: Clinical Applicationand Drug Development

EPPOSI Programming Day 2013

2012 EGM Iron Summit

63rd Session of WHO Regional Committee for the Western Pacific

British Blood Transfusion Society – Annual Conference

Unlocking the Potential of Migrants in Europe: From isolations to Multi-LevelIntegration – A Public Policy Exchange Symposium

1st Pan-South China Thalassaemia Workshop in Nanning, Guangxi

15th European Health Forum

EPHA Conference on Complementary and Alternative Medicine- Innovation andAdded Value for European Health Care

International Workshop ‘Rare Diseases & Orphan Drug Registries’ (EPIRARE)

9th International Conference of the Egyptian Society of Haematology and ResearchUpdate in Haematology

ISBT – II Ukrainian Transfusion Medicine Congress & Exhibition

3rd Pan European Conference on Haemoglobinopathies and Rare Anaemias

EURORDIS – Council of National Alliances (CNA) Workshop

EURRDIS- Council of European rare Disease Federations Workshop

3rd Asia Pacific Iron Academy (APIA) Conference

1st Pan- Indonesian Thalassaemia Workshop for Health professionals, Policy makersand patients/parents

Union of International Associations – Round Table 2012

EPPOSI Annual General Meeting

5th International Conference on Thalassaemia

•••••

•••••••••••••••••••••••••

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By organising local, regional and international conferences within the context of its highly successful andinternationally renowned Educational Programme, TIF aims to spread knowledge on the treatment and preventionof thalassaemia according to international standards, focusing on new research and clinical trials. Part of TIF’sstrategic plan for addressing health inequalities across the world, each Regional Conference takes into accountthe particular necessities of the region and is formulated in a way that addresses the needs of the region bybringing together patients/parents, medical professionals, policy-makers and other interested parties for thepurpose of exchanging methods of best practice in patient care, prevention and patient empowerment whilstproviding opportunities to network and share experiences.

WHY rEGIONAL


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Registration & Accommodation forms on our website


and country office representatives, keymedical specialists, patients’ leadersparticipated, from 19 countries.

In addition, in the course ofthe patients/ parents ‘get-together’meeting, patients had the opportunityto get to know each other, air theirgrievances, the difficulties they face,and the things they could do for eachother in order to move forward; moreimportantly many expectations forTIF’s support were raised.

The 1st Pan-Asian Conference on Haemo-globinopathies took place on 8-10 Feb-ruary 2012, at the Royal Orchid SheratonHotel and Towers in Bangkok, Thailand.

The participation exceeded our ex-pectations with numbers reachingmore than 350 participants. The pro-gramme of the meeting was formu-lated in a way that the overall event wasan ‘impact-making regional summit’ ofall relevant stakeholders. Ministries ofHealth Public Officials, WHO Regional

As an outcome of he meetingthe participants signed a declarationform with the “consensus on movingforward to address this majormedico-socio-economic problemand support in promoting services,in close collaboration with NationalHealth Authorities, to providespecific and timely diagnosis andmanagement to our patients withHb disorders, in order to improvetheir health and quality of life.”

The outcome of the meeting andthe Conference report, which includedan evaluation of the objectives of thisConference, has been presented to thenational health authorities of eachparticipating country.

A proceedings and an abstractbook has been compiled and are avail-able through TIF upon request.

up-to-date epidemiological data is alsostill needed in some countries of theregion in order to support betterplanning, as well as consensus on themethods and technologies to beimplemented to address social, culturaland religious factors in achievingeffective prevention.

Pre-marital screening legislationhas been effected in several countries,but there are remaining weaknesses inthe enforcement of this legislation.Countries in the region should ‘tailor’prevention programmes in accordanceto their specific needs, cultural factors,existing health infrastructures andavailable resources.

Pre-marital screening programmesshould be accompanied byappropriate genetic counselling toenable couples to identify and followthe most suitable reproductive option,as well as education of the publicregarding the health status of carrierindividuals, in order to avoidstigmatisation and discrimination.Prenatal diagnosis in some countriescarries the dilemma of termination ofpregnancy, which may only be allowed

Conclusions and recommendationsIn Asia and the Western Pacific regionsthere are certain needs andweaknesses common to almost everycountry, albeit to a variable level. Thosethat have been identified as being themost common, include the following:

National prevention programmes These need to be urgently developedand/or strengthened. Some countriesin these regions have been successfulin developing and implementingeffective prevention with outstandingresults, while others have identifiedsome weaknesses and are in theprocess of rectifying and refining theircontrol programmes. These countriescould constitute a model for others tofollow. Finally, there are also othercountries in these regions that wouldrequire support for establishing andimplementing their national controlprogrammes. The development ofservices for effective preventionnecessitates the active politicalcommitment and involvement by thegovernment for securing the qualityand sustainability of services. Reliable,

under limited circumstances.PGD technology can therefore

offer a viable solution to circumventsuch ethical problems. Expertise in thearea of PGD exists in countries of theregion, but co-ordination andassignment of areas of responsibilityare lacking, pending politicalcommitment.

Reference centres / centres ofexcellenceThere is a lack of designated referencecentres, or centres of excellence inmany countries of the region, whichdirectly affects the access of patients toquality clinical management. Referencelaboratories are also needed for theimplementation of effective preventionprogrammes. Only a number ofcountries have focused efforts andresources on this, like Singapore,Thailand, Taiwan-China, Hong Kong-China and Australia. In the majority ofthe rest, there are hospital or universitydepartments (India, Vietnam) orcentres (Sri Lanka, Bangladesh, etc) thattreat and provide basic treatment to asatisfactory level with a need to focus

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Pan Asian conference

Pre-maritalscreening

legislation hasbeen effected in

several countries,but there are

remainingenforcementweaknesses


on improving the quality and range ofavailable services. Networks of medicalspecialists and patients’ associations.There is a general absence of effectivenetworks among patients’ associationsand between patients’ associations anddoctors. To encourage such networkingand collaboration, TIF has embarked onan initiative to establish a Pan-AsianNetwork of patients and medicalspecialists in the field of haemoglobindisorders. Such regional networks weresuccessfully launched in Europe and theMiddle East, recently.

The aims of such a network wouldbe to improve awareness amongmedical professionals abouthaemoglobin disorders; to gain a moreaccurate picture of the availableservices and any existing problems; togather up-to-date epidemiologicalinformation, including numbers andlocations of patients in the region; toraise awareness among patients andparents of the existence of aninternational support network and peergroups in other countries; and to createnetworks of collaboration and closecontacts between patients and medicalprofessionals within and betweencountries.

Through the collection of data asolid evidence base will be built, andthis will help focus TIF’s activities on thespecial needs of the region, but more

importantly to support governmentsand Regional WHO’s efforts towards thecreation of recommendations andguidelines for Hb disorders.

Recommendations and next steps The goal and focus of activities shouldbe to establish optimal treatment forpatients, as well as effective preventionwith minimal affected annual births,across the region.1. This regional conference should be

repeated every 2-3 years.2. National workshops for both medical

professional and associationmeetings running in parallel areinvaluable. These workshops wouldimprove knowledge and awareness,but more importantly wouldstrengthen the collaborationbetween the various stakeholders.Another advantage is that servicedeficiencies in both casemanagement and prevention wouldbe brought to the surface and mightlead to the adoption of correctiveactions by policy adjustments andaction plans, from policymakers.

TIF, depending on availability of funds,will support :a) A specific number of

medical/scientific specialists fromeach country (according to theneeds) to participate in educationalworkshops organised by TIF.

b) The organisation of workshops/camps for patients associations toempower patients, to educate themof their rights and interests, to informthem of the ways to collaborateeffectively and productively withhealth authorities and how tostrengthen the psychosocial supportof the patients, would be the primarygoal of these activities.

3. A Regional Network as describedabove, to build close and effectivecommunication between theassociations and the TIF, as well asbetween associations and medicalspecialists. This will help to maintainawareness and action, and give TIFearly warning on important issuesthat require interventions.

4. Collaboration initiatives and mainte-nance of close communicationsbetween TIF, the Ministries of Health,national blood banks, national thalas-saemia associations, and WHO ineach country to:a) Encourage development of pre-

vention and treatment protocolsb) Support programmes for training

and education of scientists andhealth professionals

c) Establish whether blood needs aremet and adequate standardsapplied

d) Promote the establishment ofreference centres.

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of such strategies. It is our objective toobserve significant progress in five (5)years’ time, monitored in two years’time in the context of the 2nd Pan-Asian Conference on haemoglo-binopathies that will take place in Asia.We will be aiming to achieve:a) 25% increase in the development of

prevention programmes;b) 40% increase in patients receiving

appropriate treatment – blood trans-fusion and iron chelation accordingto TIF’s guidelines and upcomingguidelines on the management ofThalassaemia Intermedia, HbE/β-tha-lassaemia and SCD’

c) 60% increase in Patients’ Organisationsin the region;

CONFERENCE DECLARATION“We, the participants of the 1st Pan-Asian Conference on Haemoglo-binopathies, 8-10 February 2012,Bangkok, Thailand, have consensuson moving forward to address thismajor medico-socio-economic prob-lem and support in promoting services,in close collaboration with NationalHealth Authorities, to provide specificand timely diagnosis and managementto our patients with Hb disorders, inorder to improve their health andquality of life.

We are interested to form national,regional and international networks topool knowledge, expertise and resourcesto better support the implementation

d) 40% increase in Patients’ registries,and;

e) 25% increase of Reference centresacross the region and networksbetween the medical specialists ofand within countries.”

These activities should be linked toWHO’s 2008-2013 Action plan for theglobal strategy for the prevention andcontrol of non-communicable diseases(www.who.int/nmh/publications/9789241597418/en/index.html) – Non-com-municable diseases prevention andHealth Promotion V.2 (www.who.int/hpr/gs.strategy.country.shtml) and to thetwo WHO Resolutions on Thalassaemiaand Sickle Cell Anaemia (EB118_R1 andWHA59_R20, respectively).

National workshopsfor both medicalprofessional andassociationmeetings runningin parallel areinvaluable. These workshopswould improveknowledge andawareness, but moreimportantly wouldstrengthen thecollaborationbetween the variousstakeholders.


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TIF & WHO

The six days ofdiscussions

involved nearly3000 delegates,

including healthministers, public

health officialsfrom amongst the194 WHO Member

States

globinopathies, such as thalassaemiaand sickle cell disease.

Furthermore, TIF will be rep -resented by Dr Victor Bolujenkov, inthe 62nd session of the EuropeRegional Committee, to be held inMalta on September 10 – 13, 2012,where the WHO European ActionPlan for strengthening public healthcapacity and services will bediscussed. TIF will participate with athree-minute statement supportingthe initiatives taken by WHO/EURO inthis field. Please read the three-minute statement in the box.

The six days of discussionsinvolved nearly 3000 delegates,including health ministers, publichealth officials from amongst the 194WHO Member States, as well asrepresentatives from civil society,non-governmental organisations andother stakeholders.

TIF’s three-minute statement,which also can also be found on ourwebsite (www.thalassaemia.org.cy)fo cused on our commitment toimprove the quality of life and lifeexpectancy of patients with non-communicable and inherited haemo -

TIF participated in the 65th WHOWorld Health Assembly held inGeneva on 21 – 26 May 2012 with athree-minute statement on thePrevention and Control of Non-communicable Diseases.

Dr Victor Boulyjenkov, former Co -ordinator of the Human GeneticsDivision at the WHO Headquartersrepresented TIF at the this importantmeeting, which focused primarily onthe development of a global strategyand action plan regarding theprevention and control of non-communicable diseases.

Request to the Chairman of the WHA65 (21-26 May, 2012) Agenda item 13.1 “Prevention and control of NCDs”Statement of TIF (Thalassaemia International Federation)by Dr Victor Boulyjenkov

Mr Chairperson, Ladies and GentlemenThalassaemia International Federation (TIF) is a non-profit organization, comprised of one hundred and four memberassociations from 60 countries, in official relations with the WHO since 1996. TIF is committed to improving the quality oflife and life expectancy for patients with inherited haemoglobinopathies, such as thalassaemia and sickle cell disease,which fall within the scope of non-communicable diseases (NCDs). Our mission is to promote activities of existing nationalpatients/parents’ organizations as well as creating ones, and to support national health authorities in their efforts to developeffective national control programmes on the prevention and control of haemoglobin disorders.

The global public health burden of haemoglobin disorders continues to increase and available updated epidemio-logical data clearly demonstrate that the magnitude of the problem is grossly underestimated, worldwide. In this context,TIF greatly appreciated several WHO resolutions (WHA59.20, EB117.R3, EB118_R1), adopted by the WHA and EBs on sicklecell anaemia and on thalassaemia in 2006. These Resolutions facilitate the work of TIF and support our activities at countrylevel. However, we would be pleased to see a following WHO progress report, as a result of these resolutions, includingmember states activities on developing national programmes on the control of haemoglobin disorders.

You are aware that haemoglobin disorders belong to the genetic diseases family and as such require specific strategiesand interventions based on advanced research. To this end, TIF deeply appreciates the ongoing WHO initiatives outlinedat the document A65/8 and fully supports the proposed global strategy and the action plan for the prevention and controlof NCDs, especially in the research area.

TIF would be pleased to further provide WHO with relevant information on the progress achieved on the preventionand control of haemoglobin disorders worldwide, as a part of the NCD’s global health burden.

Thank you.

Mr Chairperson, Ladies and Gentlemen

TIF (Thalassaemia International Federation) is a non-profit organization dedicated to improving quality of life and lifeexpectancy for patients with inherited haemoglobin disorders which fall within the scope of non-communicablediseases. TIF is comprised now of 108 member associations from 55 countries cooperating, in official relations with WHOsince 1996.

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The global burden of haemoglobin disorders including thalassaemia and sickle cell disease continues to increase.Current epidemiological data demonstrate a gross underestimation of the magnitude of the problem taking intoaccount an increasing migration of populations worldwide.

In this context, TIF was delighted to accept several WHO resolutions on sickle cell anaemia (WHA59.R20) andthalassaemia (EB118.R1) requesting member states to develop and implement comprehensive national programmes forthe prevention and management of these diseases.

Following these resolutions, TIF has taken steps towards raising awareness across Europe for haemoglobin disordersthrough the organisation of Pan-European conferences on this topic since 2007, formulating European policies on ‘RareDisease and Anaemias’. The 3rd Pan-European conference with support of the WHO/EURO will be held in Cyprus thisOctober.

In conclusion, TIF, therefore, deeply appreciates WHO/EURO initiatives for strengthening public health capacities thatfall in line with TIF’s activities and fully supports the presented document, especially in the area of disease prevention.

Thank you.

ObITuArIESProfessor Antonio CaoProf Antonio Cao, paediatrician, geneticist and founder of the Ospedale Regionale Microcitemie in Calgiari, Sardinia,passed away on the 24th of June 2012 at the age of 83. One of the pioneers of modern understanding and manage-ment of thalassaemia, his contribution has been immense both on the academic and practical aspects of haemoglobindisorders. In the early 1970s he initiated the thalassaemia control programme in Sardinia, which included screeningand prenatal diagnosis becoming one of the first centres to advocate for the prevention of thalassaemia. A memberof the WHO, he worked on hereditary anaemias within the WHO Human Genetics Programme, through which manyguidelines on the control of haemoglobin disorders were developed. He became full professor of paediatrics andchaired the Paediatric Department of the University of Calgiari since 1974. His work has been recognised internationallyand he received several international and Italian awards. The thalassaemia community owes much to this tireless pioneer and researcher. In recognition of his lifetime contri-bution to the alteration of the natural history of thalassaemia from a fatal disease to one that can be both preventedand treated, Professor Cao was to be the keynote speaker at this year’s TIF Pan-European Conference in Cyprus. TIF, the Board of Directors and all who knew him as a friend over so many years will miss him and will never forget.He is part of our history.

Gargi PahujaOn the 21st of July, Gargi Pahuja passed away at the age of 37 and we lost an irreplaceable loving human being. Fora number of years Gargi served on the board of Cooley’s Anaemia (CAF) Foundation, and on the board of the Thalas-saemia International Federation (TIF). She was also a president of the Thalassaemia Action Group (TAG), and aPatients’ Services Director for CAF.In 1996 Gargi graduated from the University of Richmond as a Virginia Presidential Scholar with a degree in biologyand women’s studies. She earned a master’s degree in public health in infectious diseases from the University of Cal-ifornia at Berkley in 1998, and in 2004 she earned a degree in Health Law from Setom Hall University Law School inNew Jersey.Gargi’s battle for life was against all odds, but she spent it by waging her own personal crusade to give hope to otherthalassaemia patients. Her empowering and inspiring voice echoed not only in international patients’ conferencesbut also in governmental committees and official fora.The Board of Directors, members and staff of TIF, together with our global thalassaemia family mourn the lost of agifted and very special woman and express their sincerest condolences to her family.


WORLD THALASSAEMIA DAY

ARGENTINATo mark International Thalassaemia Dayon 8th May, ATA organised a talk on Pa-tients’ Rights in the lobby of the RamosMejia Hospital in Buenos Aires.

Speaking at the event was Dr Maria

Cristina Cortesi, a lawyer specializing inMedical Law and Legal Regulation ofMedicines, the Vice President of theHealth Law Committee of the Bar Asso-ciation of Buenos Aires, a member ofthe Iberoamerican Society of MedicalLaw (SIDEME), and Legal Adviser in theLegal Affairs division of the Superin-tendent of Health Services.

A wide audience actively partici-pated in the event, with patients’ rightsin Argentina explained by Dr. Cortesi,who also distributed copies of therelevant law to attendees.

AUSTRALIAAs part of International ThalassaemiaDay/Week, Thalassaemia Australia wasout and about during the month ofMay, starting with a children/familyfunction on the 6th of May at Kidspacein Cheltenham. This was a great oppor-tunity for families to get together andshare experiences, whilst enjoying a funday out.

The Thalassaemia Society of NSWhad an information stand at the Princeof Wales Hospital, Sydney on 6th of May,arranged an event to help raise aware-ness and increase understanding aboutthalassaemia amongst the local com-munity at Sydney’s Vivo Cafe, and alsoinvited the public to “FIX Thalassaemia”

on Saturday May 26 at the Fix LiveFunction Centre in Rockdale. Thalas-saemia Australia also organised aninformation table at Monash MedicalCentre on the 8 May.

BANGLADESHInternational Thalassaemia Day was ob-served in Bangladesh on 8th May by theBangladesh Thalassaemia Samity (Soci-ety). The Medical Students’ social welfareorganizations of all 12 Medical Collegesin Bangladesh were involved as partnersin observing the day, which was markedwith an event at the National Press Club,presided over by Omer Golam Rabbany,President of the Bangladesh Thalas-saemia Samity. Professor Dr M.R Khanalso graced the occasion as the ChiefGuest. Eminent Specialists and Paedia-tricians, Haematologists and othermedical practitioners participated indiscussion, while a multimedia presen-tation was made on the disease, itsprevention and treatment.

Speakers urged the Government tocreate facilities for treating thalas-saemia patients in every GovernmentHospital throughout the country, withspecial emphasis put on screening ofblood before transfusion.

INDIAOn the eve of ThalassaemiaInternational Day, strategies werediscussed that would make India athalassaemia free country, somethingthat will not be possible without activegovernment involvement. To markThalassaemia International Day a variedprogramme was arranged byFoundation Against Thalassaemia atCentral Park Sector 12 Faridabad.

Foundation Against Thalassaemiahas urged the private as well as thegovernment sector to speed up effortsto promote blood screening processesto eradicate Thalassaemia as more than3.5% of the country’s population arethalassaemia carriers and round 10,000

babies are born with Thalassaemiamajor every year.

Speaking on the occasion of In-ternational Thalassaemia Day, Gen-eral Secretary of F.A.T. RavinderDudeja said all out efforts should bemade to create awareness among thepublic regarding this deadly disease.He explained that taking the Thalas-saemia Carrier Test [HbA2] beforemarriage could play a vital role in pre-venting thalassaemia.

Thalassemics India celebratedInternational Thalassemia Day at SriSathya Sai International Centre, LodhiRoad, New Delhi with a culturalprogramme that attracted more than700 people. Shri. Anshu Prakash,Principal Secretary, Department ofHealth and Family Welfare,Government of NCT of Delhi attended,along with Guest of Honour, the formerIndia cricketer Mr. Kapil Dev.

The event consisted of song anddance performances, speeches, and in-troductions to various adult thalas-saemics who have excelled in differentfields.

IRANThe Iranian Thalassemia Societymarked International Thalassaemia Dayat the conference hall of the Iranian

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Next year’s theme:“Beyond B-thalassaemiamajor”


The national prevention pro-gramme of the Chronic Care Centerand the implementation of thepre-marital law in Lebanon contributedto a 70% reduction in the number ofnew cases in the last 18 years, whichdecreased to less than 6 cases per yearin a country where abortion is illegal.

It is hoped that a sustainable “zeronew case” status will be achieved, thusenabling the Center to eradicate thedisease and use all its resources to op-timize the treatment of thalassaemicsand contribute to ongoing improve-ments in their quality of life.

PAKISTANInternational Thalassaemia Day was ob-served by the Thalassaemia Society ofPakistan (TSP) with great enthusiasm.The day started with an awareness walkfrom Punjab Assembly to Fatima JinnahMedical College Lahore (FJMC). A largenumber of Thalassaemia patients andtheir families participated in this event.The walk was led by Ms. Zakia Shah-nawaz, advisor to Chief Minister Punjab,Professor Yasmin Raashid, SecretaryGeneral of the TSP, and Andleeb Abbas,the Vice President of the TSP. Partici-pants of the walk carried cards andbanners about Thalassaemia.

Subsequently, a colourful pro-gramme was organized at the audito-rium of Fatima Jinnah Medical College(FJMC). The function started with a wel-come address from Professor JovariaMannan, who warned that if the gov-ernment fails to implement a successfulprevention programme, an enormousburden on the country’s economy canbe expected in the coming yearsSpeeches were followed by a pro-gramme of songs, skits, dramas,dances, with the ceremony brought toa close by an address from the chiefguest, Mrs Zakia Shahnawaz.

Pakistan Thalassaemia Welfare So-ciety celebrations for International Tha-lassaemia Day on 8th May were even

Deputy of Labour and Social Welfare inTehran. Around 1000 patients and theirfamilies, specialists, health profession-als, nurses and pharmaceutical agentswere invited to the event, which wassponsored by various pharmaceuticaland medical equipment companies.

The President and Executive Man-ager of the Iranian Thalassemia Society,Dr. Arasteh and Mr. Mashhadi, wel-comed all the attendees, ahead of shortpresentations on the importance of theoccasion, healthcare issues and theconditions for thalassaemic patients inIran during the recent years.

With celebrations lasting morethan 5 hours, the event included livetraditional and pop music, theatricaland comedy performances, and more.

IRAQIraq’s Nineveh Thalassaemia Society(NTS) celebrated this year’s Interna-tional Thalassaemia Day with patientsand staff at the NTS centre, wherepatients and their families were also ad-vised on how to cope with the condi-tion as well as their rights to treatment,education and work. A gallery of paint-ings was also displayed, much to theenjoyment of all who attended.

LEBANONLebanon’s Thalassaemics came to-gether on the 8th May to not only markInternational Thalassaemia Day, butalso to celebrate that, for the first time,the Chronic Care Center did not registera single new case of Thalassemiaduring the first 5 months of the year.

more special this year as 2012 marks 20years since the Society’s founding. Tomark the occasion, the 8th NationalConference and Workshop (organizedby the Pakistan Thalassaemia Federa-tion every year) had its activities spreadover a full week to deliver increasedpublic awareness through the activeinvolvement of print and electronicmedia. All other societies working forThalassaemia in the Rawalpindi/ Islam-abad area joined in to make the occa-sion a great success.

To mark Thalassaemia InternationalDay 2012, a major function was organ-ized in the auditorium of the PakistanNational Council of Arts Islamabad. Theevent was well attended by thalas-saemics and their families, along withthe elite of the medical profession,social workers, officials and others.

PALESTINEEvery year, the Thalassaemia Patients’Friends Society of Palestine (TPFS)marks International Thalassaemia Daywith activities running through thewhole month. This year, the central cel-ebration took place on Saturday 5thMay in the district of Ramallah andal-Bireh, centre of West Bank.

20




The festivities began with thesigning of a Memorandum ofUnderstanding between PaltelCommunications Group and the Society,which entails launching a six monthcampaign on raising communityawareness through training and mediaactivities about thalassaemia.

The signing was followed by asolidarity march for MohammedSuleiman, a thalassaemia patientimprisoned by the Israeli army withoutcharge for more than a year. The marchcontinued to the hall where the officialceremony took place.

The opening ceremony conductedunder the auspice of the Governor, wasattended by 400 individuals fromaround the West Bank; patients andtheir families, volunteers, TPFS partnerhealth and youth organisations,international and local organisationsincluding UNRWA, along with theGovernor, the Paltel grouprepresentative, and the TPFS president.The programme included key notespeakers who emphasized thenecessity to collaborate more to reacha Palestine free of new thalassaemiabirths, and to continue building on thesuccess achieved till now. Calls tocontinue pre-marriage tests weremade. The ceremony included culturalactivities by students and patients,followed by recognition of specialvolunteers who supported TPFS effortsover the the past year. The ceremonywas followed by recreational activitiesfor patients and their families at thedistrict park.

SOUTH AFRICAIn celebration of International Thalas-saemia Day, a conference was held inUmhlanga organised by the SouthAfrican Thalassaemia Association(SATA). SATA Chairman Sunil Soni andDoctor Yasmin Goga urged the publicto donate blood at South Africa Na-tional Blood Service facilities and tolearn more about thalassaemia. SaidGoga: "As doctors, we can only do somuch. If there's no blood, there'snothing that we can do. Get onto so-cial networks and ask your friendsand family for support, to donatetheir blood."

SUDAN

A meeting to mark International Thalas-saemia Day in May was well attendedby patients, parents, doctors, the mediaand all those concerned with the treat-ment of thalassaemia patients. Thetheme of this meeting was the avail-ability of drugs, mainly iron chelatingagents, and ongoing efforts to ensurethat those who are suffering get accessto the treatment they need.

UAEOn the 6th & 7th May 2012, the two

days preceding InternationalThalassaemia Day, the EmiratesThalassemia Society organised the1st Emirates Thalassemia Forum forArab & Gulf Region at the prestigiousDubai World Trade Center.

Her Highness Sheikha SheikhaBint Seif Al Nahyan officiallyannounced the opening of the 2-dayforum and joined patients for thepresentations till the afternoon. This

was a social forum where patients,doctors, nurses & social activistswere invited from the Arab & GulfRegion to unite under one roof andshare issues and experiences relatedto the social aspects of Thalassemia.

On 8th of May, InternationalThalassemia Day was marked withhappiness and a fun time forThalassemia patients from UAE andelsewhere. Free games, musicalperformances, gifts and an openlunch buffet were the highlights ofthis day. Patients and their familiesenjoyed every second of the day,with an arts and crafts workshop,henna designing, face paintingand much more provided foryounger attendees, courtesy ofHamleys of London.

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loons, leaflets and a TV camera crew.The day was organised around the set-ting up of an information stall in the inthe main atrium of the Royal Manches-ter Children’s hospital, where care pro-fessionals and members of NEBATAjoined together to bring the festivities

UKNEBATA - the North of England BoneMarrow and Thalassaemia Association- is an organisation run by volunteersand funded by donations. It providessupport and information to people af-fected by thalassaemia through a net-work of medical experts. Operatingfrom Manchester, NEBATA providessupport to families across England andScotland, reaching as far as Bristol, Glas-gow and Leeds.

International Thalassaemia Daywas celebrated by NEBATA in a suc-cessful and colourful event, with bal-

to the people of Manchester. The verybusy atrium was an excellent venueand backdrop for a television inter-view given by NEBATA chairperson,Dr. Andrew Will, which aired on theBBC regional news show North WestTonight. The programme highlightedthe issues encountered by those withThalassaemia and raised awarenessamongst the greater public. NEBATA’smessage to “pay attention, discovermore and understand the condition”was spread far and wide, and the newsreport also appeared as a feature onthe BBC’s national Breakfast Show.

22



presidents of the event. Various topicswere discussed and interactive panelswere organised, while social eventswere also organised for representativesof all thalassaemia associations in Iran.

IRAQNinava Thalassemia Society HoldsSuccessful ConferenceNinava Thalassemia Society in Mosul, incollaboration with the provincial healthdepartment of Ninavah district, held its2nd Conference of Thalassaemia Preven-tion in Mosul city’s Oprawi Hotel. Theevent marked a successful programmeof thalassaemia prevention in 2011.

LAOSLaos Children's Hospital Opens Thalas-saemia ClinicOn 4th May, the Vientiane Lao PDR Chil-dren's hospital opened its new Thalas-saemia clinic. The clinic is supportingpatients with transfusion and ironchelation, through a budget currentlysponsored by KOICA which will be suc-ceeded by a NGO named HHA. It is thefirst dedicated clinic for the treatmentof thalassaemia in Laos. A patientsassociation is currently being formed.

LEBANONExtending Thalassaemia coverage forrefugee children at Lebanon’s Chronic

MEMBER NEWS AND COUNTRYADVANCES

INDONESIATIF Welcomes JAMKESMAS InitiativeThe Thalassaemia International Feder-ation (TIF) has welcomed the exem-plary initiative by the Government ofIndonesia with its JAMKESMAS nationalhealth insurance plan. TIF is grateful tothe Government of Indonesia for itsforesight and its genuine interest inameliorating the pain and suffering ofpatients with chronic diseases.

The implementation of theJAMKESMAS policy confirms that theGovernment of Indonesian has recog-nised thalassaemia, with its knownmedical-socio-economic repercussions,as a major public health problem, andhas had the vision to address it.

IRANIran Holds First Thalassaemia WinterSchoolIran’s first 3-day thalassaemia winterschool was held between 1-3 March2012 at Ramsar’s Grand Hotel. This sem-inar was held for about 200 adultthalassaemia patients from variousprovinces of Iran. Organised by theIranian Thalassaemia Society, Dr MajidArasteh and Mr. Mohammadreza Mash-hadi were the scientific and executive

Care CentreIn an agreement signed with Lebanon’sChronic Care Centre, the United Na-tions Relief and Works Agency(UNRWA) is to cover the health carecosts of Palestine refugee childrenreceiving treatment for Thalassaemia.Refugee children up to 11 years of agewill be fully covered at the Centrethrough UNRWA’s Catastrophic AilmentRelief (CARE) programme, in addition to70 per cent coverage of chronicmedications at discounted rates.

Mouna Haroui, president of theCentre, welcomed the announcement:“Through this co-operative effort,patients will be provided with properdiagnosis and the appropriate spe-cialised care. Along with the medica-tion supplied by UNRWA, improvedaccess to comprehensive treatment willallow Thalassaemics to cope betterwith their chronic condition and toavoid long-term complications. We alsoplan to work with UNRWA on preven-tion efforts, as we have demonstratedsuccess at decreasing new birthsassociated with the condition.”

Through a joint health awarenesscampaign, UNRWA and the ChronicCare Centre will conduct “train thetrainer” sessions for staff of the refugeeagency, helping to disseminate infor-mation about Thalassaemia among its


beneficiaries in Lebanon.“We are very pleased that this agree-

ment will help us to provide betterhealth care for Palestine refugee chil-dren”, said Salvatore Lombardo, speakingon behalf of UNRWA. “With the conditionshowing high prevalence among therefugee community in Lebanon, this col-laboration will be of great importance tothe families we serve.”

PAKISTANKarachi’s Kashif Iqbal Thalassamia CareCentre Shows Impressive Progress Karachi’s Kashif Iqbal Thalassaemia Care

Centre was established in 1996 by MrMuhammad Iqbal in memory of hisson, Kashif, a Thalassaemia Major suf-ferer who passed away at the age ofjust 16 years.

The achievements of this centrehave been many. It established its ownlaboratory, blood bank and transfu-sion centre in 2007 and now has 350patients registered for blood transfu-sion and more than 650 patients foriron chelation therapy. The KITCC is thefirst thalassaemia centre to haveinvented its own low-cost InfusionPump which is now in use across Pak-

istan for iron chelation. As well asstarting free family screenings forthalassaemia patients, KITCC has alsomade ultrasound available, andarranged for free screenings at cities,universities and factories across Pak-istan. Along with providing assistanceto flood-hit areas, KITCC has alsoarranged recreational activities for tha-lassaemia patients to mark occasionssuch as Independence Day, Eid, WorldThalassaemia Day, and more. KITCChas also initiated Thalassaemia Centresin Khanpur, Muzaffar Garh, Sargodha,Abbotabad and Lahore.

OTHER NEWS

Cure2Children Foundation OffersInternational AssistanceCure2Children (C2C) is a non-profit,non political and secular organizationproviding support to developingcountries' health professionals caringfor children with cancer and blooddisorders.

C2C provides free support to healthprofessionals, families, volunteers andinstitutions in developing countriescaring for children with leukaemia, can-cer and other severe diseases in com-pliance with shared principles andvision. The services offered are perti-nent to local realities and social values.The ultimate goal is to contribute to thedevelopment of worldwide, evidence-based diagnostic and managementstandards that may improve the cure ofall children with cancer.

C2C was created by paediatrichaematologist-oncologist, Dr. LawrenceFaulkner, whose experience drove himto promote a project aiming at bringinga cure to children with cancer,leukaemia and other common severeblood disorders such as thalassaemiaand sickle cell disease in underprivi-leged areas.

For more information, visitwww.cure2children.org

INDIAHunger Strike To Protest Denial OfTreatmentFollowing the denial of treatment tothalassaemia patients over the age of 18years by the Maternal and Child Health(IMCH) Medical College Hospital atKozhikkode, the Blood Patients Protec-tion Council (BPPC) observed a 4-daycontinuous hunger strike in front of theadmission desk of the IMCH hospital.

The hunger strike started on the2nd May and ended in 5th May.

Previously the IMCH had agreed tocontinue the treatment of bloodpatients irrespective of age, until theestablishment of a fully-fledged haema-tology centre at the Medical CollegeHospital for the treatment of various

blood disorders including thalassaemia,sickle cell anaemia, haemophilia andleukaemia.

Violating the agreement madebetween the IMCH Superintendentand the BPPC, the hospital authoritydenied treatment to three thalas-saemia patients - Manjusha (21),Vpinkumar (20) and Hasna (19). Thepatients were unable to attend theadult general medical departmentdue to a proven lack of sufficienthaematological care. The hungerstrike was led by Kareem Karassery,Gen. Convener of BPPC, and was sup-ported by the patients who did not re-ceive blood transfusions for four days.

M.K. Ragavan, a member of Indianparliament and member of the parlia-mentary standing committee of health,contacted Mr Kareem Karassery by tele-phone in support of the hunger strike.

On 5th May, Medical College po-lice arrested Mr Karassery along withM.P. Moideen and took them to theMedical College police station, admit-ting them for treatment for giddinessand fatigue and bringing the hungerstrike to an end.

For more than two decades,BPPC has been fighting for a well-equipped haematology centre inKerala state for the expert treatmentof blood disorders.

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Denial of treatmentto thalassaemiapatients over theage of 18 years bythe Maternal andChild Health (IMCH)Medical CollegeHospital atKozhikkode.

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22 August 2012

His Excellency, Mr. Ban Ki-MoonSecretary GeneralThe United Nations1 United Nations PlazaNew York, NY10017-3515Fax (212) 963 - 7055

Your Excellency,

Subject: Urgent Request for UN Support to Thalassaemia Patients in Iran

We understand that sanctions on the banking transactions of Iran affect immensely the country’s ability to buy andsell and in this way will limit the provision of essential elements of care of patients including life saving drugs. Eventhough there is no direct embargo on health products, the imports of drugs, equipment and consumables are expectedto be severely limited.

Experience of the embargo in Iraq in previous times, confirms the dramatic impact such limitations have on thecare of patients suffering from lifelong disorders such as thalassaemia. In Iraq common drugs like insulin and commonlyused consumables such as blood bags for transfusion and even disposable syringes underwent procedures such as“boiling” to secure sterilising and the possibility to be reused. The effect on both morbidity and mortality was devastatingfor quite a long time.

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TIF speaks out for Iranian patients

Success for Rhavindra ChhabraDr. Ravindra Chhabra, a thalas-saemia major patient who has re-quired blood transfusions every 25days since 1985, recently com-pleted his PhD degree from India'sreputed Sanjay Gandhi Post Gradu-ate Institute of Medical Sciences,Lucknow. The topic of his thesis was"Genetic Determinants of Thalas-

saemia". Dr Chhabra currentlyworks as a scientist at the Sri Au-rbindo Institute of Medical Sci-ences, Indore, and hopes that hissuccess will “… encourage all ourcolleagues who are thalassaemicsand their parents to come out fromall the agony in which we are livingand think that there is hope [forthe] future.”

In support of the efforts of the CharityFoundation for Special Diseases(CFFSD), TIF has expressed the concernsof the whole thalassaemia family to theUN Secretary Mr. Ban Ki-Moon,regarding the political situation in Iran,which is now facing intensifiedsanctions and how can these decisionscompromise the treatment andconsequently the lives of patients, in

particular those suffering from chronicdiseases such as thalassaemia andhaemoglobinopathies. TIF hasrequested from its memberthalassaemia associations to expresstheir own letter of concern in the hopethat with our combined efforts thesituation will soon be resolved,allowing for medical equipment anddrugs to be imported in the country.

Experience of theembargo in Iraq in

previous times,confirms the

dramatic impactsuch limitations

have on the care ofpatients suffering

from lifelongdisorders such as

thalassaemia.


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Send us your news!

If you have important news you want to share with the worldthalassaemia community, let us know!

[email protected]

Iran is a high prevalence area for thalassaemia. The carriers of this hereditary disease constitute 6% of the totalpopulation and there are at least 22000 affected patients registered. There is an effective prevention programme in Iranwhich was built up with considerable efforts, hard work and undivided commitment on behalf of all stakeholders:government, medical and patient communities and the community at large. If this collapses because of lack of resources,as many as 1000 new affected births can be expected every year.

Similarly and for some time now, the government of Iran provides all treatment modalities free and this has madethis country an example for other to follow. If patients are to pay out of pocket on the black market or simply not besupplied with essential elements of their treatment the effect will be as tragic as it was in Iraq some decades ago.

Thalassaemia care requires multidisciplinary approach and as such essential elements include:• Adequate and safe blood for transfusions – will this continue if essential tests for hepatitis C and HIV cannot be

provided?• Consumables such as blood filters, various types of infusion pumps, laboratory reagents etc.• Essential drugs which for thalassaemia are expensive:• Antibiotics since these patients are prone to infections• Vaccines – all thalassaemia patients should be protected from pneumococci, meningococci, haemophilus influenza

and hepatitis B• Iron removing drugs: these need to be taken daily and throughout life. They constitute the most expensive element

of treatment. Interruptions in treatment will lead to accumulation of iron and damage to vital organs, mainly the heartand the liver with subsequent increased rates of morbidity and hospitalization. Even in well-treated patientsinterruptions (for example due to non-compliance) can prove fatal and heart disease remains the major cause ofdeath. If these drugs become unavailable then the death rate will increase and affect the younger patients.

• Complications of iron overload can be detected early and prevented or their impact reduced by careful monitoringby specialists in cardiology, liver disease and endocrinology. This multidisciplinary approach is supported bytechnology such as MRI and echocardiography. At present thalassaemia care is at good level in Iran at least for the majority of patients, and there is active research by

medical professionals. Can this pro-active and holistic approach be sustained in a state of embargo? Will the death of patients serve to change the politics of a country or will it be a stain on those who chose to ignore

their health in order to triumph in a political arena? Is an economic embargo selective enough to protect the weaker andinnocent members of a community? Experience of the past does not confirm such an assumption.

As a Federation with exclusive patient orientation we request extremely urgent attention and action on behalf of theUnited Nations (UN) and the agency especially concerned with health, the World Health Organisation (WHO).

On behalf of the Board of Directors of the Thalassaemia International Federation (TIF), we remain deeply in expectationof your comments and directions and rely whole-heartedly on the capacities and authorization you are entitled throughyour position as the General Secretary of the United Nations.

Sincerely,Panos EnglezosPresident, Thalassaemia International Federation

As a Federationwith exclusivepatient orientationwe requestextremely urgentattention andaction on behalf ofthe United Nations(UN) and theagency especiallyconcerned withhealth, the WorldHealthOrganisation(WHO).


such as academic institutions and non-governmental organizations.

Heavy criticism and decline inclinical research: the need forrevisionDirective 2001/20/EC on the imple-mentation of good clinical practice inthe conduct of clinical trials on medici-nal products for human use, is based onArticle 114 of the Treaty on the Func-tioning of the European Union. Its aimis to harmonize the regulatory frame-work for clinical trials across the 27Member States. The Directive went along way to ensure a high level of pa-tient safety, but its complicated rulesand procedures led to an unfavourable

Clinical trials are an integral part ofclinical research. They are essentialfor the development of new medici-nal products and the improvement ofexisting treatments. Approximately4,400 applications for authorizationto conduct clinical trials in the Euro-pean Union are submitted every yearby researchers.

A clinical trial in the EuropeanUnion is defined by Directive2001/20/EC as a clinical study thataims to investigate the therapeu-tic effect(s) of medicinal productsthat have not yet been authorizedby regulatory bodies such as theEuropean Medicines Agency, orthe value of authorized drugs incases other than those for whichit has been authorized. In addi-tion, clinical trials are consideredto be studies that seek to assignspecific products to a therapeuticstrategy or propose to investigateeffective diagnostic or monitoringprocedures.

In contrast to popular belief, not allclinical trials are sponsored by pharma-ceutical companies – indeed nearly40% are funded by other stakeholders

regulatory framework for clinicalresearch. Indeed it has been the mostcriticized EU legislation where pharma-ceuticals are concerned, as indicated bystakeholders including professionalbodies, the patients’ community andindustry. In fact a decrease of 25%between 2007 and 2011 was noted inapplications for clinical research attrib-uted to the combined factors ofincreased costs and the launchingaugmented time delays of 90%!

Therefore a boost in clinicalresearch by combating these issueshas been necessary for some timenow, in order to promote the pur-suit of new therapeutic avenuesthrough simplified, modernized

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Policy update: clinical trials

Clinical Trials RegisterLast year’s launching of an online register by the European Medicines Agencyhas, for the first time, given public access to information on clinical trials formedicinal products authorised in the European Union. The database also allowsthe public to search for information on clinical trials authorised to be conductedoutside the EU if these trials are part of a paediatric investigation plan.

Lise Murphy, co-chair of the European Medicines Agency Working Party ofPatients’ and Consumers’ Organisations, of which TIF is a member, has said thatthe register ‘increases transparency of medical research and makes it mucheasier for patients to find information about clinical trials taking place in Europe’.

In contrast topopular belief, not

all clinical trials aresponsored by

pharmaceuticalcompanies – indeed

nearly 40% arefunded by other

stakeholders suchas academic

institutions andnon-governmental

organizations.


and expedited procedures.In this context, and after an exten-

sive consultation period, the EuropeanCommission adopted on 17 July a Pro-posal of the European Parliament andof the Council for the revision of theDirective 2001/20/EC on clinical trials.

John Dalli, European Commissionerfor Health and Consumer Policy hasstated in the press statement releasedby the Commission ‘today's proposalsignificantly facilitates the manage-ment of clinical trials, while maintainingthe highest standards of patient safetyand the robustness and reliability oftrial data’.

The proposal seeks to accelerateand simplify the authorization and re-porting procedures whilst maintainingpatient safety to the highest standard.In addition, the robustness and reliabil-ity of data collected during the clinicaltrial is safeguarded and transparency issecured, including information on trialsperformed in third countries.

A unified, harmonized legislationfor clinical trials was deemed necessarysince, under the old Directive, legisla-tion for authorization, application pro-cedures and marketing authorizationprocesses differed in Member States.The new legislation, which is now aRegulation rather than a Directive, andis mandatory to be adopted in allthe 27 Member States, will ensure oneregulatory framework is adhered to.

Directives present specific goalsthat must be achieved in every EUMember State and the national author-ities have to adapt their laws to meet

these goals, but are free to decide howto do so. Unlike Regulations, Directivesmay concern one or more MemberStates, or all of them. Each Directivespecifies the date by which the nationallaws must be adapted.

This will stimulate ‘academic’ andmulti-national clinical trials, thusstrengthening the Commissions’ dedi-cation in encouraging the develop-ment of cross-border consortia anddata collection. In addition it will hastenthe distribution of medicinal productsauthorized for human use as there willbe no heterogeneous legislation hin-dering the completion of clinical trials.

Stepping into the future

The new Regulation will ensure that therules for conducting clinical trials will beidentical throughout the EU. Thus, theauthorization procedure will progressmore rapidly. Moreover, simplified re-porting methods will avoid duplicationof submitting similar information tovarious bodies. These two proposalswill facilitate the Commission to verifythat all the rules are being adhered toand allow for greater transparency.

The new flexible and fast assess-ment procedures will not demand theestablishment of any more bureau-cratic bodies as the European Commis-sion, Member States and the EuropeanMedicines Agency will undertake theimplementation and supervision of var-ious aspects of the new Regulation. Forexample, the assessment procedurewill be controlled by Member States,whereas the portal for submitting anapplication will be managed by theCommission. This makes it easier formulti-national consortia to submit ap-plications or for existing clinical trials tobe extended to another Member State.

In view of the Commissions en-couragement of research by consortialocated in more than one MemberState, thus strengthening the robust-

ness of the data collected, most clinicaltrials are initiated by networks locatedacross several Member States. In somecases, for practical or legal reasons,these networks have difficulties in es-tablishing who amongst them wouldact as the single legal entity responsiblefor the trial (‘single sponsor’). To addressthis difficulty whilst ensuring the effec-tive supervision of a clinical trial, theproposed Regulation introducesthe concept of ‘co-sponsorship’. Allco-sponsors are responsible for theentire clinical trial, and also individualparts of the trial. In addition, allco-sponsors remain responsible forestablishing a sponsor who can takemeasures requested by a MemberState, and who can give information onthe clinical trial as a whole.

Maintaining high standards ofquality and safety are at the heart of thenew Clinical Trial Regulation,safeguarding the health and well-beingof clinical trial subjects whilstdeveloping innovative methods ofdiagnosis and treatment.

27FEATURE


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FAST FACT:

Regulations are the most directform of EU law and have bindinglegal force throughout everyMember State as soon as they arepassed.

What happens now?

The legislative proposal will bediscussed further in the EuropeanParliament and in the Council foradditional comments and / oramendments.It is expected to come into force in

2016.

The new Regulationwill ensure that therules for conductingclinical trials will beidentical through-out the EU.


By Dr. Androulla Eleftheriou

Strengthening our support topatients with thalassaemiabeyond β-thalassaemia majorIn the past three decades, extensiveresearch and focus on the preven-tion, diagnosis, management andcure of β-thalassaemia major haveresulted in better health, survival andquality of life for patients with thisdisease.

Curative methods (HSCT forsome time now, and gene therapy inmore recent years), have demon-strated impressive results andprospects, justifiably raising thehopes and expectations of the pa-tient/parent community worldwide.

Similarly, the essential elementscomprising the other component ofcontrol, ‘prevention’, have been wellidentified and implemented in anumber of countries albeit still toofew. But these countries have devel-oped successful models and inte-grated control strategies into theirnational health policies. They are pri-marily countries of Southern Europebut are currently being followed byothers both from within and outsideEurope. If there’s one ‘safe’ statementthat can be made today aboutβ-thalassaemia major it is that, wheremainly political commitment exists,this disease can be successfullyprevented and appropriately treated.

The struggle for better health andquality of life is an ongoing one andas the patient population is aging,new issues are constantly emergingthat call for more attention, researchand clinical studies/observations.These, are mainly heart complica-tions - different from those on whichfocus has been given in the pastdecades - the management of whichhas indeed brought about a revolu-tion in the survival and quality care ofpatients across the globe. But there

tional health agendas as a priority, isthe “serious lack of reliable and up-dated epidemiological data and in-formation about the clinical course,complications and mortality rates ofthe other thalassaemias, such as β-thalassaemia intermedia, HbE/β andHbH respectively.

Indeed the focus in more recentyears is on the elucidation and bet-ter understanding of the patho-physiology, natural history andmanagement of what are to dateconsidered as the β-thalassaemia in-termedia forms.

Relatively few population analy-ses of gene frequencies and studieson the natural history of differenthaemoglobin disorders beyondthose of β-thalassaemia major havebeen carried out. Rather, in the ma-jority of the countries of the world,considerable work has been focusedmainly on the underlying mutationsthat are involved. Today, we knowwith sufficient certainty that the seri-ous forms of β-thalassaemia are al-most equally divided betweenβ-thalassaemia major and intermediaand other forms until now referred toas “intermedia” thalassaemias includ-ing HbH and HbE/β thalassaemia.The latter, originally occurring at ahigh frequency in populous parts ofthe world including the Indian sub-continent, Bangladesh, Myanmar andthroughout South East Asia, whilethe α-thalassaemia (HbH) is moreprevalent in South East Asia.

Worldwide HbE/β-thalassaemiamay be the most important form ofhaemoglobinopathy because of thehigh gene frequencies for both HbEand β thalassaemia. As a result of largepopulation movements, HbE/β-thalas-saemia has replaced β-thalassaemiamajor and emerged as the most com-mon disorder in many regions beyondthe boundaries of the countries wherethey originally occurred.

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New Focus – Emergingalassaemia Syndromes

are many other long-term problemsand complications that need atten-tion, such as liver disease, infectionswith Hepatitis C and/or B, thrombo-sis, fertility, pain and osteoporosisjust to mention a few.

The sad side of this global story isthat successful strategies and impres-sive survival rates with high quality-of-life indices are only in place in veryfew countries, the majority being inEurope, North America and Australia.Unfortunately, the reality is that al-though the “know how” and techni-cal knowledge exists and is availablein the majority of the countries (in-cluding those of the developingworld where thalassaemia is mostprevalent, either as a result of highgenetic frequency or large popula-tions), national control programmesare very scarce and the status of de-velopment and/or implementationof the different components of theseprogrammes is extremely heteroge-neous within and between thesecountries.

The true picture today is that inthe majority of “affected” countries:• this disease is still not a priority on

the national health agendas;• significant number of affected in-

dividuals are still being born anddie at early ages;

• patients voice and active involve-ment is still at infancy level;

Despite: • considerable political movements

forward e.g. WHO Resolutions, EUrecommendations and

• existence of knowledge and ex-pertise on the part of themedical/scientific communities inmany parts of the world

One of the main problems in es-timating the global disease burdenfor thalassaemia, which is the prereq-uisite to bringing it to the interna-

The sad side of thisglobal story is that

successfulstrategies and

impressive survivalrates with high

quality-of-lifeindices are only in

place in very fewcountries, the

majority being inEurope, North

America andAustralia.


In the same way and contrary topast years, HbH disease is seen tobe moving and spreading acrossthe world in countries where it waspreviously rarely seen, such asNorth America and Europe (withthe exception of Southern Euro-pean countries where HbH, to-gether with β thalassaemia major,occurred at high prevalence in theindigenous populations).

As a result of changing demo-graphics, these disorders (HbE/ β andHbH) have now become more com-mon than β-thalassaemia major inmany regions and, together with ho-mozygous α-thalassaemia, affect atleast a million people worldwide. Al-though considerable knowledge hasbeen compiled on β-thalassaemia in-termedia, the other main forms ofthalassaemias (HbE/β and HbH), havebeen always been considered as the“intermedia”, “milder” or “clinically lesssignificant” thalassaemia syndromesin terms of management and care. Assuch and because of their geographicconfinement in the older times, theirdiagnosis and management has re-mained under-recognised and poorlyaddressed or entirely unaddressed.

In β-thalassaemia intermedia, thenatural history of which has beenstudied for sometime now and morecomprehensively in recent years,three main factors appear to beresponsible for the clinical sequel: (i)ineffective erythropoiesis, (ii) chronicanaemia and (iii) iron overload. Theseare linked to the variouscomplications that are more frequentin this form of thalassaemia ascompared to β-thalassaemia major,such as PHT, with secondary heartfailure (Hf ), thromboembolicphenomena, endocrinopathy, bonedisease, liver disease, fibrosis orcirrhosis and risk of HCC.

Current models for ironmetabolism in these patients suggest

that the combination of ineffectiveerythorpoesis, anaemia and hypoxialead to a compensatory increase inserum erythropoietin (EPO) and adecrease in hepcidin resulting inincreased intestinal absorption andincreased release of recycled iron fromthe reticuloendothelial (RE) system.This subsequently leads tomacrophage iron depletion, relativelylow serum feritin and preferentialportal and hepatocyte iron loading.

This proposed model is similar tothat described for the hereditaryforms of haemochromatosis and is incontrast to the regularly transfusedthalassaemia major patients, who donot have low hepcidin levels and ironis preferentially distributed to the REsystem, stimulating ferritin synthesisand its release into the circulation.

Thus, the thalassaemia interme-dia patients’ serum ferritin levels, al-though correlating with LIC,underestimate iron load as comparedwith thalassaemia major patientswith equivalent LIC. Direct LIC meas-urement or use of a better indicatorthan serum ferritin, are currently rec-ommended by experts. It is evidentthat these patients require appropri-ate monitoring, assessment andmanagement as more and more in-formation is accumulated, hence the

need for focusing efforts and work ondeveloping specific guidelines.

Today, novel findings from largecohorts of patients are providinginformation that will hopefullybridge the gap between evidenceand practice by calling forprospective clinical trials that willevaluate the efficacy, safety and costeffectiveness of proposed/recommended therapies. Suchstudies are expected to evaluate theoptimal timing, dose and duration oftransfusion, iron chelation, orhydroxyurea therapy. Although mostof the studies leading to the conceptof the iron metabolism model asdescribed above and the naturalhistory of the disease and itscomplications have concentrated onand have been extracted frompatients with β-thalassaemiaintermedia, it is expected that a greatdeal of this knowledge will also applyto the other “intermedia forms” suchas HbE/β-thalassaemia and HbH(α-thalassaemia). In spite of this,considerably more studies areneeded in these patient populationsin order to better define the naturalhistory of these forms, which arehighly variable and poorly studied.Their phenotypes can also rangefrom asymptomatic to transfusion

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In the same wayand contrary topast years, HbHdisease is seen to bemoving andspreading acrossthe world incountries where itwas previouslyrarely seen, such asNorth America andEurope.

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dependent as with β-thalassaemiaintermedia.

Approximately 50% of the pa-tients with HBE/ β-thalassaemia arephenotypically similar to β-thalas-saemia major patients and as suchthey require regular transfusions. Theclinical course for the other 50% re-sembles that of β-thalassaemia inter-media. The marked variability andclinical course in HbE/ β-thalassaemiais still largely unexplained. The samefactors that lead to β-thalassaemia in-termedia, including: heterozygositywith β + thalassaemias (mild muta-tions), co-inheritance of α-thalas-saemia mutation (which occurs in15% of patients with this form) and in-heritance of the XmnI+/f genotype ofthe g-gamma globin gene, may alsocontribute to milder clinical course inthese forms of thalassaemia.

Ineffective erythropoesis, apop-tosis and oxidative-damage are cen-tral components of the disease asdescribed in β-thalassaemia interme-dia and the instability of HbE and theexpression of α-haemoglobin stabi-lizing protein do not appear to bemajor factors in the pathophysiologyof HBE/ β-thalassaemia intermedia.

In the absence of markedly ele-vated ferritin levels, non-transfusioniron load in HbE/β-thalassaemia in-termedia can result in widespreadorgan injury. For example, currentevidence suggests that in the ab-sence or presence of transfusion ther-apy, LIC levels between 6-7mgFe/galone are associated with an in-creased occurrence of morbidity,suggesting maintaining LIC levelsbelow 5mg/Fe. Thus, it is critical tounderstand when to initiate, knowhow to monitor and how to managechelation therapy in these patients.

It is of immense importance,given the wide variety of complica-tions associated with the pathophysi-ology of these previously thought of

intermedia (β or HbE/ β) withmoderate anaemia. It is commonlyfound in South East Asian, MiddleEastern and Mediterraneanpopulations. There is a widespectrum of genotypes andphenotypic presentations. These, asin the case of β-thalassaemiaintermedia and HbE/β, range fromthose who appear clinically to beasymptomatic, to others who aremore anaemic, having significanthepatosplenomegaly and requiringoccasional or even regulartransfusions, to the severe HbHhydrops faetalis syndrome that cancause death in affected foetuses latein gestation.

This hereditary disorder is usuallycaused by deletions removing all butone single a-globin gene (detectionalHbH disease). A small proportion ofpatients have deletions removing 2a-globin genes plus a nondeletionalmutation affecting a third a-globingene (nondeletional HbH disease). Ingeneral, non-deletional HbH diseasehas a more severe clinical course thanthe deletional form.

Review of recent literature sug-gests that HbH disease is not as be-nign a disorder as previouslythought. It can bring about growthretardation during childhood andiron overload in adults regardless ofprevious transfusion history, leadingto hepatic, cardiac and endocrinedysfunction. Significant anaemiamight occur during infections, fever,hypersplenism, or pregnancy thatmay necessitate the need for bloodtransfusions. An essential part of thematernal/child healthcare should in-clude screening the partners of allpregnant women with HbH diseasefor their thalassaemia carrier status,and providing these and other cou-ples who are at risk of conceiving off-spring with HbH disease withappropriate genetic counselling.

30


as “Non-transfusion dependant tha-lassaemias” and the model of theiriron metabolism, to consider accurateand proactive monitoring of LIC anddevelopment of complications inorder to allow for prompt initiation ofblood transfusion and iron chelationtherapies. It has been demonstratedin one of the biggest studies, albeitonly on patients with β-thalassaemiaintermedia, that transfusion therapywas protective for thrombosis EMH,PHT, heart failure, cholelithiasis andleg ulcers. While at the same time itwas associated with an increased riskof endocrinopathy. Iron chelation wasin turn protective for endocrinopathy,PHT and iron-related liver disease.

Hydorxyurea treatment on theother hand was associated with an in-creased risk of hypogonadism andwas protective of EMH, PHT, leg ul-cers, hypothyroidism and osteoporo-sis. Splenectomy in the same studywas, shown to be independently as-sociated with an increased risk ofmost disease-related complications,the role of splenectomy, however de-serving revisiting.

It is essential for the medical/ re-search community in the field tomove forward and identify reliable,accessible and considerable lesscostly tools in order to monitor regu-larly and accurately the LIC in thesepatients.

Finally, it is essential to put to-gether, as soon as possible, all knowl-edge and information available in thecontext of Guidelines specifically ad-dressing the needs of patients withthese forms of thalassaemias, whilecontinuing to collate data, includingepidemiological ones, that will fill in themany gaps still present today with re-gards to these forms of thalassaemias.

HbH disease deserves a slightlyseparated reference, as this is a formof a-thalassaemia that is oftenmanifested clinically as thalassaemia

It is essential for themedical/ researchcommunity in the

field to moveforward and

identify reliable,accessible and

considerable lesscostly tools in order

to monitorregularly and

accurately the LIC inthese patients.


Prospective and systematic stud-ies of the natural history of HbH dis-ease, particularly during infancy andchildhood, as well as during preg-nancy, have not been carried out todate and are much needed. Informa-tion derived from these investiga-tions can lead to better insight topotential risk factors associated withsevere disease and can help to for-mulate future medical interventionsand treatment strategies.

Conclusions:The prognosis for patients with thehaemoglobinopathies that are newlyemerging in Western and NorthernEurope, in North America and else-where where there are robust healthinfrastructures and capacities to pro-vide treatment, is strongly depend-ent on the healthcare system’sapproach to these disorders. Prenataldiagnosis, neonatal screening, com-prehensive care and access to newtherapies including transfusion andchelation therapy, stem cell trans-

References Correspondence

“Age-related complication in treatment-naïve patients with thalassaemia intermedia” 2010 Blackwell Publishing Ltd, British Journal of Haematology, 150,

pg480-497

David H.K. Chui, Suthat Fucharoen and Vivian Chan “Hemoglobin H disease: not necessarily a benign disorder”, Blood, 1 February 2003, Volume 101,

Number 3, pg.791-800

Taher A., Mussallam K.m. et al “Overview on practices in thalassaemia intermedia management aiming for lowering complication rates across a region of

endemicity: the OPTIMAL CARE study”. Blood, 11 March 2010, Volume 115, Number 10, pg.1886-1892

Mussallam M.K, Cappellini, M.D, et al “Iron overload in non-transfusion-dependent thalassaemia: a clinical perspective”, Blood Reviews, 26S (2012), 16-19

Taher A, Hershko C and Cappellini M.D, Review: “Iron overload in thalassaemia intermedia: reassessment of iron chelation strategies”, British Journal of

Haematology. 2009. Blackwell Publishing Ltd, 1-7

Faulkner L, Yqub N, et al “Transplantation in Low Resource Countries” abstract from the 1st Pan-Asian Conference on Haemoglobinopathies, February 2012,

Bangkok, Thailand

Bread L, Casu C et al “Therapeutic Hemoglobin levels after gene Transfer in β-Thalassaemia Mice and in Hemotopoietic cells of β-Thalassaemia and Sickle

Cell Disease Patients”, PloS ONE, March 2012, Volume 7, issue 3, e32345

Mussallam K M, Taher A and Rachmilewitz A, “β-Thalassaemia Intermedia: A Clinical Perspective”, Cold Spring Harb Perspect Med 2012; 2a013482

Treatment of β-Thalassaemia Major with Autologous CD34+ Hematopoietic Progenitor Cells Thalagen TM , a Lentiviral Vector Encoding the Normal Human

β-Globin Gene”, ClinicalTrials.gov, identifier: NCT01639690

plantation and HbF–enhancingagents are important components ofcare. Addressing the economic andcultural barrier and increasing theknowledge of physicians about thesedisorders should improve the overallcare of affected patients.

However, the real problem is stillin the countries where the disordersare most prevalent and occurringamongst the indigenous populationsand which unfortunately include themajority of the countries of thedeveloping world. It is therefore nowimportant to move beyondβ-thalassaemia major and addressthese forms of thalassaemia moreeffectively at the national andinternational level.

TIF is committed to embark onimportant services for these patients,such as the preparation of guidelines,awareness and training campaigns,both for the patient and medicalcommunities and importantly for thenational, regional and internationalhealth bodies.

It is the responsibility of us all toimmediately address the needs of pa-tients with HbE/β, HbH and β-thalas-saemia intermedia, in terms ofappropriate education, awareness,medical and other care, in order forthem to enjoy to the maximum ex-tent possible, good quality, fulfillinglives. Through this brief article, whichis a combination of information pub-lished by experts in the field, TIF takesthe first steps forward to offer sup-port to these patients globally.

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For more details, please readTIF’s position paper on Non-Trans-fusion Dependent Thalassaemiasavailable on our website. Other TIFposition papers include:

• Counterfeit medicine• Patients rights• Information to Patients• Chronic Hepatitis C

The real problem isstill in the countrieswhere the disordersare most prevalentand occurringamongst theindigenouspopulations andwhichunfortunatelyinclude themajority of thecountries of thedeveloping world.


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