ukts honours prof bernadette modell · • dr josh wright, consultant haematologist, ... prof...

A word from our President . . . . . . . . . . 2

Latest News . . . . . . . . . . . . . . . . . . . . . . . . 3

Medical News . . . . . . . . . . . . . . . . . . . . . 7

News from around the World . . . . . . . 17

Patient News . . . . . . . . . . . . . . . . . . . . . . 19

Office News . . . . . . . . . . . . . . . . . . . . . . . 21

Standing Order Form . . . . . . . . . . . . . . . 23

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UNITED KINGDOM THALASSAEMIA SOCIETY

A Charity OrganisationRegistration Number: 275107

19 The BroadwaySouthgate Circus, London N14 6PH

Telephone: 0208 882 0011 • Fax: 0208 882 8618Email: [email protected] • www.ukts.org

The UK Thalassaemia Society’s third national medical conference “Thalassaemia – a childhood condition comes of age” was held on 10th June 2008 at the Royal Society of Medicine in London W1. Like the family conference held in Leeds in March 2007, this conference was held in association with the Royal Society of Medicine. We are honoured to have been associated with the RSM in these two major conferences.

In accordance with our usual practice, our conference organising committee was part medical specialists, part UKTS – we were particularly lucky that the following

doctors, all leading figures in their own specialities, agreed to assist us –

Prof Geoffrey Dusheiko, Professor of •Medicine & Honorary Consultant Centre for Hepatology, Royal Free & University College School of Medicine, Royal Free HospitalDr Malcolm Walker, Consultant •Cardiologist & Clinical Director of the Hatter Cardiovascular Institute, University College Hospital LondonDr Josh Wright, Consultant •Haematologist, Royal Hallamshire Hospital Sheffield & Vice-Chair of the UK Forum on Haemoglobin Disorders

The team from UKTS were: Treasurer George Constantinou, President Mike Michael and Coordinator Elaine Miller; all of whom are extremely grateful to Prof Dusheiko, Dr Walker and Dr Wright for giving up their time for planning meetings, chairing at the conference and innumerable emails; especially the invaluable advice they gave us in planning the programme.

When we started planning this conference in summer 2007 we had two main aims in mind. The first was to emphasise the fact that, as indicated by the title of the conference, in the UK thalassaemia can no longer be considered a childhood condition. Of course this is

well known by our members and those who are treated in or work in specialist centres; but far away from these centres, even health professionals have been known to express great surprise that thalassaemics gain university degrees, play sports and have careers and families! Which is a source of wry amusement to our thal Committee members, most of whom are in their forties and even (dare I say it) fifties! The second main point we wished to make was the importance of involving doctors from fields other than haematology in thalassaemia

Thalassaemia – a childhood condition comes of age

UKTS Honours Prof Bernadette Modell

September 2008 ISSUE NUMBER 112

L to R – Dr Malcolm Walker, George Constantinou, Andy Charalambous, Neelam Thapar (front), Prof Bernadette Modell, Dr Chris Sotirelis, Mike Michael, Prof Geoffrey Dusheiko

Continues on page 3 ➡

www.ukts.org2 Thalassaemia Matters ...continuing the fight against Thalassaemia

Dear Members,

Welcome to the latest issue of the UKTS newsletter. As always we try to bring you interesting and informative items regarding life with thalassaemia, from home and abroad. In this way we hope to remind ourselves that we are part of a global community and that our hopes, our fears and our needs are the same whether we live in the developed or the developing world. So let’s use the Singapore conference to re-acquaint with old friends, make new ones, and remind each other that no matter what race, culture or religion we belong to we are all part of the same global community and that we should all work together to build common knowledge and common goals.

To highlight this lack of knowledge let me ask a simple question; “How many thalassaemia patients do you think there are globally?” I would be surprised if we could even come close to an answer. And maybe that’s the first step.

This quarter’s edition contains articles from some of the speakers at our academic conference last June on infertility and hepatitis C and also a comprehensive article on the optimum diet for those living with thalassaemia.

We also have updates regarding

thalassaemia in Nepal, an item from

Morocco and a story from a Malaysian

mother. On the home front we cover the

Janmasthami festival, the new electronic

MSc in Haemoglobinopathies and the

wedding of our own Marathon Man

Gabriel Theophanous and his new wife

Nishel. Last but certainly not least, the

“latest news” section brings you notice of

the new, revised edition of our publication

Standards for the Clinical Care of Children

and Adults with Thalassaemia in the

UK. Contact the UKTS office if you are

interested in receiving a copy.

As usual we would like your comment on

any of the stories in this issue or if you have

any comment to do with thalassaemia in

general then email us [email protected]

Finally remember that your membership

is used to part fund the office and the

newsletter, help us continue our work by

sending in your membership payment or

donations.

Until the next issue,

Mike Michael

President

UK Thalassaemia Society

A word from our President

Mike Michael President

Dr Christos Sotirelis Vice-President

Menuccia Tassone Secretary

George Constantinou Treasurer

Philip Agathangelou Assistant Treasurer

Costas Kountourou Committee

Bharat Nathwani Committee

the UKtsManagementcommittee

Our Mission

Statement

■ To be the definitive source of information, education and research for those affected by, or working with thalassaemia.

www.ukts.org Thalassaemia Matters ...continuing the fight against Thalassaemia 3

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treatment. The modern, holistic approach to treatment demands the skills of doctors who specialise in, for example, the heart, liver, bones and fertility issues. We therefore included doctors working in these specialities in the conference invitations. Although the programme was aimed primarily at doctors, we also made a point of including nurse specialists.

Registrations totalled 200 with participants travelling from all parts of the UK, a fairly impressive total for a condition still relatively rare in this country. The venue, the New Lecture Theatre at the Royal Society of Medicine, comprises the latest and best in technology and comfort; and the participants settled comfortably into their red leather seats to enjoy a programme from clinical specialists of international repute. Dr David Misselbrook, the Associate Dean (and Dean-elect) of the Royal Society of Medicine gave the welcoming address. The programme was separated into 4 sessions

Living with thalassaemia – in the long 1. term chaired by Dr Josh Wright, UKTS chair Dr Christos SotirelisThalassaemia; the reversible 2. cardiomyopathy chaired by Dr Malcolm Walker, UKTS chair George ConstantinouManagement of liver disease 3. in thalassaemia chaired by Prof Geoffrey Dusheiko, UKTS chair Andy CharalambousManagement of infertility and 4. osteoporosis in thalassaemia chaired by Prof Bernadette Modell, Emeritus Professor of Community Genetics

University College London & UCL Centre for Health Informatics & Multiprofessional Education (CHIME), UKTS chair Neelam Thapar.

The presentations were as follows:

The importance of clinical governance •and auditing of performance in improving outcomes in the NHS - Prof Sir Bruce Keogh, Medical Director of the NHS, Professor of Cardiac Surgery, University College LondonLiving as an adult with thalassaemia• – Mr Michael Michael, President, UK Thalassaemia SocietyThalassaemia; a blood condition •of heart, liver & glands – Dr Philip J Darbyshire, Consultant Paediatric Haematologist, Birmingham Children’s Hospital & Chair of the UK Forum on Haemoglobin DisordersThe National Haemoglobinopathy •Registry – Dr Rob Hollingsworth, Head of NHS Medical Data Solutions and ServicesCardiomyopathy – risk stratification •and long term management – Dr Mark Westwood, Specialist Registrar in Cardiology, London Chest HospitalThe investigation and treatment of •cardiac arrhythmias in thalassaemia – Dr Pier Lambiase, Senior Lecturer & Honorary Consultant Cardiologist, the Heart Hospital, LondonEmerging therapies for hepatitis •C – Prof Stefan Zeuzem, Professor of Medicine & Chief of Department of Medicine, J.W. Goethe University

Hospital, FrankfurtManagement of advanced liver •disease in thalassaemia (cirrhosis, hepatocellular carcinoma and assessment of patients for transplantation – Dr James O’Beirne, Consultant Hepatologist, Royal Free Hospital, LondonInfertility and IVF in thalassaemia• – Prof William Ledger, Professor of Obstetrics & Gynaecology, Academic Unit of Reproductive & Developmental Medicine, University of SheffieldNovel data on the pathogenesis and •management of thalassaemia-induced osteoporosis – Dr Evangelos Terpos, Consultant Haematologist, Director of the Department of Medical Research, General Air Force Hospital, Athens & Honorary Senior Lecturer, Faculty of Medicine, Imperial College, London

The formal presentations were followed by a question and answer session chaired by Prof Bernadette Modell.

Just before he closed the conference, UKTS President Mike Michael presented Prof Bernadette Modell with a special award from the Society in recognition of her outstanding contribution to thalassaemia throughout her long career. Said Mike; “Professor Bernadette Modell was the first person in the UK caring for children with thalassaemia who said “Maybe there IS something we can do for these kids. Maybe we shouldn’t just tell their parents to take them home and keep them happy for what life remains to them. Maybe we should actually try to treat them.” She inspired the parents of those children and, crucially, inspired other doctors to think that there is some hope, some future to aim for. That spirit of hope led to a change in attitude towards thalassaemia, which brought ground-breaking advances in treatment. It also led to the formation of the UK Thalassaemia Society. Professor Modell, the fact that so many of us who were children in that era are now in our forties (and even fifties) is in no small measure thanks to you.”

UKTS thanks the following for their sponsorship of this event (alphabetical order) ApoPharma Inc, Clinovia, Novartis, Swedish Orphan International

Conference participants in the New Lecture Theatre, Royal Society of Medicine.

Continues from page 1


New Revised Edition (October 2008)Now available to download from the UKTS website www .ukts .org .uk .

For a hard copy or CD-ROM contact the UKTS office .

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UKTS Annual General Meeting 2008

This year’s AGM took place at the UKTS office on the evening of 8th July. 23 voting members were present at the meeting.

Outgoing Assistant Secretary Katerina Read chaired the meeting and welcomed everyone. All members present were provided with copies of the minutes of the AGM held on 15th May 2007, which were approved and signed by outgoing Secretary Vava Tsioupra. Members were also provided with copies of the audited accounts (financial year ending 30th November 2007).

As only 8 persons were nominated to the Management Committee they were elected without the need for a vote. They were:

Philip Agathangelou, Andy Charalambous*, George Constantinou, Costas Kountourou, Mike Michael, Bharat Nathwani, Christos Sotirelis and Menuccia Tassone (see “Meet the Committee” on page 6).

Messrs Lyons Leonidou, Chartered Accountants & Registered Auditors of Galla House, 695 High Road, London N12 0BT were re-appointed as auditors of the Society for the forthcoming year.

*Mr Charalambous subsequently tendered his resignation, which was accepted by the Committee.

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Philip Agathangelou (Assistant Treasurer)

Philip is a 32 year old beta thalassaemia major patient who has held the office of UKTS Assistant Treasurer for the past two years. He works as an Account Manager for an Internet service provider and is responsible for managing corporate relationships. Philip feels as a patient he has benefited strongly from the Society’s hard work over the years and is keen to put something back and help the Society fulfil its aims and objectives going forward, through adding his skills and experiences to their efforts. His interests include e-commerce (he has completed an MA in E-Business) and additionally spending time travelling when possible. He is currently the UKTS Assistant Treasurer.

George Constantinou (Treasurer)

George is a beta thalassaemia major patient. He is a founder member of the UKTS, having served on the Management Committee from 1976-1985 and again from 1999 to the present day. George has been a tireless campaigner on behalf of thalassaemia all his adult life and has conceived and been involved with many UKTS projects including the Compliance Workshop and Doctors’ Workshop in 2002; also

the thalassaemia patients’ personal organiser. More recently he has been involved in producing the revised version of the UK National Standards for thalassaemia treatment and the 3rd National Doctors’ Conference, which will take place in June 2008. He served UKTS in the office of Treasurer during the last 3 terms. George is a hotel manager by profession and is married and has a daughter.

Costas Kountourou

Costas is a beta thalassaemia major patient. He has been a member of the UKTS Management Committee for many years, being actively involved in fund raising, events organising and the administration of the Society. During the 2005/2006 and 2006/2007 terms Costas served UKTS as Secretary. He is also involved in the editing of the UKTS quarterly magazine Thalassaemia Matters. Costas feels that his many years on the Committee have given him valuable experience, which will help to carry forward the aims of the Society. He is a travel agent by profession.

Mike Michael (President)

Mike is a beta thalassaemia major patient and since his marriage in 2004 he has also become a thalassaemia parent, as his stepdaughter Valerie has the same condition. He has been

President of the UKTS for the last eight years and has served on the Management Committee for several terms in the past. He has represented the Society at many national and international conferences and functions and is a Board Member of the Thalassaemia International Federation. Mike also provides invaluable IT support to the UKTS by installing and maintaining our office computers. He is an IT consultant by profession.

Bharat Nathwani

Bharat’s daughter Mena is a beta thalassaemia major patient. Bharat is a life member of UKTS and a regular attender at past AGMs. Bharat hopes that his experiences as a parent of a young child with thalassaemia then growing up to adulthood will give him a valuable insight into the experiences of other parents in the same situation. He is an accountant by profession.

Dr Christos Sotirelis (Vice President)

Chris is a beta thalassaemia major patient. He is a 43 year old qualified aeronautical engineer who has completed a doctorate in Air Transport Engineering as well as being a licensed private pilot. He works as an air transport consultant on a variety of projects, involving economics, management and planning issues for airlines, airports and associated

organisations. His interests outside aviation include: languages, music, and history, as well as travelling around the world. Chris has served UKTS as Vice President during the 2006/2007 term. Chris believes that he is able to provide some additional skills towards further improving the position and prestige of the UKTS in the UK and abroad, so that it is better able to respond to the complex challenges which present today both nationally and internationally. He is currently the UKTS Vice-President.

Menuccia Tassone (Secretary)

Menuccia is a former beta thalassaemia major patient who underwent a successful bone marrow transplant at the age of 22. She gave a presentation on this subject at the UKTS national conference in 1999. This is her fourth nomination to the UKTS Management Committee. In the past she has served as Assistant/Acting Treasurer and has been involved in many sub-committees organising conferences and fund raising events. She has also been very active in the redesign and editing of Thalassaemia Matters. In her work for the Society, Menuccia aims to serve the UKTS to work towards the objectives of research, fundraising and improvements in the current standard of care for thalassaemia patients.

Meet the UKTS Committee of 2008/2009

(In alphabetical order)


The consequences of thalassaemia major and of its treatment are many, affecting a number of systems within the body. The reproductive system is no exception and many women who suffer from the disorder find themselves troubled by absent or infrequent menstrual periods, failure of ovulation and infertility. Progress in understanding the reasons why these phenomena occur, along with significant improvements in the management of anaemia and iron overload have improved the prospects of such a patient being able to start a family, although concerns about the safety of pregnancy and childbirth in the more severely affected patients remain.

The textbooks tell us that patients with thalassaemia are infertile because of the effects of iron accumulation on the hypothalamus and pituitary glands at the base of the brain. If the hypothalamus fails to produce sufficient GnRH (gonadotropin releasing hormone) or the pituitary fails to produce enough FSH (follicle stimulating hormone) and LH (luteinising hormone), the hormone drive to the ovaries to ovulate, or to the testes to produce sperm, is lacking and the woman or man can become infertile. Lack of FSH and LH is termed hypogonadotropic hypogonadism, and in women is associated with a lack of oestrogen hormone. Young girls with this condition may fail to go through puberty normally, either with no periods at all or with infrequent periods. Others will not see absence of periods until they are older, depending on the severity of their disorder. Men with thalassaemia are only rarely lacking in testosterone so they usually have normal facial and body hair growth, and normal erections, although their sperm quality may be suboptimum.

Investigation of infertility and absent periods in a woman with thalassaemia will involve blood tests, an ultrasound

scan of the pelvis and an MRI (magnetic resonance imaging) of the hypothalamus and pituitary. None of these are particularly unpleasant, apart from the claustrophobia that some patients experience during the MRI scan. Sedation can be used if necessary in such cases. The likely results of these tests will be a low level of FSH, LH and oestrogens in the blood sample, a small uterus and ovaries seen on ultrasound and possibly evidence of pituitary damage on MRI. Such tests are usually easy to interpret and explain why the patient is having problems. Doctors may also give a single injection (or a nasal sniff) of GnRH to see whether this produces a rise in FSH and LH in the bloodstream. This is termed a GnRH challenge test, is free of side effects and can be useful in planning treatment.

More recently, it has become clear that some patients with thalassaemia also suffer from iron accumulation in the ovaries or testes. Hormone treatment is less likely to be effective here, since the ability of the woman to ovulate or the man to make sperm is irrevocably damaged. These patients will have a different hormone profile in their blood tests and will be warned ahead of treatment that there are low chances of success. Thankfully these cases seem uncommon.

Infertility is a problem for a couple, and it is important to remember to check the male partner’s sperm count if the woman is affected, or conversely to check that she ovulates and has healthy uterus and fallopian tubes if the man is affected. Such tests are routine in a fertility clinic setting and can be organised by an interested GP in some cases. We would also want to be sure that the woman has had a recent normal smear test, is immune to rubella (German measles), and screening for chlamydia, hepatitis B & C and HIV might

be offered in addition, particularly if IVF is planned.

Perhaps the most important aspect of the management of infertility in the patient with thalassaemia is to make sure that the health of the patient is optimised before treatment begins. The essentials of pre-treatment assessment are listed in table 1, and require a close liaison between the team of doctors and nurses managing the thalassaemia, and the fertility clinic. It is advisable to refer to a clinic that is based in a hospital setting, within the NHS in UK, rather than to a free standing clinic out of the hospital system, for reasons of patient safety.

Pre-treatment medication also needs careful review. Folic acid supplementation, possibly in higher than routine doses, should be started. Some medications commonly used to treat patients with thalassaemia are possibly unsafe in early pregnancy, including desferrioxamine, biphosphonates (for bone protection), high dose vitamin C and ACE inhibitors (for high blood pressure). These medications should only be stopped by the lead clinician in the thalassaemia team, and not by patients without prior discussion with their doctors. Thalassaemia patients with diabetes can safely continue Metformin, but may need to switch to insulin in pregnancy as diabetic control may deteriorate. Careful monitoring will be needed. Calcium and vitamin D supplements can be continued.

So how might a fertility clinic go about treating infertility in a patient with thalassaemia? The answer will depend on the cause of the problem. If the routine investigations reveal hypogonadal hypogonadism then the simplest approach is to provide a once daily injection of FSH and LH. This can be done using a subcutaneous injection through a pen

Management of Infertility in Patients with Thalassaemia

William Ledger, Professor of Obstetrics and Gynaecology, University of Sheffield, Centre for Reproductive Medicine and Fertility

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device and is not as unpleasant as it sounds. By replacing FSH and LH, the ovaries (or testes) will begin to work again, since they have been lying dormant rather than being destroyed. The woman will ovulate, often with the help of a single injection of hCG (human chorionic gonadotropin) hormone to ensure the egg is released, and can conceive naturally after intercourse at the correct stage of the menstrual cycle. The clinic will monitor the ovaries response to the drugs by ultrasound scans and blood tests – the scans can see the egg follicle growing in the ovary and the blood tests monitor the hormones produced by the growing egg follicle.

There are two significant risks to this approach. The major problem is that the drugs are powerful and can often induce growth of two or more follicles, with risk of twin or triplet pregnancy. Most of the triplets and quads reported in the UK press in recent years have been caused by this treatment, known as ovulation induction, rather than by IVF. Multiple pregnancies have many drawbacks, the most significant being the high risk of premature birth and need for admission to a neonatal intensive care unit. Although many babies will come through this ordeal unscathed, some, particularly the very premature, may have a long term handicap such as cerebral palsy, and some will not survive. It is obviously best to avoid this risk if possible, and responsible clinics will use low doses of drugs with careful monitoring to do their best to produce ‘one baby at a time’. The website of the Human Fertilisation and Embryology Authority has useful information on this topic (www.hfea.gov.uk). The second complication which can result from overdosage with these powerful drugs is OHSS (ovarian hyperstimulation syndrome). The ovaries enlarge and the woman experiences fluid retention with bloating, breathlessness and nausea. Although uncommon in ovulation induction, serious cases require hospital admission and would be particularly complicated to manage in a patient already unwell with thalassaemia. Again, these treatments can be safe if carried out carefully with close liaison with the medical team.

The chances of a pregnancy and live birth after ovulation induction depend on the response of the ovaries to the drugs,

and on the age of the woman. Although seemingly unfair, biology makes it very difficult for women over 40 to achieve a healthy live birth, with rapid increases in rates of infertility, miscarriage and chromosome problems such as Down’s syndrome above this age. For this reason, women with thalassaemia who wish to have children should discuss their plans with their doctors and be seen in a fertility clinic before 35 when possible.

The response of the ovaries or testes to injections of FSH and LH may be surprisingly poor. This would indicate damage to the ovaries or testes themselves, and treatment may be unsuccessful. If this is the case then little can be done, other than to consider treatment with donated sperm or eggs. The clinic would provide counselling to the couple involved, with careful explanation of the benefits and drawbacks to this approach before treatment was given.

Should ovulation induction fail to result in pregnancy despite satisfactory ovulation, or if there are other infertility factors involved, then the clinic may suggest IVF (in vitro fertilisation). IVF has come a long way since the birth of Louise Brown 30 years ago this year, and is more straightforward and less stressful than many believe. Again, the safest clinics for patients with thalassaemia are those which are hospital based. IVF again involves a daily injection of FSH, possibly with use of a nasal spray or injection of a second drug, a GnRH analogue, to prevent early ovulation. The FSH injections last for 10 – 14 days and are followed by a single injection of hCG and then collection of eggs from the woman’s ovaries. Egg collection is done using a needle guided by ultrasound scan control. The scanner is a long thin probe that is placed into the vagina. The ovaries can be easily seen on the scanner and the needle runs inside the probe and is pushed through the top of the vagina into the ovary. This involves a sharp pain in the lower part of the tummy, so the procedure is carried out under anaesthetic or sedation. It takes 10 – 30 minutes to do, and will produce anything from one or two to over twenty eggs.

The male partner will be asked to produce a sperm sample on the same day as egg collection, and the laboratory

scientist (embryologist) will incubate the eggs with the sperm and induce the egg to fertilise. Fertilised eggs (embryos) are grown in the lab for 2 – 5 days. The best one or two embryos will then be replaced into the cavity of the womb (uterus) in a procedure similar to having a smear test taken, a quick and fairly painless, if emotionally charged, procedure. The couple then have to wait for two weeks to see if they have a pregnancy. The clinic will often prescribe a daily progestogen during this time, given as a vaginal pessary, which may help implantation.

If the pregnancy test is positive then there is still a possibility of miscarriage or, rarely, an ectopic pregnancy. A good clinic will perform early pregnancy scans to check that the pregnancy is healthy before sending the couple for antenatal care through their GP. Early pregnancy scanning will also check if there is a twin pregnancy!

Younger patients and those with medical problems that would be made worse by twins (including thalassaemia) would be well advised to have a single embryo transfer to avoid risk of a multiple pregnancy. Although the chances of a baby are reduced with single embryo transfer, the second embryo can be frozen and replaced later in the woman’s natural monthly cycle, often without drugs and without the stress of an egg collection. This “one plus one” approach will give a similar chance of pregnancy to the two embryo transfer, without significant risk of twins.

The chance of a live birth after a single cycle of IVF, based on the average of all treatments in UK in 2006, falls from over 28% for women under 35, to less than 10% for women over 40. Again, useful information can be obtained from www.hfea.gov.uk.

Preimplantation genetic diagnosis (PGD) is a new approach which aims to avoid the chances of a baby having both copies of the gene for thalassaemia. IVF allows embryos to be studied in the laboratory. One or two cells are removed from the embryo and analysed to see if they have normal or thalassaemic genes. Only those embryos that are clear of thalassaemia genes would then be replaced. The technology needed to carry out these tests

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is complex but becoming more widely available in UK. www.laboratoriogenoma.it is a useful website that describes PGD in more detail.

Of course, having a positive pregnancy test is only the end of one chapter, not the whole book. Pregnancy can be complicated in women with thalassaemia and close liaison with the medical team is essential. Many centres will have a dedicated Consultant Obstetrician who works with this group of patients. It is particularly important to monitor cardiac function closely, to maintain haemoglobin levels at or above 10g/dl and to check blood pressure and for diabetes regularly. Ultrasound will be used to monitor foetal growth. As some women with thalassaemia are of short stature, there is an increased need for Caesarean delivery. In the largest UK case series published to date, Susan Tuck and her colleagues at the Royal Free Hospital in London reported 24 live births from 29 pregnancies. Complications included deterioration in cardiac and liver function, and in diabetes. Sadly, there were two deaths from

cardiac disease, highlighting the need for high level medical care for this group of patients. Some of the more severely affected patients with thalassaemia may be best advised to avoid pregnancy because the risk to their life, and potentially also to that of their unborn baby, is too great.

One continuing problem is that the UK NHS has little interest in infertility, and many patients are surprised to learn that they will be expected to pay for their own treatment. Politicians from both major parties have repeatedly promised to improve NHS support but little progress has been made. The position varies greatly across the UK, being determined entirely at local level by PCTs (Primary Care Trusts). Patients who are contemplating treatment can find out about their eligibility for funding from their GP or fertility clinic. Costs of private IVF vary considerably and it can be difficult to establish the true cost of treatment from some websites of private clinics. It is important to establish exactly how much a cycle of treatment will cost, including drugs, scans, consultations and many other “add-ons” before starting

treatment.There have been many improvements in

the medical management of thalassaemia in recent years. Many patients can expect a near normal lifespan and can enjoy a good quality of life. For many of us, this includes having a family and seeing one’s children grow. With a careful, collaborative approach, most women with thalassaemia should be able to be helped to conceive and to have a successful pregnancy.

Table 1 – Pre treatment assessment of patients with thalassaemia

Optimise chelation•Cardiac function•Liver function•Thyroid function •Virology•Optimise diabetic control•Review medication•Screen for acquired red cell antibodies •(risk of haemolytic disease)Check male for haemoglobinopathy•Arrange genetic counselling if necessary•

Prof John Porter

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Frequently Asked Questions About ExjadeReplies by Professor John Porter

Professor of Haematology, University College London

Is there clear evidence that Exjade 1. removes iron from the heart?There is clear evidence in cell culture and in animal models that the drug removes iron from heart and heart cells. There is also evidence in individual patients treated for up to 4 years with Exjade where T2* has improved. This needs to be confirmed in a formal study so that the best dose, the chances of success and the effects relative to Desferal can be precisely documented. There are at least two ongoing trials designed to look at this

question in more detail. One study will examine the rate of change in heart iron with Exjade using T2* and another randomised study will compare the effects of Exjade with desferrioxamine. Data recently obtained from a large scale prospective study confirms that Exjade can remove iron from the heart in Thalassaemia patients.

Is there clear evidence that Exjade 2. removes iron from the liver?There have been at least five large scale trials designed to answer this question.

Replies reviewed by:

Dr Bernard Davis, Consultant Haematologist, Whittington Hospital, London

Dr Farrukh Shah, Consultant Haematologist, Whittington Hospital, London

Dr Paul Telfer, Consultant Haematologist, Royal London Hospital, London

Dr Anne Yardumian, North Middlesex Hospital, London


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It is clear that Exjade removes iron from the liver in a dose dependent manner.

I heard that Exjade has caused liver 3. failure in some people, is that true?There have been over 3000 patients studied in formal controlled trials without any case of liver failure. These include extension phases of these trials that have now reached up to 5 years of follow up. Since the drug has been licensed, over 36,000 patients have received the drug; these include a wide range of diagnoses including elderly patients on multiple medications. Hepatic failure has been reported in several patients who were not on a trial and a causal relationship of this complication to Exjade treatment is therefore difficult to establish. Because of the lack of liver failure in the carefully controlled large-scale clinical trials of more than 3000 patients, liver failure in properly monitored patients is very unlikely. Never the less, it is recommended that, as in the trials, all patients on Exjade have monitoring of liver function monthly.

I heard that Exjade may cause 4. kidney problems, is that true?One way in which kidney function is monitored is to measure the levels of creatinine in the blood. There are several reasons that these levels may increase. In about one third of patients who start on Exjade the creatinine increases by about 30%. This usually happens within a few weeks of starting treatment or increasing the dose. About 6% of thalassaemia patients have shown values above the upper limit of normal and no patient above twice the upper limit of normal. Studies for up to 4 years show that this creatinine increases are not progressive and rarely require interruption of treatment.

Is it safe for my child to take 5. Exjade? Exjade has been tested in formal clinical trails in more children than any other chelator and found to have an acceptable tolerability and that is

why it has been licensed as first line therapy for children over 6 years old. There seem to be no special problems of giving treatment to children: in particular growth appears to be unaffected by Exjade (unlike Desferal above doses of 40mg/kg/day). Follow up is up to 5 years at this time.

I have heard that Exjade can be 6. given to children as young as 2 years, but that the child must be started on Desferal therapy before Exjade can be applied for. Why can’t my child’s start his/her chelation regime with Exjade without having to endure the Desferal infusions first?The original licensing of Exjade in Europe allowed first line treatment for children above six years old and second line (i.e. only in patients in whom desferal is contra-indicated or inadequate) between the ages of two and six. There is no hard and fast definition of what is ‘inadequate’ however.

I am on Ferriprox & doing fine, do 7. I have to change over to Exjade if my doctor suggests it? What reason is there to change?If you are’ doing fine’ with deferiprone: your liver iron, serum ferritin, heart iron, heart function and liver function are all OK and you are not having problems with arthritis there is no reason to change unless you find Exjade easier to take.

How long has Exjade been tried for 8. & how many patients are currently using it in the UK?Exjade has been in long term clinical use for up to 5 years. Patients in the UK have been treated for over 4 years on clinical trials and now as part of their regular treatment. Currently about 500 patients are being treated with Exjade in the UK.

I have heard that the dose at which 9. Exjade acts effectively as a chelator is dangerously close to a dose which may be toxic to the kidneys and/or liver. Need I be concerned?

This is not usually true. Most patients in the clinical trials have shown a decrease or control of body iron at doses that do not cause toxicity. A small number of patients do not tolerate treatment and need to stop but this is generally less than with other chelators.

If I am changed onto Exjade 10. therapy, which monitoring tests should be done, and how often (e.g. liver function tests, serum creatinine and any other necessary tests).Liver and kidney function tests are done at the same time as the cross match (i.e. about once a month). For the first 4 weeks after starting treatment these are checked weekly but thereafter only monthly. When you visit the clinic it is advisable to produce a spot urine sample to check the urine for protein. As with Desferal, testing of ears and eyes are recommended. Unlike deferiprone there is no requirement to do weekly blood count monitoring.

Are there any other side effects, 11. major or minor when taking Exjade that I should be aware of & look out for?The commonest side effects are diarrhoea and abdominal discomfort. These usually settle spontaneously. By changing the timing of the dose until after meals, these symptoms are often improved if symptoms persist. Skin rash, usually at the start of treatment is seen in up to 1 in 10 patients. Mild and moderate rashes usually resolve without interrupting treatment whereas severe rashes require temporary interruption followed by gradual reintroduction of treatment. Only very occasionally is it necessary to permanently discontinue treatment. Other rarer side effects are listed in the patients information leaflet.

Arthritis does not seem to be a problem with Exjade.


Part 1: Reducing the iron absorbed from food .

Diet is not just about losing weight! Being

a dietician, I often have to try and convince

people I meet socially that, no, I don’t spend

my time handing out diets to perfectly

healthy ladies who need to lose 3kg in order

to fit into their evening dresses! Diet is the

way we need to eat to optimise our health.

This is different for different people and

different ages. Thalassaemia is a complex

condition and the ideal diet would need to

take account of many factors. This article

concerns the iron present in food and also

discusses Antioxidants in food, diet for the

prevention of Osteoporosis and Diabetes,

Zinc in the diet and diet for children with

thalassaemia.

In thalassaemia, although most of iron

overload is due to blood transfusion,

increased absorption of iron from the diet

is also important. Only a small amount

of iron from the diet is absorbed into our

body. The amount absorbed is higher when

haemoglobin in the blood is low. People

with low haemoglobin such as those with

thalassaemia intermedia or those with

thalassaemia major, in-between transfusions

could therefore adapt their diet so that not

only the total amount of iron in their diet is

low but also the amount of iron absorbed

into their body is low. There are two kinds of

iron in the diet: iron which is present in red

meat (Meat iron) and iron which is widely

distributed in the diet (Non-meat iron).

Meat IronMeat iron is present in red meat such as beef,

lamb and pork and the dark meat of chicken

as well as in seafood such as sardines, cockles

and mussels. Liver is a very rich source of

meat iron Try to cut down on these and

perhaps substitute meat with soy protein.

It is not, however, a good idea to exclude

meat, chicken and fish completely from your

diet because they contain other important

nutrients, particularly for children. Choose the

white part of chicken rather than red meat as

it contains less iron.

On average, after a meal with red meat,

about 35% of iron will be absorbed into

our body. However, this may vary between

10-40%, depending mainly on whether the

meal contains milk or milk products. The

calcium, present in milk, cheese, yoghurt,

cream decreases the absorption of meat

iron. Try to drink a glass of milk with a meat-

containing meal and to use milk in cooking.

Good examples are the white cheesy sauces

in lasagna, pasticcio, mousaka and cannelloni,

adding lots of cheese in spaghetti bolognaise

and using yoghurt and milk to cook your

curries.

Milk intake should be at least one pint

daily, particularly because it helps to prevent

osteoporosis, as it will be discussed later. If

you are worried about your weight, semi-

-skimmed or skimmed milk are just as rich

sources of calcium as whole milk.

Non-Meat IronNon-meat iron is widely distributed in the

diet, present in eggs, chocolate, cereals,

vegetables, fruits roots (potatoes, parsnips),

beans and lentils. In the UK several foods are

fortified with iron, such as breakfast cereals,

wheat flour and bread. However this may not

be the case in other countries.

The absorption of non-meat iron from the

diet into our body is much less than that of

meat iron, but it may vary more than 20 fold,

depending on the composition of a meal.

The foods which decrease its absorption

are: (i) cereals (ii) dairy products. The foods

which increase its absorption are: (i) fruit

and vegetables rich in vitamin C, (ii) meat,

fish, shellfish and poultry and (iii) pickles,

sauerkraut, soy sauce, vinegar and alcohol.

It is difficult to avoid taking non-meat iron

because it is present in most foods. However,

diet can be modified by taking more of the

foods which decrease and less of the foods

which increase the amount of iron absorbed

into our body. These foods will be discussed

below.

Foods which decrease non-meat iron absorption .

1 . Cereals:Wheat bran, maize, oats, rice and soy,

decrease the iron absorbed into our body

and fight the effect of vitamin C. Foods rich

in vitamin C increase iron absorption. It is

good to eat a lot of cereals in your diet, but

remember not to take a vitamin C-rich food

with them, like orange juice. Try to combine

milk and cereals (e.g. cheese sandwich,

French toast, macaroni cheese, cereals and

milk). In the UK, all wheat flour other than

wholemeal is required by law to be fortified

with iron. The fortification of breakfast cereals

is voluntary. It may therefore be better to

choose unfortified wholemeal wheat flour

and bread, and to look carefully at the label

of your favourite breakfast cereal. Unfortified

breakfast cereals include porridge oats and

some cereals in health shops but look at the

label to make sure you choose an unfortified

variety. In other countries, flour and breakfast

cereals may not be fortified. Is this a good

excuse for more holidays abroad then?

Soy protein also decreases the amount of

iron absorbed into your body. Soy protein

can work well in many recipes (e.g. spaghetti

bolognaise, stews and casseroles) and the

taste can be improved by adding spices.

2 . Tea, coffee and spicesTea, coffee and some spices (e.g. oregano)

decrease iron absorption. Drink plenty of tea

and coffee daily, particularly with your meals.

Better yet, if you take it with milk. Tea is also

a very good source of antioxidants as will be

discussed later. And, keep adding oregano to

spice-up your souvlaki!

3 . Dairy productsMilk, cheese and yoghurt decrease the iron

absorbed into our body. Calcium is also

important for osteoporosis, so it is good

to include as many dairy products as you

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Diet for Thalassaemia

By Dr Dona Hileti, Senior Dietitian Great Ormond Street Hospital, London

Prolypsis Medical Centre, Nicosia, Cyprus


can in your diet. Lower fat varieties of milk

(skimmed or semi-skimmed) and cheese are

just as high in calcium and may be preferred

if you are watching your weight. At least one

pint of milk should be taken every day.

Foods which increase non-meat iron absorption .

1 . Vitamin CVitamin C is present in fruit, fruit juice and

vegetables. It is better to avoid drinking fruit

juice, such as orange juice, with your meal or

your toast in the morning. Instead, a cup of

tea or coffee is better options as they inhibit

iron absorption. Alternatively, have a glass

of milk! Beer increases iron absorption so it

is better to avoid drinking it with your meal

too often, but you could always have it on

its own with some nuts! Fruit and fruit juice

are, however, good sources of antioxidants

and should be taken on their own as snacks.

Boiled vegetables contain much less vitamin C

because the vitamin leaks in the water.

2 . Meat, poultry, fish and seafoodMeat, poultry, fish and seafood not only

contain a lot of meat iron but they also help

to absorb more of the non-meat iron from

your food! It would be unwise, however, to

omit them from the diet altogether as they

contain other vital nutrients, particularly

important for children and adolescents.

3 . Pickles, sauerkraut, soy sauce, vinegar, alcohol

Sauerkraut, pickled onions, turnips and

carrots as well as fermented soy products

(e.g. miso and soy sauce) enhance iron

absorption. The amount of iron absorbed is

even higher when the pickled vegetables are

added to bread and rye- containing meals.

In general, a low iron diet would contain

cereals (maize, whole-grain flour, beans) and

root vegetables with little meat, fish or foods

rich in vitamin C. A moderate iron diet would

consist of cereals and root vegetables but

would also contain some vitamin C-rich foods

and meat. High iron diets contain generous

quantities of meat, poultry and fish. They also

contain foods with high levels of vitamin C

such as citrus fruits and some vegetables. A

high iron diet can be reduced to a moderate

one by the regular consumption of foods

which decrease the amount of iron absorbed

by our body, such as dairy products, cereals,

beans, coffee and tea. Right, says Fred, let’s

have a cup of tea!!!

Part 2: Antioxidants in Food

Paradoxically, oxygen is essential for life but

is also lethal! This is because normal oxygen

molecules can convert into different chemical

forms known as ‘free radicals’. When the

activity of free radicals is harnessed and

controlled, they have important uses in the

body. Uncontrolled free radical reactions,

however, can do great damage and lead to

disease.

Antioxidants are important in any diet,

because as their name suggests, they prevent

oxidative damage in the body. In doing so,

they play an important role in the prevention

of diseases such as coronary heart disease

and cancer. In thalassaemia, because of the

excess iron in the body, there is a higher risk

of oxidative damage. In this article, I will

concentrate on the four main antioxidants:

Vitamin E, Vitamin C, Carotenoids and

Flavonoids

1 . Vitamin EVitamin E is the most important dietary

antioxidant. Several studies have found that

many thalassaemics have lower levels of

Vitamin E in their blood compared to non-

thalassaemics. This could be either because

thalassaemics do not take as much Vitamin E

in their diet or because their needs are higher.

In many studies, when Vitamin E was given

as a supplement, Vitamin E levels in the blood

improved. However, even if your doctor or

dietician recommends you take a supplement,

the best way for any vitamin to enter your

body is through your food.

Vitamin E is fat-soluble which means that it

is present in foods which have a high amount

of fat. The best sources of Vitamin E are

vegetable oils (olive, safflower, palm and soya

oil). The best one to use is probably olive oil

because the type of fat it contains can help

to prevent heart disease. In Mediterranean

countries where olive oil is used a lot (Greece,

Portugal, Spain, Italy) heart disease is lower

than in Northern Europe. Remember,

however, that the vitamin is destroyed slowly

with frying. Therefore, the best way to get

the most out of your olive oil is to add it

to food towards the end dressing. Olive oil

mixed with lemon, for example, can make

a delicious dressing for fish, chicken, boiled

vegetables and salads. Being Greek Cypriot

myself, I can give you many recipes where

olive oil features as the main ingredient! You

can probably do better, however using you

own imagination. Choose the extra virgin

olive oil if you like the intense flavour and you

tend to use it as a dressing, or experiment

with more refined varieties if you want to

use it for cooking, making cakes etc. Ghee

also contains Vitamin E but since olive oil has

additional health benefits, you may like to try

using it in cooking.

Other sources of Vitamin E are dairy

products, cereals, nuts, eggs and meat. Dairy

products are particularly good to include

in the diet not only because they contain

Vitamin E, but also because they inhibit iron

absorption from our food into our body and

also because they contain a lot of calcium

which can help to prevent Osteoporosis

(weak bones). You can try to use milk in

cooking or to have a glass of milk with your

meal. Skimmed milk has lower levels of

Vitamin E than full-cream milk although the

amount of calcium is the same.

2 . Vitamin CYou might remember from my previous article

that Vitamin C increases the absorption of

non-meat iron. Therefore, although Vitamin

C is a very powerful antioxidant, I will not

advocate using many Vitamin C-containing

foods in combination with foods that are

high in non-meat iron. This is important for

those with thalassaemia intermedia and are

not regularly transfused.

Remember that non-meat iron is widely

distributed in the diet, present in eggs,

chocolate, cereals, vegetables, fruits, roots

(potatoes, parsnips), beans, and lentils. In the

UK, several foods are fortified with iron, such

as breakfast cereals, wheat flour and bread,

although this may not be the case in other

countries.

Vitamin C is mainly found in fruit, fruit

juices and vegetables. It might be better to

have your piece of fruit or glass of fruit juice

on their own, in-between meals and not

during or immediately after your meal. As

health professionals we recommend people

to eat 5 portions of fruit and vegetables daily.

Examples of what is one portion are: a glass

of fruit juice, a piece of fruit such as apple,

pear, banana, orange, half a grapefruit,

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one tomato, a helping of vegetables such

as carrots, courgettes, French beans or a

small salad. Vitamin C is water-soluble, so if

boiling vegetables it will leak out in water.

Light steaming preserves the vitamin better.

Cooked vegetables with olive oil and lemon

can make a very tasty snack or a light meal.

Vitamin E and Vitamin C work better when

they are together and therefore remember to

fuel your vegetables with olive oil!

3 . CarotenoidsCommon dietary sources of carotenoids

are carrots, yellow squash, corn, tomatoes,

papaya, oranges and dark-green leafy

vegetables. Again, most of these foods are

high in Vitamin C and therefore the same

caution applies as above. It is worth pointing

out that the absorption of carotenoids from

the diet is much higher when the food

contains fat or oil. So, keep adding that olive

oil!! Carotenoids can be destroyed when

cooking at high temperatures, therefore keep

the cooking low and short if you can.

4 . FlavonoidsThese are found in tea, red wine, fruit and

vegetables. What better excuse to include

a glass of red wine with your meal! If it is a

more sober occasion, have your meal with

a cup of tea! I am sure the English amongst

you are rejoicing now! Tea will not only

give you lots of antioxidants, but it will also

inhibit the absorption of iron from your food,

especially if you take it with milk. Try to have

several cups of tea daily. Remember that we

need about 8 glasses of fluid daily to be well

hydrated.

SummaryVitamin E is mainly found in vegetable oils •

such as olive oil and safflower oil. The best

one to use is probably olive oil because it

can help to protect against heart disease.

Add it towards the end of cooking, after

the food is cooked or on raw vegetables

because heating can destroy the vitamin.

Vitamin C is present in fruit and •

vegetables. It is best not to consume

many of those in combination with foods

that are high in non-meat iron, if you

have thalassaemia intermedia and are not

being transfused. You could have fruit and

vegetables in-between meals. Add olive oil

to your vegetables because Vitamin C and

Vitamin E work better together.

Carotenoids are found in carrots, yellow •

squash, corn, tomatoes, papaya, oranges

and dark-green leafy vegetables. As these

foods are also high in Vitamin C, the

above caution applies again to olive oil.

Tea and red wine contain flavonoids which •

are also antioxidants. Furthermore, tea

inhibits iron absorption.

PART 3: The role of Zinc

Zinc has important biological functions

which are still not fully understood. Among

other roles, it is important for the growth of

children, sexual maturation of adolescents, a

strong immune defence system and healthy

skin. Several studies have shown that people

with thalassaemia tend to have low levels

of zinc in the blood, probably because they

excrete more zinc in the urine.

This is partly because iron chelators (such

as Desferal and Deferiprone) not only bind

iron but also some zinc and excrete it in the

urine. So what can we do to help maximise

the amount of zinc we get from our food

and prevent us to get a deficiency? Unlike

iron, zinc is not stored in our body. We are

therefore dependent on a regular supply

of zinc from our daily diet to provide our

requirements. In this article, I will highlight

the main sources of zinc in our diet and

explain how to get the most out of the zinc

in our food. Zinc supplements can be bought

across the counter but it is not safe to use

them unless recommended your doctor or

dietician. Taking zinc supplements above our

requirements can interact with other nutrients

such as copper and can also be bad for the

immune system. The main nutritional sources

of zinc are animal foods (meat and dairy

products) and wholemeal cereals. These will

be discussed below.

1 . Animal foodsBeef, pork, chicken and fish contain large

amounts of zinc. Zinc is present in the

lean part of meat and not the fatty part.

Therefore, chose lean cuts of meat and

skinless chicken, especially if you need to

watch your calorie intake. As red meat is

also high in iron, it may be better to chose

chicken or fish instead.

Dairy products (milk, cheese, yoghurt) and

eggs also contain a lot of zinc. Milk and milk

products are very important in thalassaemia

for a variety of reasons; they inhibit the

absorption of iron, they can help to prevent

osteoporosis, are important for growing

children and are also useful sources of zinc. I

cannot stress it often enough how important

it is to take plenty of milk every day, either

as a drink or as part of your meal recipes.

Choose low-fat cheese, yoghurt and skimmed

or semi-skimmed milk if you are watching

your weight.

2 . CerealsZinc is present in the outer part of the

grain of most cereals. Therefore, unrefined

varieties of wheat, maize and rice are good

sources of zinc, while refined cereals are

poor sources. Try to switch to wholemeal

bread for sandwiches and use brown rice

and wholemeal flour and pasta in cooking.

Cereals contain a substance called phytic

acid which inhibits the absorption of zinc

from food. What is interesting is that the

animal food sources described above (meat,

chicken, fish, milk, eggs) can prevent this.

It is therefore beneficial to include some

chicken, fish or milk with the unrefined

cereals. Examples are chicken, tuna or cheese

sandwiches and wholemeal bread, chicken

curry with brown rice, wholemeal spaghetti

with grated cheese, chicken or cheese pies

made with wholemeal flour. I am sure you

can add your own examples to this list!

Taking your meal with a glass of milk or a cup

of milky tea will give you an extra star!

3 . Food preparationThe way we prepare our food can also affect

the amount of zinc we get from our diet.

Like many other nutrients, zinc can leach into

cooking water during food preparation. It is

better to avoid using too much water when

boiling and to try steaming instead. When

cooking chicken or fish, we can use the

juices after roasting, frying or boiling to cook

sauces which can compliment the meal. In

this way, any zinc leaching into the cooking

media will still be included in the meal. A few

weeks ago, I was watching one of the TV

chefs preparing a pasta meal and much to

my surprise he added the water from boiling

the pasta into the sauce! Apparently, it is

supposed to make it more creamy! Certainly

from the zinc point of view, it is not such a

bad idea!

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Stefan Zeuzem, MD

Professor of Medicine

Chief – Department of Medicine I

J.W. Goethe University Hospital

Theodor-Stern-Kai 7, 60590 Frankfurt

Germany

Phone: +49-(0)69-6301-6899 or 4544

Fax: +49-(0)69-6301-6448

Email: [email protected]

A broad spectrum of agents are under investigation for treatment of chronic hepatitis C, and it is hoped that these drugs will ultimately increase cure rates, reduce the required duration of therapy, improve tolerability and possibly simplify therapy. There are presently no compounds in clinical trials that have the potential to replace the current standard of care when used alone. Rather than supplanting the standard of care, it is now clear that the new agents will be utilized, at least initially, against a backbone of PEG-IFN plus ribavirin.

Protease inhibitors

TelaprevirClinical data available to date with telaprevir have been obtained in treatment-naive patients with genotype 1 infection. When telaprevir is administered at a dose of 750 mg every 8 hours, a >2 log drop in serum HCV RNA within 72 hours is typical and maximal reductions in serum HCV RNA levels, on the order of 4.4 log10 IU/ml, are achieved after 14 days. Telaprevir-resistant HCV emerges rapidly during monotherapy. Some variants isolated after 2 weeks of treatment have a >60-fold increase in half maximal inhibitory concentration as compared with wild-type HCV. This shows that telaprevir has a low barrier to genetic resistance; however, when telaprevir is combined with PEG-IFN alfa-2a plus ribavirin, the antiviral activity is improved and the incidence of resistance is greatly reduced.

When telaprevir is administered with PEG-IFN alfa-2a, rapid additive reductions in viral load are achieved and, in some patients, serum HCV RNA is suppressed to below the limit of detection (<10 IU/

ml) within 14 days. In a small Phase I trial (n=12) in which telaprevir was administered for 28 days in combination with the standard of care, all patients had undetectable HCV RNA at the end of treatment with PEG-IFN alfa-2a, ribavirin and telaprevir triple therapy.

In the randomized, double-blind, placebo-controlled Phase II PROVE-1 (n=250) and PROVE-2 (n=323) trials, telaprevir is being administered for 12 weeks with PEG-IFN alfa-2a 180 µg/week with and without (only PROVE-2) ribavarin 1,000 or 1,200 mg/day. Final SVR data are now available for patients in most treatment groups in these trials. In PROVE-1, 6 of 17 patients treated for 12 weeks with telaprevir-based triple therapy achieved an SVR, and among those treated for 12 weeks with telaprevir-based triple therapy followed by 12 weeks of PEG-IFN alfa-2a plus ribavirin (24 weeks total) the SVR rate was 61%. Consistent with these findings, SVR rates of 62% were obtained in a larger group of patients (n=82) treated for 12 weeks with telaprevir-based triple therapy and 68% of patients (n=81) treated for 12 weeks with triple therapy then 12 weeks with the standard of care in the PROVE-2 trial. The SVR rate in patients treated with telaprevir plus PEG-IFN alfa-2a for 12 weeks was 36% (n=78). Collectively, the results of PROVE-1 and PROVE-2 demonstrate that ribavirin is essential to maximize SVR rates in patients treated with telaprevir, and that SVR rates as high as 68% may be possible in genotype 1 patients treated with a 12-week triple-therapy regimen followed by a 12-week standard combination-therapy regimen.

Detailed genotypic and phenotypic analyses have been conducted to characterize the resistance profile of telaprevir. Six resistant variants have been detected to date. The data suggest that the steep initial Phase 1 decline in HCV RNA is due to suppression of wild-type virus, that viral rebound is either due to pre-existing resistant variants or rapid de novo selection of resistant variants not present at baseline and, importantly, that telaprevir-resistant variants are susceptible

to PEG-IFN alfa-2a and ribavirin. In PROVE-2, virological breakthrough was much less common in patients treated with telaprevir-based triple therapy (2%) than with telaprevir plus PEG-IFN alfa-2a (24%). Viral sequence analysis showed that breakthrough was associated with selection of telaprevir-resistant mutants. Patients who relapsed after the end of treatment have been shown to harbour telaprevir-resistant variants, although the role of these mutants in precipitating relapse is unclear.

The incidence of telaprevir-resistant HCV was related to low trough serum drug levels of both PEG-IFN alfa-2a and telaprevir in the PROVE-1 study. However, interestingly among those patients with a low trough serum drug levels of telaprevir but high serum drug levels of PEG-IFN alfa-2a or a low serum drug levels of PEG-IFN alfa-2a but high serum drug levels of telaprevir virologic breakthrough was rare suggesting that maintenance of higher drug levels of one or both drugs would be expected to limit the emergence of resistance in the setting of combination therapy.

Telaprevir was associated with increased rates of certain adverse effects including rash, gastrointestinal events and anaemia. The rate of discontinuation for adverse events during the first 12 weeks of PROVE-1 and PROVE-2 was 2–3-fold higher in recipients of telaprevir-based triple therapy than with the standard of care. The maculopapular rash has generated the most concern, but this event resolved upon treatment discontinuation in all patients.

BoceprevirBoceprevir (SCH 503034), an orally active protease inhibitor, produces dose-related reductions in HCV RNA levels when administered alone or in combination with PEG-IFN alfa-2b. In a Phase I dose-ranging study, the drug was studied at doses of 100 mg twice daily to 400 mg every 8 hours for 14 days. Administration every 8 hours produced greater reductions in HCV RNA levels than twice-daily administration and 60% of patients treated with the 400

Targeted Therapy for Chronic Hepatitis C


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mg every 8 hours daily dose had reductions >2 log10 from baseline (versus <20% with twice-daily administration).

Boceprevir has been studied in HCV genotype 1 patients who did not have a virological response (<2 log10 decrease in HCV RNA) after 12 weeks of treatment with PEG-IFN alfa-2b with or without ribavirin. Patients (n=26) received boceprevir 200 or 400 mg every 8 hours for 1 week, PEG-IFN alfa-2b 1.5 µg/kg/week for 2 weeks and the two drugs in combination for 2 weeks during this randomized three-period crossover study. The highest reductions in HCV RNA level occurred after 2 weeks of treatment with the combination of boceprevir 400 mg three-times daily plus PEG-IFN alfa-2b 1.5 µg/kg/week. A total of 4 of 10 patients treated with this combination regimen became HCV RNA negative during 2 weeks of treatment. The effects of the two agents were additive and boceprevir did not dampen the characteristic fluctuations in serum HCV RNA levels that occur during once-weekly treatment with PEG-IFN alfa-2b.

Mutations that confer resistance to boceprevir have been selected during in vitro experiments and have been isolated from patients treated with the drug. The variant (A156T) with the highest level of resistance (>100-fold) to boceprevir had significantly reduced colony formation efficiency in a replicon system and is less fit than wild type virus.

In the Phase II SPRINT-1 study, boceprevir 800 mg thrice daily is being evaluated in combination with PEG-IFN alfa-2b 1.5 µg/kg/week plus ribavirin in treatment-naive HCV genotype 1 patients. In two treatment groups, boceprevir is being introduced after 4 weeks of treatment with PEG-IFN alfa-2b plus ribavirin 800–1,400 mg/day, after which triple therapy will be continued to complete a total of 28 or 48 weeks. In a third group boceprevir is started concurrently with PEG-IFN alfa-2b plus ribavirin 400–1,000 mg/day and will be administered for 48 weeks. A control group will receive the standard of care for 48 weeks. According to a preliminary report, 54–79% of patients in the triple-therapy groups had undetectable serum HCV RNA levels (<15 IU/ml) after 12 weeks compared with 34% of control patients.

The rate of treatment discontinuations due to adverse events was higher in patients receiving triple therapy (8–12%) than in the control group (5%).

Nucleoside Analogue Polymerase inhibitors

R1626R1626 is an oral prodrug of a potent and selective nucleoside analogue polymerase inhibitor (R1479). Dose-dependent reductions in HCV RNA of up to 3.7-log10 were obtained after 14 days when R1626 was administered at a dose of 1,500–4,500 mg/day in patients infected with HCV genotype 1. After 14 days of treatment, five of nine patients treated with the highest dose had undetectable HCV RNA (<50 IU/ml). Reversible reductions in haemoglobin levels (mean 0.9 g/dl decrease at day 14 with 4,500 mg/day relative to placebo) and white blood cell counts were detected in patients treated with the drug. The combination of R1626, PEG-IFN alfa-2a and ribavirin produces synergistic reductions in serum HCV RNA levels in patients infected with HCV genotype 1. An interim analysis of an ongoing Phase II trial showed that 4 weeks of treatment with the combination of R1626 1,500 mg twice daily plus PEG-IFN alfa-2a 180 µg/week and ribavirin 1,000/1,200 mg/day reduced serum HCV RNA levels beyond that achieved with the combination of R1626 plus PEG-IFN alfa-2a (5.2 versus 3.6 log10). The standard of care (PEG-IFN alfa-2a plus ribavirin) produced a 2.4 log10 reduction in HCV RNA over 4 weeks. A total of 81% of patients had undetectable HCV RNA after 4 weeks of treatment with R1626-based triple therapy. Neutropenia (<0.5 x 109 cells/l) was detected in 12 of 31 of patients during treatment with this regimen and 2 of 20 patients treated with the standard of care.

No evidence of resistance to R1626 has been detected in clinical isolates obtained from patients treated with the drug. Thus, R1626 appears to have a high barrier to the development of resistance. R1626 is being studied at doses of 500–1,500 mg twice daily in combination with PEG-IFN alfa-2a at a dose of 90 or 180 µg/week and ribavirin in an ongoing 48-week Phase II trial.

R7128R7128 is a prodrug of PSI-6130, a cytidine analogue inhibitor of HCV polymerase inhibitor that produces at least additive reductions in HCV RNA levels when administered with the standard of care. In a Phase I trial the combination of R7128 1,500 mg twice daily plus PEG-IFN alfa-2a and ribavirin reduced serum HCV RNA levels by a mean of 5.12 log10 IU/ml in 20 treatment-naive patients with HCV genotype 1 infection (versus 2.95 log10 IU/ml in 10 patients treated with placebo plus the standard of care). A total of 17 of 20 (85%) of patients in the triple-therapy group had undetectable HCV RNA (<15 IU/ml) at week 4 compared with 1 of 10 (10%) in the control group. Headache, chills, fatigue, nausea and fever, were the most common adverse events across all treatment groups. Grade 4 neutropenia was reported in 1 of 20 patients in each dosing cohort and in 1 of 10 placebo recipients.

Non-nucleoside polymerase inhibitors

GS-9190GS-9190 is a non-nucleoside polymerase inhibitor that is being evaluated in treatment-naive genotype 1 patients enrolled in an ongoing Phase I dose-escalation study. The plasma pharmacokinetic profile was dose proportional in patients who received single oral doses of 40–2,400 mg. The mean terminal elimination half-life of the drug was estimated to be 10–13 hours, which suggests the drug is suitable for once- or twice-daily dosing. Maximum median reductions in serum HCV RNA levels of 0.46 to 1.49 log10 IU/ml were obtained 24 hours after administration of a single dose and mean reductions of 1.4 log10 IU/ml and 1.7 log10 IU/ml, respectively, were obtained after 8 days of treatment with 40 mg and 120 mg twice daily. Further development of GS-9190 is on-hold as a possible but not confirmed cardiovascular adverse event (QT prolongation) was observed. A specific electrophysiological study in healthy volunteers has been initiated to further evaluate this finding.


Nepal is one of the poorest countries in the world…

The Nepal Thalassaemia Society was founded, along with a few other members, by an incredible man named Durga Pathak who lives in Kathmandu. I only became involved myself when one of the children from the mountain village school I support became critically ill and was diagnosed with thalassaemia… that was the beginning of mine and Durga’s wonderful friendship.

Last year I was most thrilled to be asked to write a little article about thalassaemia in Nepal for your UKTS news letter; and I am now once again delighted to write and give you all a little update on our progress.

As you are all well aware having to live with thalassaemia is no easy task… its a 24/7, 365 day a year illness that requires lots of attention….. that’s why living with thalassaemia in Nepal is almost impossible - and that is why most children die before they reach their teens.

In Nepal there is no specific place for patients to go and receive advice, help, care and treatment for their condition. There are only a few places scattered over Nepal where even a blood transfusion is available – it’s hard to believe. The main place to receive a transfusion is in the capital of Kathmandu (Ktm), in a hospital called “Kanti Childrens Hospital”. The hospital is over populated and far from hygienic and there are no facilities for the

children and their parents during their transfusions. Each child has to make a special journey (usually 8 – 16 hours) to Ktm once every 2 weeks… on arrival they have to go to the Red Cross Blood Bank on one side of Ktm to collect a packet of blood (now free to all thalassaemic children through donations from the UK) and then make the gruelling, often 2 hour journey to the other side of heavily polluted Ktm where the hospital is situated. On arrival they are checked in and allocated a bed so the transfusion can begin… there is nowhere for the parent to sit, let alone lie down. Often the parent will sleep under the bed or on the bed with the child while the transfusion is taking place. Parents pray that there are no fevers or complications during each transfusion as doctors are few and far between and extremely costly.

We want to change all that !!!The Nepal Thalassaemia Society has a

dream… a dream to open a desperately needed clinic especially for our growing numbers of thalassaemic children…

We have a plan to open a small but perfectly planned clinic very close to the Red Cross Blood Bank in Kathmandu. It will offer heavily subsidised transfusions and treatment for all thalassaemics. Proper patient records can be kept; and a fully qualified and experienced doctor will be available to give specific medical advice. Transfusions will be carried out hygienically and under much more comfortable conditions than are available now - we even plan to have a TV and DVD player to play educational and medical programmes (many of the children would never have access to this in their life time !!) and to educate the parents further on their children’s condition… it will be a clinic solely dedicated to the Thalassaemic Children of Nepal.

I have spent this year in the UK on a mission to raise money to fund our

dream… I hope to raise £25,000 which would not only fund the set up of the clinic but secure the next 3 years of running costs. Approximately a quarter of the necessary funds have been donated thanks to friends, family and local people and I hope to reach my target by the end of the year… we will achieve our goal and our children of Nepal WILL have a better quality of life.

At the moment only a handful of children are having regular transfusions and Desferal treatment… little Harimaya Upreti who you met in my last article is one of those, she is financially supported by my Uncle, Patrick Stephens here in the UK… she is one of the luckiest of them all… last month she received one of the 5 desferal machines donated by UKTS. Durga Pathak invited her and her brother to stay at his house for 2 weeks during which time he taught Harimayas brother to administer the Desferal treatment… now Harimaya has her treatment in her little mud hut in the foothills of the Himalayas !!! its a miracle !! most will never have the chance to even try Desferal… its just financially not an option.

I would very much like to take this opportunity to say a massive thank you to Elaine Miller and the UKTS for the kind donation of 5 Desferal machines to our Nepal Thalassaemia Society… I can assure you they are already in full time operation !!!! they have already been a great source of delight to the children who are benefiting from them… thank you UKTS from all our children of Nepal.

I would also like to thank Hina Raithatha and her father for their very generous donation toward our clinic… you touch my heart with your kindness.

Finally… thank you all for your love, support and kindness… a little goes along long long way in The Kingdom of Nepal x

Wendy Pinker

On Behalf of The Nepal Thalassaemia Society

Wendy with her adopted Nepali children, Arjun and Yashoda.

news from around the world


Continuing our tradition of forming links with thal patients all over the world, UKTS was delighted to recently donate 2 Medis pumps to the Moroccan Association of Thalassaemia and Haemoglobin Diseases. Professor Mohammed Khattab (pictured left) received the pumps on behalf of the Moroccan Association.

news from around the world

News from Morocco


When you bring a child into this world, you make a commitment to nurture and ensure this baby grows up to be a successful adult achieving his/her highest potential. It is no different if the child has a chronic blood disorder like thalassaemia. You have the same hopes and aspirations for her and expect her to achieve her full potential. And you provide her with the best opportunities that you are capable of. Thalassaemia is only part of your child. Just like having teeth that must be brushed and hair that must be washed. And for her, she has to top up her blood when it is low and get rid of her excess iron

accumulated from the multiple transfusions. Thus, you incorporate her hospital visits into your daily life, together with her daily chelation regime and the many monitoring tests. AND ferrying her to kindergarten, music lessons etc…

You have the same expectations from her as from your other children and do not allow her to use thalassaemia as an excuse not to do her best. She has to follow the same rules as her siblings and bear the consequences of her mistakes or reap the rewards from her hard work.

Living with thalassaemia makes you realise, as a parent, that you’ve that you’ve got to make sure that the family is familiar with her care and you are not the sole carer, in case something happens to you. You must train her to be responsible for her own management when she is old enough, to be knowledgeable about her condition and be aware of her treatment regime. In other words, she must be able to look after herself and know what it takes to be in the best of health.

As a mother, I take the initiative to learn as much about thalassaemia as I possibly can.

It is unfair to expect the medical team to do so. They have to look after all the other illnesses that exist. As my daughter grows older, we work as a team and specialise in thalassaemia care and management. So long as we understand and are knowledgeable about thalassaemia, it is no longer a burden. We deal with any complication that arises and try to overcome it in the best possible way. Isn’t this what life is all about? Each and every one of us has our own challenges. However, we are really privileged to have a whole team of doctors, nurses, lab technicians, volunteers and family (both thalassaemia family and our own) to support us in dealing with ours.

Thalassaemia is not a problem. It is only a problem if we allow it to be. We can accept that thalassaemia is in the family and deal with it, or we can let its problems consume us and curl up in a corner and fade away in self pity. From the way I see it, I owe it to my daughter to incorporate it into our lifestyles and live the lives that we are blessed with, with a positive and optimistic outlook. In fact, thalassaemia has made us stronger, with greater empathy to everything else.

Having a Thalassaemic ChildBy Khoo Swee Hong, Malaysia

Khoo Swee Hong (left) with daughter Janice Kua.

patient news

On Saturday 20th September 2008 a terrific party was held in aid of UKTS. Nearly 200 people packed the function room of the Fox Pub in Palmers Green, North London, for a fun evening which included dancing, karaoke, raffle prizes and of course, a lot of laughter! The organiser was our supporter (and thal patient) Tanya Yucel, who did a fantastic job of bringing all the elements of the event together. I should mention that there was also a delicious buffet supper, most of which was prepared by Tanya, her mum and friends. We all had a great time and in the process raised the amazing sum of £2,020 for UKTS!

A massive thank you to Tanya and all those who helped her to make the evening such a great success, including those

named below who donated goods, services or raffle prizes.

Andy Charalambous • – DJ and karaoke host (assisted by Barry Demetriou)Angela Reynolds • – balloon decorationsAroma Patisserie of Palmers Green •– goodsBurgéon Floral Design • – raffle prizeDivine House of Barnet • – raffle prizeEpping Physiotherapy Clinic • – raffle prizeIos Restaurant of Southgate • – raffle prizePortrait Studio • – raffle prizeToni & Guy of Enfield • – raffle prizeTop Discount of Palmers Green •– loan of television for karaokeYasir Halim of Palmers Green • – goods and raffle prizeZehra • – raffle prize

Party Night in Palmers Green


patient news

Gabriel Theophanous made history in April 2006, when he became the first thal major in the world to run a marathon! We are now delighted to report the next stage in Gabriel’s personal history; which he tells in his own words.

Nishel and I got married on 28th June 2008, at a venue called One Great George Street in Westminster, London. We had the whole day at the venue, starting off with a civil ceremony followed by a Greek & Indian reception where our guests enjoyed a real mix of both our cultures. We had a wonderful day; it was filled with emotion and happiness, and was filled with our closest friends and family.

A bit of background to our story is that we met 12 years ago at Brunel University, where we both became friends as part of a bigger circle. Within our group of friends we’re known as the ‘Ross & Rachel’ as we both liked each other but never quite got it together properly until after university - and even then it was

on and off until almost 4 years ago when everything fell into place and we knew it was completely right for us to be together!

We live in Southgate and have loved doing up our flat together. I work as a management accountant for the restaurant industry while Nishel works in television research for the Discovery Channel.

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Congratulations to Gabriel & Nishel

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Congratulations to Congratulations to Gabriel & Nishel






























































































































































































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As usual, UKTS were present to raise awareness of thalassaemia at the annual Janmasthami Festival, held at Bhaktivedanta Manor, Watford, on 24th and 25th August 2008.

After a miserable, sodden August the weather finally decided to smile on us; making it a colourful and enjoyable occasion for the many thousands who flocked to the world famous Hindu temple to celebrate the birthday of Lord Krishna.

UKTS Coordinator Elaine Miller attended on the 24th; accompanied by Committee member Bharat Nathwani and his wife Nima. Members Sonoo and Prem Malkani also helped on the 24th; but the hardest worker by far was Preeti Dhanak-Steele (a UKTS member from Cambridge who is both a thalassaemia patient and a nurse), who bravely manned stall on both days (with help from her niece Priya). Many thanks to all our volunteers.

Janmasthami Festival

L to R Nima Nathwani, Sonoo Malkani, Bharat Nathwani, Prem Malkani, Preeti Dhanak-Steele

office news

Date:22 November 2008

Venue:Penbridge Banqueting Suite

470 Bowes Road, New Southgate, London N11 1NL

Come and help us raise awareness for our charity. Join in with our evening’s entertainment and let the

band ‘Millennium’ keep you dancing until midnight.

UKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSUKTSAnnual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual Annual

Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Thalassaemia Dinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & DanceDinner & Dance

Tickets priced at £45including drinks are available from:

UK Thalassaemia Society

19 The Broadway, Southgate, London N14 6PH

Organisers: Maria Gavriel and Photoulla Wilson.

Call them for further details

Phod: 07966 386375Maria: 07946 663485


UKTS Welcomes NEW-MEMBERS

AnnualMr Hussain Jassat

Mr James Miller

Mrs Brigid Offley-Shore

Ms Aneesa Patel

Ms Mital Patel

Mr Syed Rahman

Mr Yameen Rasul

Mrs Betty Why

LifeMr David Hayes

Ms Romaine Maharaj

Our most grateful thanks to all our donors for their generosity.

Mr S Gandhi £300.00

Mrs B Lysandrou £60.00

Mrs Niki Patapiou-Smith(in memory of her mother) £50.00

DONATIONS

The Editorial Committee reserves the right to alter any articles for publication where necessary and accept and reproduce or copy on good faith.

Neither the Editorial Committee or the Society accept any responsibility for any inaccuracies or omissions.

The views expressed are not necessarily that of the Society.

E- MSc in Haemoglobinopathies

University College London (UCL) in collaboration with Thalassaemia International Federation (TIF) is thrilled to introduce a unique online MSc course in Haemoglobinopathies, scheduled to start in September 2008. This is the first e-learning course in this field, and it offers to health professionals from anywhere in the world an affordable opportunity to pursue an advanced degree without interruption of family or work responsibilities.

The aim of this MSc course is to train health professionals in all aspects of the effective control – including prevention and holistic management – of haemoglobinopathies. For further details, please visit the UCL website at www.instituteforwomenshealth.ucl.ac.uk/Education/Haemoglobinopathies/index.htm

If you teach a

child who has

Thalassaemia – You need to read

this leaflet

office news

At UKTS we are well aware that many parents of thalassaemic children experience difficulty in explaining the condition to their child’s teachers or carers. We have therefore designed a new leaflet which gives all the necessary information in an easily accessible format so that parents can give these to the teachers at their child’s school. The leaflets are available to parents FREE of charge from the UKTS office – call us now on 020 8882 0011.

UKTS is grateful to the following for their assistance with this project

Isabel Adams, > Thalassaemia Nurse Specialist, Birmingham Children’s Hospital (BCH)

Munira Bharwani, > parent

Jane Carrington-Porter, > teacher, of James Brindley School based at BCH

Susan Crawford, > Specialist Nurse Haemoglobinopathy, Birmingham Sickle Cell/Thalassaemia Services

Dr Philip J Darbyshire, > Consultant Paediatric Haematologist, BCH

Pamela Hayes, > School Nurse, Swanshurst School, Birmingham

Nazam Rehman, > parent

Notice to Parents and Teachers

The UK Thalassaemia Society thanks Jeans for Genes for funding this project


To the Manager [Name of Your Bank]

Address

City Postcode

Please pay: Bank of Cyprus UK, PO Box 17484, 87 Chase Side, London N14 5WH

For the credit of: UK Thalassaemia Society, Registered Charity No: 275107 Sort Code 30-00-42 Account Number 00593812

The sum of : £2.00 £5.00 £10.00 Other £ _____________________ (amount)

On the ___________________ (day), ___________________ (month), ___________________ (year)

And thereafter every month until further notice and debit my account accordingly.

Name(s) of account holder(s) to be debited:

Account Number:

Sort Code:

Signed Date

Signed Date

Your Address

Tel Number:

Email address:

I would like tax to be reclaimed on my donation under the Gift Aid Scheme. I am a UK tax payer and pay an amount of income tax and/or capital gains tax at least equal to the tax that can be reclaimed on my donation. Please tick.

YES NO

Please call 020 8882 0011 if you have any queries. When completed, please return to:UK Thalassaemia Society, 19 The Broadway, Southgate Circus, London N14 6PH.

We will then send this form on to your bank.

Thank you for your valued support.

Please Support The UK Thalassaemia Society by Making a Monthly Donation

STANDING ORDER FORM

office news

www.ukts.org

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membership application form

UK Thalassaemia Society, 19 The Broadway, London N14 6PHCharity Reg No. 275107

Your Personal Details

ALL DETAILS AND INFORMATION WILL BE KEPT ON OUR COMPUTERS AND WILL REMAIN IN THE OFFICE AND WILL NOT BE MADE AVAILABLE TO ANYBODY OUTSIDE OF THE UKTS.

If you however do not wish your details kept on our computers please tick this box

OFFICE USE: Date Paid Receipt No. Approval Date

Title (Mr/Mrs/Miss/Ms/Other):

First Name(s):

Surname:

If you are a patient or parent of a patient please complete the section below

Patient’s Name(s):

Hospital where‑treated:

Address:

Date of Birth:

Consultant’s Name:

Consultant’s Telephone:

GP’s Name:

Address:

Telephone:

Transfusion Frequency:

Units received at each transfusion

Blood Type

Type of thalassaemia: (e.g. Major, Intermedia, Haemoglobin H etc)

Sex: Male Female

Address:

Contact Details

Telephone: Home:

Work:

Mobile:

Fax:

Email:

Are you a:

Patient Parent/Relative

Healthcare Professional Association

Other (Please state)

Membership Required (please tick)

ANNUAL (£10.00) LIFE (£100.00) (Please make your cheque payable to U.K.T. Society)

Blood Transfused (please tick)

Whole Washed Frozen Filtered

Chelation (please tick)

Desferal Deferiprone Desferal & Deferiprone

Post Code:

Occupation:

Ethnic Origin:(Optional)

ukts honours prof bernadette modell · • dr josh wright, consultant haematologist, ... prof...

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