ukts launches ground-breaking new publicationthe book has been designed to provide guidelines for...

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

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Medical News . . . . . . . . . . . . . . . . . . . . . 4

News from around the world . . . . . . . . 8

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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: office@ukts .org • www .ukts .org

14th June 2005 was one of the most important dates in the UKTS calendar for a long time. Our second national conference for doctors who treat thalassaemia patients (Thalassaemia in the 21st Century – A National Standard of Care, King’s Fund, Cavendish Square London W1) took place;

an event specifically designed to coincide with the release of our new Standards for the Clinical Care of Children and Adults with Thalassaemia in the UK.

This publication was commissioned in 2002 after our first national doctors’ conference; when it became apparent that many doctors felt, as we did, that there were significant variations in the quality of treatment available to thalassaemics in the UK. This document provides comprehensive clinical guidelines and performance indicators for the monitoring of treatments available and outcomes. It is the first set of clinical guidelines to be published in the UK relating to a condition which does not affect the indigenous population. The document has been welcomed by the Department of Health and described as “excellent and timely” by the Royal College of Pathologists. Although the book has been designed to provide guidelines for doctors, we also hope that it will be a means of showing thalassaemia patients what standard of care they should be receiving and encouraging them to seek it. For this reason the book will be available free to thalassaemia patients and parents

on application to the UKTS office; as well as being distributed free to healthcare professionals throughout the UK. Contact us today if you would like a copy.

This project, which took three years to complete, has taken up many hours, days and weeks of work from all involved. Everyone in the Writing Group has worked extremely hard and we are very grateful to all the doctors who gave up their time to help us. Particular thanks are owed to Dr Anne Yardumian, Dr Paul Telfer and Mr Matthew Darlison, who gave up many of their all too rare leisure hours to ensure that the document was finished on time. Our grateful thanks to all the Writing Group, listed below.

• Dr Anne Yardumian, Consultant Haematologist, North Middlesex University Hospital (Chair of Writing Group)

• Dr Paul Telfer, Senior Lecturer in Haematology, St Bartholomew’s & The Royal London Hospitals NHS Trust, Queen Mary, University of London

UKTS launches ground-breaking new publication

– Standards for the Clinical Care of Children and Adults with Thalassaemia in the UK

July 2005 ISSUE NUMBER 102

Continues on page 3 ➡

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

Mike Michael President

Menuccia Tassone Vice-President

Costas Kountourou Secretary

Olga Demetriou A Secretary

George Constantinou Treasurer

Philip Agathangelou A Treasurer

Maria Gavriel Committee

Chris Sotirelis Committee

Andreas Yiannikou Committee

Dear Friends,

This issue of TM introduces a brand new UKTS publication – the first Standards for the Clinical Care of Children and Adults with Thalassaemia in the UK. This book has been one of our “invisible” UKTS projects for the last three years. Of course, we could never have produced a document like this without the help of the writing group, chaired by Dr Anne Yardumian, Consultant Haematologist at the North Middlesex Hospital. Thank you again to everyone who gave up their time to help us with this project.

At UKTS we are in contact with patients from all areas of the UK, some of them living very far from the nearest hospital which has a thalassaemia specialist. Obviously those centres which treat a large number of thals have excellent services and their staff have developed a high level of expertise, but this cannot be the case in a hospital where they only see one or two thals. The Standards can be used by any hospital to provide clear guidelines for the management of

thalassaemia and its complications. By making the document available to patients as well as healthcare professionals we hope to show them what level of service they are entitled to expect. If you would like a copy, you simply need to call or email our office.

The book was launched at our second national conference for doctors involved in the treatment of thalassaemia on 14th June 2005 at King’s Fund in Cavendish Square, Central London. We were privileged to welcome over 120 guests, some of whom had literally travelled from the other side of the world to attend the conference. It was an inspiring day and a pleasure to meet so many people dedicated to improving the treatment available to thalassaemics.

This has been a very busy time for UKTS, with the final stages of producing the Standards, organising the conference and also managing to send delegations as far away as Taiwan and Malaysia, to collect information on behalf of the Thalassaemia International Federation. You will be able to read about these visits in this issue and the next.

May I wish everyone a happy and restful summer and to those of you who will be travelling, a safe and pleasant journey.

Until the next issue,

Mike MichaelPresident, UK Thalassaemia Society

A word from our President

the UKtsManagementcommittee

Aims & Objectives

of UKTS

■ The relief of persons suffering from thalassaemia.

■ The promotion and co-ordination of research in connection with thalassaemia.

■ To educate people on the problems of thalassaemia.

■ To offer counselling to sufferers and carriers.

■ To bring together patients, their families and well-wishers to exchange ideas and information.

■ To raise by any legal means the funds required for the above activities.

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

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• Mr George Constantinou, patient representative, UK Thalassaemia Society

• Dr Phil Darbyshire, Consultant Paediatric Haematologist, Birmingham Children’s Hospital

• Mr Matthew Darlison, Senior Research Fellow Clinical & Applied Bioinformatics, University College London Centre for Health Informatics & Multiprofessional Education (CHIME)

• Dr Sally E Kinsey, Consultant Paediatric Haematologist, St James’s University Hospital, Leeds

• Mrs Elaine Miller, Co-ordinator, UK Thalassaemia Society

• Professor Bernadette Modell, Emeritus Professor of Community Genetics, University College Hospital London & UCL Centre for Health Informatics & Multiprofessional Education (CHIME)

• Dr Melanie Pollitzer, Associate Specialist in Paediatrics, Royal Berkshire Hospital, Reading

• Professor John B Porter, Professor of Haematology, University College Hospital, London

• Sr Emma Prescott, Thalassaemia Nurse Specialist, Whittington Hospital, London

• Mrs Katerina Read, patient representative, UK Thalassaemia Society

• Dr Christos Sotirelis, patient representative, UK Thalassaemia Society

• Dr Rachel Spector, Clinical Psychologist, Birmingham & Solihull Mental Health Trust

• Dr Christine Wright, Consultant Haematologist, Sickle Cell & Thalassaemia Centre, City Hospital, Birmingham

Continues from page 1

Thalassaemia In The 21st Century– A National Standard Of Care

Our second national conference for doctors who treat thalassaemia patients took place on 14th June 2005 at King’s Fund, Cavendish Square, London W1.

The conference generated enormous interest from the outset. Registrations came in from all parts of the UK and some

from much further away, with visitors from China and Taiwan among the delegates. Long before 14th June there were no further places to be had; and on the day there was not a spare seat in the hall.

The opening ceremony was conducted by our President Mike Michael and the Rt Reverend Dr John Sentamu, Bishop for the Diocese of Birmingham and Chair of the NHS Screening Programme for Sickle Cell & Thalassaemia (we are delighted to report that since the conference, Bishop Sentamu has been appointed Archbishop of York, so taking up the second highest office in the Church of England).

Professor Sir David Weatherall was to have presented a global view of thalassaemia in the 21st century, but sadly was unable to be with us due to illness. Dr Kate Ryan of Manchester Royal Infirmary, Co-Chair of the conference organising committee, instead opened the conference with an overview of thalassaemia treatment in the UK. Then followed the keynote address by Dr Anne Yardumian of the North Middlesex Hospital, which presented the new Standards for the Clinical Care of Children and Adults with Thalassaemia in the UK. Copies of the new document were included in the conference packs distributed to the delegates. Further presentations followed on:

• Management of bone disease – Dr Ersi Voskaridou, Laikon Hospital Athens

• Sexual development & fertility – Professor Pierre-Marc Bouloux, Royal Free Hospital London

• Pre-implantation diagnosis in the UK – Dr Mary Petrou, Regional Haemaglobinopathy Genetic Centre (Perinatal Centre) UCLH

• Update on the National Screening Programme – Dr Allison Streetly, Programme Director

• Bone marrow transplantation – Dr Phil Darbyshire, Birmingham Children’s Hospital

• The patient’s perspective – Professor Bernadette Modell, CHIME

• Goals & monitoring in chelation therapy – Professor Renzo Galanello, University Hospital, Cagliari, Sardinia

Delegates at the conference

Dr Christos Sotirelis of UKTS and Dr Kate Ryan of Manchester Royal Infirmary, co-chairs of the conference organising committee.

The Rt Rev Dr John Sentamu, Bishop for the Diocese of Birmingham, opens the conference.



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• Optimal use of desferrioxamine – Dr Bernard Davis, Whittington Hospital London

• Deferiprone alone or in combination – Dr Paul Telfer, The Royal London Hospital

• T2*MR of myocardial iron & chelator efficacy – Professor Dudley Pennell, Royal Brompton Hospital London

• ICL670 & other new chelators – Professor John Porter, University College Hospital London

N.B. If anyone would like copies of the slides/abstracts used in the presenta-tions, please contact the UKTS office.

The evaluation forms given in by the doctors at the end of the conference were vastly encouraging and made us feel that the hard work which went into organising the conference was very worthwhile. 75% of respondents found the meeting highly relevant to their educational needs, with the remaining 25% finding it mostly relevant. 64% rated the quality of education offered as excellent and the remainder rated it as good. Even more

significantly, a staggering 93% those who submitted an evaluation stated that after attending the conference they would modify their practice; 39% saying that their practice would be modified in a major way.

Thank you to all those who gave up their time to give presentations and/or chair sessions; and helped us to make the conference a success. Particular thanks go to the conference organising committee – Dr Kate Ryan, Dr Christos Sotirelis, Prof Pierre-Marc Bouloux, Mr George Constantinou, Mrs Elaine Miller, Prof John Porter, Dr Paul Telfer, Dr Adrian Williams.

On the evening of 14th June UKTS hosted a celebration dinner at Lemonia restaurant in Central London to thank those who contributed to the Standards book and the conference.

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Summary of a presentation given by Dr Michael Antoniou at the UKTS AGM, 10th April 2005As I’m sure most of us know, at the moment a bone marrow transplant from a tissue matched donor (e.g. a brother

or sister) offers the only hope for a cure of a haemoglobinopathy (thalassaemia or sickle cell disease). However, within the last 5 years and especially the last 24 months, there have been some major developments, which suggest that gene therapy for thalassaemia or sickle cell disease may at last be an option in the not too distant future.

How is gene therapy for the haemoglobinopathies envisaged to take place?All of our different types of blood cells

Gene Therapy for the Haemoglobinopathies:

Current Status and Future Trends

Dr Anne Yardumian, Chair of the Writing Group, presents the new “Standards for the Clinical Care of Thalassaemia”



latest news medical news

including the red blood cells, which are faulty in thalassaemia or sickle cell disease, are produced from what are known as “stem cells” that are present within our bone marrow. So the idea of how thalassaemia or sickle cell disease can be cured by gene therapy is quite simple; if you correct the genetic defect in the bone marrow stem cells of the person with either thalassaemia or sickle cell disease, then they will now produce normal rather than defective red blood cells for the rest of their life! Technically, gene therapy for β-thalassaemia or sickle cell disease will take place by what is known as an “ex vivo” procedure, which is basically a three-step process:Step 1: Bone marrow is removed from the person with β-thalassaemia or sickle cell disease and the stem cells within the bone marrow are isolated or purified.Step 2: The therapeutic gene unit consisting of a normal functioning copy of the β-globin gene is then introduced into these isolated bone marrow stem cells. During the few days that this takes place, the person being treated undergoes a mild form of chemotherapy to partially destroy their diseased bone marrow, a procedure known as “conditioning”.Step 3: The now corrected bone marrow stem cells are returned to the person from where they came from. Since the chemotherapeutic conditioning has largely destroyed the diseased bone marrow, the gene therapy corrected bone marrow stem cells are able to readily establish themselves within the vacated bone marrow spaces. As their bone marrow stem cells now contain a normal functioning copy of the β-globin gene, they will produce normal red blood cells rather than those deficient in β-globin and abnormal haemoglobin as before they were treated.

Key features to note about this procedure are (i), you are correcting the person’s own bone marrow stem cells and therefore you do not need a donor as in the case of a bone marrow transplant and (ii), you are not correcting the faulty β-globin genes that the person with either β-thalassaemia or sickle cell disease has inherited; you are simply adding or introducing a normal functioning extra copy of the β-globin gene into their bone marrow stem cells. As a result any person

with either β-thalassaemia or sickle cell disease is treatable by this ex vivo gene therapy procedure, as long as their bone marrow is in good shape!

The β-globin therapeutic gene unitWe have known for some time the components that would need to be included to make an efficient proper functioning, therapeutic β-globin gene unit. Firstly, we of course need a normal copy of the β-globin gene, which carries the information for β-globin protein, which in turn is part of normal haemoglobin. Secondly, we need what are known as genetic control or regulatory elements. These are basically elements that will allow the β-globin gene to be powerfully “switched-on” in the red blood cells to produce enough β-globin protein to be of therapeutic value. The β-globin gene genetic control element or “on-switch” is known as the “locus control region” or “LCR”. We know from research conducted a number of years ago that a LCR-β-globin gene combination delivered to bone marrow stem cells will produce the minimum 25% of normal levels of β-globin protein that is sufficient to make someone with β-thalassaemia independent of having to have blood transfusions.

Advances in the delivery of the β-globin therapeutic gene unitIt is perhaps obvious from what we have discussed so far that we have known how to bring about a cure for β-thalassaemia or sickle cell disease by gene therapy for some time. The major obstacle, which has prevented us from realising this goal, has been the lack of a gene delivery or “vector” system that could efficiently introduce the therapeutic LCR-β-globin gene combination into bone marrow stem cells. However, in the year 2000 Professor Michele Sadelain in New York (USA) finally achieved a major breakthrough in this area. Professor Sadelain adapted a vector based on the human immunodeficiency virus (HIV), which under normal circumstances causes AIDS. Perhaps ironically, one can convert this otherwise dangerous virus into a potential lifesaver by removing all the virus disease-causing genes and replacing them with your therapeutic gene unit, which in our case is the LCR-β-globin

gene combination. Professor Sadelain showed for the first time that an HIV-LCR-β-globin gene vector could be used to efficiently infect bone marrow stem cells and completely cure the disease condition in a mouse model of severe β-thalassaemia intermedia. Within the last 12 months groups in North America led by Professors Punam Malik, Connie J. Eaves and Keith Humphries have published reports where they describe using similar HIV-LCR-β-globin gene vectors to completely correct the genetic defect in human bone marrow stem cells obtained from people with β-thalassaemia major. These groups showed that when these corrected human stem cells are introduced into mice, these animals survive and live very well with this human bone marrow transplant, which further demonstrates the therapeutic potential of this procedure if repeated in human subjects. In addition and not too be forgotten, HIV-LCR-β-globin gene vectors have also been shown by the group of Professor Tim Townes in the USA to be effective at curing sickle cell disease again in a mouse model experimental system.

Finally, based on work sponsored by UKTS in my laboratory a few years ago, we have built and tested our own version of the HIV-LCR-β-globin gene vector. In collaboration with Professor Giuliana Ferrari of the Telethon funded gene therapy institute in Milan (Italy), we have shown that our HIV-LCR-β-globin gene vector is just as effective as other versions at curing the severe β-thalassaemia intermedia mice. Experiments with human bone marrow stem cells from people with β-thalassaemia major are currently underway. However, importantly our data to date suggests that the much simpler (and smaller) design of our HIV-LCR-β-globin gene vector gives it two very significant advantages over those of other groups. Firstly, it is much easier to produce, which is not an insignificant consideration when you are contemplating scaling up manufacture of the vector for use in human clinical trials. Secondly, the smaller size of our vector means there is more space remaining within the virus to allow us to add other genetic components that would improve the efficacy of our vector to the point where we thought it

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would be feasible to go into the clinic to treat people.

How does the HIV-LCR-β-globin gene vector work and are there any associated problems?The HIV-LCR-β-globin gene vector has the property of inserting its genetic material (the genes that it is carrying) into the DNA of the bone marrow stem cells that it will be used to infect. Consequently, as these bone marrow stem cells divide and grow they retain their copy of the HIV-LCR-β-globin gene vector since this is now part of their genetic makeup. This is why the genetic correction of bone marrow stem cells of people with β-thalassaemia or sickle cell disease with the HIV-LCR-β-globin gene vector, in principle will be a life-long cure.

However, there is one major problem that arises from this otherwise very beneficial process. At present we have no control over where within the DNA of the bone marrow stem cells the HIV-LCR-β-globin gene vector will insert itself. As a result it is quite possible that a host stem cell gene or genes will be disrupted in their function (turned up, down, on or off) by the HIV-LCR-β-globin gene vector inserting somewhere nearby. The fear is that this in turn could lead to a loss in normal cell growth processes giving rise to a cancer type condition. We now know that this is not just a hypothetical possibility.

Since about 1999, gene therapy clinical trials have been conducted in Paris and London to treat boys with a condition known as severe combined immune deficiency-X or SCID-X. The same ex vivo gene therapy procedure we described above for the haemoglobinopathies was used to treat these boys with SCID-X; that is, bone marrow stem cells were isolated from the person being treated; these bone marrow stem cells were then genetically corrected by introducing into them a normal functioning copy of the therapeutic gene, in this case known as gamma-C (γC), using a virus vector similar to HIV known as an MuLV retrovirus, which as in the case of HIV permanently inserts its genetic material or cargo into the stem cell DNA; the corrected stem cells were then returned to the person being treated from where they originally came from. Normally

boys with SCID-X, which totally lack an immune system, live only to about the age of 2 years, succumbing eventually to an infection that you or I would have no trouble fighting off. However, with gene therapy the immune system of these boys is gradually restored and they can begin to live a normal life, with the first two boys treated in this way now 6 years old and doing fine!

Although overall these trails have been a resounding success with the gene therapy being effective in all cases, they have unfortunately also highlighted potential dangers. Out of a total of 15 boys treated, 3 have now come down with a leukaemia-type condition. The main cause of this seems to be that the MuLV retrovirus carrying the therapeutic γC gene has inadvertently switched-on a cell growth regulating gene (called LMO-2) by inserting itself nearby. As a result a single class of immune system white blood cell started to grow in an uncontrolled manner very similar to a leukaemia. We are still not sure how general this problem is going to be. At the very least this phenomenon seems to be a hit or miss affair depending on where the therapeutic virus inserts itself in the DNA of the bone marrow stem cells. The field of gene therapy has nevertheless reacted very strongly and much research effort is currently being expended to address and find solutions to the safety concerns caused by the insertion of therapeutic virus vectors into target cell DNA.

Future prospectsOthers may differ in their view but although the work conducted over the last 5 years has been very encouraging, I personally believe that certain important issues need to be resolved before we embark on gene therapy clinical trials for β-thalassaemia and sickle cell disease. Firstly, there is a major pragmatic consideration; we need to develop technology to efficiently produce large amounts the HIV-LCR-β-globin vector. Secondly, we need to still improve the efficiency with which the LCR-β-globin therapy gene unit switches-on once delivered to the bone marrow stem cells; at the moment there is still too much variation between one stem cell and the next. Thirdly, we need to try and “insulate” the HIV-LCR-β-globin vector

once it has inserted itself into the stem cell DNA. This will do two things. It will minimise the possibility of the HIV-LCR-β-globin vector inadvertently switching on or off a host cell gene that could lead to problems such as the leukaemia found in a few of the cases in the SCID-X trials, and it will help to allow the HIV-LCR-β-globin vector to function more efficiently. We and others are in the process of testing HIV-LCR-β-globin vectors with additional genetic elements that possess this “insulation” effect.

Finally, overall, I feel we need to be near 100% sure that the gene therapy for β-thalassaemia and sickle cell disease will work with minimal risks. Why do I say this? In the case of say the boys with SCID-X, the only hope for life is a bone marrow transplant if you are lucky to have a matched donor. So for most SCID-X boys, gene therapy holds the only hope of maintaining life and as a result it is generally thought that potentially higher risks are acceptable in such cases since the alternative is a very short and poor quality life. However, this is certainly not the situation with β-thalassaemia and sickle cell disease, where there are reasonable albeit rather painful and inconvenient treatments available that can keep you going in pretty good shape for decades! Because of the availability of these non-genetic but effective maintenance treatments for β-thalassaemia and sickle cell disease, I feel a much lower level of risk of failure and/or side effects such as leukaemia are acceptable compared to something like SCID-X.

Despite all this we have without a doubt reached a very exciting time in the development of gene therapy for β-thalassaemia and sickle cell disease. After many frustrating years, I would say that the advances in the HIV-LCR-β-globin vector system has at long last given us a clear direction to go in with a very good chance of success. The next few years of research will I think be crucial as we can in all likelihood expect to see a continuing fast rate of progress towards our first clinical trials.



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Two members of the UKTS Committee, George Constantinou and Chris Sotirelis, were asked by TIF to attend this camp as TIF representatives as well as a way to keep up with the developments on the care and treatment of thalassaemia in Malaysia. This year’s Thalassaemia Camp took place on the island of Langkawi off the northern coast of Malaysia. It is a 3-day event, organized regularly in a suitable resort of Malaysia where patients from all of the states of Malaysia are invited to attend together with nurses, carers and some doctors. This idea originated from the members of the Pulau Penang Thalassaemia Society (Penang T.S.) and has been running every year since the mid nineties. The idea behind this is to give the patients time together to have a good time, enjoy the company of others with a common problem and at the same time to learn about thalassaemia and to discuss ways of solving everyday problems they face, living with thalassaemia. Adolescents and young adults who can travel independently and unaccompanied are particularly targeted. Parents are not encouraged to attend unless they are accompanying a young child. The cost of travel, registration, accommodation and catering is entirely borne by the Penang T.S, but only for the Penang members. Members of other associations have to pay themselves or their associations on their behalf.

A number of presentations were given to the patients, parents, doctors and nurses in the audience over the 3-day period. These included :

On day 1, Dr. See Ching Mey of the School of Educational Studies, University of Sains Malaysia, Pulau Penang, gave a presentation entitled ‘Positive Living’ on the psychological aspects of life with thalassaemia. She concentrated on explaining areas such as psychological burden, the subconscious effects on present behaviour, commitment making, control, challenges and tools for positive thinking. This was followed by an hour-long break out session / group therapy workshop where patients tried to find their current levels of positive or negative state of thinking. There was also a discussion amongst each group as to what are the main obstacles patients face daily making increasing their mental misery.

On day 2 Dr. Lim Shueh Lin’s (Consultant Endocrinologist, Hospital Pulau Penang) presentation entitled ‘Fertility and Reproduction’ gave an overview of the main areas of growth, development, fertility and bone disease treatments for patients in adolescence as well as for adults. She continued further in ‘The Endocrine Issues – Growth and Development’, an overview of how the endocrine system works and is affected by thalassaemia and the lack of iron chelation. The afternoon session contained presentations by George Constantinou of the UKTS, with title ‘Past Present, Future - My Life’ where he examined ways of living with thalassaemia and the issues it generates, using a different point of view. This was followed by The Psychological Impact of Thalassaemia – A patient’s perspective’ given by Chris Sotirelis of the UKTS. This presentation aimed to provide an understanding of the main drivers behind life with thalassaemia, how they interact and affect the patients and their families’ lives, their growing up and personal development.

The final day involved presentations by Dr. Goh Ai Sim (Consultant Haematologist, Hospital Pulau Penang) on how to ensure patients understand the issues and feel motivated to follow the proper treatment

protocols, and followed by a look at career and employment programs, dealing with the challenges of seeking, finding and getting a job in Malaysia by a state Employment & Careers advisor.

It closed with an interactive session with the audience where George and Chris developed some of the issues that were concerning patients during the visit and individual discussions on areas such as social acceptance, relationships, and marriage, having children etc.

The Camp was a great success, as confirmed by the feedback received by the organizers. These camps are a wonderful idea as they offer an opportunity for patients to take a break away from their everyday routine and meet other persons fighting against the same kind of problems they are fighting for. It also gives them the chance to exchange experiences and ways of dealing with their condition based on a better understanding. During our stay we were treated with the wonderful traditional Malaysian hospitality and had a chance to meet and make friends with a lot of people. During the evening of the second day there was a gala dinner organized where a group of patients created an impromptu play, which they performed acting with songs for all present. It was truly amazing to see their great creativity.

The Penang Thalassaemia Society must be congratulated for the excellent work they have done in providing these camps for the patients. Over the last 10 years since the previous visit they have been instrumental in maintaining the difficult tasks of giving hope and motivation to their patients to keep them wanting to improve their treatment and quality of life. Their dedication and energy in the achievement of these goals have been amazing and truly creative, under very difficult conditions.

We are truly grateful to all we met, and those who became our friends. We hope that we will be able to meet again either in Malaysia or in a future conference.

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7th Thalassaemia Camp, Malaysia:30 April – 2 May 2005

by Chris Sotirelis

UKTS’s George Constantinou (standing, 2nd left) pictured here with some of our Malaysian friends.


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Mrs Zanib RasulMany of our members in the North of England will be familiar with Mrs Zanib Rasul, the founder of NEBATA (North of England Bone Marrow & Thalassaemia Association). We are grateful to Mrs Rasul for sharing her memories and experiences as a mother of two children with thalassaemia; and of how NEBATA was established.

Mrs Rasul, could you please tell us something about your background – where/when you were born, when and why you first came to the UK, anything else about your family you would like to share with our readers.

I was born in the Pakistani district of Jehlum on 24th November 1954. I was the fifth child in my family and went to school up to the end of primary school, after which I went into Islamic studies at Havelian Mahboobabad in the district of Abotabad. Between 1971 and 1976 I began teaching Arabic and Urdu as well as Religious Studies to girls aged 8 to 14. I came to the UK when I got married to my husband of 28 years, in June 1977. The following year we had our

first of four children, and I again took up teaching, while enrolling in college to study English and maths as well as gaining a qualification for teaching. Since then I have achieved several qualifications including, The City Guild Diploma in Community Care, Counselling level 1, Child Care and Introductory Course in Management. My husband grew up in the UK and graduated from the University of Salford with a BSc. [hons] in 1980. We now run our own post office business together.

My oldest daughter, Zurqa, now 27, qualified as a medical doctor in 2004 and is working as a house officer. Yameen, 23, my only son and a thalassaemic, will be graduating from UMIST this summer, with a first class master’s degree in Computer Systems Engineering. I am very much looking forward to celebrating his graduation. My third child, Sajida, 20, was born with Thalassaemia but was cured with a bone marrow transplant from her eldest sister. She has just finished her second year as a medical student at the University of Manchester. My youngest daughter, Majida, 17, is currently doing her A-levels at Manchester High School for Girls. I am very proud of my children and when I count my blessings from Allah I count them twice.

Would you like to share any of those memories of being the parent of a newly diagnosed thalassaemic baby with our readers and other parents? And how did you manage as the mother of a family with 2 young thal children?

When Yameen was born he appeared to be a normal, healthy baby, but after a while he became pale in colour, couldn’t sleep, cried all the time and was referred by our GP to the hospital. After his first transfusion he was visibly much better and we were given another appointment for second blood transfusion and although

I had no idea what was going on I was happy that Yameen was feeling better. After about four transfusions it was suggested that Yameen might have Beta Thalassaemia Major. This was the first time had ever heard of the disorder. I was told nothing excepting that the only cure was a bone marrow transplant and that this was more successful if the donor were a sibling. We had ourselves and our oldest daughter tested, with no perfect matches. We hoped that perhaps our next child, Sajida, would be a compatible bone marrow donor. Unfortunately, she too was diagnosed with Beta Thalassaemia Major. We were distraught to hear that Sajida would also need transfusions like Yameen and at the time I couldn’t accept that she too was ill. At this difficult time we were offered no counselling or any other help to make the situation easier for us. There was no written information available as it is nowadays. It was when Sajida started having transfusions that I began to find out what the future held for my children. One particular day stands out in my memory. When I was in hospital I tried to ask the nurse when my daughter was likely to get a bone marrow transplant. I was bounced between doctors and nurses, and on this day I became so fed up and so frustrated that I cried to the point that I almost fainted. Then Sajida was referred to Royal Manchester Children’s Hospital for her bone marrow transplant, as her tissue type luckily matched with her eldest sister, Zurqa. Everyday I saw my children being pricked five or six times, to be canulated, for blood transfusions. After Sajida had her bone marrow transplant, it was a little easier to deal with, firstly because Yameen was able to face his treatment with a great deal of bravery, as he still does to this day, and secondly because there was renewed hope for Yameen.


www.ukts.org10 Thalassaemia Matters ...continuing the fight against Thalassaemia

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What gave you the strength to keep going?

Love for my children, my faith and last but certainly not least the support of my husband always kept me going.

Can you tell us briefly about your daughter’s BMT?

Sajida spent three months in hospital isolation and three months in home isolation (sterile diet, her own room etc), following strict instruction from the nurses, who were more helpful and much kinder than what we had experienced before and I respected them for that. Dr. Evans, the consultant haematologist and his team performed the transplant and we are forever grateful. It was with their help and professionalism that we realized that the relationship between patient, their families and the medical teams was very important, and should be built on, as it is a long term connection.

What inspired you to start your work in support of thalassaemia and to ultimately start NEBATA? Could you please tell us something about the time when you were setting up NEBATA.

In 1988, the year of Sajida’s bone marrow transplant, the doctors introduced us to some other families who were facing the same struggle of finding a

donor for a transplant, as we had done for both Sajida and Yameen. Doctors and social workers, who had dealt with us, suggested that we set up a local group to give moral (and if necessary financial) support, as well as supporting research in to bone marrow transplantation. We followed this advice and so the Blackburn and District Bone Marrow Trust Fund was born. We registered as a support group for Thalassaemic patients and their families in 1989, with fewer than ten members, including myself and my husband, Mr. and Mrs. Patel and our chairman, the late Jamal Bhatti. We started holding meetings in our living rooms and sometimes in the community centre. As the Blackburn and District Trust Fund, we slowly raised £6000 for bone marrow research at the Royal Manchester Children’s Hospital

The establishment of our Association was possible with the kind help of health care professionals at the time, to bring families and carers together. Over the past seventeen years, we have worked hard to support thalassaemic patients and their families, through counselling, distribution of written information as well as attendance at UKTS conferences. I still enjoy working closely with everybody who is families and carers together. I still enjoy working closely with everybody who is involved with Thalassaemia.

Tell us something about your

international travels in pursuit of your charity work.

I have attended many international TIF conferences in different countries, where I have gained a wealth of knowledge and met with many families from developing and developed countries. I have seen how they cope with out the facilities we are lucky to enjoy in England

What do you miss most about your native land? How often are you able to visit the land of your birth and do you have family there? Are they able to visit you in the UK?

I miss my family: brothers and sisters and nieces and nephews, for I have only my own children and my husband here in England I miss the natural lifestyle, the food and my childhood friends. I particularly miss my class that I taught. I am able to visit my family in Pakistan every two to three years, because I am very involved with the lives of my own children and want to spend as much time with them as possible. Unfortunately nobody in my family has been to England except my late father, who came to visit in 1987, but he didn’t like the cold weather.

Is there any particular person you admire?

The person I most admire is my son.

What is your favourite hope for the future and what is your greatest joy in life?

The hope I cherish for the future is that everything the association has come to stand for is fulfilled and nobody feels as lost and alone as we did in the early days, and to have a nice big house. My greatest joy in life is looking after my children and seeing them happy and flourishing in their life.

If any of our readers would like to know more about NEBATA, their contact details are:

North of England Bone Marrow & Thalassaemia Association352 Oxford Road, Manchester M13 9NLTel: 0161 273 7200 / 0161 745 7671Website: www.cmmc.nhs.uk/nebata


Congratulations toMumta &

ShaneWhen was the last time you saw such happy smiles? This exotic picture shows UKTS member (and thalassaemia patient) Mumta Dutt on her wedding day. Mumta and her husband Shane Walters were married on 3rd September 2004 on the beautiful island of Antigua. Mumta, who works as a secretary at British Airways, certainly knows how to choose a romantic destination! Congratulations and best wishes from all at UKTS.

www.ukts.org Thalassaemia Matters ...continuing the fight against Thalassaemia 11

My name is Rachael Walker, I am 24 years old and have sideroblastic anaemia. I have intravenous desferal 24 hours a day, 6 days a week via my Portacath; and Ferriprox tablets 3 tim1es a day. If I do not effectively manage this treatment I will suffer the consequences of iron overload, in exactly the same way as thalassaemia patients.

I am writing this personal experience because I was non-compliant with my treatment for about 3 years. It is hard for me to publicly admit this, but as I go on to explain how I got through this

difficult time I hope you will agree that I have managed to overcome it. This is my final step on the road to recovery and permanent compliance. I also hope that if you or someone you know can relate to any of the experiences, thoughts and feelings I had, it will help by giving you the confidence to realise that there is a way out.

Until 3 years ago I was a model patient. I was 99% compliant with my treatment. A number of lifestyle changes then began to interfere with my treatment regime. I moved out of my parents’ home, where I had the stability, support and routine of my treatment down to a fine art. I left

university and started working full-time as a nurse, I moved in with my boyfriend Mark and later we got engaged. While all this was happening within a short period of time, my compliance began to slowly decrease. It happened so slowly I didn’t actually want to believe it was true. I remember thinking about my illness constantly. I would dwell on my misfortunes and trick myself into thinking that I would do my treatment tomorrow. One minute I would feel angry and depressed and the next I would be consumed with guilt. It was as if, because

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A Patient’s Struggle with Adherence to Chelation Therapy


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all the other things in my life were falling into place and I genuinely felt happy, there wasn’t enough of me left to do the treatment.

Eventually I stopped doing my treatment altogether and my promises of tomorrow and next week faded until I didn’t feel as if I cared. I lied about my treatment, I didn’t want to know my ferritin levels and it wasn’t very often it was checked without my prompting and I got caught out. I didn’t feel supported and cared for by my family, friends and hospital staff. I felt like a burden to everyone and because of this I felt intense guilt and embarrassment. I was ashamed of my actions and it became such a problem that I didn’t know or even care how to get out of the situation. I felt like a failure because I could not will myself into taking my medications. I believed that people thought I wanted to die and that it was ll my own fault – nobody could do it for me. I started to doubt my lust for survival, to think that deep down, I really did want to die.

Last summer, my consultant decided that it was time to intervene and I was secretly glad. I started attending the hospital overnight for 3 nights and later 5 nights a week to have the desferal done for me. I did this for 5 months. My treatment was taken out of my hands and I was referred to psychological health for an assessment to see if there were any treatments that could help. I started seeing Maggie, a cognitive behavioural therapist, once or twice a week. Cognitive behavioural therapy (CBT) has helped me to examine my thoughts, feelings and behaviours and make the necessary changes. There have been various techniques that have helped my recovery.

• Thought records – this is a process of examining a situation by looking closely at the emotions felt during that situation. Writing down thoughts and identifying the key thought (the thought which causes the most intense emotions - called a “hot thought”). The next stage is to examine the arguments for and against that thought to see if what is experienced is a true reflection or has been distorted. The final stage involves finding a balanced alternative thought, weighing up the arguments

for and against in order to understand what it was that determined how the situation occurred and progressed. It also enables the changing of thinking and behaviours.

• Behavioural changes – if I did not believe the alternative balanced thought, I then had to think of ways to prove or disprove the new thought. Usually this involved using experiments. One such occasion was when I believed that complying with my treatment should be easy. I believed that others thought this and would find it easy to carry out. Maggie and I carried out a survey to see if what I believed was true. By the end of the experiment, my thoughts had changed and I realised that complying with my treatment is hard and would be hard for others.

• We identified high-risk situations so that I could find ways to cope.

• I started a treatment chart to record my progress daily. This was kept on my fridge and it was to help me see my successes rather than focussing on the times I didn’t comply with my treatment.

• We designed flash cards to remind me of why I needed to comply with my treatment. These included consequences of non-compliance and my desire to comply and survive. It really helped to be able to read my incentive cards during times of loss of direction and motivation.

• My biggest problem was thinking that I had to cope with the treatment by myself, that it was my problem to deal with. I found it impossible to ask for help, I couldn’t discuss my feelings and difficulties with anyone because I was embarrassed and ashamed. For me, admitting difficulty was to fail. By practising asking for help I began to realise how I had projected my feelings into what I believed others felt about me and that these thoughts were false.

• Acknowledging that the treatment is hard and that others would also find it

difficult to cope with.

• Allowing myself to feel angry and upset, but to consider the positive aspects as well.

• Realising that I can’t always be perfect, I have experienced a blip and it may happen again. Making sure that I do not set my standards so high I am guaranteed to fail.

• Having Maggie to tell me that it is OK to find it hard and for me to start believing her has been a real turning point.

The most helpful aspect of CBT has been talking through my problems and anxieties with Maggie. Realising that I had the answers to my questions, I just needed help finding them. Going home and testing methods and finding that they work has given me the confidence that I can do my treatment again. I found the thought records very difficult at first. I sometimes felt uncomfortable examining my thoughts and feelings so closely, admitting things I had kept hidden for a very long time to someone I hardly knew. Thought records are very labour-intensive and it was hard not to want a “quick fix” solution. My thoughts and behaviours changed so slowly that sometimes it felt that I hadn’t actually made any progress. Looking back, I can start to see how small changes in thinking and behaviour have vast results.

Finally, I would just like to praise Maggie, my consultant and my haematology specialist nurse. They never once got angry with me. They listened to me cry and tried to do everything to help. They didn’t give up on me, even when I believed I was not worth helping. They always listened and encouraged, but never judged.

Thank you Rachael, for sharing your experiences with such openness and honesty. We wish you all the very best.

www.ukts.org Thalassaemia Matters ...continuing the fight against Thalassaemia 1�

Life Assurance and Thalassaemia– some questions answered

Life assurance involves paying a premium (usually monthly or annually) to ensure that a cash sum is paid in the event of the death of the insured individual. Most policies of this kind are taken out to provide for dependants, for example, a husband who is the family’s main wage earner may wish to provide for his wife and children. Life assurance policies may also be taken out for a specific purpose, such as to pay off a mortgage in the event of a death. There are many different kinds of policies on the market. Here are a few examples –

Term assurance – this is a life insurance policy which covers the life of the insured person for a cash sum (the sum assured), in return for a payment (usually monthly but possibly annually) known as a premium. This is the cheapest form of life cover. Assurance is provided for the term of the policy only, i.e. the sum assured is payable only if the insured dies within the term of the policy. There is no investment value in the policy.

Policies can cover a single life or be taken on a joint life basis, typically on a “joint life first death” basis. In this case, for example, a husband and wife may take out such a policy to provide for the surviving partner. Some term policies can be renewed or extended.

Whole of life assurance – in this case premiums are paid throughout the life of the insured person and the sum assured is paid out on death. Again, these policies can be taken out on a joint life basis.

Endowment policies – these are savings policies which also provide life assurance. They are for an agreed term, with the minimum usually being 10 years. A cash sum is paid at the end of the term (“on maturity”) or in the event of the death of the policyholder. These policies are usually taken out to repay an interest-only mortgage. However, always remember that an endowment policy does NOT guarantee to repay a mortgage.

Why do thalassaemics find problems getting life assurance?

Under the Disability Discrimination Act 1975 it is unlawful to discriminate against people with a disability in connection with the provision of goods and services. However, insurance companies are allowed to offer different terms to different people according to circumstances. Each time a life assurance contract is negotiated, the insurance company must calculate the risk they are entering into, that is, what is the likelihood of them having to pay out the full sum insured. For example, in a case where no disability is involved, a healthy person of 25 wanting a 10 year term policy for £50,000 would pay a lot less than a 60 year old person taking the same policy, as statistically they are far more likely to survive for 10 years. By the same principle insurance companies are allowed to differentiate between those who have a disability and those who do not; they can also differentiate between people who have a certain disability according to its severity.

Insurance companies look at each case on its own merit. The company must base their calculations on information relevant to the assessment of the risk, which is from a reliable source. This can be; actuarial or statistical data, medical research information and medical reports on an individual. If you have a condition (such as thalassaemia) which the insurance company consider to be a high risk, the company can exclude that condition from the policy. It may be possible for a thalassaemic to obtain life assurance but it is likely to be “loaded” i.e. have a higher premium, as in the view of the insurance company they are taking a bigger risk. It is also more likely to have a restricted term. Basically, if you can find an insurer to give you life cover, the policy is likely to exclude death relating to any existing health problems AND is likely to be very expensive.

It is no longer mandatory to take out life insurance when obtaining a mortgage (although some lenders, especially banks, still demand it; it is also required if you take out an endowment mortgage). When applying for a mortgage, be sure to ask whether life assurance is required so that you have the option of choosing a lender or type of mortgage which does not require it.

It is important to realise that a contract of insurance is taken out according to the doctrine of utmost good faith, which means that you are obliged to disclose any facts which might affect the insurer’s judgement in accepting/declining the contract, fixing the terms or adjusting the premium, even if you are not specifically asked for them. Therefore always ensure that you provide all the facts on any application and NEVER state a deliberate untruth. If you do the contract is null and void, the policy will not pay out in the event of a claim and you will not get a refund of the premiums you have paid.

You may wish to seek the advice of a reputable Independent Financial Adviser (IFA). IFAs must be registered with the Financial Services Authority.

• Are you a thalassaemia patient/parent of a thalassaemic child?

• Do you have the UKTS specially designed personal organiser for thalassaemia patients?

If not why not – all it takes is a call to our office.This valuable aid to keeping your own patient – held medical record is FREE to patients and parents/carers of children with thalassaemia.

Call 0208 882 0011 to order your copy now!

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UKTS Marathon Man

UKTS Marathon Man Marios Ioannides, pictured here with his sister-in-law, thalassaemia patient Chriso (nee Papayiacovou). Heartfelt thanks to Marios and all our Marathon runners for their magnificent support.

UKTS Personal Organiser

www.ukts.org1� Thalassaemia Matters ...continuing the fight against Thalassaemia

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When travelling abroad it is important to feel secure and have the peace of mind that will allow you to fully relax and unwind. Freedom Travel Insurance has worked in conjunction with the United Kingdom Thalassaemia Society and leading clinicians in the field to offer people living with thalassaemia the benefit of an A1 insurance product.

Freedom already works with other medical charity groups and our expertise spans many conditions from diabetes to cancers. Our aim is to provide a wide range of benefits with an excellent level of security: this is achieved by the policy being 100 per cent underwritten by AXA, one

of the largest insurance companies in the world.

Premiums are individually calculated and dependent on such factors as when and for how long you are travelling, your intended destination, your age and how well your condition or conditions are managed. Medical screening and policy document issue are carried out over the telephone with trained staff in our head office. Freedom is not a call centre operation. There is a policy excess of only £50.00 including for claims relating to conditions which we have screened and agreed to cover. NB Quotations will vary subject to medical screening

Price is one thing but the true value of an insurance contract is often realised if a claim is made. Our programme is backed up by a team of clinical staff, claims experts and linguists available round the clock and planes fitted with specialist equipment in case of the need for medical evacuation or repatriation.

We do not claim to be able to help everybody with their insurance requirements however we are hopeful of providing a solution in the majority of cases.

For a quotation call 0870 774 3760

Travel Insurance For Thalassaemia Patients

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

Mr M Ashfaq £5

Bank of Cyprus £100

Mrs H Castillo-Binger £10

Mr A Gandhi £30

Mr S Gandhi £150

Mr J Hill £750

Mr M Ioannides £1,027 .70

Mr Y Ioannides £2,353 .20

Mr Isaac £50

Katon family £35

Mrs A Katsouris £50

Mr M Michael £70

Mr D Phillips £855

Miss M Purvis £20

Dr A Tsikoudas £200

DONATIONS

UKts Welcomes neW‑MeMBeRs

AnnualDr R W EvansMr D NikolovMr I Vasiliev

LifeDr H N AhmadMr M Moller

The UK Thalassaemia Society Annual Dinner & Dance

Date: Saturday 5th November 2005Venue: The Brewery, Chiswell Street, London EC1Y 4SD

Tickets available shortly from the UKTS office

Thalassaemia International Federation• 10th International Conference on Thalassaemia &

Haemoglobinopathies &

• 12th International Conference for Thalassaemia Patients & Parents

7-10 January 2006, Dubai, United Arab EmiratesFor further details please contact UKTS or refer directly to TIF on 00357 22 319 129Conference website www.tif.ae

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.

Have you visited the UKTS website lately? If not, why not give it a go – we have a number of new features for you to browse through. You can now read Thalassaemia Matters (both current and past issues) on-line, post messages in our guest book and get personalised careers advice!

NOW IT IS EVEN EASIER TO SUPPORT UKTS! Our website has a brand new

“Give Now” feature, which enables you to make easy, hassle-free donations on-line using your credit card (donations remain anonymous if you so wish). No more stressing about stamps, envelopes, or walking to the letter box - you can make a donation as easily as clicking a switch.

In addition to the new features there is lots more, including dietary advice (under Living With Thalassaemia) and Society information. Visit us at www.ukts.org and see for yourself why the UKTS website has so many visitors from all over the world. We love to hear from our friends – sign our guest book and you will get a message back from us. Hoping to hear from you soon!

Many thanks to our website committee Mike Michael, Chris Sotirelis and Philip Agathangelou for their continuous work in maintaining and updating the website.

events

www .ukts .org– On-line Donations and other

exciting new features on our website

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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)

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