may 2009 issue number 114 ukts holds first ever ... · of stem cell therapy was given by dr peter...

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

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

Medical News . . . . . . . . . . . . . . . . . . . . . 6

News from around the World . . . . . . . 10

Events . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12

Patient News . . . . . . . . . . . . . . . . . . . . . . 14

Office News . . . . . . . . . . . . . . . . . . . . . . . 15

CO

NTEN

TS

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

On 21st April 2009 representatives from the UK Thalassaemia Society met Under-Secretary of State for Health Services, Ann Keen. Medical advisory panel member Dr Anne Yardumian joined Vice-President Dr Christos Sotirelis, Treasurer George

Constantinou and Coordinator Elaine Miller at the Minister’s office in Whitehall.

Much of the “unseen” work carried out at UKTS involves working with the Department of Health and government officials to raise the profile of thalassaemia at the highest possible level; and needless to say we were delighted to be offered this opportunity to speak personally to the Minister about the issues which affect thalassaemia patients and their families. We were warmly welcomed by Ms Keen who, as a former nurse herself, showed a real concern and appreciation of the challenges faced by our patients. The Minister was particularly interested in the difficulties faced by most patients in getting routine transfusions done outside Monday to Friday, nine to five hours – an issue which continues to blight the career and education prospects of all too many patients. In the words of the Minister, the NHS should work “7 days per week, 365 days per year”. It was encouraging to note the Minister’s commitment to improving the availability of services and we look forward to working further with the Dept of Health towards this end.

The Minister was very interested to hear

that in 2006 Dr Yardumian’s patient Gabriel Theophanous became the first thalassaemic in the world to run a Marathon. This resulted in a frank discussion about the health inequalities which exist in the UK – where our patients are treated in a holistic manner by experienced clinicians, the care they receive is as good as, or better, than that available anywhere in the world. It is our goal that every patient in the UK should have access to this high standard of care. We may not all be Marathon runners but we all deserve the opportunity to reach our own goals and maximise our potential.

We were very grateful for this opportunity to raise the profile of thalassaemia within the Dept of Health and we will keep the Minister informed of our activities and the issues which are important to our patients.

The UKTS continues to further its Parliamentary lobbying activities by a consistent presence in the All Party Parliamentary Group for Sickle Cell and Thalassaemia, which will be officially launched at a Reception at the House of Commons on 14th July 2009. More on this in the next issue.

UKTS Holds First Ever Ministerial Level Meeting

May 2009 ISSUE NUMBER 114

Ann Keen MP, Under-Secretary of State for Health Services

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

Dear Friends,

Hello and welcome to issue 114 of Thalassaemia Matters. First and foremost, please be sure to note the notice of our AGM which will be held on 7th July 2009. If you are a member and you feel you would like to help the Society by serving as a Trustee, please fill in the form enclosed with this issue (you will need to be nominated by another UKTS member). We are always looking for new blood so if you have the skills and experience (for example fundraising, legal, accounting or marketing experience, managerial and organisational skills) why not stand for the Committee? Be sure that your nomination form arrives with our office no later than 19th June 2009.

Our front-page story this issue concerns the meeting we held with Under-Secretary of State for Health Services, Ann Keen. This meeting was the culmination of months of hard work - an example of the “unseen” work carried out by UKTS. We feel this is a real mark of progress. It is essential to raise the profile of thalassaemia at the highest level so that decision makers and policy makers are aware of the issues that are important to

us. Don’t forget, however, that we work at all levels so if there are any local issues which concern you, please don’t hesitate to get in touch with us.

I hope you will enjoy this issue; we have some topical advice about precautions against swine flu (please take note) and some very interesting articles and reports. Thanks to all who gave up their time and effort to raise funds for us, such as the Marathon runners and the Young Lohana Association of North London. And don’t miss the article in “News Around the World” by our brave young friend from Kenya, Mohammed Bachani – as thals we are sometimes in danger of feeling isolated; but imagine being the only thal in the entire country! (If anyone would like to write to Mohammed please contact our office.)

Finally, I wish you all a very happy summer and safe travels.

Until the next issue.

Mike MichaelPresident UK Thalassaemia Society

A word from our President

Mike MichaelPresident

Dr Christos SotirelisVice-President

Menuccia TassoneSecretary

George ConstantinouTreasurer

Philip AgathangelouAssistant Treasurer

Costas KountourouCommittee

Bharat NathwaniCommittee

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

latest news

Well, we have certainly been hearing a lot of warnings on this subject in the press recently; and knowing how the press like a good, sensational story, sometimes it is hard to know how seriously to take them. Here are a few tips for the benefit of our members.

�■ If you have thalassaemia, like all persons with an underlying medical condition, you need to take extra special care of yourself to minimise the risk of infection. Some of you will have had a splenectomy and therefore have a higher susceptibility to infection. If so, make sure you are taking your prophylactic penicillin as it has been prescribed by your doctor. As a person with thalassaemia, you are probably not at greater risk than others of catching flu, but you may be at somewhat greater risk of complications if you do.

�■ Common sense will carry you a long way to protect you from swine flu.

�■ If possible, avoid travelling to affected areas (Mexico, the USA).

�■ Carry an anti-bacterial hand gel (available from chemists) and use it frequently when using public transport or in public areas which are used by a lot of people.

�■ If somebody nearby has to sneeze or cough, ask them always to use a tissue, dispose of the tissue and wash their hands.

�■ The regular flu jab does not offer protection against the swine flu virus.

�■ Face masks are unlikely to be of any use in protecting you from other people’s germs.

�■ If you have heard of cases of swine flu in your area, you might want to avoid places where large numbers of people are crowded together for long periods of time (e.g. cinemas, theatres, sporting events).

�■ If you develop flu-like symptoms (fever, chills, sore throat, headache, aching limbs) seek medical attention

immediately by telephoning your hospital doctor or nurse specialist who will advise you on what to do. Avoid going into crowded areas such as doctor’s surgeries, A & E etc.

�■ Don’t panic. Remember that, as well as swine flu, there are still just as many of the usual coughs and colds going around, as well as hayfever which affects many people at this time of year. We all fall prey to these from time to time. A useful distinction to remember is that flu would almost always come with a fever, whereas a common cold and hayfever won’t.

You can find more information here: http://www.dh.gov.uk/en/index.htm - download the “Swine flu information leaflet” or call 0800 1 513 513 to hear the latest information on swine flu.

With thanks to Dr Achim Schwenk, Consultant in Infectious Diseases, North Middlesex University Hospital

Swine Flu – Some Practical Advice

NOTICE TO ALL UKTS MEMBERS

The Annual General Meeting (AGM) of the UK Thalassaemia Society will be held on :Date: Tuesday 7th July 2009Time: 7.30 pmVenue: UK Thalassaemia Society officeALL MEMBERS WELCOMEPlease contact the office for further details


Genetic Interest Group Conference 14th May 2009Innovative Therapies and Rare Diseases . Are we there yet?

The Genetic Interest Group (GIG) conference

took place on 14.05.09 at the Clifford

Chance building in Docklands, London. The

theme of the conference was “Innovative

Therapies and Rare Diseases. Are we

there yet?” As Alastair Kent, Director of

GIG, explained in his introduction, we are

constantly hearing from the popular press of

“breakthroughs” in research which lead us

to believe that new treatments and cures are

“just around the corner”. In reality, however,

research into treatments and cures for genetic

diseases is extremely complex and takes many

years of lab research and safety trials before

the scientists involved can even contemplate

extending their studies into human trials.

Despite what we read in the press, cures for

rare genetic diseases are unlikely to be widely

available within the short or even medium

term.

Needless to say, this time consuming

process is incredibly expensive. So who foots

the bill? The fact is that most investment in

drug development is private; that is to say,

funded by drug companies. Drug companies

naturally make most profit when they can

develop a drug which is needed by many

thousands of people; so where is their

incentive to fund research into developing

drugs for these very rare conditions? (N.B. According to NICE guidelines the definition

of a very rare “ultra-orphan” disease is

that there should be less than 1 patient per

50,000 people in the population; in effect,

this means less than 400 patients in the UK.)

Sometimes research that has been done to

develop a drug for another condition can turn

out to be useful. We heard a presentation

from Prof Peter Hillman (University of Leeds)

on how a drug (Eculizumab) was originally

developed as a treatment for rheumatoid

arthritis; but eventually became a recognised

treatment for paroxysmal nocturnal

haemoglobinuria, a rare, acquired genetic

disease of the blood. The drug was used in

research and patient trials from 1992 until

2007, when it was licensed for use in the

USA and Europe. During this time the pharma

company (Alexion Pharma Inc) had spent over

$800 million in development. Since 2007 the

company has realised $77 million – although

it must be emphasised that this figure refers

to sales, not profit. Once a drug becomes

available on the market it is only a matter of

time before “rival” drugs come along; so the

pharma companies have only a few years to

realise their investment.

An interesting presentation on the use

of stem cell therapy was given by Dr Peter

Hollands (University of Westminster). In

line with Alastair’s introductory remarks, Dr

Hollands also stressed the fact that the press

often make unrealistic claims for stem cell

treatment. He explained that stem cells are

cells which can do a variety of jobs. They

come from a number of sources:-

Embryonic stem cells are derived from

human embryos. They are difficult to create

and potentially unstable, leading to safety

concerns. Obviously there is a great deal of

ethical debate (and media hype) around the

use of embryonic stem cells). To date they

have not been used clinically.

Bone marrow stem cells constantly

make/renew our blood cells. The first stem

cell transplant from bone marrow was

carried out in the 1950s, since when many

transplants have been carried out. They can

be used to treat blood disorders (such as

thalassaemia); however there is a snag in that

the donor and the recipient must be a tissue

match. There is currently research under

way into the possibility that bone marrow

stem cells may repair other tissues, e.g. heart

tissue.

Peripheral blood stem cells – in certain

cases drug therapy can be used to release

stem cells from the bone marrow into the

circulating blood. These are then collected

by apheresis and used as an autologous

graft (given back to the same patient).

This treatment may be used on adults who

have undergone chemotherapy for cancer

treatment.

Skin, gastro-intestinal tract, liver and central nervous system stem cells are

differentiated and are essential to normal

health. They have no clinical use.

Foetal stem cells have the potential to

repair all tissues. Obviously there is a huge

ethical debate about the use of foetal stem

cells. At the present time the Ukraine, China

and India are the only countries doing foetal

stem cell transplants.

Cord blood stem cells (from the umbilical

cord of a newly born baby) like bone

marrow stem cells, can make all types of

blood cells. They can be used to treat blood

diseases and after chemotherapy for cancer

treatment. Cord blood can be collected after

both natural and caesarean deliveries. Up

to 200ml of cord blood can be collected

from each donation. After donation, the

stem cells are isolated (approximately 25ml),

treated with a protective substance and then

frozen in a computer controlled freezer at

-196°C. Theoretically the cells can be stored

indefinitely. In order to carry out a cord blood

stem cell transplant, the patient must first be

treated with chemotherapy which means that

the procedure carries a risk (just as in bone

marrow transplant). However cord blood

stem cell transplants carry a lower risk of

GVHD (graft versus host disease, or rejection).

At the moment, in the UK less than 1% of

cord blood is collected and stored and Dr

Hollands called for a national scheme to

ensure that all expectant parents have the

right to have their baby’s cord blood collected

and stored. At the moment there are only

2 public cord blood banks; one in Edgware

(London) and the other is the Anthony Nolan

cord blood bank in Nottingham. There are

a number of private companies offering this

service but a national scheme would be far

preferable. The Royal College of Midwives

have stated their support for a national

scheme and in the USA it will soon be law

latest news

GIG staff Helen Parr, Melissa Hillier, Ben Francis and Krystle Kontoh


that doctors and midwives must inform all

pregnant women about cord blood collection.

Finally, Dr Hollands told the amazing story

of Patrizia Durante, a Canadian woman, who

was diagnosed with acute myeloid leukaemia

(an aggressive form of blood cancer) while 26

weeks pregnant. When she did not respond

to chemotherapy, labour was induced so

further doses could be given without harming

the baby. The umbilical cord of Patrizia’s baby

(a girl named Victoria Angel) was preserved

in liquid nitrogen. Patrizia’s cancer returned

in 2002 after a short period of remission

and her doctors decided to perform a cord

blood transplant from her own daughter.

The transplant carried a risk as the baby

(carrying 50% of her father’s genes) was only

a 50% match; but happily the outcome was

a success and mother and baby are alive and

well today. This ground-breaking procedure

was carried out by Prof Pierre Laneuville and

his colleagues at McGill University Health

Centre in Montreal. Apparently the story is

now being made into a Hollywood film so

look out for it!

Patrizia Durante with her daughter Victoria Angel.

Prof Adrian Thrasher (Professor of Paediatric

Immunology, Great Ormond Street Hospital)

gave an update on the use of gene therapy,

used in the treatment of children with

severe immunodeficiency (“bubble” babies

who cannot have any contact with the

outside world as have no immunity against

infection). These children can traditionally

only be cured by a matched bone marrow

transplant, which is risky. Gene transfer

technology enables the clinician to place a

corrected gene inside the patient’s own cells.

A modified virus (now usually HIV) is used as

a vector to take the therapeutic gene to the

cells. This is known as an autologous graft,

where the patients own cells are used – the

patient’s bone marrow cells are harvested

from the hip bones (approx 100ml), modified

and transfused back to the child 3-5 days

later. This can be done as an out-patient.

As the patient’s own cells are being used,

there is no need for chemotherapy (as in

other transplants, which require the patient

to spend around 3 months in hospital).

So far 19 immunodeficiency children have

been treated with gene therapy; most have

normal immunity today. However, there are

still problems with the technology – the

function of other genes can be affected. Five

of the nineteen children treated developed

leukaemia; 4 were successfully treated but

one unfortunately died. Another complication

is that in some cases the transplanted genes

can “switch off” so that long term therapy is

needed. “Next generation” vectors are now

being trialled which it is hoped will be safer

and have a better long term effect.

Prof Thrasher pointed out that research

into gene therapy can be problematic as

the regulatory authorities place increasingly

restrictive requirements on the academic

sector. Currently there is a lack of connection

between the academic/research sector and

the NHS. Future developments may be limited

by a lack of collaboration and funding. The

2003 Genetics White Paper pledged £50

million over 5 years for research into gene

therapy. This has come to an end and there is

currently no designated funding in the NHS.

There were further presentations and

workshop sessions throughout the afternoon

– sadly I left rather early as I had work to

do in the office, but the day was extremely

interesting and enjoyable. Many thanks to

all the staff of GIG for their hard work in

arranging the conference.

Elaine Miller

1st ANNOUNCEMENT – 19th International Conference on Oral Chelation

latest newslatest news

Venue of the 19th ICOC: Bath University, Bath, United Kingdom.

Deadlines for Abstract submission and Early Registration: Abstract Submission Deadline: 14th September, 2009. Early Registration Deadline: 1st September, 2009.

Dates of the Conference: The 19th ICOC will be organised in Bath University on the 11-14th December 2009.

Preliminary ProgrammeFriday 11th December: Early Evening: Registration Evening: Keynote LecturesLate Evening: Welcome Reception by the Mayor of Bath

Saturday 12th December: Scientific sessions and Social Programme

Sunday 13th December: Scientific sessions and Social Programme

Monday 14th December: Morning – Free time and Social programmeAfternoon – Scientific sessionsEvening – Closing

PUBLICATION OF THE PROCEEDINGS OF THE 19th ICOC IN THE JOURNAL HEMOGLOBIN:

Arrangements have been made for the publication of selected papers of the proceedings of the 19th ICOC in the

journal Hemoglobin. The papers will be selected from the submitted abstracts for oral or poster presentations or keynote lectures by a panel of the scientific committee. The authors of the selected papers will be informed about the selection of their papers after the completion of the conference. Guest Editor of the 19th ICOC proceedings in the journal Hemoglobin will be George J Kontoghiorghes.

Contact information: George J. Kontoghiorghes Fax: 00357 26271434 Tel: 00357 26272076 e-mail: [email protected] www.pri.ac.cy www.icoc-isocam.org


medical news

Diabetes in Thalassaemia

Dr Maria Barnard, Lead Consultant in Diabetes, The Whittington Hospital NHS Trust, London & Honorary Senior Lecturer, University College London Medical School (email: [email protected])

Diabetes – The Global ChallengeDiabetes mellitus is a major global health challenge. In 2007, around 246 million people worldwide were living with diabetes and that number is predicted to increase to 380 million by 2025. In the UK, the number of people with diabetes could reach three million by 2010. Already each day in the UK, £10 million is spent on treating diabetes and diabetic complications, including heart disease, stroke, blindness, kidney disease and amputations. People with diabetes may also develop severely abnormal blood glucose (sugar) levels, requiring urgent medical attention. All this means that people with diabetes have a reduced life expectancy. Worldwide, every ten seconds, a person dies from diabetes-related causes.

How is diabetes diagnosed?Diabetes is diagnosed by detecting an increased glucose level on a blood sample. If this does not give a definite answer, an

oral glucose tolerance test (OGTT) is done, when blood glucose is measured before and 2 hours after having a glucose drink. In some people, the blood glucose level is not high enough to diagnose diabetes, but is not low enough to be classified normal. This “impaired glucose tolerance” or “pre-diabetic syndrome” can be a precursor to developing diabetes.

Diabetes Diagnosed:

Random blood glucose

≥ 11.1 mmol/l

Fasting blood glucose

≥ 7.0 mmol/l

OGTT: 2 hour glucose

≥ 11.1 mmol/l

What causes diabetes?Insulin lowers blood glucose levels. It is released from islet ß-cells in the pancreas into the bloodstream. It controls the transfer of glucose out of the blood and into cells, where it is used to create energy. Diabetes occurs when there is not enough insulin in the body (insulin deficiency) or when the body becomes resistant to the effects of insulin (insulin resistance).

Some famous people with diabetes:

Christopher Biggins

Sir Steve Redgrave

Halle Berry

Type 1 diabetes affects 5-10% of people with diabetes and is most commonly diagnosed in childhood. It is caused by autoimmune destruction of the ß-cells, when antibodies are made that directly attack these insulin-producing cells. People are born with a genetic tendency to develop type 1 diabetes, which is then triggered possibly by exposure to a viral infection.

Type 2 diabetes affects 90–95% of people with diabetes and is usually diagnosed in people aged over 40 years. It is caused mainly by resistance to the effects of insulin. Obesity is a major risk factor for type 2 diabetes, which is therefore greatly on the increase. Other risk factors include

having a family member with type 2 diabetes. People from some ethnic groups are at risk of developing type 2 diabetes: in the UK, there is a significant risk of diabetes occurring in people of Indo-Asian, Black-Caribbean or Black-African descent.

There is a large collection of other conditions that can lead to diabetes, including thalassaemia. Diabetes in thalassaemia is thought to be caused mainly by iron overload, from repeated blood transfusions, damaging the pancreas. However, there may be other factors. Insulin secretion may be reduced, possibly because iron is directly toxic to the insulin producing pancreatic ß-cells. But there is also evidence in some people with thalassaemia for an autoimmune reaction against ß-cells. Insulin resistance may occur in thalassaemia, caused by liver disease from iron overload or hepatitis C virus infection. Some patients with thalassaemia and diabetes have both insulin deficiency and insulin resistance. Furthermore, with the worldwide diabetes epidemic, a person with thalassaemia and raised blood glucose could have developed type 1 or 2 diabetes. The situation can be very complex. However, understanding the underlying cause of diabetes in an individual is essential for logical treatment decisions.

What symptoms does diabetes cause?Excess glucose in the blood is cleared by being excreted in the urine, carrying water with it. Diabetes can cause symptoms of being thirsty, passing a lot of urine, weight loss and tiredness. When there is a severe insulin deficiency, as in type 1 diabetes, these symptoms can occur rapidly over 2-4 weeks and if untreated, can lead to profound dehydration and kidney failure. With insulin resistance, as in type 2 diabetes, there is a more gradual increase in blood glucose levels. These symptoms tend to develop much more slowly and people may have type 2 diabetes for years before being diagnosed.

For people with thalassaemia who develop diabetes, their symptoms will depend on how insulin deficient and how

Dr Maria Barnard, Lead Consultant in Diabetes, The Whittington Hospital NHS Trust, London & Honorary Senior Lecturer, University College London Medical School


medical news

insulin resistant they are. However, with modern iron chelation therapy, generally people with thalassaemia gradually develop diabetes and many have no initial symptoms. Some thalassaemia services will therefore screen their patients once a year, measuring the fasting blood glucose level or performing an OGTT.

Why is it important to treat diabetes?

Advanced cataract

The main aim in treating diabetes is to prevent diabetic complications. Diabetes damages blood vessels and various organs. In the eyes, diabetes can cause early cataracts (a cloudy lens in the eye). It can also damage the blood vessels supplying the back of the eye with oxygen and nutrients. This is called diabetic retinopathy and it is the commonest cause of blindness in working-age adults in the UK.

In the kidneys, diabetes can damage the small blood vessels which carry blood through for the kidneys to clean. This is called diabetic nephropathy and in the early stages it causes no symptoms. It is detected by measuring increased protein levels leaking through into the urine (microalbuminuria, then proteinuria). As the damage progresses, the kidney function deteriorates and dialysis or kidney transplant may be needed.

Retinal photograph (early retinopathy)

Diabetes can damage the small blood vessels supplying nerves, causing loss of

function. This is called diabetic neuropathy and it particularly damages sensation in the feet, causing numbness and possible severe damage, such as foot ulcers.

Diabetes can damage the larger blood vessels in the heart and brain. Lumps of fatty material containing cholesterol narrow these blood vessels (atherosclerosis). Eventually blood flow can be completely blocked by a small overlying blood clot. This can damage the heart muscle (heart attack) or brain (stroke).

Preventing these complications is a critical aim of diabetes management. This can be done by both tight blood glucose control and by controlling all other factors that can damage blood vessels.

There has been a belief that people with thalassaemia and diabetes have a much lower risk of developing diabetic complications. However, I think that will be challenged in the future. The successful management of thalassaemia is now leading to a significantly increased lifespan. If diabetes is also present, there will be a much greater opportunity for diabetic complications to develop, which we are beginning to see in our patient population.

How should diabetes in thalassaemia be managed?Ideally, with the best iron chelation therapy, iron overload diabetes will not occur in thalassaemia. This is the great future hope.

However, worldwide diabetes is increasing so much that type 1 or 2 diabetes may occur in a person with thalassaemia. In thalassaemia, screening allows raised blood glucose levels to be detected at an early stage. At this stage of abnormal glucose tolerance, intensive iron chelation may prevent diabetes developing or can even revert blood glucose levels to normal. In addition, weight loss and exercise are both established ways to reduce insulin resistance and to prevent progression to diabetes.

Once diabetes develops, some oral antidiabetic drugs have been tested in thalassaemia. There are reports on the successful use of sulphonylureas and acarbose. Sulphonylureas (e.g. glibenclamide, gliclazide, glimepiride) lower blood glucose by increasing insulin release from pancreatic ß-cells. Acarbose inhibits the digestion of starch and sugars in the gut, reducing the rise in blood glucose after food. For type 2 diabetes, metformin is the first choice treatment but there is little research on its use in thalassaemia. Metformin reduces insulin resistance and can protect against cardiovascular disease. It could be equally promising in thalassaemia and we certainly consider using metformin.

For the many patients with thalassaemia

Measure Target Measure TargetFructosamine [*HbA1c

< 299 umol/l< 6.5 %]

Blood pressure- with nephropathy

< 130 / 80< 125 / 75

Blood glucose before mealsBlood glucose after meals

4 – 7 mmol/l5 – 8 mmol/l

Aspirin if > 40 years of age or cardiovascular risk

75 mg daily

Smoking status Non-smoker Total cholesterol < 4.0 mmol/l

Exercise Daily LDL cholesterol < 2.0 mmol/l

Body mass index 20 – 25 kg/m2 Triglycerides < 1.7 mmol/l * HbA1c is the most widely used measure of average blood glucose control in diabetes. It is not used in thalassaemia because repeated blood transfusions make it inaccurate.

Normal artery Artery narrowed by cholesterol deposits

Continues on page 8 ➡


who are insulin deficient, insulin is an essential daily treatment. Insulin must be injected (into the fat immediately below the skin). There are two main treatment regimens. The first uses biphasic insulins (e.g. NovoMix 30, Humalog Mix50) injected twice daily. These contain a mixture of rapidly acting and slower acting (intermediate) insulins, given before breakfast and before the evening meal.

Breakfast insulin

The basal/bolus insulin regimen is the other main option. A very slow-acting (basal) insulin is given once a day (e.g. Glargine {Lantus}, Detemir {Levemir}) to give background 24 hour insulin cover. Then, every time a person eats carbohydrate they inject a rapid acting (bolus) insulin (e.g. NovoRapid, Humalog), which controls the blood glucose rise from the food. This requires multiple daily injections of insulin. Also, to get good blood glucose levels,

patients need to learn how to adjust their insulin dose according to the carbohydrate content of the food.

For people with type 1 or 2 diabetes, there are education courses based on small groups of people working together, discussing all aspects of living with diabetes with trained tutors. These courses can improve diabetes control and quality of life. There has been no research into developing courses for people living with thalassaemia and diabetes. However, people with thalassaemia and insulin dependent diabetes have attended our type 1 diabetes course (WINDFAL) and found this a useful and valuable experience.

One of the most effective settings for clinicians to look after a person with thalassaemia and diabetes is in a joint clinic. Here members of both diabetes and thalassaemia teams work together with patients. This enables staff to manage all the complex needs of these two conditions and it allows staff to learn from each other and provide a consistent approach.

ConclusionManaging diabetes is one of the greatest challenges that a person with thalassaemia can face. Training people to self-manage their diabetes and providing support from

specialist teams working together are critical for excellent diabetes control. Only then will we see people with thalassaemia and diabetes achieving a long and healthy life.

References and useful websites:1. http://www.whittington.nhs.uk/diabetes

‘Guidelines for the Management of Diabetes’

2. http://www.diabetes.org American Diabetes Association: clinical practice recommendations

3. http://www.idf.org International Diabetes Federation: the global impact of diabetes

4. http://www.who.int/diabetes World Health Organisation Diabetes Programme

5. http://www.nscretinopathy.org.uk National Screening Committee for Retinopathy in the UK

6. http://www.runsweet.com Useful practical site for advice on diabetes and exercise/sport

7. http://jdrf.org.uk Juvenille Diabetes Research Foundation: information for children and families

8. http://www.diabetes.org.uk Diabetes UK: information, policy statements and campaigning

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Hi Everybody,For those of you who don’t know

me I’m John Wilson from Hamilton in Western Victoria. Although I don’t have thal I will explain a little bit about my haemoglobinopathy. I have CDA or in short CDA. I make red cells properly but my body lacks the essential erythropoietin to mature those cells into useful oxygen carriers. As a result I have a blood transfusion of red cells, usually 4-5 units every 3 weeks and chelate the excess iron with Exjade which I started 2 years ago. Before that we used Desferal and the painful nightly injections. So when it all boils down I have the same treatment as thalassaemics. For those who are wondering, yes I do still have a spleen! I should also mention that my older brother Tim has the same condition as me.

3 years ago during a normal school day, I walked into Yr 10 Biology and was

welcomed by the smiling faces of Sarah and Maria from TSV. I was absolutely shocked that they were talking about haemoglobinopathies, chelation and blood transfusions. Living in a small country town and only seeing a few transfusion patients at the hospital meant I had no idea that thalassaemia even existed and that so many other patients live the same life I do. The girls got quite confused when I started asking them all sorts of interesting questions that they usually don’t get from the students. I think they thought I was being a smart Alec!

Who would have thought that we would become such good friends and travel overseas together? Funnily enough Louisa had spent hours with my older brother entertaining him during his transfusions while mum and dad were busy looking after me. So 18 years later we caught up again.

I decided to join the Thal Society so that I could be in a group that shared a common bond. The newsletter put out by TSV is invaluable and we all look forward to learning from it. I really appreciated the sponsorship that I received from TSV on behalf of the Stan Kastoras memorial fund to attend the TIF conference in Singapore last month.

It was wonderful to meet so many other people living with haemoglobinopathies and feeling like part of a big group. Singapore is a vibrant clean city which made it a wonderful destination for the conference. The individual meetings and seminars were held in the massive Suntec convention centre/ridiculously huge shopping centre. It goes without saying that we had a ball attending the conference. Listening to keynote speakers, patients, parents, haematologists, cardiologists and pharmaceutical reps was mind boggling but very interesting. It was comforting to realise just how thorough and competent our medical treatment is in Australia. I learnt heaps about Exjade and the importance of taking it properly. It was disappointing to hear in some countries people with haemoglobinopathies are disadvantaged and sometimes even discriminated against. I walked away from the conference with a ton of knowledge and a heap of new friends.

Whilst there we made the most of the opportunity and had an extra weeks holiday at the end of the conference. The 6 of us travelled and experienced Singapore to the max with fun activities, food and SHOPPING!

Summing up I would like to thank TSV for giving me this fantastic opportunity to broaden my horizons. I had a great time meeting and spending time with another young patient, Eugie.

I look forward to attending the next TIF conference in 2010.

Thank you and best wishes, John

Singapore 2008 ConferenceReport by John Wilson, Thalassaemia Society of Victoria

news from around the world

John with friends (L to R) Antonella Alvaro, Eugenia Pantazis and Louisa Di Pietro.


On May 9, a new Thalassaemia Centre was inaugurated in Baku, Azerbaijan.

President Ilham Aliyev and the First Lady Mrs Mehriban Aliyeva attended the event and cut the red ribbon opening the Centre.

The launch of the Centre will contribute to the creation of a specialized medical centre and blood bank, the development

of a voluntary donor service so thalassaemia-affected children can get safe and high quality blood, the enlightenment of the population and deepening of cooperation with foreign states fighting thalassaemia to prevent the expansion of this disease and finally the training of highly-qualified personnel.

The opening of this new treatment Centre in the capital is one of the worthiest contributions by the Heydar Aliyev Foundation to the health of our population.

President Ilham Aliyev expressed his satisfaction with the facilities available at the Centre.

Thalassaemia Centre Inaugurated in BakuBy Nailya Guliyeva, President of the Azerbaijan patient society “Savab Dunyasi” and

Board Member of the Thalassaemia International Federation

Hi Readers,This is Mohammed Bachani from

Mombasa, in Kenya, along the shores of the Indian Ocean. I have thalassaemia. I am a boy aged 13 years, currently studying in Nasserpuria Primary School. My hobby is teasing my elder sister who normally gets angry when I tease her. Actually I like it.!!!

I am in Class Seven. In school, I am commonly known as ‘King’ because I am the top student. In fact I am proud of my six years achievements. God willing, when I grow up, it’s my dream and ambition to become a paediatrician.

First and foremost, every time I need a transfusion my Father has to go round looking for donors from friends or relatives. Sometimes relatives refuse to donate, others say they would die if they donate blood. I was diagnosed when I was six

months old, I started having a fever which couldn’t be controlled. In my town, Mombasa, thalassaemia is not recognized at all, so we had to travel to other town called Nairobi and there it was discovered that I have thalassaemia. My parents were heartbroken, they couldn’t believe it but it was destiny and so it happened.

There are many problems which I face in the hospital, I get a lot of injections as my veins are thrombosed (which means a lot of pain in both my hands). Sometimes if my Father does not get donors, we then have to take blood from hospitals. The blood reacts on my body, I get fever which is uncontrollable, I get nausea, that’s all from allergies to blood, and sometimes I get malaria. My haemoglobin really drops rapidly and within one or two weeks I am back to the hospital, and hospital bills are very expensive. After every six months my parents request the Doctor to test my iron and we get the results after two months.

During my blood transfusion, I go from 8 a.m. till late night with both my parents, so my sister is all alone at home. I am away from school and this really affects me and my studies. That’s why I want to come to U.K. and settle there. I know some problems which I face here don’t exist there as there are expert Doctors, Nurses etc.

Sometimes I get frustrated and say

why only ME??? But my parents calm me down, I really cry even now as I am writing my tears are rolling down my cheeks. It’s very difficult for me to go through all this at this teenage age.

I am very glad that the UK Thalassaemia Society is helping people to overcome this disease. I wish there was even one society here in Kenya, but I thank Aunt Elaine who supplies us with articles or any problems we ask she is very supportive to us. God blesses her.

In 2002, I had a bone marrow transplant in Birmingham Children’s Hospital but it failed. My only sister donated her narrow. It was a terrible moment for my family and me, when we got to know it had failed. Even at that time we got a lot of support from the UK Thalassaemia Society and that is when we came to know about this Society.

Finally, my request to UKTS – if possible please find a way that me, and my family we can come and stay in the U.K. so that I can benefit from the modern medication like my fellow thalassaemic patients are benefiting, since I am the only one in Kenya.

A special vote of thanks to Aunt Elaine and all the Thalassaemia Society Members. I pray to God to make this Society a very successful one in the world. Amen.

Love from Mohammed Bachani

My Life as a Thalassaemia Patient in Mombasa

news from around the worldnews from around the world


UK Thalassaemia SocietyDinner Dance

2009

SAVE THE DATE!

DATE: Saturday 21st November 2009

VENUE: The Regency Banqueting Suite113 Bruce Grove, London N17 6UR

MUSIC: Dancing to the music of Diastello

Contact the UKTS office for further details

The 2009 Flora London Marathon took place on Sunday 26th April. Teacher Deirbhle Mannion ran the London Marathon for the UK Thalassaemia Society. Her inspiration for the 26-mile run was five-year-old Sabrena Afridi, a thalassaemia patient, who attends Chandlings School in Oxford where she teaches. Says Deirbhle; “When Sabrena joined our school and I learned about her condition and daily challenges, I decided to run for the UK Thalassaemia Society.”

A huge THANK YOU to all our brilliant runners for their brave effort and for the funds they have raised to help us with our work.

Running the Marathon for Thalassaemia

events

Runners and times

Tony Lambrou 5 hrs 01 mins

George Malekos 6 hrs 52 mins

Deirbhle Mannion 4 hrs 22 mins

Dinos Stavrou 5 hrs 43 mins

George Stavrou 5 hrs 43 mins


After last year’s success, the Young Lohana Association of North London (YLANL) once again presented ‘The L Factor’ on Sunday 7th December 2008 at the Zoroastrian Centre in Rayners Lane. The L Factor is a showcase for the talent amongst the youth of our community, with performances by 3 year olds to 30 year olds! The YLANL believe it is essential to encourage today’s youth, allowing them to develop the confidence to become tomorrow’s leaders. The evening raised money for the UK Thalassemia Society, as thalassaemia is a condition prevalent not only in our community but throughout society yet there is little awareness of this life-

changing condition. Our aim was therefore to both encourage the Lohana youth to display their talents and to raise awareness of thalassemia within the community.

Our event was supported by special guests on the night, improved versions of Simon and Louis (from the X Factor), MP Tony McNulty and Goodness Gracious Me actress/ patron of the Asian Awareness Campaign of the UK Thalassemia Society, Nina Wadia. Our other guests included the chief speaker on thalassemia, Mrs Sonoo Malkani. Our guests all showed great support for the L Factor and got well into the spirit of the event!

Hosted by myself and our Social Secretary (Jeet Rughani), the night started off with some spectacular dancing, vocal medleys and orchestral acts. The night continued with an array of dazzling performances from all the participants, from song and dance to traditional devotional hymns accompanied with live instruments. With the sparkling array of bright costumes, the routines were performed and the audience voted for their favourite act of the evening. After a delicious meal served during the show’s interval, the crowd gathered waiting in anticipation; as to who would be crowned the winner of L factor 2008 ?

During the interval, we made a pledge to collect further donations and any cash donations made on the night were doubled by the YLANL.

The winners of the night, a group named Badsha, who provided incredible entertainment by blending the best of east and west dance moves, were presented with a trophy by our main sponsor, Blue Ginger and Blue Zenzer.

2008’s L Factor was an immense success, raising in the region of £2,500 for the UK Thalassemia Society. Our efforts would not have been materialised without the talent of these young people and the generosity of the Lohana Community.

We hope that our donations have made a difference to assist the funds required by the UK Thalassemia Society. We believe that we have also achieved our other goal in helping to raise awareness of Thalassemia and the effects of the illness. We hope that others will be inspired by our efforts and endeavour to help UK Thalassemia Society in any way that they can.

Ronak Paw, President YLANL 2008 - 2009

All of us at UKTS sincerely thank everyone at YLANL for making us their chosen charity and for working so hard to make this event such a success – thanks also go to our Patron Ms Nina Wadia for giving up her time to attend and to Mrs Sonoo Malkani for representing UKTS at the event.

The Young Lohana Association of North London present ‘The L Factor’

in aid of UK Thalassemia Society

Some of the talented acts who took part. Centre right (in blue) is Patron of the UKTS Asian Awareness Campaign, actress Ms Nina Wadia

events


Dear FriendsIt was with some surprise that I received

an invitation to a party to be held on 11th April last. The event was to celebrate EID and was also a belated 40th birthday bash for one of the North’s best loved thalassaemia patients – ME!!! The event was organised by Mrs Rasul of the North of England Bone Marrow and Thalassaemia

Association (NEBATA). I arrived at the venue (a posh restaurant

in Rochdale) with my family, but had left my beloved wife Fouzia at home due to some news we had received some weeks earlier (the news and the party together made it a great day in my life!) – we are expecting!!!!! We are officially having another baby thanks to the wonders of IVF. Most of my family, including my two sisters, arrived at the bash a little late; having been held up in traffic due to a football match.

I knew most of the people present and they all hugged me and gave me birthday cards, presents and kisses! I made a short speech about living with thalassaemia which I hoped would be an inspiration to all the younger thals at the party – a lot of them asked me questions and I tried to answer them as best I could. Also, my sister (mum of my niece Noshaba, who is also a thal) gave a parent’s account; which included how our parents brought me up

and how they coped with it all.After a quiz and a few jokes lunch

was served, which was followed by my nephew Haroon Hanif who did a brilliant performance of his Michael Jackson dance routine – this brought the house down (he is available for bookings, please enquire!).

Finally there was a birthday cake which I cut – everyone had a piece and I received lots of good wishes from all. Mrs Rasul presented me with a card and present and said “Now we look forward to the next 40 years!”

I would like to thank Mrs Rasul and NEBATA for organising this fantastic event and for everything they have done for me in the years I have known them.

‘Bye for now and I hope to see many of you at the UKTS conference! Best wishes, Tanver

P.S. Throughout all this my son Abdur-Rehman was mingling with the young girls and showing me up – not bad for a two-year-old – he’s one to watch out for girls!

Hello to everybody! My name is Roberto. I am Italian from Naples and since October 2008 I have been a patient at the Whittington Hospital, after moving to the UK to start my PhD in astronomy at the University of Hertfordshire. I am affected by beta thalassaemia major and need a blood transfusion every 18-20 days. Before coming to the UK, I used to have transfusions done mainly by my father at home, being himself a doctor, but he referred me to specialists for the most important treatments.

At first, moving to another country everything was hard, moreover if you consider our health status and the linguistic difficulties, but after these first months

everything looks easier than I thought before.

Differences exist between NHS and the Italian Health Board and they both have different pros and cons. High quality care is offered by the Whittington Hospital, doctors and staff nurses are always kind and thoughtful, as expected from EU standards or even more. At the second medical examination, after the 7 months I have been living here, the clinical status appears to be good and further examinations are going to be booked. Previously, I was taking Exjade for 2 years and, luckily, I can continue the therapy in the UK as well.

My Birthday BashBy Tanver Khan (Bradford)

From Naples to HertfordshireBy Roberto Raddi

Tanver (with his son Abdur-Rehman) is presented with a birthday gift by Mrs Zanib Rasul of NEBATA

Roberto getting used to English customs.

patient news


UKTS Welcomes NEW‑MEMBERS

AnnualMs Gulcan Birdane

Mr Roberto Raddi

LifeMs Roanna Maharaj

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

Mrs S . Afridi £100 .00

Ms Shreena Bathia £105 .00

Mr J . Bewes £50 .00

Mr D . Bolster £40 .00

Chelsea F.C. £4,475.00

Mr Alan Coombs £15 .00

Mrs Elida Joannou £343 .32 (in memory of Mr Andreas T. Nicolaou)

Mrs M . Kyriakkides 50 .00

Lohana Ladies of North London 650 .00

Met Police Greek Staff Assoc 250 .00

T . Perry 50 .00

Ms D Ptohopoulos 50 .00

Square Conduct Lodge 100 .00

Dr R .P . Tahalani 55 .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.

Mike is presented with a donation from the MPGSA by the President of Cyprus Demetris Christofias

Recent Events

•The Met Police Greek Staff Assoc Dinner 30 April 2009 — Attended by UKTS President Mike Michael

•Thalassaemia International Federation Board Meeting & Workshop, Damascus, Syria 29 April – 3 May 2009 — Attended by UKTS Vice-President Dr Christos Sotirelis

•2nd Pan-American Thalassaemia Conference, Sao Paolo, Brazil 5-11 May 2009 — Attended by UKTS Vice-President Dr Christos Sotirelis

•Dept of Health Workshop; Long Term Conditions – good service provision and how to deliver it, London 12 May 2009 — Attended by UKTS Vice-President Dr Christos Sotirelis

•Genetic Interest Group Trustee Awayday London 11 February 2009 — Attended by UKTS Coordinator Elaine Miller

•Rare Disease UK Reception, House of Commons 25 February 2009 — Attended by UKTS Coordinator Elaine Miller

•Friends of Thalassaemia Blackburn with Darwen Health & Well Being Event 7April 2009 (presentation) — Attended by UKTS Coordinator Elaine Miller

•SSCATS Foundation Thalassaemia Awareness Event Sheffield 7 May 2009 (presentation) — Attended by UKTS Coordinator Elaine Miller

•Genetic Interest Group annual conference London 14 May 2009 — Attended by UKTS Coordinator Elaine Miller

•British Society for Disability & Oral Health 8 May 2009 Eastman Dental Hospital (presentation) — Attended by UKTS UKTS Assistant Coordinator Katerina Loizi-Read

office news

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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):

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

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may 2009 issue number 114 ukts holds first ever ... · of stem cell therapy was given by dr peter...

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