Issue 114

May 2009 ISSUE NUMBER 114
UKTS Holds First Ever
Ministerial Level Meeting
Ann Keen MP, Under-Secretary of State for
Health Services
CONTENTS
On 21st April 2009 representatives from
the UK Thalassaemia Society met UnderSecretary 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
A word from our President . . . . . . . . . . . 2
Events. . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12
Latest News. . . . . . . . . . . . . . . . . . . . . . . . . 3
Patient News. . . . . . . . . . . . . . . . . . . . . . . 14
Medical News . . . . . . . . . . . . . . . . . . . . . . 6
Office News. . . . . . . . . . . . . . . . . . . . . . . . 15
News from around the World. . . . . . . . 10
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.
UNITED KINGDOM
THALASSAEMIA SOCIETY
A Charity Organisation
Registration Number: 275107
19 The Broadway
Southgate Circus, London N14 6PH
Telephone: 0208 882 0011 • Fax: 0208 882 8618
Email: [email protected] • www.ukts.org
A word from our President
Our
Mission
Statement
■ To be the definitive
source of information,
education and research
for those affected
by, or working with
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
2
www.ukts.org
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 Michael
President
UK Thalassaemia Society
The UKTS
Management
Committee
Mike Michael
President
Dr Christos Sotirelis
Vice-President
Menuccia Tassone
Secretary
George Constantinou
Treasurer
Philip Agathangelou
Assistant Treasurer
Costas Kountourou
Committee
Bharat Nathwani
Committee
latest news
NOTICE TO ALL
UKTS MEMBERS
The Annual General Meeting (AGM)
of the UK Thalassaemia Society will
be held on :
Date: Tuesday 7th July 2009
Time: 7.30 pm
Venue: UK Thalassaemia Society office
ALL MEMBERS WELCOME
Please contact the office for further details
Swine Flu – Some Practical Advice
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.
�■ 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
Thalassaemia Matters
...continuing the fight against Thalassaemia
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.
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
3
latest news
Genetic Interest Group Conference 14th May 2009
Innovative Therapies and Rare Diseases. Are we there yet?
GIG staff Helen Parr, Melissa Hillier, Ben
Francis and Krystle Kontoh
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
4
www.ukts.org
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
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
Venue of the 19th ICOC:
Bath University, Bath, United Kingdom.
Saturday 12th December:
Scientific sessions and Social Programme
Deadlines for Abstract
submission and Early
Registration:
Sunday 13th December:
Scientific sessions and Social Programme
Abstract Submission Deadline:
14th September, 2009.
Early Registration Deadline:
1st September, 2009.
Monday 14th December:
Morning – Free time and Social
programme
Afternoon – Scientific sessions
Evening – Closing
Dates of the Conference:
The 19th ICOC will be organised in Bath
University on the 11-14th December 2009.
Preliminary Programme
Friday 11th December:
Early Evening: Registration
Evening: Keynote Lectures
Late Evening: Welcome Reception by the
Mayor of Bath
Thalassaemia Matters
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
...continuing the fight against Thalassaemia
5
medical news
Diabetes in Thalassaemia
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 “prediabetic syndrome” can be a precursor to
developing diabetes.
Diabetes Diagnosed:
Dr Maria Barnard, Lead Consultant in
Diabetes, The Whittington Hospital NHS
Trust, London & Honorary Senior Lecturer,
University College London Medical School
Dr Maria Barnard, Lead Consultant in
Diabetes, The Whittington Hospital
NHS Trust, London & Honorary Senior
Lecturer, University College London
Medical School (email: maria.barnard@
whittington.nhs.uk)
Diabetes – The Global Challenge
Diabetes 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
6
www.ukts.org
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
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.
Halle Berry
What symptoms does diabetes cause?
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
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
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)
Normal artery
Measure
Fructosamine
[*HbA1c
Blood glucose before meals
Blood glucose after meals
Smoking status
Exercise
Body mass index
Artery narrowed by cholesterol deposits
Target
< 299 umol/l
< 6.5 %]
4 – 7 mmol/l
5 – 8 mmol/l
Non-smoker
Daily
20 – 25 kg/m2
Measure
Blood pressure
- with nephropathy
Aspirin if > 40 years of
age or cardiovascular risk
Total cholesterol
LDL cholesterol
Triglycerides
Target
< 130 / 80
< 125 / 75
75 mg daily
< 4.0 mmol/l
< 2.0 mmol/l
< 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.
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?
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
Diabetes can damage the small blood
vessels supplying nerves, causing loss of
Ideally, with the best iron chelation therapy,
iron overload diabetes will not occur in
thalassaemia. This is the great future hope.
Thalassaemia Matters
...continuing the fight against Thalassaemia
Continues on page 8 ➡
7
medical news
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.
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
Breakfast insulin
clinic. Here members of both diabetes and
Swedish Orphan A5 landscape ad 4/2/09 09:27 Page 1
The basal/bolus insulin regimen is the other thalassaemia teams work together with
main option. A very slow-acting (basal)
patients. This enables staff to manage all
insulin is given once a day (e.g. Glargine
the complex needs of these two conditions
{Lantus}, Detemir {Levemir}) to give
and it allows staff to learn from each other
background 24 hour insulin cover. Then,
and provide a consistent approach.
every time a person eats carbohydrate
they inject a rapid acting (bolus) insulin
Conclusion
(e.g. NovoRapid, Humalog), which controls
Managing diabetes is one of the greatest
the blood glucose rise from the food. This
challenges that a person with thalassaemia
requires multiple daily
injections
face. Training
people
to self-manage
Swedish
Orphan of
A5 insulin.
landscape adcan
4/2/09
09:27 Page
1
Also, to get good blood glucose levels,
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
SWEDISH ORPHAN INTERNATI
www.swedishorphan.com
SWEDISH ORPHAN INTERNATIONAL
www.swedishorphan.com
...quality care for the rare disease patients!
8
www.ukts.org
medical news
Thalassaemia Matters
...continuing the fight against Thalassaemia
9
news from around the world
Singapore 2008 Conference
Report by John Wilson, Thalassaemia Society of Victoria
John with friends (L to R) Antonella Alvaro, Eugenia Pantazis and Louisa Di Pietro.
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
10
www.ukts.org
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
news from around the world
Thalassaemia Centre Inaugurated in Baku
By Nailya Guliyeva, President of the Azerbaijan patient society “Savab Dunyasi” and
Board Member of the Thalassaemia International Federation
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.
My Life as a Thalassaemia Patient in Mombasa
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.
Thalassaemia Matters
...continuing the fight against Thalassaemia
Love from Mohammed Bachani
11
events
Running the Marathon for
Thalassaemia
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.
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
SAVE THE DATE!
UK Thalassaemia Society
Dinner Dance
2009
DATE: Saturday 21st November 2009
VENUE: The Regency Banqueting Suite
113 Bruce Grove, London N17 6UR
MUSIC: Dancing to the music of Diastello
Contact the UKTS office for further details
12
www.ukts.org
events
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
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-
Thalassaemia Matters
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.
...continuing the fight against Thalassaemia
13
patient news
My Birthday Bash
By Tanver Khan (Bradford)
Tanver (with his son Abdur-Rehman) is
presented with a birthday gift by Mrs Zanib
Rasul of NEBATA
Dear Friends
It 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 AbdurRehman was mingling with the young girls
and showing me up – not bad for a twoyear-old – he’s one to watch out for girls!
From Naples to Hertfordshire
By Roberto Raddi
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
14
www.ukts.org
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.
Roberto getting used to English customs.
office news
Recent Events
DONATIONS
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
Mike is presented with a donation from the MPGSA by the President of Cyprus
Demetris Christofias
50.00
Lohana Ladies of North London
650.00
Met Police Greek Staff Assoc
250.00
T. Perry
50.00
•The Met Police Greek Staff Assoc Dinner 30 April 2009
— Attended by UKTS President Mike Michael
Ms D Ptohopoulos
50.00
•Thalassaemia International Federation Board Meeting &
Workshop, Damascus, Syria 29 April – 3 May 2009
— Attended by UKTS Vice-President Dr Christos Sotirelis
Dr R.P. Tahalani
•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
Thalassaemia Matters
Square Conduct Lodge
100.00
55.00
UKTS Welcomes
NEW‑MEMBERS
Annual
Ms Gulcan Birdane
Mr Roberto Raddi
Life
Ms Roanna Maharaj
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.
...continuing the fight against Thalassaemia
15
membership application form
UK Thalassaemia Society, 19 The Broadway, London N14 6PH
Charity Reg No. 275107
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
Your Personal Details
Contact Details
Title (Mr/Mrs/Miss/Ms/Other):
Telephone:
First Name(s):
Home:
Surname:
Mobile:
Address:
Fax:
Email:
Are you a:
Post Code:
Occupation:
Patient
Parent/Relative
Healthcare Professional
Association
Other (Please state)
Ethnic Origin:
(Optional)
Membership Required
ANNUAL (£10.00)
(please tick)
LIFE (£100.00) (Please make your cheque payable to U.K.T. Society)
If you are a patient or parent of a patient please complete the section below
Patient’s
Name(s):
Consultant’s
Name:
Date of Birth:
Consultant’s
Telephone:
Sex:
Male
Female
GP’s Name:
Type of thalassaemia: (e.g. Major, Intermedia, Haemoglobin H etc)
Address:
Hospital
where‑treated:
Telephone:
Address:
Blood Transfused
Whole
Chelation
Desferal
Transfusion
Frequency:
OFFICE USE: Date Paid
Units received at
each transfusion
Receipt No.
www.ukts.org
(please tick)
Washed
Frozen
Filtered
(please tick)
Deferiprone
Blood
Type
Approval Date
Desferal & Deferiprone
Designed and printed by Orion Design & Print Ltd. Tel: 020 8351 3222 Email: [email protected]
Work: