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OUR BLOOD
September 2013
3FACTSABOUT YOUR BLOOD
FURTHER AWARNESS IS VITAL FOR DIAGNOSIS
Ex-footballer Geoff Thomas talks about his battle against leukaemia and how he overcame the odds of survival
PH
OT
O: P
RIV
AT
EAN INDEPENDENT SUPPLEMENT DISTRIBUTED WITHIN THE GUARDIAN ON BEHALF OF MEDIAPLANET WHO TAKE SOLE RESPONSIBILITY FOR ITS CONTENTS
RARE CONDITIONS The need for accurate diagnosis and support
BLOOD PRESSURE How to minimise the risks
of complications
A spotlight on
2 · SEPTEMBER 2013 AN INDEPENDENT SUPPLEMENT BY MEDIAPLANET
A bright future ahead
WE WON’T STOPUNTIL WE’VE BEATENBLOOD CANCER
30,000 people are diagnosed with a blood cancer each year.
We won’t stop until every single one of them survives.
To find out more visit beatingbloodcancers.org.uk or call 020 7504 2200
“As President of the British Society for Haematology I am delighted to support this campaign to raise awareness of blood conditions,” says Professor Graham Jackson.
Many are aware of what hap-pens to people who have prob-lems with their heart, lungs, liver and kid-
neys but are not as informed about how important it is to have healthy blood and bone marrow.
The blood serves the vital func-tions of transporting oxygen from the lungs to the rest of the body, fi ghting infection and ensuring that blood clots form in the right place at the right time. When the blood is unhealthy, patients can experience breathlessness, se-vere life threatening infection and either excessive bleeding or clots forming in the wrong place and thrombosis.
Disorders of the blood can be transient and benign or severe, malignant and life threatening. We are keen to raise awareness of asso-ciated diseases.
We are also keen to trumpet the very signifi cant improvements in treatments for serious blood dis-orders. One very important devel-opment is that the vast majority of children with leukaemias are now
cured, as are the vast majority of pa-tients with Hodgkin’s disease.
Haematology has introduced the closest thing to the ‘magic bullet’ in the form of tyrosine kinase inhibi-tors to treat chronic myeloid leukae-mia. Haematology has always been at the forefront of stem cell technol-ogies, and we have developed stem cell transplantation into a vitally im-portant therapy for patients with se-vere haematological disorders. Stem cell transplantation has been used to develop and harness the immune system to destroy cancer cells and keep patients in remission.
There is still much to do, howev-er, as there are still many patients who suffer from significant ill health or even die from their hae-matological disorder — sometimes because the disease is recognised too late in the course of the illness to save the patient.
The picture remains very excit-ing with many new excellent tar-geted anti-cancer agents becom-ing available and we at the British Society for Haematology are keen to promote education and research around disorders of the blood and bone marrow, to continue to im-prove the outcome for our patients.
Mediaplanet contact information: Phone: +44 (0) 203 642 0737
E-mail: [email protected]
OUR BLOOD 3RD EDITION, SEPTEMBER 2013
Managing Director: Chris EmbersonEditorial and Production Manager:
Faye GodfreyBusiness Developer: Sara Downes
Responsible for this issueProject Manager: Katie Dolamore
Phone: +44 (0) 203 642 0737E-mail: [email protected]
Professional contributors:Professor Graham JacksonProfessor Gareth Beevers
Dr Graham CollinsDr Anne Yardumian
Professor Stephen O’Brien
CHALLENGES
@MEDIAPLANETUK
EDITOR’S PICK
Page 9Maria Walker shares her experience of living with von Willebrand disease
Professor Graham JacksonPRESIDENT, THE BRITISH
SOCIETY FOR HAEMOTOLOGY
“One very important development is that the vast majority of children with leukaemias are now cured, as are the vast majority of patients with Hodgkin’s disease”
*patients surveyed July 2013
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Blood pressure is the force exerted by the blood on the inner walls of the ar-teries, as a result of the pumping ac-tion of the heart. It is written as two numbers, such as 120/80 (120 over 80). A blood pressure below this value is normal, but readings above 120 over 90 may indicate high blood pressure.
“High blood pressure, or hyperten-sion, puts extra strain on the arteries and heart, leading to heart attacks and strokes,” says Gareth Beevers, Professor Emeritus at the Universi-ty of Birmingham, and a trustee of Blood Pressure UK. “Therefore, keep-ing blood pressure as close to nor-mal as possible reduces the risk of these conditions.”
Silent killer“The problem is that hypertension doesn’t cause any symptoms until se-rious damage is done,” continues Pro-fessor Beevers. “That’s why it is called the ‘silent killer.’
“The only way to prevent this from happening is to have your blood pres-sure checked regularly by a doctor.
How often depends on your blood pressure. If it is normal, you should have it checked every two years. If it is above 140 over 90, another check will be necessary a few days later, and then again the following week. And if it’s still above 140 over 90 you might need treatment. If your blood pressure is below normal but near to 140 over 90, you need a check every few months.”
Self-monitoring “In between appointments with a doctor, you can measure your blood pressure with a home monitor,” adds Professor Beevers. “Choose one that has been ‘clinically validated’ and is listed on the British Hypertension Society website. It will have a cuff that fi ts around your arm and will measure blood pressure at the upper arm. Ask a doctor how to use it cor-rectly, for example, you shouldn’t talk while taking your readings.”
Other recommendations include always using the same arm, placing the cuff on the arm at the heart’s level, and not taking readings after drinking
LORENA TONARELLI
Question: What can be done to keep blood pressure under control and minimise the risk of associated heart attacks and strokes?Answer: Monitor your blood pressure regularly and follow a healthy lifestyle.
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* World Health Organization, May 2012, World Health Statistics report 2012, Geneva Switzerland, www.who.int/gho/publications/world_health_statistics
ONE IN THREE ADULTS* HAS HIGH BLOOD PRESSURE
Keeping your blood pressure in check
NEWS
coff ee or eating. It’s best to take three readings every time, and to discuss them with a doctor.
Professor Beevers says: “People can prevent hypertension in the fi rst place by cutting down on salt, eating more fruit and vegetables, drinking less alcohol and exercising regularly.” These simple changes are also key for those with the condition. In addition, these individuals can benefi t from prescription medication, so it is im-portant to seek professional advice.
“Modern drugs are more eff ective and safer than old ones,” says Pro-fessor Beevers. “We have uncontro-versial proof that they save lives.
“In recent years, we have seen a reduction in strokes and heart at-tacks in this country, partly as a re-sult of improved blood pressure con-trol. So, increasing public awareness of hypertension and its preven-tion and management is important and worthwhile.”
8.5 MILLION PEOPLE ARE DIAGNOSED
WITH HIGH BLOOD PRESSURE IN THE UK
1FACT
Gareth BeeversEMERITUS PROFESSOR
OF MEDICINE, THE
UNIVERSITY OF
BIRMINGHAM AND
TRUSTEE OF BLOOD
PRESSURE UK
“The problem is that hypertension doesn’t cause any symptoms until serious damage is done”
An estimated 16 million people in the UK suffer from high blood pressure. Thats
SEPTEMBER 2013 · 5AN INDEPENDENT SUPPLEMENT BY MEDIAPLANET
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Sickle Cell Disease, a genetic condition affecting the shape and efficacy of our oxygen-carrying red blood cells, is the most common inherited blood condition in England and globally.
■ In the UK, it affects
between 12,500 – 15,000 people
■ It occurs in 1 in 2,000 new
births annually
■ A further 250,000 - 300,000 are thought to carry
the sickle cell trait
■ Numbers are rising due to
population diversity
■ It is most common among
people of African and African-
Caribbean descent, but it can affect any ethnic group
■ Symptoms range from
crippling pain to severe or
chronic anaemia
■ Health implications
include strokes, acute chest
syndrome and organ damage
■ Antenatal screening is available for women whose
pregnancy is at risk of the
disorder
■ As yet there is no cure, despite research and
development
SOURCE: THE SICKLE CELL SOCIETY
Did you know?
but only a third are aware they have the condition as it is almost always symptomless. The only way to detect high blood pressure is through a blood pressure check
GET CHECKEDBlood Pressure UK highlight the importance of having regular checks for high blood pressurePHOTO: SHUTTERSTOCK
1 in 3adults
6 · SEPTEMBER 2013 AN INDEPENDENT SUPPLEMENT BY MEDIAPLANET
Graham Collins, Consultant in Hae-matology at the Oxford Cancer and Haematology Centre, Oxford, and Trustee of the Lymphoma Associa-tion says: “Raising awareness is im-portant because lymphoma is the fi fth most common cancer in the UK, after breast, lung, bowel and prostate cancers. And the number of new cases diagnosed each year is in-creasing. Further, some lymphomas are easier to treat if detected early, which is more likely if people are aware of signs and symptoms.”
Diagnosis: the challengesLymphomas are cancers of the lymphatic system, whereby the lymphocytes (a type of white blood cell) are ‘out of control’ and can divide in an abnormal way or not die off when they should.
“The condition can be straightfor-ward to diagnose, if it presents with lumps in the neck, armpits or groin,” says Dr Collins. “But sometimes the lymphoma develops in the chest, abdomen or bone marrow, where it can’t be felt. There may be non
specifi c symptoms like tiredness, night sweats and weight loss, which can be overlooked.Many other symptoms can occur, like cough or diarrhoea. These can initially look more like other illnesses, so diagno-sis may take some time.”
Further research is vital“The good news is that lymphoma is a treatable and, in many cases, a curable condition,” continues Dr Collins. “We can cure about 80
per cent of patients with Hodg-kin lymphoma, the most com-mon type in teenagers and young adults, and about 60 per cent of patients with high-grade non-Hodgkin lymphoma.
“Immunochemotherapy, which combines chemotherapy drugs with antibody drugs, is the cor-nerstone of treatment and the biggest advance of the last decade. However, promoting research into new treatments is absolutely vi-tal. There are many exciting drugs being studied that can target the underlying biology of the lym-phoma and be tailored to increas-ingly specifi c patient groups.”
Ongoing research eff orts and in-creased awareness can make a dra-matic, positive diff erence in terms of saving lives. That’s why they matter.
For information and support visit
www.lymphomas.org.uk or call Freephone
0808 808 5555
Search ‘blood’ on Patient.co.uk patientuk@patientuk
8 million people a month trust our health informationis the most common type ofanaemia worldwide
Taking your iron tablets with orange juicewill help your gut absorb them.
Iron deficiency
This week is Lymphatic Cancer Awareness Week. The initiative aims to raise awareness of lymphoma and to promote research into new therapies for it, to improve patients’ quality and duration of life.
Saving lives through awareness and research
LORENA TONARELLI
INSPIRATION
“When I was 16 I began to feel tired and developed a chesty cough. My GP put me on antibiotics but they didn’t help. Eventually I couldn’t keep food down and rapidly lost weight. My family thought I had an eating disor-der — even I wondered if I did.
“After fi ve months of my symptoms worsening, Mum took me to A&E. I had a chest X-ray which showed that my left lung had collapsed. I was also found to have swollen lymph nodes above my collarbone and in my neck and armpits, so I had a lymph node biopsy.
“I was diagnosed with advanced Hodgkin lymphoma. I had large lymph nodes in my chest, which were causing my left lung to collapse. When lymphoma was fi rst mentioned, I didn’t understand what it meant as I’d never heard of it before — this was probably the scariest time for me.
“During my chemotherapy and ra-diotherapy I lost my hair, was sick, lost even more weight and had high tem-peratures, infections and nerve prob-lems. But it was all worth it in the end — my hair’s grown back and I’ve been in remission for over two years.”
My story
Rachel BithellDiagnosed with Hodgkin
lymphoma aged 16
Dr Graham CollinsConsultant in Haematology, Oxford
Cancer and Haematology Centre and
trustee of the Lymphoma Association
AROUND 1 IN 5 PEOPLE DIAGNOSED WITH
HODGKIN LYMPHOMA ARE UNDER THE
AGE OF 24
2FACT
Delete Blood Cancer UKWe launched in February with a key aim: to increase the pool of lifesaving stem cell donors.
We’re calling on all health professionals to help us to achieve our aim by encouraging more people to sign up to donate some stem cells.
As you probably know, a stem cell donation is often the only successful lifesaving treatment if a perfect match is found. With this in mind, we believe that not one single person suffering with blood cancer should die because of a lack of a match.
Be part of our nationwide stem cell donor
recruitment initiative. Together we really
can save lives.
Find out what you can do: Twitter: @DeleteBC Facebook: www.facebook.com/deletebloodcanceruk
Delete Blood Cancer UK Charity no: 1150056 T 020 3176 7620 [email protected]
www.deletebloodcancer.org.uk
8 · SEPTEMBER 2013 AN INDEPENDENT SUPPLEMENT BY MEDIAPLANET
Beta thalassaemia major (BTM) is an inherited blood disorder in which the body does not make properly-func-tioning red blood cells. It occurs when genes fail to code for a protein in our haemoglobin, the iron-rich molecule found in red blood cells.
Dr Anne Yardumian, consultant haematologist, explains: “We are born with foetal haemoglobin. With-in the first year of life we switch to making adult haemoglobin. Babies with thalassaemia don’t switch, and develop severe anaemia, which puts them at risk of complications such as heart failure. Early diagnosis is there-fore crucial. All babies in England are tested at about five days of age. Those
with BTM need blood transfusions for life, usually once every two to four weeks. This keeps their haemoglo-bin near normal, allowing for a good, healthy existence.”
Preventing iron toxicityBlood transfusions are generally safe and the risk of infections such as hepatitis C is minimal, as all donated blood is carefully screened. But, treat-ment with chelating drugs is needed to prevent toxic iron overload due to frequent blood transfusions.
“Like any medication, chelating drugs can have side effects and re-quire close patient monitoring,” says Dr Yardumian. “Yet they are vital. Be-fore they became available children with BTM would die in their teens from iron damage.”
Awareness needed“The problem with thalassaemia is that there are many carriers,” contin-ues Dr Yardumian. “These have the disorder’s gene but are healthy. If two have a baby together, there is a one in four chance of it having BTM. Great-er awareness would allow carriers to make informed decisions. Some have a termination if they find out their ba-by will have thalassaemia. Some don’t. Others opt for in vivo fertilisation
techniques that allow selecting em-bryos without the BTM gene before implanting them in the womb.”
Thinking positivelyGabriel Theophanous, President of the UK Thalassaemia Society, adds: “Sadly, there is a taboo about the con-dition. We must spread the message that, with appropriate treatment and a healthy lifestyle, people with thalas-saemia can live a normal life.”
Theophanous was born with be-ta thalassaemia major. In May, he ran his second Virgin London Marathon, beating his personal record by an amazing 30 minutes.
“The condition has made me stronger and given me a positive out-look on life. Transfusions are a minor inconvenience; they take only about three hours and I can have them af-ter work. Of course, it’s not all plain sailing. It’s normal to feel low some-times. I find that the best way to cope is talking with another patient, who can fully understand what I am going through. We share our experiences and motivate each other.
“It’s all about thinking positively — you can do anything you want to do.”
LORENA TONARELLI
Dr Anne Yardumian Consultant haematologist
When science and passion connect, innovation happens.Collaboration with people inside and outside of Novartis enables us to explore biomarkers and targeted drug development focused on individual patients. These connections accelerate the development of medicines with potential for greater patient benefit. Together, we discover innovative medicines that transform the way people live with cancer and targeted diseases.
www.novartis.co.uk
Thalassaemia: living life to the full
INSPIRATION
■ Question: How important are early diagnosis, the right treatment, and a positive attitude in the management of thalassaemia?
■ Answer: They are crucial, as they enable people with the condition to enjoy a full and healthy life.
LIVING A NORMAL LIFEGabriel Theophanous, President of the UK Thalassaemia Society, was born with beta thalassaemia major. He says that the condition has given him a positive outlook on life and this year he completed his second Virgin London MarathonPHOTO: PRIVATE
SEPTEMBER 2013 · 9AN INDEPENDENT SUPPLEMENT BY MEDIAPLANET
Our heritage, your future
Sobi has one goal – to ensure that people with haemophilia live the lives they wish for. Our innovation will be based on taking the time to understand what that looks like. And then acting on that understanding together with you.
With more than 30 years’ experience in the field, we are coming back to our heritage now with fresh eyes. This, coupled with our commitment, drives us towards innovation for improved treatment for haemophilia that is aligned with your goals.
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Sobi is an international specialty healthcare company dedicated to rare diseases. Our mission is to develop and deliver innovative therapies and services that improve the lives of patients.
Despite common belief, bleeding disorders do not only affect men. Some 295,000 women in the UK are thought to have one called von Willebrand disease. Of these women, only about 5,000 have received a diagnosis.
Maria Walker has the condition. She says: “I bruise easily and deal-ing with cuts can be a challenge, but this does not prevent me from leading a normal, active life. I just avoid putting myself in unneces-sarily dangerous situations, and I always ensure that I take preven-tative measures wherever I go, so as not to interfere with my work-ing life, for example, carrying suf-ficient sanitary protection for ex-ceptionally heavy periods. When I feel it’s required, I also make the people around me aware of my condition, but in a very matter of fact way. My message to other women with the condition? Live life to the full!”
Affecting about 6,000 people in the UK, haemophilia is a genetic condi-tion that reduces the blood’s ability to clot. It is caused by lack of clotting factors — substances that help change blood from liquid to solid after an inju-ry, thereby stopping the bleeding.
Symptoms include bruises and frequent joint bleeds accompanied by pain and stiffness. Their severity depends on the amount of clotting factors in the blood. Children born with less than one per cent of the normal amount suffer significant-ly, whereas those born with great-er amounts can be symptomless for years. The condition is usually de-tected by accident when they have a dental procedure or are injured and start to bleed heavily.
Who does it affect?Haemophilia mostly affects boys (but not exclusively). It is passed to them from one or both parents. Tests can help determine whether a child will have haemophilia. However, about a third of children born with the condi-tion have no family history of it.
Severe haemophilia, the most common form of the condition, is treated with regular intravenous injections of clotting factor. These usually start at under two years of age and continue for life. They can be given by parents at home and help prevent bleeds occuring, allowing children with haemophilia to run around, play football, and so on — just like any other child. As a result, the condition doesn’t really have a big impact on quality of life, which remains good.
Also, novel long-acting clotting factors are under study that can be injected once a week, instead of three times a week as current versions. They will be routinely available in the next year or two, leading to a fur-ther improvement in the quality of life for children with the condition.
Some parents find it difficult to come to terms with the fact that their child has a genetic illness, es-pecially if there is no family histo-ry of it. They may also find inject-ing their little ones a daunting task. But, once they recognise that this stops their children from bleeding and therefore suffering, they can cope with, and manage, the condi-tion quite well. So, educating par-ents about the importance of treat-ment is crucial.
LORENA TONARELLI
■ Question: Does a diagnosis of haemophilia mean that children with the condition will have a poor quality of life?
■ Answer: No – parents can help them grow happily and healthily, says nurse consultant for haemophilia, Kate Khair.
Educating parents about haemophilia is crucial
Spotlight
Maria WalkerLiving with von Willebrand disease
10 · SEPTEMBER 2013 AN INDEPENDENT SUPPLEMENT BY MEDIAPLANET
Stephen O’Brien, Professor of hae-matology at the Northern Insti-tute for Cancer Research of New-castle University, explains: “Un-like acute leukaemia, CML devel-ops slowly over time, often with-out symptoms or with non-specif-ic symptoms like tiredness, bruis-es, enlarged spleen or infections that don’t respond to treatment. So people may feel relatively well despite having the condition. This delays the diagnosis and con-sequently treatment initiation,
hindering a positive clinical out-come. Increasingly, we are pick-ing up CML just because people are having tests for other reasons. It is important that anybody who experiences unusual symptoms sees their GP as soon as possible.”
Huge leap forwardChronic myeloid leukaemia is di-agnosed in about 600 people each year in the UK. It is characterised by over proliferation of immature white blood cells in the bone mar-row. These eventually prevent the production of healthy white blood cells, which help the body fight infections.
Early diagnosis and treatment initiation are worthwhile. With modern drugs the prognosis is ex-tremely good and most patients
can now expect to have an almost normal life span.
“Back in the 90s, CML was mainly treated with injections of interferon, which is associat-ed with a 5-year survival rate of about 45 per cent,” says Professor O’Brien. “Modern tyrosine-kinase inhibitors, which have been the standard treatment for CML since 2003, has a 5-year survival of about 85 per cent. So we made a huge leap forward in the last ten years, which is unprecedented in CML.”
Most patients in the advanced stages of CML eventually need chemotherapy, either with tablets or injections. A bone marrow trans-plant can help control the con-dition long term, but is only suitable for a limit-ed number of patients.
Improving cure ratesProfessor O’Brien is the chief in-vestigator of the SPIRIT 3 study, a randomised trial involving thou-sands of patients from hundreds of clinical research sites across the UK, which aims to evaluate and compare the efficacy of new-er drug treatments for chronic myeloid leukaemia.
He says: “The evidence so far seems to indicate that, while they do not provide any signif-icant improvement compared with existing treatments, in terms of survival, they could po-tentially improve our ability to stop treatment in the long term
and cure the condition.”
LORENA TONARELLI
AWARENESS FOR CML IS KEY TO SURVIVAL
Stephen O’Brien Professor of haematology
INSPIRATION
Question: What are the major challenges of chronic myeloid leukemia (CML) and how can they be overcome?
Answer: Symptoms may be absent or not obvious, so increased awareness may prompt people to seek a diagnosis, increasing their chances of recovery.
OVERCOMING THE ODDSGeoff Thomas was given just three months to live, but 10 years on, he is still going strong and using his experience to help others PHOTOS: PRIVATE
AROUND 600 PEOPLE IN THE UK ARE DIAGNOSED WITH CML EVERY
YEAR
3FACT
Former England and Crystal Palace footballer, Geoff Thomas, was diagnosed with blood cancer in 2003. He talks about his experience and how he survived against the odds.
“I had just retired from football and was getting used to my new life when I was given the shock-ing news that I had leukaemia. I was told I only had three months to live. I was devastated.
“That was in 2003, yet here I am 10 years on from that dreadful di-agnosis. This is largely thanks to my sister. She proved to be a bone marrow match and a transplant in 2004 quite literally saved my life.
“I now work closely with the charity Leukaemia & Lymphoma Research to increase awareness of blood cancer and raise money. Every year around 30,000 people are diagnosed with a blood cancer in the UK and up to half of these people do not survive because the right treatment isn’t available.
“New treatments are continu-ally being developed in research laboratories, but only about six out of 100 blood cancer patients are entered into clinical trials to test these treatments. This means many are denied poten-tially life-saving drugs. Being di-agnosed with leukaemia was not
part of my game plan, but since my diagnosis I have met hun-dreds of inspiring people af-fected by blood cancer. While I am no longer a footballer, I still chase a goal: that one day we will beat blood cancer.”
SOURCE: LEUKAEMIA & LYMPHOMA RESEARCH
SEPTEMBER 2013 · 11AN INDEPENDENT SUPPLEMENT BY MEDIAPLANET
Thousands of disorders fit the definition of “orphan diseases” established by health authorities around the world. Each orphan condition is rare, affecting hundreds or thousands of people — a far cry from the millions of patients with high blood pressure or type 2 diabetes. The collective impact on public health is immense, however, and the vast majority of rare diseases still lack effective treatment options.
After decades of neglect, research on rare diseases has exploded in recent years. According to a recent report by the Institute of Medicine, a branch of the US National Academy of Science, “scientific and technological advances on many fronts — combined with supportive public policies and private initiatives — offer opportunities to intensify research on the causes and mechanisms of
rare diseases and to reduce the number of rare diseases with no or inadequate means of prevention.”1
Novartis is a leader in the development of orphan drugs with a particular focus on blood disorders ranging from chronic myeloid leukaemia (CML) to iron overload in people with sickle cell disease, thalassaemia and myelodysplastic syndrome to myelofi brosis — a blood cancer that affects the bone marrow and has symptoms that can severely compromise patients’ quality of life.2
What can be achieved by a focus on such rare disorders is best illustrated in chronic myeloid leukaemia. A diagnosis of CML would have meant an average life expectancy of 6-7 years3, however today, CML has truly become a chronic disease that is managed through medicines taken in tablet form with most patients now being able to look forward to a longer term prognosis with at least three out of four patients alive at 10 years.3
Novartis is taking the unprecedented step forward in its ongoing commitment to the CML community with the launch of a clinical trial programme that evaluates the potential for patients with a particular form of CML to remain in remission after stopping drug therapy. It should however be emphasised that stopping treatment is not a clinical recommendation and should only be considered in the context of a well conducted clinical trial study.
“This type of trial raises important questions for patients and clinicians alike as it requires patients to stop taking a proven treatment that is well known with largely manageable side eff ects and move towards a situation where they can live treatment free; but there is a need to acknowledge the risk of a recurrence of the disease. For each patient this is a very individual decision and not every patient will want to go down that route. However, these trials off er the hope of treatment free remission to
many patients who are currently on long-term treatment” said Sandy Craine of CML Support.
“The ability to live drug-free without relapse of leukaemia is an exciting prospect for patients, nurses and physicians alike. It is important to note that these trials are designed for patients who have responded exceptionally well to treatment and will inform us as to how feasible this approach will be for this select group. The proportion of patients in this group increases with time on treatment so is an added incentive for the majority of patients to ‘keep taking the tablets’!” said Professor Jane Apperley of the Centre for Haematology, Imperial College London.
Novartis remains committed to improving treatment options for patients with CML and is hoping to bring many new treatments to patients with rare diseases in the next few years.
A step forward in rare disease research
PHOTO: SHUTTERSTOCK
COMMERCIAL FEATURE
1IOM (Institute of Medicine), 2010, Rare Diseases and Orphan Products: Accelerating Research
and Development, Washington DC: The National Academies Press. (Page 18)
2Mesa RA, Schwagera S, Radia D, et al. The Myelofi brosis Symptom Assessment Form (MFSAF):
an evidence-based brief inventory to measure quality of life and symptomatic response to
treatment in myelofi brosis. Leuk Res. 2009;33:1199-1203.
3Leukaemia and Lymphoma Research. Chronic Myeloid Leukaemia. https://leukaemialym-
phomaresearch.org.uk/sites/default/fi les/cml_august_2013.pdf. Last accessed August 2013
Date of preparation: August 2013 / TAS13-C060
