Blood Health IE Q3 2019 by Mediaplanet UK&IE

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DR SIOBHAN GLAVEY Clinical trials allow patients to access treatments that have not yet been approved for use within Ireland. » p4

DONAL BUGGY, IRISH CANCER SOCIETY Almost two out of every three carers themselves received no support on how to carry out their role. » p6

DR MÁIRÍN RYAN, HIQA A new assessment oﬀers hepatitis C testing to all people in Ireland born between 1965 and 1985. » ONLINE

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Greater access to new, targeted therapies and innovative trials offers great hope to Irish haematology patients DR PHILIP MURPHY Head of Haematology Department, Beaumont Hospital and Clinical Director, Blood Cancer Network Ireland

‘The people who are crazy enough to think they can change the world are the ones who do’ - Steve Jobs. The prognosis for patients in Ireland with blood cancers continues to improve dramatically because of greater access to new, targeted therapies and to important new, collaborative clinical trials.

here are also many dedicated scientists and doctors in this country who are doing cutting edge, laboratory research into a wide variety of haematological conditions, including blood cancers and bleeding and thrombotic conditions. Blood cancer clinical trials into myeloma Blood Cancer Network Ireland (BCNI) is a collaborative national ne t work , s e t up a nd le d by Professor Michael O’Dw yer of University College Galway. BCNI runs early phase blood cancer clinical trials, as well as providing a national biobank for blood cancers and promoting blood cancer registration. Myeloma is a cancer of plasma cells, which usually manifests with severe bone disease and/or kidney failure. An antibody therapy that directly targets plasma cells has recently been developed. A re c ent pha se 1 s t udy by

BCNI has just been published in a prestigious American journal, which shows that addition of this antibody therapy to standard therapy for myeloma results in better outcomes. Current trials r un by BCNI include the use of an E-selectin inhibitor in patients with relapsed acute myeloid leukaemia. In an ex tremely impor tant development, Cancer Trials Ireland have recently joined the HOVON international cooperative group to participate in trials for acute myeloid leukaemia. Through this partnership, the use of targeted therapies in addition to chemotherapy is being trialled for the fi rst time, for a condition that has not seen any great improvement in survival rates in recent years. Innovative research into residual myeloma Dr Siobhan Glavey, Consultant H ae m at olo g i s t at B e au mont Hospital and Senior L ecturer at Royal College of Surgeons in

Ireland (RCSI), has had extensive research experience at the Dana Farber Institute in Boston, the leading centre for myeloma in the world. She is currently leading a national collaborative study to assess response of myeloma patients to therapy. Dr Glavey will use state-of-theart next generation sequencing to look for evidence of residual myeloma in patients, with the aim of fi ne tuning therapy for individual patients. In a fur ther impor tant research project, the Department of Haematolog y at Beaumont Hospital is collaborating with Dr Triona Ni Chonghaile and her team at RCSI to assess the response of patients’ myeloma cells to the combination of chemotherapy and a recently developed molecule which promotes cell death (apoptosis) by inhibiting BCL-2. Chimeric antigen receptor T-cell (CAR-T Cell) therapy CAR-T cells are patient T cells that

are genetically modified in the laboratory to become active against the patient’s cancer cells and then reinfused into the patient. Th i s exc it i ng new for m of immunotherapy is showing great promise worldwide in patients with leukaemia and myeloma who have not responded to conventional therapies. It is expected that CAR-T cell therapy will be available for the first time in Ireland later this year in the Children’s Hospital Crumlin and in St James’s Hospital, Dublin. Bleeding disorders Von Willebrand Disease is the most common inherited bleeding disorder, affecting both men and women equally. The f i rst recombi na nt von Willebrand Factor replacement has recently been licensed for use in Europe, thus reducing use of blood products to treat this condition. Dr Michelle Lavin and Professor James O’Donnell, of the National Coagulation Centre, St James’s

Hospital and Irish Centre for Vascular Biology, RCSI, had the distinction of publishing a review paper on the management of low von Willebrand factor levels in ‘Blood’, the most prestigious journal in haematology. In haemophilia, an X linked disorder affecting males, low levels of clotting factors VIII or IX lead to severe bleeding. Recent gene therapy studies are showing that higher levels of Factor VIII or IX can be maintained in the longer-term, holding out promise of cure for haemophilia. For haemophilia patients who have inhibitors and are therefore resistant to factor replacement therapy, the monoclonal antibody emicizumab has shown marked reduction in frequency of bleeding.

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Make your blood count Patients with Hereditary Haemochromatosis (HH) can now give blood and save lives, at any IBTS blood donation clinic nationwide, including mobile clinics.* For more information visit our website www.giveblood.ie or call 1850 731 137 *Subject to certain HH maintenance and normal blood donation eligibility criteria.

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A decade of progress improves outcomes for myeloma patients The outlook for patients with relapsed and refractory myeloma has never been better and even more new treatments are on the horizon. “Twenty years ago, the average life expectancy of someone diagnosed with myeloma was just three years,” explains Paul Browne, Professor of Haematology at Trinity College, Dublin. “Now people’s life expectancy is at least 10 years.” In fact, survival rates for patients with myeloma, a form of bone marrow cancer, have improved more rapidly than for most other cancers, according to statistics published by the Irish National Cancer Registry. While there remains no cure for this relapsing-remitting cancer, which recurs after periods of remission, a diagnosis no longer comes with the sense of fear it once did. With regular blood tests, doctors can track the disease more accurately enabling timely and effective treatment. “Although you may not be cured,

with the right treatment you can live well for many years. It’s more like living with a chronic disease, such as diabetes,” says Professor Browne. Advancements in myeloma therapy The i mproved out look c a n be attributed to the range of drugs now available to treat the condition. Over the last decade, there has been a move away from chemot herapy a nd stem cel l therapy in favour of new classes of drugs that can be used together to achieve higher success rates. These include proteasome inhibitors that block the development of proteasome within a cell, resulting in a protein build up that eventually kills the myeloma completely. There are also immunomodulatory drugs that work by killing myeloma cells directly, reducing

the blood supply to myeloma cells, preventing myeloma cells sticking in the bone marrow, and boosting a patient’s immune system. Another group of drugs added to the list are known as monoclonal antibodies. These antibodies are designed to work against certain proteins found in the body, and can be used to target and attack myeloma cells. They can be used at different stages of myeloma. All these treatments are highly targeted, improving success rates and reducing damage on surrounding healthy cells. They’ve also proven to be highly effective in patients with relapsed and refractory myeloma. Progress continues but requires further funding While access to these therapies is generally good, Professor Browne

PROFESSOR PAUL BROWNE Professor of Haematology, Trinity College, Dublin warns against complacency. “We do have a challenge with access to monoclonal antibodies and we also need to consider funding structures for developments that are coming.” he says. Professor Browne is specifically referring to CAR-T therapy, re c ent ly l ic en s e d for u s e i n adult patients with relapsed and refractory B-cell lymphoma and children with acute lymphoblastic leukaemia. The highly complex treatment involves collecting and using patients’ own white blood cells, modifying them to attack the cancerous cells and then putting the cells back into the body by means of a special transfusion. CAR-T therapy is not yet available to treat myeloma, but clinical trials are promising. “Within the next 12-18 months, we hope to see

CAR-T therapy licensed for use,” confirms Professor Browne. With advancements in technical knowledge, clinical developments and a spirit of col laboration, Professor Browne believes there is no reason why the current trajectory of progress can’t continue. And, if it does, perhaps that elusive cure won’t be a pipe dream for much longer. WRITTEN BY: KATE SHARMA

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How blood cancer affected me

Retired bank manager, Liam McGonagle, was enjoying his retirement. From golf days to spontaneous trips out, he made the most of life, so, when his shoulder started to ache in January 2019, he felt confident it would pass. “When the doctor told me I had myeloma, I thought he was talking about sun damage; I’d never heard of it before,” says Liam. The 61-year old from Ireland only went to see his GP after suffering from a chest infection. Within days, he was in his local hospital and had a biopsy to confirm the diagnosis. “A side effect of the myeloma was a negative impact on my kidney function, which I later discovered was a major concern for my haematology team.” He says: “I can’t remember those first few days, but when I came round and they showed me my chart I thought it was someone else’s!” I’d never been ill before Although Liam had regular checks for his blood pressure, he had never taken more than three days off sick in his career. “It was a huge shock,” he says. “The medical team were amazing though and I think we caught it just in time.” Multiple myeloma is often hard to diagnose because the symptoms are vague. Early treatment is key, as is positivity and determination from the patient. Liam set himself goals/milestones to focus on as his chemotherapy treatment commenced, with the big prize being stem cell transplant treatment, which is due to take place shortly. I have to really think about situations now While his condition has improved, Liam has had to go through a huge change. He says: “It’s hard not being able to just jump in the car any more, to attend football matches, concerts etc, as I have to be very aware of possible infections from being in crowds. Family and friends have been a tremendous support to me throughout my journey and, truthfully, without that support network, I am not sure I would be as far on as I now am.” Liam believes that, had he not had that chest infection, his myeloma diagnosis may have come too late. “I was just putting up with the shoulder, as you do. But now I see it as a warning light like you get in a car. And even if it goes LIAM MCGONAGLE away again – you should always get Patient and Multiple Myeloma a warning light checked out.” Ireland Ambassador WRITTEN BY: GINA CLARKE

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Lack of awareness and the indolent nature of early disease make this cancer more challenging to detect DR SIOBHAN GLAVEY MB PHD FRCPATH Consultant Haematologist, Beaumont Hospital and Senior Clinical Lecturer, Royal College of Surgeons in Ireland

Multiple myeloma is the second most common bone marrow cancer in Ireland, but of the 250 people who will be diagnosed with this condition in 2019, most of them will not have heard of it before.

his lack of awareness, along with the fact that this disease can be silent initially, means that, for some patients, complications of the disease have already occurred at the time of diagnosis. Multiple myeloma is a cancer that develops from bone marrow cells called plasma cells. These cells normally function as part of our immune system and make proteins needed to fight infection. In multiple myeloma, though, these cells make an unhealthy protein that can be detected using a blood or urine test, and this protein can build up in the blood and cause problems like kidney failure. ‘Multiple’ refers to the many sites of bone marrow that are affected, leading to cavities in bone that can be seen on scans, or to painful bone fractures. Back pain could be a symptom of bone disease Patients who need treatment for multiple myeloma have usually developed at least one of what are known as the CRAB criteria; C – high Calcium levels, R – kidney/ renal failure, A – Anaemia or B – bone disease. Bone disease can cause back pain, which is a common symptom in the general population, and therefore may not be attributed to multiple myeloma initially.

New treatments for multiple myeloma have improved survival The average age of diagnosis is 75, but one third of patients are under 65 years of age. Due to improvements in new treatments for multiple myeloma over the last 15 years, the outlook for patients has greatly improved. Currently in Ireland, survival rates for multiple myeloma are reaching best international standards, with over 50% of newly diagnosed patients living five years or longer. This is due, not only to new drug treatments, but due to their successful combination with autologous stem cell transplant for some patients. However, t h i s rem a i n s a n incurable disease and the vast majority of patients will eventually relapse and require further treatment, therefore new treatments and treatment strategies are needed. Clinical trials allow patients to access treatments that have not yet been approved for use within Ireland, and therefore would not otherwise be available to them. They are essential to the development of cancer treatment programmes and the advancement of cancer care. Due to the work of Blood Cancer Network Ireland and Cancer Trials Ireland, new treatments and drug combinations have been made

available to multiple myeloma patients in Ireland. But further investment is needed to attract clinical trials and make them available to the patients who will benefit most. Refinements in genetic testing for a personalised approach to treatment In recent years, it has become apparent from several studies that the fewer cancerous plasma cells present in the bone marrow following initial treatment, the longer the remission and survival for that individual patient. This is known as ‘minimal residual disease’ – MRD. We also know that certain genetic markers found in the plasma cells can help to predict survival. These biomarkers are specific to individual patients and might help us to identify which patients might benefit from which treatments and when. At Beaumont Hospital and Royal College of Surgeons in Ireland (RCSI), we will soon open a new study looking at a combination of MRD and a genetic test that incorporates 92 genes from plasma cells, to try to identify how patients will respond to treatment, and to learn more about predicting prognosis. We hope that in the future this will lead to personalised medicine for multiple myeloma patients in Ireland.

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Will immunotherapy New therapies will become ﬁrst line for boost poor relapsed ALL outcomes childhood ALL? AN INDEPENDENT SUPPLEMENT BY MEDIAPLANET

DR LARRY BACON Consultant Haematologist, National Allogenic Bone Marrow Transplant Unit, St James’s Hospital, Dubliin

PROFESSOR OWEN SMITH CBE Professor of Paediatric and Adolescent Medicine, University College Dublin

Intensive research and discovery over the last 75 years have pushed overall survival rates in childhood acute lymphoblastic leukaemia up to around 90% – but the cure has come at a cost.

ersonalised medicine is the key to reducing the long- and shortterm impact of cancer treatment on children with acute lymphoblastic leukaemia (ALL). That’s according to Professor Owen Smith CBE, Professor of Paediatric and Adolescent Medicine at University College Dublin, who says a combination of drug discoveries and risk stratification from the 1940s onwards resulted in event-free survival rates of 87% by the early 2000s. “But there is a price, and that’s the longand short-term side-effects. Multi-agent chemotherapy is not very nice as a significant number of these patients experience life-threatening toxicities. Then we started to notice there was a chronicity to survivorship as well: bone disease, heart disease, secondary cancers and neurotoxicity. “We want all of our patients to grow up and have their own children and to do all of those things that you and I can do,” he says. Personalised antibody therapies to tackle ALL That’s where new, personalised treatments such as antibody and chimeric antigen receptor T-cell (CAR-T) therapies come in. Developed thanks to our deeper understanding of the underlying biology of ALL, two new antibody therapies each target separate specific surface antigens on the leukaemia cells. CAR-T therapy genetically modifies the patients’ own T-cells and

returns them to the body to fight the cancer cells. Crucially, they do not appear to have the same long-term side effect profile as multi-agent chemotherapy. While the antibodies are currently only funded for relapsed or refractory disease and CAR-T therapy has not yet received the green light for funding, Professor Smith believes they will soon become the mainstay of first line treatment. “I firmly believe that, in the next five to ten years, we're going to be introducing antibodies and CAR-T upfront for standard risk patients. The only thing that's stopping us from doing that at the moment is the cost. Cost is the only barrier to CAR-T therapy becoming standard “But, as with new technologies, as they get better, the price plummets. It took 13 years and $3 billon to sequence the human genome in 2013, but I could sequence your genome tomorrow for €1,000,” he says. Ultimately, these new breakthrough treatments will change the landscape of ALL treatment, he concludes, pushing up survival while reducing toxicity. WRITTEN BY: GINA CLARKE Read more at healthnews.ie

Antibody and CAR-T cell therapies are using the body’s own immune system to fight acute lymphoblastic leukaemia (ALL) – and it’s changing the game for people with relapsed and refractory disease.

flurry of innovative new treatments are giving fresh hope to people facing relapsed or refractory lymphoblastic leukaemia (ALL). Two new antibody therapies were given National Centre for Pharmacoeconomics backing in June, and the green light for chimeric antigen receptor T-cell (CAR-T) therapy is hoped for in the coming months. It represents a huge step forward for previously difficult-to-treat patients, says Dr Larry Bacon, Consultant Haematologist at the National Allogenic Bone Marrow Transplant Unit in Dublin’s St James's hospital. “These drugs are hugely significant and represent a complete change in the landscape for the therapy of relapsed and refractory disease,” he says. One of the drugs binds to both T-cells and ALL cells to boost immune response, and the other binds to cancer cells before unleashing a fatal chemical pay load. Both treatments induce remission in up to 80% of patients. When the patient achieves remission and they can be considered for bone marrow transplantation, the treatments act as a bridge to a bone marrow transplant, which would not be possible with active disease. One of the new antibodies can lead to cytokine release syndrome or central nervous system toxicity, whereas veno-occlusive disease of the liver has been seen in patients treated by the other.

Another option for patients who previously have not been able to get into remission CAR-T therapy, which genetically modifies the patients’ own T-cells to recognise and destroy leukaemia cells, can be used in relapsed disease either pre- or post-transplant. Dr Bacon calls the treatments “a huge breakthrough.” “Because CAR-T cell therapy is so new, we don’t know how much impact these drugs will have on patient outcomes. But it will be significant, as they allow people who historically couldn't get into remission to have the option for another line of therapy.” Of course, these new treatments do come with potential side effects, most of which are linked to their method of ‘supercharging’ the body’s T-cells. CAR-T therapy can have significant side effects, as it generates a huge cytokine storm in the blood, says Dr Bacon. “But,” he added, “as we're learning more about these new drugs and how best to use them, they are becoming much safer.” WRITTEN BY: AMANDA BARRELL

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At Amgen, we believe that the answers to medicine’s most pressing questions are written in the language of our DNA. As pioneers in biotechnology, we use our deep understanding of that language to create vital medicines that address the unmet needs of patients fighting serious illness – to dramatically improve their lives. For more information about Amgen, our pioneering science and our vital medicines, visit www.amgen.co.uk

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Innovation and education in the haemophilia community BRIAN O’MAHONY Chief Executive, Irish Haemophilia Society

Prior to 2016 in Ireland, treatment for haemophilia consisted of regular intravenous infusions of clotting factor concentrates (CFCs). In the last three years, innovative treatment options have significantly developed.

n 2016, we began to use a new generation of extended half-life (EHL) recombinant CFCs where the half-life in the blood was extended. This allowed for less frequent intravenous infusion and higher trough levels of factor in the blood, providing more protection from bleeding into joints or muscles. New interventions see less bleeding or pain in patients Data demonstrates these new products resulted in less bleeding, less chronic pain in damaged joints, less acute pain and less frequent use of pain medication. There was also a significant reduction in the number of daily activities adversely affected by haemophilia. A new treatment, based on a monoclonal antibody has been licensed by the European Medicines Agency for use in those with inhibitors or antibodies to FVIII. It has seen a very significant reduction in bleeding – and avoids intravenous infusion as this therapy is injected subcutaneously. This treatment has also been licensed for haemophilia A without inhibitors and offers an excellent option to improve quality of life, reduce bleeding and reduce burden of treatment. Other treatments in clinical trials include an RNAi inhibitor and several tissue factor pathway inhibitors. These products interfere with the mechanisms that prevent too much coagulation in people without haemophilia and can be used for both types of haemophilia and for those with inhibitors. A cure for haemophilia could be close A cure for haemophilia, after more than 20 years of false starts and false hopes, finally looks to be close. Gene therapy clinical trials for both haemophilia A and B are showing impressive results. A record number of trials are underway. One trial demonstrated factor levels of 5% to 7% consistently for over seven years after a single infusion of gene therapy using an adeno-associated viral vector. Other trials are showing expression levels between 30% and 70%, with estimates of duration of expression varying from eight years to a lifetime after a single infusion. In Ireland, the community are beneficiaries of bespoke publications on novel therapies, educational videos, regular published updates and, most importantly, meetings and conferences where new treatment options are explained. This has resulted in a culture of innovation where we were the first country in the world to switch all people to EHL products and we have a high participation rate in clinical trials, leading to early adaption of novel approaches. We live in interesting times. Read more at healthnews.ie

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Living well with and beyond blood cancer DONAL BUGGY Head of Services, Irish Cancer Society

For people living with blood cancer, their treatment journey can take a number of paths. A diagnosis of blood cancer can bring fear and anxiety. But for many there is hope.

lood cancer is an umbrella term for cancers that affect the blood, bone marrow and lymphatic system. There are more than 140 different types of blood cancers, but most fall into three main groups: leukaemia, lymphoma and myeloma. It is little known that, together, blood cancers represent almost one in ten cancer cases, with more than 2,000 people across Ireland diagnosed every year. Blood cancer is the fourth most common cause of cancer-related death in Ireland but, thankfully, new treatments are improving survival rates and giving fresh hope to patients. Treatment options vary according to the aggressiveness of the cancer For people living with blood cancer, their treatment journey can take a number of paths, with many different treatment options now available. Acute, aggressive forms of blood cancer require immediate treatment, whereas slow-growing blood cancers can often be managed as a chronic condition. In cases where the cancer is not aggressive, patients can opt to be monitored by their doctor through ‘ac t ive sur vei l la nce’ – where regular check-ups and blood tests are carried out in lieu of immediate treatment. Whether treatment is required at a later stage depends on progression of the disease. For those who’ve had treatment, people with blood cancer may enter a period of remission where the cancer cells are completely or partially gone and they are monitored closely to ensure the cancer does not come back. Carers need support in giving mental health, dietary and exercise advice With cancer survival often comes additional needs. Research commissioned by Janssen Sciences Ireland last year to mark Blood Cancer Awareness Month found that nine in ten people with blood cancer felt that the care they received from a carer was of huge or high impor-

tance to them. But almost two out of every three carers themselves received no support on how to carry out their role. They identified areas of training and suppor t that would have helped them better prepare for their role including: mental health or mindfulness training (46%); information on diet and exercise (28%) and how to get the most out of an appointment (33%) with a healthcare professional. In August, we helped launch the the National Cancer Survivorship Needs Assessment, ‘Living With and Beyond Cancer in Ireland’. This report sets out the model of care needed for cancer survivors in Ireland. Surviving cancer can bring with it real challenges. While there is often an expectation people will return to ‘normal life’ after their treatment has stopped, the reality is often quite different. While many people return to good health, others experience ongoing issues for years afterwards. Short- and long-term effects of cancer treatment can affect every aspect of daily life. Physical issues include incontinence, weight changes, sleep disturbance and fatigue. Emotional and psychological effects include shock, distress and fear of recurrence, low self-esteem and depression. Social and intimacy issues include lack of support, fear of burdening family and friends, loss of identity and altered relationships. And financial issues include increased stress due to financial difficulties, lack of (or reduced) household income, and an increase in costs to help manage side effects of treatment. While working to deliver better State supports, the Irish Cancer Society is also committed to doing more to help survivors through our own services. We currently provide assistance through our Freephone Nurseline, Daffodil Centres, counselling service, Living Life group and educational events. The Needs Assessments recently published will help us enhance these services and develop new ones to support survivors and their families.

The I r i sh C a nc er S o c iet y ’s annual survivorship conference ‘Living Well With and Beyond Cancer’ provides information for patients and carers, and support on all aspects of the cancer journey. Each year the event features talks, workshops and seminars covering topics such as managing treatment side-effects, mindfulness, returning to work after cancer, diet and exercise, and living with advanced cancer. The conference is for cancer patients and survivors, those who care for them, healthcare professionals and support service staff. For questions or concerns about any aspect of cancer, you can also call the Irish Cancer Society’s Cancer Nurseline on Freephone 1800 200 700 (lines open Mon-Fri 9am-5pm), or drop into one of our 13 Daffodil Centres in hospitals nationwide.

Irish Cancer Society annual conference: Living Well With and Beyond Cancer is our annual national conference and educational event for cancer patients and survivors, those who care for them, health care professionals, and support service staff. This conference aims to provide information and support to enable people to live well after a cancer diagnosis. This year’s Living Well with and Beyond Cancer conference will take place in Limerick on 21st September. Attendance is free and you can register at cancer. ie/living-well or by contacting the Irish Cancer Society’s Cancer Nurseline on Freephone 1800 200 700 (lines open Mon-Fri 9am-5pm).

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A decade of development in chronic lymphocytic leukaemia has prolonged lives and shifted expectations

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DR AMJAD HAYAT MBBS MRCP FRCPATH PHD Consultant Haematologist, University Hospital Galway

DR MARK GURNEY MB BCH BAO MRCPI Haematology Specialist Registrar, Wellcome-HRB ICAT Fellow, NUI Galway

The treatment of chronic lymphocytic leukaemia (CLL) has changed dramatically over the past decade with a growing understanding of the disease biology guiding the use of established therapies and driving the introduction of new and effective chemo-free treatments.

hronic lymphocytic leu k aem ia i s t he most c om mon leu k aem i a i n adults. In Ireland, 250-300 new cases are diagnosed annually. CLL develops when a normal immune cell called a B-lymphocyte undergoes a series of genetic changes promoting growth and sur vival. These cells accumulate within the bone marrow and enter the blood where they may be detected on a routine blood test. Many pat ients do not need immediate treatment and are managed by watchful waiting. Over time, the accumulation of cells can cause enlargement of the spleen and lymph glands and impair the function of the bone marrow with anaemia, bleeding and infection risks. Many patients also experience tiredness, sweats and weight loss. Treatments aim to eliminate the leukaemic cells, resolve symptoms and restore bone marrow function. Developments in CLL treatment: Chemotherapies have been the mainstay of CLL treatment for over 50 years. Clinical trials established the best combination therapies. Development of antibodies that target a common marker on the leukaemia cell surface improved treatment further. Modified forms of chemotherapy are used for older patients for whom treatment is harder and riskier to endure. These approaches to treatment are effective in eliminating leukaemic cells but seldom lead to long-term cure. The discovery that genetic and molecular changes can predict the effectiveness of chemotherapy laid a foundation for the modern personalised approach to chemotherapy use. The past decade has seen radical new CL L t reat ments emerge. Targeted drugs that inhibit the signals leukaemic cells rely upon for survival or signals that prevent cell death have become available. MEDIAPLANET

They have shown dramatic benefits in clinical trials. These tablets are taken continuously and possess a very different but generally milder side effect profi le. There are now ma ny patients for whom CLL is controlled on long-term therapy. This has required a coordinated approach across medical specialties and primary care in managing longer-term effects such as cardiovascular changes and infections. Looking to the future: The dramatic achievements of the past decade have raised further questions and shifted the goals for patients and researchers alike. Quality of life for patients receiving long-term therapy has become a key consideration. The range of treatments now ava i lable requ i res add it iona l knowledge and understanding from patients and carers to enable informed decision making in conjunction with clinical teams. The patient-led group, Chronic Lymphocytic Leukaemia Ireland (CLLI), has emerged as a key resource for patients and families navigating this new landscape of CLL care in Ireland. Irish patients have benefited from and contributed to clinical t r i a l s e s t abl i sh i n g t a r g e te d therapies in CLL. This process continues through Cancer Trials Ireland’s involvement in the international CLL13 trial. The new generation of trials assess chemotherapy-free combinations given for a fixed period of time aiming to suppress the disease to undetectable levels. New therapies and combinations have inherent and considerable cost associated. It is vital that we advocate for prompt and equitable economic assessments to maximise the benefit of future developments to Irish CLL patients.

Chronic lymphocytic leukaemia (CLL) patient shares her incredible story CIARA O’DONOVAN Communications Manager, CLL Ireland

It was 2011 and Jan Rynne, a busy mum of four, was contemplating returning to work when she developed a ‘red eye’. Jan says: “I’d been back and forth to eye clinics and eventually went to ophthalmologist who diagnosed episcleritis. He said he could fix it but something underlying was causing it.”

he n e w s t h at he r e ye problem was due to a failing immune system was a huge shock. “I did have a lot of infections, and I did tell my GP I was tired a lot - but I had four young kids, so tiredness was expected. Eventually the results from a simple blood test showed I had something called chronic lymphocytic leukaemia (CLL)” - and it was blood cancer. “I’d the ‘Big C’ and I was terrified” CLL shares many symptoms with other conditions and can often be overlooked, especially in younger patients. Jan, from Drumcondra in Dublin, recalls that in the immediate aftermath of her diagnosis, she felt her prospects were poor: “Online articles were saying that life expectancy with CLL was just five years - I was 39 years old and my youngest was only three.” There was very little information available on CLL, and what she did find was out of date or was written for someone much older. “I was scared all the time. My husband and I knew that our only hope was to educate ourselves, so we got in touch with online support groups to learn more.” Jan was one of the lucky ones Jan’s tenacious husband, Michael, was instrumental in helping her find Irish haematologist, Professor Patrick Thornton, one of Ireland’s few CLL specialists. “Professor Thornton had all the latest information about new, more effective treatments - including non-chemotherapy options”.

JAN RYNNE Co-Founder, CLL Ireland and CLL Patient Advocate

But, critically, these treatments weren’t ava i lable i n Irela nd. Jan was referred to see the top European consultant Professor Peter Hillmen in Leeds and, in September 2014, she started a clinical trial for a new drug – a non-chemotherapy novel agent. It was almost too late… Jan recalls: “The day I took my first capsules, my bone marrow was 96% infiltrated by CLL cells. I was exhausted. My platelets were low, I was anaemic, my spleen was swollen, and I had developed further complications.” I nc re d ibly, w it h i n days of st a r t i ng her new t reat ment, symptoms started to improve and the lymph nodes in her neck started to shrink. Still it would be six months before Jan began to feel like herself again. It was arduous but slowly things improved.

Treatment has not been without its side effects Every day, Jan has to take three capsules to keep her leukaemia at bay. She receives regular infusions to fight infections, and frequently requires trips to the GP and courses of antibiotics. Jan must travel to Leeds every six months to get another supply of drugs. And Brexit is a looming worry. “I’ve been told my care won’t be affected - but information is unclear and who knows what it will all mean. I’m comforted by the knowledge that several new drugs are in trials and that newer drugs are reporting fewer or no side effects. If things change with my disease, I hope I will be able to access one such drug.”

Jan’s charity to support others with CLL: Jan set up Chronic Lymphocytic Leukaemia Ireland, a registered charity that aims to support others with CLL. “Hundreds of people have benefitted from our information days, hearing first-hand from CLL medical experts that there is hope!” The next information event will be in November in Galway City. Visit www.clli.ie to learn more. Read more at healthnews.ie HEALTHNEWS.IE

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Celebrating 35 years of bone marrow transplantation in Ireland Over 1,400 family donor and unrelated donor transplants have been performed in Ireland for life-threatening blood cancers including leukaemia, lymphoma and myeloma.

he first bone marrow transplant was performed in St James's Hospital Dublin in 1984 under the supervision of Professor Shaun McCann. These patients who have had a donor transplant include young adults and, more recently, patients up to 68 years old. Patients are referred to St James's for transplant from blood and cancer specialists all over Ireland. Susceptibility to blood cancers can increase with age and, with improved longevity in Ireland (as highlighted in the recent TILDA project 1 under the direction of Professor Kenny, Trinity College Dublin), older, fitter patients are seeking out this high risk – but potentially life-changing – procedure to cure their disease. What is a transplant? Allogeneic bone marrow transplan-

WRITTEN BY: DR CATHERINE FLYNN Consultant Haematologist, National Bone Marrow Transplant Centre

tation involves transferring the stem cells from a healthy donor to a patient following chemotherapy or radiation. The cells can be harvested from a family member, usually a sibling or an unrelated matching donor. With decreasing family sizes and increased ethnic diversity in modern Ireland, more unrelated donors are required than ever before. Close links between our Nat iona l Adu lt B one Ma r row transplant centre and the Irish unrelated donor registry (IBMTR) are critical in finding the best matched unrelated donors for Irish patients.

excessive bruising or internal bleeding), infection (repeated infections that fail to respond to antibiotics) or swollen lymph glands (in the neck, under the arms or hidden in the chest or abdomen). Several diseases can have similar sy mptoms to these including viral illnesses such as glandular fever, HIV or other cancers and it is important to get medical advice to assist in making the correct diagnosis. Typically, an examination and blood tests will lead to referral to a specialist centre where additional scans and tests including a bone examination will make a definite diagnosis.

The most successful transplants happen when the disease is well controlled at the beginning of the procedure. The advantage of a donor cell transplant is that, when successful, the patient has a new bone marrow and a new immune system. Many patients return to the workplace and family life and some pursue new adventures with their second chance. All transplant survivors in St James's hospital are invited to attend an annual medical review to optimise their long-term health. Looking after our increasing number of transplant survivors is a growing challenge.

What are the symptoms of blood cancer? When a patient presents with leukaemia or lymphoma, they will often have bleeding (nose bleeds,

Technology is supporting patients after transplant Technology is advancing to make diag noses quicker and assess responses to initial treatment.

Help us celebrate To celebrate 35 years of bone marrow transplantation in Ireland and World Marrow Donor day 2019, The Second All-Ireland Bone

Marrow Transplant conference is being held on Friday 20th of September 2019 for healthcare professionals involved in the field and a patient and family day will take place on the morning of Saturday 21st September 2019. Both these events will be held in Durkan Lecture Theatre, Trinity Centre, St James's Hospital, Dublin 8. All are welcome.

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1: tilda.tcd.ie/publications/reports

Early relapse in follicular lymphoma patients not fully understood

Life expectancy for patients with follicular lymphoma is good, but there are significant gaps in knowledge, support and provision that need addressing.

at ient s w it h adv a nc e d forms of follicular lymphoma can expect to live 20 years or more. However, given the biological and clinical heterogeneity, research is needed to better understand the complexity of the disease and ensure that patients receive the best possible (and ideally tailored) treatment according to risk factors. Despite the excellent outcomes for the group as a whole, the life-expectancy of patients who experience a progression of the disease within 24 months is considerably lower: “Only 50% overall survival as compared to 90% in patients without early relapse,” confirms Dr Amjad Hayat, Con su lta nt Haematolog ist at Galway University Hospital. More needs to be done to identify these individuals early, understand why their outcomes are so much worse, and investigate the best treatment options. Assessing long-term outlook to tailor therapy The ability to accurately assess

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the long-term outlook for each patient and then tailor therapy to the individual is an area of ongoing research in cancer. The Follicular Lymphoma International Prognostic Index (FLIPI), which takes into account five key factors that affect prognosis, has gone some way to providing this but is not used to tailor treatment or predict early relapse. Further refinements of the prognostic score also fail in accurately predicting early progression of disease and prognostic scores remain research tools. More recently, the use of PET scans at the end of treatment has been reported as an independent prognostic marker. Positive PET scans at the end of immunochemotherapy have been shown to be useful in identifying individuals at high risk of an early relapse. A refinement of PET, that refers to the total volume of active disease pre-treatment “total metabolic tumour volume” (TMTV) could be a useful predicting tool. But there are still many unknowns.

INTERVIEW WITH:

DR AMJAD HAYAT MBBS MRCP FRCPATH PHD Consultant Haematologist, University Hospital Galway Uncertainty around diagnosis can be very distressing for patients Even for those patients who go on to live relatively normal lives, the weight of uncertainty that comes with a diagnosis of follicular lymphoma can be hugely detrimental to their quality of life. Since the early stages of the disease are asymptomatic, by the time a patient seeks help a high percentage will already have advanced stage lymphoma. As a slow-developing cancer, that remains incurable,

despite significant improvements in standard treatment, the first course of action could be ‘watchful waiting,’ which often adds to a patient’s anxiety. This is an issue that Hayat believes needs to be addressed. “Most patients are waiting for something dreadful to happen and we need to do more to provide psychological support for them,” he says. Improved funding for clinical trials is needed to improve patient access When it comes to treatment, there are plenty of effective therapies out there. First line treatments can include radiotherapy, if the condition is localised, along with a combination of chemo-immunotherapy. Maintenance drugs are also commonly given to patients in the two years following treatment. New options, such as new monoclonal antibodies, immunomodulatory agents, drugs targeting selective cellular pathways and CART-T cell therapy, are on the horizon too,

but Hayat believes many patients in Ireland could be missing out on academic clinical trials, allowing early access to these newer developments due to a lack of funding. As part of the Lymphoma Forum of Ireland, Hayat is advocating for greater access, particularly or patients who experience an early relapse. There is no denying the progress that has been made, but greater research and better support are needed to help patients living with follicular lymphoma. WRITTEN BY: KATE SHARMA