MyelomaMatters
• Medical Matters Allogeneic transplantation Volume 8 Issue 4
• Retail Therapy Walk raises £70,000 www.myeloma.org.uk
LETTERS
Editor’s letter Dear Readers Welcome to this issue of Myeloma Matters – I hope you find the articles interesting and informative. The Myeloma Matters team works hard to develop each issue and would value your feedback on any of the features. We are grateful to Prof Nigel Russell from Nottingham City Hospital for his comprehensive article about allogeneic transplantation and to Kevin Brownless, a patient from Dorset, for sharing his experience of receiving this type of transplant. This issue also includes a summary of the results from a Myeloma UK survey sent to over 80 patients which emphasises the importance of patient choice when decisions are made regarding the type and location of treatment. With National Myeloma Week just passed, I am positive that the efforts of everyone across the country helped to make this year's week the most successful one yet. Thanks to all who took part. In the next issue of Myeloma Matters we will update you on what went on around the country to raise both funds and awareness. If at any time you have any questions, comments, or would like more information on our current work and future plans, please get in touch. In the meantime, I hope you get a lot out of this issue and thank you for your continued support. With best wishes
Jude Watson Editor
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CONTENTS
Contents 3–4 5 6 7 8 – 10 11 12 – 14 15 16 – 17 18 – 19 20 – 21 22 – 23
Newsround Special feature Myelomascope Research Medical matters Ask the nurse Living with myeloma AL amyloidosis Patient experience Policy and politics Fundraising in action Myeloma UK news
Letters I was recently asked if Myeloma Infodays are worthwhile. Don't hesitate, do it! I went to the day in Newport a couple of years ago and was at one in Bournemouth in April and they were both excellent. There were medical experts there to update us with the latest information and (separate) patient and carer workshops, where we could discuss our various problems with like-minded people. They laid on tea, coffee, cake and a nice lunch; they even laid out bottles of water for us, reminding us that we had to keep drinking! One of the most important things that I took away from both days was that you're not alone with this disease. There are lots of people out there who want to help and be helped. An Infoday is a worthwhile thing; Myeloma UK is to be congratulated on bringing us all together. Kevin Brownless, patient, Dorset Thanks for your recent mailing which included an update on your plans for 2009 – I was very impressed with the information I received, specifically the news that Myeloma UK are going to announce new research initiatives this year. Keep up the good work. Edward Mitchell, Birmingham Thank you to all the team at Myeloma UK for your support and help throughout our contact with you and your commitment to the cause. It is certainly a charity that achieves a great deal and a significant rapport with its supporters in such a competitive world. Anon I was disappointed to read that Revlimid is not available for myeloma patients in Scotland but will be for patients in England and Wales at second relapse. Although I have only recently been diagnosed, it is a worry that if the time comes when my doctor thinks I need Revlimid, I may not get it. Mrs Adams, Inverness
Send us your feedback Send your letters to: Jude Watson, Myeloma UK, Broughton House, 31 Dunedin Street, Edinburgh EH7 4JG jude@myeloma.org.uk
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NEWSROUND
Rare Disease Day at the Scottish Parliament Myeloma UK recently attended a Rare Disease Day event organised by Rare Disease UK at the Scottish Parliament. Similar events were also held at the Westminster Parliament and the Welsh Assembly. Rare Disease Day is observed across the world on the 28 February. The main aim of the day is to raise awareness of rare diseases such as myeloma and to reinforce their importance as a public health priority. The event held at the Scottish Parliament had a focus on rare diseases across the European Union (EU). There has been a growing call for European action in the field of rare diseases and a push for national strategies to be developed for rare diseases in each EU member state. So far only three EU countries have implemented national plans for rare
diseases (Belgium, the Czech Republic and France). Rare Disease UK took the opportunity provided by Rare Disease Day to reinforce its position that a rare disease strategy should be developed in Scotland and each of the other home nations, in order 'to improve care for people with rare diseases and to increase the chance of finding new treatments and cures'. The national strategies proposed would work to develop a more coherent research framework in each nation, improve the diagnosis and treatment of rare diseases and increase information provision for patients. Myeloma UK will be following the progress made with interest and will keep you up-to-date on any developments relating to proposals specific to the UK.
Launch of European myeloma survey: have your say A survey has been developed by Myeloma Euronet, the European network of myeloma patient groups of which Myeloma UK is a member, to collect information about 'myeloma treatment compliance' among myeloma patients.
New 'ring-leader' gene identified in myeloma
patient and medical communities across Europe to recognise the issues and challenges patients face in complying with their treatment.
'Treatment compliance' is a term used to describe when a patient correctly follows treatment advice, which is essential in ensuring that treatment is effective.
Please get involved with this valuable survey – either contact Jude at Myeloma UK (jude@myeloma.org.uk) to get a survey sent out to you, or fill it in online by visiting: www.myeloma-euronet.org/ en/survey/myeloma-patientcompliance.php
Myeloma Euronet hope that the results of the survey will encourage
The information you provide will be completely confidential.
A team of international researchers has identified a new gene involved in the development of myeloma. The gene, called UTX, is located on the X chromosome and has been identified in 10% of myeloma patients. Normally, the UTX gene plays a role in basic gene regulation. It has the ability to turn other genes on and off, through altering the structure of something called a 'histone', a sub-unit of our genetic material that plays a big part in gene activity. In myeloma patients, the UTX gene is expressed in an altered form and becomes deactivated. Unlike other altered genes involved in cancer, UTX is not involved in cell division or cell death. Instead, it alters the function of other genes in the cell, allowing them to 'run riot'. This is the first example of an alteration of this type indicated in cancer. According to Dr Andy Futreal, co-leader of the Cancer Genome Project, it "shows the depths that cancers will plumb in order to get themselves ready to go". Ongoing research into this gene will take place in a select few locations around the world, including the Institute of Cancer Research (ICR) in London, as part of the myeloma genetics and drug development programme funded in part by Myeloma UK. The discovery of the UTX gene represents another very important treatment target.
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NEWSROUND
Health Select Committee publishes report on access to NHS drugs A report has been published by the House of Commons Health Select Committee following its recent inquiry into 'top-up fees'. The inquiry was launched to examine the recent package of reforms, including copayments or 'top-up fees', introduced by the Government to improve access to some NHS drugs. The package of reforms was announced by the Health Secretary, Alan Johnson, in November 2008 and included: • Allowing patients to pay privately for a drug that is not available on the NHS without having the rest of their NHS care withdrawn • Taking a new approach to allow NICE greater flexibility when it assesses whether expensive drugs for rare and severe diseases should be made available on the NHS • Introducing a framework for NICE to consider patient access schemes, put forward by pharmaceutical companies as a way to effectively reduce the cost of a drug
• Giving a commitment to greater transparency and standardisation within the exceptional case funding system. This is the system by which patients – under exceptional circumstances – may obtain local funding approval for drugs that are not yet routinely available on the NHS The Committee expressed its concerns that the Government's policy on co-payments would be very difficult to implement and could have an impact on patient safety. It also expressed its reservations about the reforms to the NICE process.
In particular, it felt that the new, more flexible approach to appraising certain drugs may not be a fair or efficient way to make them available. Similarly, it considered that patient access schemes may over-burden the NHS. In relation to the exceptional case funding system, the Committee agreed that the system should be standardised and made more transparent for patients. The Department of Health will shortly respond to the outcome of the inquiry, and Myeloma UK will continue to monitor developments.
Myeloma UK funds two new clinical guidelines Two new clinical guidelines have been developed with the support of educational grants from Myeloma UK. They contribute to a series of myeloma clinical guidelines prepared by the UK Myeloma Forum (UKMF) and endorsed for use by the British Committee for Standards in Haematology. The guidelines summarise available evidence and best practice to help inform healthcare professionals on all aspects of the diagnosis, prognosis and management of myeloma and its related disorders.
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The first set of guidelines, developed by the UKMF, is a 2009 update on plasmacytoma. The update incorporates new information on solitary plasmacytoma of bone and extramedullary plasmacytoma (outside of bone) as well as an additional section on the management of multiple solitary plasmacytoma. The second set of guidelines, jointly prepared by the UKMF and the Nordic Myeloma Study Group, focuses on the investigation of newly detected M-proteins and the
management of Monoclonal Gammopathy of Undetermined Significance (MGUS). Myeloma UK keenly supports the development of evidence-based clinical guidelines to help benchmark, guide and shape a consistent approach to practice across the whole of the UK. The guidelines can be downloaded from both the UKMF and British Committee for Standards in Haematology websites – www.UKMF.org.uk www.bcshguidelines.com
SPECIAL
FEATURE
Myeloma UK survey emphasises the importance of patient choice When making treatment decisions it is vital that patients are given a say in the type and location of their treatment, wherever possible and applicable. The criteria that patients apply to making such decisions are wideranging, individual and subject to change throughout the course of their myeloma. The ability of healthcare professionals to recognise this can make a dramatic difference to a positive therapeutic partnership. The need to do this has been brought into sharp focus most recently due to a policy shift, led by the UK Government, towards moving some aspects of cancer care out of the hospital and into the home and community setting. The hope is that this move will address capacity issues in cancer centres by minimising inpatient care. To gain support for this initiative, the Government has stated that "most cancer patients want to receive as much of their care as possible close to home". Contrary to this statement, a Myeloma UK survey has shown that, if given a choice, there is a clear split in patient preference to receive treatment at home versus in hospital.
The survey Bone disease is a common feature of myeloma that is treated by a group of drugs called bisphosphonates. Myeloma UK surveyed over 80 patients taking either intravenous bisphosphonates (received at hospital) or oral bisphosphonates (taken at home) in order to gain insight into patient preference for both the type of treatment and where it is given.
Results snap shot • Nearly half of patients surveyed would opt for intravenous bisphosphonate treatment in hospital over taking oral tablets at home • 40% of patients want more information from their healthcare professional • Healthcare professionals should aim to provide information from a variety of sources, in different formats, on numerous occasions • At least 10% of patients do not comply with the drug dosing information While the shift from hospital to home suits many patients, a large proportion (35%) of respondents to our survey reported a preference to receive treatment in hospital. In fact, of those patients who had experience of both intravenous and oral treatment, there was virtually a 50 / 50 divide in patient preference, making a strong case for the need to take patient choice, where one exists, into consideration when making decisions about the setting of treatment and care. Patients who prefer to receive intravenous bisphosphonate treatment told us that they "feel more secure" and enjoy the "social interaction with hospital staff and other patients". Patients who prefer oral at-home treatment do so because it is more convenient, less invasive and takes less time. As myeloma patients are usually taking numerous drugs, the
convenience of oral bisphosphonates has to be weighed up against the likelihood of the patient committing to the drug's dosing schedule. Unsurprisingly, not all patients reported taking their pills as instructed because "it is not convenient to wait an hour before breakfast" or they "forget to take it at the right time". 97% of patients reported that oral bisphosphonates have an impact on their ability to schedule their day. Patients receiving bisphosphonates reported that the more information they received about the drug, the more confident they felt about their treatment. Although doctors and nurses are providing information on bisphosphonate treatment, more is needed. Less than 20% of patients received an information sheet to take home and 40% of patients stated a desire for more and better information, primarily on the pros, cons and long-term effects of treatment. Summary The setting of care can have a significant emotional and physical impact on patient quality of life. The Government is pushing care out of the hospital and into the home. This move, whilst sensible to deal with increasing demands on the health system, may come at the expense of patient preference for a large proportion of patients who, for various reasons, prefer to receive treatment in hospital. Myeloma UK will feed the results of this survey into appropriate forums to ensure that, wherever possible, healthcare professionals will engage patients in a dialogue to determine their preferences. 5
RESEARCH
Myelomascope New drug defibrotide shows promise for myeloma Defribrotide is a deoxyribonucleic acid (DNA) derivative being tested for myeloma in early phase (firstin-human) clinical studies with the established combination of melphalan, prednisone and Thalidomide Pharmion™ (MPT). It is anticipated that the addition of defibrotide to this combination may enhance the effectiveness of MPT in myeloma patients and help to reduce some of the side-effects of this combination. Preclinical (in the laboratory) studies conducted by the Dana-Farber Cancer Institute in Boston confirmed that defibrotide may improve outcomes for myeloma patients by: • Enhancing the sensitivity of myeloma cells to melphalan and prednisolone • Decreasing myeloma cell adhesion in the bone marrow microenvironment, therefore reducing the damaging effect of myeloma on bone • Inhibiting the expression of cytokines that stimulate the survival of myeloma cells in the bone marrow microenvironment
low levels of white blood cells) and thrombocytopenia (low platelet levels). The incidence and severity of peripheral neuropathy was low. Further studies in larger numbers of patients are required to test the drug's effectiveness.
Protein kinases provide good targets for the treatment of myeloma Samariam lexidronam / Velcade™ combination shows positive results for myeloma bone disease A Phase I study designed to assess the safety, tolerability and efficacy of a drug called Samariam 153lexidronam in combination with Velcade (bortezomib), for the treatment of patients with relapsed or refractory myeloma, has shown positive results. This small study at the Institute of Myeloma and Bone Cancer Research in California enrolled 24 patients and collected measures of the usefulness and the side-effects of the drug on myeloma when used in combination with Velcade.
Samariam 153-lexidronam belongs to a class of drugs called radiopharmaceuticals and works to reduce pain associated with bone disease. It is taken up by areas of Defibrotide has also recently shown the bone affected by myeloma and promise when used in conjunction gives off radiation to reduce bone with Granulocyte Colony-Stimulating Factor (GCSF) to increase the number pain. of stem cells available for harvest for 21% of patients responded to patients undergoing stem cell the drug, which appears to be transplantation. These initial results well tolerated with minimal or support the need for further research manageable side-effects. The into the potential of defibrotide as an side-effects that were experienced included neutropenia (abnormally anti-myeloma treatment. 6
Protein kinases modify proteins in the body by changing their activity or function and are the major regulators of cellular activity. They have a significant role to play in the development of many cancers. Consequently, protein kinases are an ongoing target in cancer research and drug development. Recently, studies have suggested that the elevated expression of the protein kinase NIK (otherwise known as MAP4KA) activates the NFkB pathway, which regulates the body's immune response and is implicated in the development of myeloma. This discovery has therefore established a new protein kinase target for research in myeloma. Studies in the near future will focus on the protein kinase NIK in order to test a new way to treat myeloma.
Any questions? Myeloma Infoline
0800 980 3332 askthenurse@myeloma.org.uk
www.myeloma.org.uk
RESEARCH
First ever myeloma patient to receive new targeted radiation now in remission The first ever myeloma patient in a UK clinical study to receive a new, targeted type of radiotherapy is now in remission. Mrs Pauline Pain is doing well after receiving the treatment which is designed to completely clear the bone marrow of myeloma cells before a stem cell transplant. Radiotherapy is now rarely used as part of the transplant process because of potentially serious side-effects. However, this is a new technique which aims to achieve similar results with minimal side-effects. Patients in the study, led by Dr Kim Orchard at Southampton University Hospital, are treated with 'highly targeted' radiotherapy that specifically destroys myeloma cells in the bone marrow but doesn't affect surrounding healthy cells. It is thought that one of the main reasons why stem cell transplants so far have had only partial success in providing long-term remission is that patients are not cleared of all the myeloma cells before the transplant. The radiotherapy currently being tested by Dr Orchard can deliver much higher doses of radiation than conventional radiotherapy, directly to the myeloma cells in the bone marrow, which will potentially delay the onset of relapse after a transplant. Dr Orchard's team is assessing whether this new treatment can prolong remission for transplant patients. Mrs Pain from the Isle of Wight was the first patient recruited to the study. It is now over a year since her transplant and Mrs Pain continues to do well. Dr Orchard said: "Obviously the excellent response of our first patient must be treated with caution. However, this study is very exciting and we hope other patients will respond in the same way. Another bonus is that we suspect that this treatment may also be triggering the patient's immune system to seek out and destroy any remaining myeloma cells after the transplant. If this is true then this approach offers great promise for the treatment of all patients with blood cancers who need a transplant". Myeloma UK provided the initial seed money for the study which is now funded by Leukaemia Research. 80 people are scheduled to take part in the study at Southampton General Hospital over a two-year period.
Case Study My name is Pauline Pain, I'm 58 years old, retired and live on the Isle of Wight with my husband Chris. I was diagnosed with myeloma in September 2007. I'd been going back and forth to the doctor for tests after a seemingly innocuous injury to my back wouldn't heal after a fall in the garden. I sensed something was not right, telling my GP, "I'm not ill but I know I'm not well.� The series of blood tests and a biopsy confirmed that I had myeloma. In April 2008, after 18 weeks of initial treatment, I started preparation for a stem cell transplant at Southampton General Hospital under Dr Kim Orchard. Dr Orchard asked if I wanted to be the first patient on a new study, and, after discussing the details with my GP, I accepted. I had a preliminary, small dose of the radiotherapy treatment on 23 April then the main dose on 7 May. The treatment made me feel tired and slightly sick, but over all I felt very, very well. It was incredible to be walking around knowing that something inside me was fighting the myeloma, but I couldn't feel it at all. After the treatment was completed, tests showed that I had no trace of myeloma left in my bone marrow. I had the transplant two weeks later. The remarkable aspect of this treatment is that I felt few ill-effects, and I was able to go home for the two weeks before my transplant rather than having to stay in hospital. I'm still going backwards and forwards from the Isle of Wight to Southampton for check-ups every month, but I am feeling incredibly well. I am able to take part in all the hobbies and activities that I did before my diagnosis.
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MEDICAL
MATTERS
Allogeneic transplantation by Prof N H Russell, Consultant Haematologist, Nottingham City Hospital The role of allogeneic transplantation in myeloma remains controversial and generates frequent debate amongst haematologists who look after patients. Although allogeneic transplants for myeloma have been performed for over 20 years, they have mainly been carried out in younger patients up to the age of 50, and are usually only suitable for the small minority of younger patients who have a brother or sister (sibling) with a matched tissue type who can act as a donor. What continues to intrigue haematologists about the use of allogeneic stem cell transplantation for myeloma is that there is evidence that this may offer a chance of cure for some patients. However, the treatment remains complicated and associated with significantly greater risks than autologous stem cell transplantation (using your own cells), which remains the other major alternative treatment option. Indeed, autologous stem cell transplantation has been the standard of care for patients with myeloma under the age of 65 years for several years. Furthermore, there are an increasing number of non-transplant options for patients with myeloma such as treatment combinations that include Velcade and Revlimid, which are effective in prolonging the survival of patients who relapse after autologous transplantation. All of these developments have given rise to clear evidence of an improving outlook for myeloma patients. Population-based studies from the United States and 8
patients with a matched sibling donor. In contrast, unrelated donor transplants are regarded as "developmental" or "generally not recommended" unless in the context of a clinical study.
Scandinavia have shown impressive improvements over the last decade in both five- and 10year survival particularly for patients under the age of 60. This is most likely to reflect the impact of recent advances in treatment and their widespread dissemination into clinical practice. So where does this leave allogeneic transplantation? The number of allogeneic transplants performed in the UK is relatively low. In 2007 just 37 were reported to the British Society of Bone Marrow Transplantation (BSBMT) across all blood cancers, compared with 756 patients undergoing autologous transplantation during the same year. The BSBMT has recently produced a table of guidance for when to consider transplantation for a variety of diseases including myeloma. Currently for those patients considered "fit for intensive therapy" an autologous transplant is considered to be a standard treatment. An allogeneic transplant could be considered as a "clinical option" in selected younger
The major reason for caution concerning the use of allogeneic transplantation remains the risk of the procedure. It is well recognised that conventional high-intensity pre-treatment for this type of transplantation in myeloma is associated with a high risk of treatment-related mortality, which has shown to be as high as 30%. The risks of allogeneic transplants are greatest in patients over the age of 50 and in patients who have been heavily pre-treated prior to receiving the transplant. There is, however, evidence of an improvement in survival and a reduction in treatment-related mortality over more recent years. Nevertheless this remains a procedure associated with significant risks. Clinical studies The high risk of transplant-related mortality associated with allogeneic transplants has lead investigators to undertake clinical studies to evaluate the role of allogeneic transplants using reduced intensity pre-treatment (often called 'mini allotransplants'). A number of these studies have been carried out throughout Europe including studies by French and Italian Myeloma study groups and the European Bone Marrow Transplant (EBMT) group. The strategy used in these studies has been to evaluate the role of an initial autologous transplant followed
MEDICAL
a few months later by a mini allotransplant using cells from a sibling donor. In these studies this tandem autologous / allogeneic transplant has been compared with either a single autologous or a double autologous transplant procedure. The concept behind this approach is to try to get the patient to achieve the best possible response with the autologous transplant prior to proceeding to a mini allo-transplant. The aim of the mini allo-transplant is to make use of a 'graft versus myeloma effect' (see Box 1) to eradicate any persisting myeloma cells, leading to long-term survival and, potentially, "cure". What are the long-term results of the mini allo-transplant studies? In a recent update, two of the studies performed by the EBMT and by the Italian myeloma study group, show statistical evidence of a reduced risk of relapse emerging at five to six years in the group of patients receiving the mini allotransplant compared with the group of patients just receiving autologous transplantation. This currently translates into a survival advantage for the tandem transplant group. In the EMBT group, for example, the results show 65% of patients surviving at six years compared with 50% in the patients receiving autologous transplantation alone. However, this apparent advantage comes at a price as a significant proportion of patients go on to develop chronic Graft versus Host Disease (GvHD – see Box 2) which can be debilitating and require ongoing immuno-suppressive therapy for some years. In the
MATTERS
Box 1
Graft versus myeloma effect An important part of any allogeneic transplant procedure and a crucial difference from autologous transplantation is to exploit a 'graft versus myeloma effect'. Graft versus myeloma is thought to be mediated by special blood cells called T cells. T cells in myeloma patients do not function as they should, so the T cells from the donor attack and kill any persisting myeloma cells in the patient following the transplant. Therefore allogeneic transplantation relies on two different ways of killing the myeloma cells: firstly, the effects of the high- or reduced-intensity treatment the patient receives prior to the transplant; secondly, the 'graft versus myeloma effect' which may operate over several months in the post-transplant period and is lacking when the patient undergoes an autologous transplant procedure. Studies have clearly demonstrated that the risks of transplant-related mortality have fallen to 10-15% with the use of mini allotransplants, therefore the procedure is considerably safer than the conventional high-intensity allogeneic transplant but still not without risk.
Patient Experience Read more about allogeneic transplantation from a patient’s point of view on page 16
Italian study 50% of patients developed this complication and at four years 28% of patients were still on immuno-suppressive treatment such as steroids for the GvHD. So there clearly are toxicities associated with this approach. Also it is important to see if this apparent advantage continues with longer follow-up. Some study groups have attempted to reduce the risk of GvHD by using specific techniques to prevent this complication from occurring; unfortunately, this approach has been associated with poorer results. What about unrelated donor transplants? The previously mentioned studies have only recruited patients with sibling donors. This means there is less information to guide us concerning unrelated donor transplants and in general these must be regarded as developmental. That said, there are some circumstances where this option might be considered, such as in a young patient with poor risk features at diagnosis (such as coexisting plasma cell leukaemia) or in a young patient whose myeloma is behaving aggressively following initial treatment. However, for patients with advanced refractory myeloma, an unrelated donor transplant is unlikely to be beneficial. So where does this leave us? In Nottingham we have not undertaken a conventional highintensity allogeneic transplant for myeloma for several years. However, we continue to consider suitable younger patients for the combination of autologous followed by mini allo-transplant. Generally we only consider this option in 9
MEDICAL
Box 2
Graft versus Host Disease The development of GvHD is a double-edged sword: severe GvHD can be life-threatening but mild or moderate GvHD is associated with the advantageous 'graft versus myeloma effect'. Therefore, some GvHD is beneficial, but too much GvHD is a serious complication of the transplant procedure. The major causes of transplant-related mortality following allogeneic transplantation in myeloma are post-transplant infections and the development of graft versus host disease (GvHD). Graft versus myeloma without severe GvHD is the holy grail of allogeneic transplantation in myeloma.
MATTERS
patients under the age of 55 years with a matched sibling donor. We have used the same approach for the mini allo-transplant as the EBMT group, which is based on that pioneered in Seattle some years ago and uses low-dose total body radiation combined with fludarabine. This type of pretreatment for a mini allo-transplant is well tolerated by the patient and often the stay in hospital is limited to a week or so. We have observed chronic GvHD in some of these patients but they have responded well to immunosuppressive therapy. The final decision about proceeding to a mini allo-transplant is difficult and the patient needs careful counselling about the risks and possible benefits of the procedure. It is often useful for them to spend time with a Myeloma Clinical Nurse Specialist who can discuss these risks and act as a patient advocate in the decision-making process. Summary Although the role of allogeneic transplant in myeloma is the subject of much ongoing debate and indeed controversy, there is little doubt that it has its place, likely a very important one, in the treatment of myeloma. This is especially true for the younger patient group.
Any questions? GvHD can be characterised by selective damage to the liver, skin and mucosa, and the gastrointestinal tract. Skin GvHD can manifest in a diffuse rash similar to the one pictured above.
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 Myeloma Infoline
0800 980 3332  askthenurse@myeloma.org.uk
www.myeloma.org.uk
Medical terms Allogeneic stem cell transplant A procedure in which stem cells or bone marrow from a compatible donor (usually a sibling) are collected, stored and given to the patient after highdose chemotherapy treatment.
Autologous stem cell transplant A procedure in which a patient's own stem cells are collected, stored and then given back following high-dose chemotherapy. Patients may have two autologous transplants – one as part of initial treatment and a second at relapse (sometimes called a double transplant), or they may just have one (single) autologous transplant.
Mini allogeneic transplant Also called mini allo-transplant or reduced intensity conditioning transplant. A type of allogeneic transplant that uses lower dose of chemotherapy than a standard allogeneic transplant and therefore avoids some of the side-effects and risks associated with higher-dose chemotherapy.
Tandem transplant A planned double transplant procedure. It can be two autologous stem cell transplants or an autologous followed by a mini-allogeneic transplant.
Total body radiation Treatment with X-rays, gamma rays or electrons to damage or kill cancerous cells. Rarely, it can be used over the whole body in preparation for a transplant.
LIVING
WITH
MYELOMA
Ask the nurse by Ellen Watters RGN,, Myeloma Information Nurse Specialist, Myeloma UK Since my transplant two and a half years ago I have been on Thalidomide as a maintenance therapy. My doctor has recently told me that she is planning to take me off it. Can you tell me why? Thalidomide has been proven to be an effective treatment for use at different stages of myeloma, either on its own or in combination with other drugs. One focus of the national myeloma study MRC Myeloma IX was to determine whether there is a beneficial effect for patients of using Thalidomide as a maintenance therapy (i.e. used continuously until the myeloma comes back). The latest analysis of the results emerging from this study has indicated that maintenance Thalidomide may not result in a better outcome for patients compared with when no maintenance treatment is used. It is important to stress that no harmful effects of using Thalidomide maintenance have been reported from the study either. Given that these recent findings suggest there are no proven benefits to remain long-term on Thalidomide, the current recommendation is that doctors discuss stopping maintenance therapy with their patients. There remain, however, clinical circumstances where Thalidomide maintenance is an appropriate treatment option. More work and analysis is necessary to give a complete picture of the role and effects of Thalidomide as a maintenance therapy.
I have had myeloma for three years and my haematologist says I may now also have AL amyloidosis. Please tell me a little more about this. The term amyloidosis is a generic term used for a group of diseases where an abnormal protein, called amyloid, is produced. AL amyloidosis is one form of amyloidosis where the amyloid is produced as a consequence of abnormal plasma cell production in the bone marrow.
I have heard that prescription charges have been scrapped for cancer patients in England. Can you tell me more about this and what is happening in the rest of the United Kingdom? From 1 April 2009, all patients in England receiving treatment for cancer or the effects of cancer have been entitled to free prescriptions. Patients seeking free prescriptions should apply for an exemption certificate from their GP surgery or haematology clinic; this certificate can then be shown to pharmacists as proof of exemption. Patients can also claim a refund for any prescriptions paid for since 1 April. The certificate lasts for five years and can be applied for again once it expires. Prescriptions have been free in Wales since 2007; they will be free in Northern Ireland by 2010 and Scotland will phase out prescription charges by April 2011 (their cost will be reduced year-on-year until that time).
Amyloid is broken down by the body only very slowly, so it builds up in tissues and organs, disrupts their function and causes symptoms. Each patient has a different pattern of amyloid deposition, with different organs affected. Amyloid can build up in the kidneys, heart, liver, spleen, nerves, or digestive system, and may affect two or more organs at the same time. AL amyloidosis is a relatively rare disease, with approximately 500 – 600 people diagnosed in the UK each year. About 10 – 20% of myeloma patients also have AL amyloidosis, or go on to develop it. Due to similarities between myeloma and AL amyloidosis they are almost always treated the same way, and, for most patients, myeloma remains the priority in terms of treatment.
AL amyloidosis information Myeloma UK has developed a range of patient information for AL amyloidosis patients. Call the Infoline 0800 980 3332 or visit www.myeloma.org.uk /amyloidosis
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LIVING
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Complementary Therapies by Eve Hallam, Myeloma Information Nurse Specialist, Myeloma UK
Although complementary therapies are increasingly accepted and integrated into conventional medicine, further research is needed to better understand exactly how they work and the ways in which they interact, positively, negatively or if at all, with conventional treatment.
Complementary medicine is based on different practices and ideas, and consists of many different approaches to both treat and understand the origin of disease. Patients choose to use a complementary therapy for a number of reasons including: to improve general wellbeing and emotional state, to gain some sort of personal control over the impact myeloma is having on their life and to help deal with the symptoms and complications of myeloma and its treatment. Complementary therapies can be used in addition to and alongside conventional myeloma treatment prescribed by your doctor. They should not be confused with alternative therapies, which are those used instead of conventional treatment.
Types of complementary therapies There are many forms of complementary therapies. The most well known and commonly used forms include acupuncture, aromatherapy, homeopathy, massage, medication, music therapy, 12
reflexology, relaxation, visualisation and yoga. Different therapies can be used to treat different problems, and the approach used depends very much on your particular illness, complaint or preference. What works for one person may not be beneficial to someone else.
Combining conventional and complementary approaches Conventional treatment (i.e. types of chemotherapy) is the treatment that almost all patients with myeloma will receive. Although conventional treatment approaches do have their limitations, there is usually a great deal of published evidence, derived from rigorously conducted clinical studies, to suggest that these treatments work and improve patients' lives. Generally, such studies have not been conducted with complementary therapies. Therefore it is very hard to make any specific recommendations about the use of complementary therapies and the likely impact they have on an individual patient or their myeloma.
While complementary therapies may help to alleviate symptoms and side-effects of myeloma and its treatment, they should be taken only alongside, and not instead of, conventional medical treatment. Always inform your doctor of any complementary therapies you intend to take or participate in. What follows is an overview of some of the more common complementary therapies:
Acupuncture focuses on improving overall wellbeing, rather than a treatment of a specific symptom. Acupuncture is part of traditional Chinese medicine and uses the balance of the body's own life force to restore wellbeing. Practitioners of Chinese medicine believe that the body has a system of chi (life force) which becomes unbalanced when someone is ill. The acupuncture needles are applied to areas where this flow has been blocked, in order to restore balance and health. During acupuncture the body releases endorphins (the body's natural morphine), which help to reduce pain, relax muscles and increase feelings of wellbeing. The principal aim of acupuncture is to recover the equilibrium between the physical, emotional and spiritual aspects of the individual.
LIVING
Aromatherapy is the use of essential oils (concentrated plant oils) and massage to promote relaxation, alleviate stress and relieve symptoms of anxiety. The scent and properties of different oils vary, and a variety of oils is used to produce different results. Oils like lavender are relaxing and soothing, whereas lemon and other citrus oils are invigorating. It may be best to avoid using oils on the skin when you are having chemotherapy or radiotherapy as the skin can become sensitive, but the perfume of lightly scented candles can be used to help with nausea and to aid relaxation. You should always be treated by a registered aromatherapist who is experienced in treating people with cancer. Homeopathy is a form of medicine in which patients are treated with small quantities of natural substances that trigger symptoms much like those that are in need of being 'cured'. The body is thus encouraged to heal itself. Homeopathic remedies come in tablet form, liquids or creams. Some remedies are tailor-made for the individual's symptoms, taking their whole physical and mental wellbeing into account. Other remedies are ready-prepared and can be bought in chemists and health food shops. Remember to discuss with your doctor any remedies you plan to take. Massage is used to relieve muscle pain and tension and can be both therapeutic and relaxing. A trained masseuse or masseur will gently manipulate problem areas to ease tightened muscles and relieve pain. Remember to tell the masseuse / masseur that you have myeloma and that forceful massage could damage your bones.
WITH
MYELOMA
Before you start treatment with a complementary therapy: • Tell your doctor • Use a reliable, qualified practitioner who has experience with cancer patients • Use a practitioner you feel comfortable with • Remember, there are no "miracle" cures • Don't stop your conventional myeloma treatment • Do not pay too much money for any therapy Meditation is a relaxing mental exercise that can help to reduce anxiety, stress and pain. Breathing techniques and concentration are used to relax each part of the body in turn. Meditation is usually carried out in a quiet room and in a sitting position for about 15 – 20 minutes. You can learn to guide your own meditation and can use music and aromatherapy candles to help relaxation, relieve tension and aid sleep. Music Therapy is the use of music and sound to promote relaxation and to reduce treatment or diseaserelated anxiety. Although music therapy does not treat physical symptoms, it aims to reduce the symptoms of disease such as pain, which are often exacerbated by anxiety. Music therapy is usually provided through a one-to-one or group session with a trained music therapist. This will usually last between 30 minutes and an hour. Music therapists have a wide-range of techniques that they may use during
sessions. Individual needs and musical preferences are discussed in the initial session, and the techniques then used are based on these. During a session, live or prerecorded music may be played by the music therapist. Pre-recorded music is sometimes provided for you to listen to between therapy sessions, or you may be encouraged to listen to your favourite pieces of music whilst relaxing at home. This is designed to induce a relaxed state of mind. Central to music therapy is the powerful association that music can have with different periods of our life; listening to specific types of music can promote pleasant thoughts and memories. Other techniques used in music therapy involve composition and song-writing, lyric analysis and improvisation. You may be provided with musical instruments such as percussion and wind instruments, keyboards, or be encouraged to sing in order to create individual sets and pieces of music. The music therapist may also encourage discussion about the chosen music and lyrics, in order to establish the emotion and meaning behind them. In this sense, music therapy is designed to promote communication with others and selfconfidence. This may be especially beneficial to people who find it difficult to talk about their disease and their emotions. You do not have to have a musical background, or be able to play a musical instrument or sing to participate in music therapy. The premise behind this type of therapy is not music learning – although this may be an added benefit – but to provide an alternative and enjoyable way of expressing emotions. 13
LIVING
WITH
MYELOMA
Reflexology is a specialised form of foot massage, based on the theory that different areas of the foot represent and are connected with the body's internal organs. Pressure is applied to different points on the sole of the foot to help relieve pain and sickness. This type of massage can be very relaxing. Relaxation is the use of breathing exercises to bring about a feeling of calmness by concentrating on your breathing rhythm and the tightening and relaxing of muscles. It is usually done lying down wearing loose clothing. Reiki is an ancient form of healing which uses the body's own energy or life force to restore a sense of balance or calmness, release tension and reduce pain. The reiki healer will channel energy through their hands to various parts of your body. Although he or she will not actually touch you, you may feel sensations of heat, cold, vibration and tingling on the skin. You are fully dressed while receiving reiki healing. Visualisation involves the use of mental imagery while you are in a state of meditation or relaxation. It can be used as a relaxation tool or to reduce stress and anxiety. Picturing yourself within a peaceful scene will encourage relaxation. Other forms of visualisation are more closely linked to the symptoms of illness. Some patients imagine their immune system destroying the myeloma cells, their blood counts coming up, or visualise themselves becoming stronger. Yoga is a form of exercise that involves stretching and breathing and is used to unite mind, body and spirit. Light yoga exercises can be useful in strengthening muscles and bone, while yoga breathing exercises can be very relaxing. 14
Summary Using complementary therapies can give you a feeling of control over your myeloma and treatment, enabling you to do something positive and pro-active to manage any symptoms and side-effects. Carers and family members may also find therapies aimed at reducing tension and promoting relaxation helpful. If you are thinking of using any of these therapies you should look for a qualified, registered therapist who has experience of treating cancer patients. The Institute for Complementary Medicine can be contacted for a list of registered practitioners in your area (contact details are provided in the box to the right). Many cancer centres offer complementary therapies, but not always free of charge, or can give you the contact details of services available locally.
“Massage is used to relieve muscle pain and tension and can be both therapeutic and relaxing. A trained masseuse or masseur will gently manipulate problem areas to ease tightened muscles and relieve pain.”
Institute for Complementary Medicine (ICM)
0207 922 7980 (Monday – Friday, 10am – 4pm)
www.i-c-m.org.uk
AL
AMYLOIDOSIS
Living with AL amyloidosis: Heart and / or kidney involvement By Eve Hallam, Myeloma Information Nurse Specialist, Myeloma UK Introduction This article focuses on the symptoms you may expect with heart and / or kidney involvement after a diagnosis of AL amyloidosis, and what can be done to manage these symptoms. This is the first in a series of similar articles looking at different aspects of living with AL amyloidosis. Future articles will include information about managing the symptoms caused by amyloid in other organs, including the nervous and digestive systems. Symptoms Amyloid and the heart In AL amyloidosis heart problems usually occur as a result of amyloid deposits within the heart muscle. This causes the heart to become stiff and affects its ability to pump effectively, leading to fatigue, shortness of breath and fluid retention often causing swollen ankles. Amyloid and the kidneys Kidney problems usually occur in people with AL amyloidosis as a direct result of amyloid deposits in the filtering system of the kidney, or sometimes by the drugs used to treat AL amyloidosis. Amyloid deposits in the kidney most often cause a condition called 'nephrotic syndrome'. This is where the damaged kidneys leak healthy blood protein (called albumin) into the urine. This loss of albumin from the bloodstream causes water to leak out from the blood vessels into the tissues. This causes swelling (oedema) which is most noticeable in the lower legs and feet.
Symptom management Low salt diet If your heart or kidneys are affected you may be advised to go on a low salt (sodium) diet. Salt is made up of two elements, sodium and chloride. These are used by the body to maintain water balance in the blood and tissues. If you have heart or kidney problems you may already be retaining more fluid than normal. By reducing your salt intake this can help reduce the fluid retention that causes a strain on your heart, increases your breathlessness, and leads to swelling in your feet, ankles, legs and abdomen (oedema). Fluid restriction You may have your fluid intake restricted to less than two litres per day by your doctor. This is also to help reduce the fluid retention discussed above. If you find the restriction difficult try sucking on ice cubes or boiled sweets, gargling with ice cold water, or chewing gum. Daily weight Your doctor may recommend you weigh yourself daily. This helps to monitor any fluid retention and allows you to manage this at home by increasing your diuretic (water) tablets as agreed with your doctor. In order to do this you will need to buy a set of accurate digital scales and weigh yourself at the same time every day. You should then keep a record of how much you weigh and note any increases or decreases. Your doctor will advise you how to manage your diuretic tablets according to any weight gain or loss.
Exercise Gentle forms of exercise such as walking, swimming, cycling, gentle aqua-aerobics, gentle gym work, yoga and tai chi are good for overall health and can help reduce fatigue and improve circulation. You may have less energy during and after treatment and feel frustrated that you cannot do as much as you could before. Try to do just a small amount of exercise to start with and see how you feel, both immediately afterwards and a day or so later. Make sure you only do what feels comfortable. Exercise may also be particularly beneficial after you have finished a course of chemotherapy treatment. Your doctor will be able to refer you to a physiotherapist who will advise you on an exercise regime. Remember to discuss any changes to your diet or level of exercise with your doctor before starting.
Summary If your heart and / or kidneys are affected by AL amyloidosis you may experience some of the symptoms outlined here. These may be alleviated through a combination of prescribed treatment and lifestyle changes, and after treatment for the underlying AL amyloidosis your organ function may improve slowly over time.
Further information Myeloma UK has developed a range of patient information for AL amyloidosis patients. To order your copy of Living with AL amyloidosis – Your Essential Guide, call the Infoline 0800 980 3332. This information is available at www.myeloma.org.uk / amyloidosis 15
PATIENT
EXPERIENCE
My experience of allogeneic transplantation Kevin Brownless, myeloma patient, Dorset dexamethasone, which got me back into remission within three months. Towards the end though, I had terrible diarrhoea and so my Poole Hospital Consultant put me on a course of Velcade injections. After eight injections though, my stomach swelled up and, again, I had very bad diarrhoea, so I stopped taking it and had a clear, drug-free month before I went into Southampton Hospital for the transplant.
I was diagnosed with myeloma in November 2005. The following March, I had a stem cell transplant using my own stem cells (autologous). This held up well until fifteen months later, when my paraprotein levels started to rise again. My Consultant at Poole General Hospital gave me a choice: have another autologous transplant and then face another problem fifteen months down the road, or consider having a donor transplant (allogeneic) using somebody else's stem cells, which could potentially cure me. I was referred to another Consultant, at Southampton General Hospital, who advised that I was in the right age bracket (I'm currently 52), albeit at the top end, to have a transplant. He told me that I was too old to have total radiation (I was relieved to hear that), then he explained the procedure to me: chemotherapy, to wipe out the system, followed by the transplant. He explained that, in many ways, it would be similar to what I had already gone through before, but more arduous. There was also the 30% mortality rate to consider. I told him that without the transplant, there was a very good chance it'd be a 100% rate in my case, so I agreed to go ahead with it. As I don't have any brothers or sisters, I was told that my tissue status would be analysed on the Antony Nolan Trust bone marrow computer system, which searches worldwide for suitable donors. 16
“My Consultant gave me a choice: have another autologous transplant and then face another problem fifteen months down the road, or consider having a donor transplant (allogeneic) using somebody else’s stem cells, which could potentially cure me.�
(I often wondered why, if I was back in remission, my Consultant had then put me on Velcade. Much later on, I learned that Thalidomide and Velcade work in completely different ways when killing the cancer cells; the latter 'mops up' anything overlooked by the former). I started to receive chemotherapy as soon as I arrived at the hospital. I was given melphalan and campath / fludarabine. I received the actual transplant four days later. Having been through it all before, it was nothing new, other than the cells were given to me fresh, as opposed to having been frozen first.
Incidentally, with 'allo' transplants, the doctors like to give men younger women's stem cells as, apparently, there's something in the cells which protects against testicular cancer.
However, I was also given a new drug called cyclosporin (an immunesuppressive drug to reduce the risk of Graft versus Host disease (GVHD), where the stem cells can reject the body) and that caused me to have paranoid psychosis. I went loopy for a while, imagining all sorts of weird things, such as I was under Police surveillance, my wife's car had been stolen and, apparently, I even tried to escape from my hospital bed!
In the meantime, I was put on a course of Thalidomide and
My whole mouth lining disintegrated and my throat swelled up, making it
Kevin Brownless I was soon advised that there were four suitable donors for me, in the UK alone.
PATIENT
very difficult and uncomfortable to swallow. I have since learned with this is called mucositis, which I don't recommend. I eventually ended up in intensive care for a few days with a lung infection while they sorted me out, which included putting a tube down my throat to feed me. Thankfully, I don't remember any of that. My blood pressure dropped (which it had also done during my previous transplant) and it took a while for them to balance it. After six weeks, I was discharged.
EXPERIENCE
they hadn't got any bugs, as I won't have a fully functioning immune system for 12 months post-transplant. After three months, the restrictions were lifted a little; for example I can now mingle with people, drink coffee, eat salad and see my grand-daughter. Needless to say, there have a small number of problems since the transplant.
I thought I'd broken a tooth inside my gum. The fragment eventually At the time of writing this, over six came out. It turned out that it months have passed since the wasn't a bit of tooth, it was a bit of transplant and I feel fine. I've put on jaw: I had necrosis of the jaw, a lot of weight, thanks to my wife's caused by the two shots of excellent cooking (I lost 4 kilos in Zometa (bisphosphonate) which I hospital) and am back to my normal had had two years before. weight now. When I was discharged, there was a long list of I also developed GvHD after receiving a top-up shot of things I couldn't do or eat / drink. lymphocytes (white cells) from my donor to boost my depleted Here are just a few examples: immune system. After my • Alcohol Consultant spotted that I looked a bit pink on my chest and legs, • Nuts what followed was two weeks of • Spices having to smear steroid Betnovate cream all over my body. My wife • Herbs had to don rubber gloves and do • Unpeeled fruit or salad my back. I assure you, the novelty soon wore off! • Seafood (apart from fish) Lately, I've noticed that my upper right hand teeth and a small area of • Meat from a delicatessen cheek above them are numb. This is under investigation; I'm not sure I also had to avoid caffeine and only what it is, maybe nerve damage drink water if it had been boiled first from the necrosis. (even though it's filtered twice in my The Consultants, however, are house beforehand). Decaffeinated pleased with my progress and, tea was fine though. apparently, my bone marrow is I had to avoid all contact with already a 98% clone of the donor's crowds and I couldn't see my four (a 34 year old woman – I'd love to year old grand-daughter for 6 know who she is); it appears I've months (what with schools being reversed the aging process! breeding grounds for germs). Anyone who wanted to see me had I am full of optimism about the first to be interrogated to make sure future. •
Honey
Finally, I would like to thank my Consultants, Dr Fergus Jack at Poole and Dr Kim Orchard at Southampton and all of the staff at Southampton and Poole General Hospitals' Oncology departments for their truly amazing work.
Medical Matters See page 8 for an article about allogeneic transplantation by Prof Nigel Russell, Consultant Haematologist at Nottingham City Hospital
Further information
High-Dose Therapy and Stem Cell Transplantation Infoguide Explains high-dose therapy and stem cell transplantation, why they are needed, and the potential advantages and disadvantages of treatments. It also discusses what will happen during the procedure, as well as outlining what to expect during the recovery process
Myeloma Infoline
0800 980 3332
askthenurse@myeloma.org.uk 17
POLICY
AND
POLITICS
Exceptional funding for drugs: patient experience and government policy by Sarah Ritchie, Policy and Advocacy, Myeloma UK would be more clinically appropriate to use an alternative drug to the one approved for a patient's disease stage, he or she has to justify their decision to the PCT using the exceptional funding route, with varied success.
Introduction The National Institute for Health and Clinical Excellence (NICE) was set up to eradicate postcode prescribing. However, the NICE process takes so long that local health bodies have been forced to make decisions about who gets what drug using a policy that was designed for something else entirely. The NHS "exceptional funding" process – which allows the 152 Primary Care Trusts (PCTs) to determine what drugs they fund based on the 'exceptionality' of patients – has created another tier of postcode prescribing. The Government has responded to many months of intense debate about this issue by developing guidance to support improved decision-making within PCTs. It is clear to Myeloma UK, however, that the actual experience of patients who are subject to exceptional funding decision-making has not been properly taken into account in the development of the guidance. 18
The experience of patients is at the heart of the issue and must form the basis of reform.
The experience of patients There are two main situations when myeloma patients need exceptional funding approval to obtain a drug. The first is during the time between a drug receiving its licence and gaining a positive NICE recommendation. The other is when the drug is approved by NICE but the patient who needs it does not fit the 'restrictions' for which the drug has been approved for routine use. The second situation is fairly common in myeloma. Myeloma is a very heterogeneous cancer and clinicians require flexibility regarding how to treat individual patients and when. However, drugs are increasingly being approved for a more restricted use than their licensed indication; not for reasons of clinical efficacy or safety, but due to cost-effectiveness. Therefore, each time a doctor considers it
Over the past two years Myeloma UK has supported around 60 patients through the exceptional funding system. We have provided practical advice and emotional support to these patients on a case-by-case basis, and have advised many more who came to us with enquiries about how the process works. Through this we have identified a number of common difficulties. For example: The decision-making process is often lengthy and distressing. The process generally takes several weeks but has been known to take months. Myeloma patients who need these drugs are unwell and the time involved in going through the system can have a detrimental impact on their health. It can also be very distressing for patients and their families who feel out of their depth in pursuing an appeal. There is rarely appropriate expertise on decision-making panels. Myeloma is a rare, complex and individual cancer. Yet there is rarely a haematology or oncology expert on the panels that consider applications, let alone a myeloma expert. How the panel, with limited or no experience of myeloma, can give due consideration to an application is questionable, especially as the panel
POLICY
will be looking at a wide array of different applications each time they meet. The lack of understanding about myeloma is worryingly clear from some PCT refusal letters Myeloma UK has seen. To refuse an application without having sought alternative relevant expertise is not the right way to be making decisions on life-extending cancer drugs. It is almost impossible to prove that someone is "exceptional". Clinicians must prove to the PCT that their patient is 'more exceptional' than other patients with the same cancer. Often this is wrongly interpreted by PCTs to mean that the patient must be clinically "unique". This is impossible to fulfil as it is always imaginable for another patient to be similar. PCTs refuse applications on improper grounds. Even though the Department of Health tells PCTs that they cannot refuse funding for individual drugs on the grounds of cost alone, or because there is no supporting NICE guidance for the drug, in practice these are often used as grounds for refusing an individual patient's request. Furthermore, with limited data on cost-effectiveness available some PCTs confuse cost-effectiveness with the drug's net price. This immediately generates a bias against patients who require an expensive drug, whether or not the drug in question is cost-effective.
The Government's proposed reforms The Government says that new NHS Constitution will help address the problem by stating that: "You have the right to expect local decisions on funding of other drugs
AND
POLITICS
and treatments to be made rationally following a proper consideration of the evidence. If the local NHS decides not to fund a drug or treatment you and your doctor feel would be right for you, they will explain that decision to you." This does not signal any changes to the way decisions are made. PCTs are already obliged under administrative law to make fair and rational decisions. The problem is the interpretation of what is a 'rationally made decision' or a 'proper consideration of the evidence'. A supporting handbook has been developed for PCTs, containing 51 pages of checklists and flowcharts to help PCTs improve their systems. However, it leaves crucial factors open to interpretation: it does not define 'exceptionality', what a 'rational' or 'justified' decision consists of, what taking account of 'appropriate specialist expertise' should involve, nor what counts as a reasonable timeframe for dealing with 'urgent' cases. Ultimately, we still expect many of the common problems that we see to continue.
Summary It is the view of Myeloma UK that the exceptional funding policy should be abolished. If here to stay for the foreseeable future, then reform needs to go much further and genuinely reflect the needs of patients. Until a replacement is found we want to see a comprehensive review of the exceptional funding system that takes full account of patient experience in developing appropriate policy. We intend to use our understanding of the system to push for evidence-based reform that will truly be to the benefit of patients.
Further information
Accessing Treatments on the NHS: An Information Pack Myeloma UK has produced an Access to Treatments Pack for patients as a guide to the processes involved in securing access to myeloma treatments. We hope it will empower patients and their family members, together with their doctors, to gain access to treatments. It provides information on: • The role of the Primary Care Trust (PCT) in deciding what treatments it funds • The role of the National Institute for Clinical Excellence (NICE) • How exceptional case funding processes work The pack also provides advice to patients on: • How to get the right decision from the PCT • Applying for exceptional funding and appealing against a negative decision • Additional action to consider • How Myeloma UK can help To order your pack or for more information contact: Sarah Ritchie 0131 557 3332 sarahr@myeloma.org.uk www.myeloma.org.uk 19
FUNDRAISING IN ACTION Sub-zero polar race in aid of Myeloma UK
After finding out one of his very close friends had myeloma, Julian Evans wanted to do something to show his support. He decided to do something truly unique and signed up to compete in the 'Polar Race', a competitive 350 mile team race across the arctic to the magnetic north pole. The race started in mid April and they completed it six weeks later. Julian raced on skis, pulling his supplies in 120Ib pulks, stopping at two checkpoints en route to stock up on essentials and he faced temperatures as low as -50 C. Julian has so far raised over £10,000 from the challenge and has just taken part in the Edinburgh marathon as well. Well done from everyone at Myeloma UK.
St Mary's College completes a six-month fundraising drive, raising £20,960 for Myeloma UK
On 9 January, Myeloma UK Chief Executive Eric Low attended St Mary's College in Hull to receive a cheque for over £20,000, raised by staff and students of the College. The fundraising took place in memory of Christine Leech who had myeloma. It was largely organised by her husband Phil Leech and Julie Carr, both assistant head teachers at the College. The College's fundraising efforts began with the Humber Bridge HalfMarathon on 29 June 2008, with over 40 students and staff taking part and raising £7,500 in sponsorship. The fundraising continued with everyone at the College getting involved in a wide range of events during the year
including a Year Seven disco, a race night and an event where students were allowed to throw wet sponges at their teachers. The fundraising target of £20,000 was reached just before Christmas at a staff pantomime. Eric Low commented: "A huge thank you to all of the staff and pupils of St Mary's College for raising such an impressive amount for Myeloma UK. The range and diversity of fundraising activities have been remarkable and it's obvious that everybody at the College has been involved in some way. It has really brought the students together towards a common goal.'' More information can be found at www.st-marys.hull.sch.uk/ MyelomaHalfMarathon.htm
Hospital Saturday Fund presents £1,000 cheque to Myeloma UK Myeloma UK was one of 24 Scottish-based charities to receive a donation from the Hospital Saturday Fund at an event at Glasgow's City Chambers in February. Community fundraiser, Lawrence Orr, took to the stage to accept the donation and joined celebrity Carol Smillie who was receiving an award on behalf of another Scottish charity.
The Hospital Saturday Fund is a registered charity which this year celebrates its 60th anniversary. Chief Executive of the Hospital Saturday Fund, Keith Bradley, said: "We're delighted to put money back into the communities around Scotland". Thank you to the Hospital Saturday Fund for their kind support. Go to www.hsf.eu.com for more information.
Pictured: Keith Bradley, Lawrence Orr and Carol Smillie
20
FUNDRAISING IN ACTION Myeloma UK Retail Therapy Walk raises £70,000
Stand up for myeloma in Manchester raises £5,000
Sunday 10 May saw 300 Myeloma UK supporters flexing their credit cards and hitting the shops in a five-mile sponsored 'Retail Therapy Walk for Research' in aid of Myeloma UK. A sea of orange t-shirts filled St Luke's church grounds as the walkers assembled before Myeloma UK Patron Maureen Lipman and celebrity ambassadors Dom and Sandi Wood officially started the walk.
On Sunday 24 May, Myeloma UK held a comedy night 'Stand up for myeloma' at the Comedy Store in Manchester. Over 400 people attended the event, with top comedians Stephen K Amos, Jack Whitehall, Ivan Brackenbury, Alex Boardman and Justin Moorhouse providing the entertainment for the evening.
The event raised in excess of £70,000, and every penny will be used to fund our research programme. Thanks to all those who took part in the day and helped to make it such a success. www.myeloma.org.uk/rtw
A brilliant night was had by all and around £5,000 was made through ticket sales and a raffle held on the night. Thanks to all those people who came along and supported the event, and to those who helped sell tickets or provided advertising for the event brochure. www.myeloma.org.uk/comedy
Entire family takes part in the Great Manchester Run for their dad
Double marathon challenge for myeloma
When Geoff Woodruff was diagnosed with myeloma in 2008 it came as a great shock to his family. They decided they wanted to do something positive to show their support for Geoff and so the entire family of seven decided that they would take part in the Bupa Great Manchester 10K Run on Sunday 17 May. The whole family completed the run successfully, and altogether they raised over £3,000.
On 31 May, Mike Derry, (pictured left) an architect from Devon, embarked on his second marathon challenge in three months. He ran the Edinburgh Marathon, having also tackled the gruelling South Devon Coastal Marathon back in February. Mike ran both marathons in aid of his dad Geoff, who was diagnosed with myeloma last year. Since announcing his challenges in January, Mike has already raised more than £2,500 through sponsorship.
Pictured right 'The Woodruff Runners' (left to right): Ian, Liz, Al, Carole, Kath,
More about the Edinburgh Marathon will
Jess, James.
follow in the next issue of Myeloma Matters.
21
MYELOMA
UK
NEWS
Myeloma UK awards two
My working day
Rebecca Wight, Patient to Patient Programmes, Myeloma UK When I accepted this job, I remember Eric saying to me, "I wish I had your job". As time goes by, and the more I learn about myeloma and Myeloma UK, I completely understand why Eric said this to me. In January 2009 I started at Myeloma UK, coordinating all of our Patient to Patient programmes. A key part of this role is working with the network of Myeloma Support Groups across the UK, helping new ones to get off the ground and providing assistance to existing groups. I am also excited about future projects we have planned such as a 'buddy' network. This programme will put patients in contact with other patients all over the UK. We are also working towards setting up a representative patient panel to help develop and progress Myeloma UK services.
I love the fact that no two days are the same. One day I am directing film clips for Myeloma TV (www.myelomatv.org.uk) and another I am designing a patient diary, a tool patients can use in conjunction with their doctors and nurses to track and record their symptoms and treatment. Every day is so busy but all it takes is a phone call or email from a patient to stop me in my tracks. The feeling of community between myeloma patients, family and friends makes this a unique environment to be part of. I was fortunate to attend the London Myeloma Support Group meeting one week into the job. By far the best part of the day was speaking to patients and their families and friends: the positive impact belonging to the group had on them was evident. On a daily basis I communicate by phone and email
with Support Group Leaders and it continually astounds me how much time and dedication they put into the running of their groups. I am really looking forward to meeting more Support Group Leaders and fellow group members at forthcoming Myeloma Support Group meetings, Patient and Family Infodays and other Myeloma UK events. If you have any queries regarding any of the patient programmes at Myeloma UK, please do not hesitate to get in touch. Rebecca on 0131 557 3332 rebecca@myeloma.org.uk
New videos added to Myeloma TV – watch now New videos have been added to Myeloma TV and are available to view now at www.myelomatv.org.uk. The new clips include Dr Paul Richardson from the Dana-Farber Cancer Institute in Boston talking about new insights in myeloma. Dr Richardson is one of the world leaders in the research and treatment of myeloma and was the principal investigator on the pivotal 'Apex' Velcade study. We also have a range of new videos from patients and carers talking about their experiences of myeloma. They include Neil Atterson who was diagnosed with myeloma in his mid-thirties, and carer Thea Scott who talks about her role as a carer for her husband, a myeloma patient. Watch these clips and many more at www.myelomatv.org.uk • Coming soon…new videos are added all the time to Myeloma TV. If you would like to be notified when they go live, please email rebecca@myeloma.org.uk and she will update you.
22
FORTHCOMING
Rat Race Urban Adventure
Join Team Myeloma UK to take part in this unique team event.
• Edinburgh 18 / 19 July • London 26 / 27 September
To register your place contact Lorna 0131 557 3332 lorna@myeloma.org.uk
2009 Raffle for Research
1st
Walking Tours Holiday in Spain
£20,000 £19,000 £18,000 £17,000
EVENTS
Patient and Family Myeloma Infodays
Meet others with myeloma, share experiences, learn from experts • Sheffield Saturday 5 September • London Saturday 17 October • Bristol Saturday 7 November • Newcastle Saturday 14 November To register your place contact Kirsty 0131 557 3332 kirsty@myeloma.org.uk or register online www.myeloma.org.uk
Join a sponsored walk with a difference –
Retail Therapy Walk for Research
£16,000
£15,000
with High Point Holidays
£14,000
2
nd
£13,000
£250 John Lewis voucher
£12,000 £11,000
£10,000 £9,000 £8,000
3
rd
Apple iPod touch
£7,000 £6,000
£5,000 £4,000 £3,000
4
th
£100 ASOS.com voucher
Runners up prizes: 10 lucky runners up will receive a £20 Oddbins voucher
£2,000
£1,000
£1 per ticket
To order your raffle books contact Lawrence 0131 557 3332 lawrence@myeloma.org.uk www.myeloma.org.uk/fr
• Bristol Saturday 19 September For details contact Sara 0131 557 3332 sara@myeloma.org.uk www.myeloma.org.uk/rtw
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Myeloma Matters editorial and production team Medical Editor: Dr Gordon Cook Director, Blood and Marrow Transplantation Programme St James’s University Hospital, Leeds Editor: Jude Watson Editorial support: Sara Morgan Design: Linda Scott-McFarlane Myeloma UK Chairman: Judy Dewinter Chief Executive: Eric Low Patron: Maureen Lipman CBE Board of Directors: Greg Allon, Dr Gordon Cook, Josie Dobrin, Jackie Green RGN, Peter Hunt, Claude Littner, Andrew McAllister, Dr Atul Mehta, Prof Gareth Morgan Myeloma Matters is published bi-monthly by Myeloma UK. The information presented in Myeloma Matters is not intended to take the place of medical care or the advice of a doctor. Your doctor should always be consulted regarding diagnosis and treatment. No part of this newsletter may be reproduced in any way without prior permission from Myeloma UK Myeloma UK Broughton House, 31 Dunedin Street, Edinburgh EH7 4JG Tel: 0131 557 3332 Fax: 0131 557 9785 Email: myelomauk@myeloma.org.uk Myeloma Infoline: 0800 980 3332 www.myeloma.org.uk Charity No. SC 026116 National Myeloma Week 21 – 28 June