Volume 8 Issue 4 www.myeloma.org.uk

MyelomaMatters

• Medical MattersAllogeneic transplantation

• Retail Therapy Walk raises £70,000

Dear Readers

Welcome to this issue ofMyeloma Matters – I hopeyou find the articlesinteresting and informative.The Myeloma Matters teamworks hard to develop each

issue and would value your feedback on anyof the features.

We are grateful to Prof Nigel Russell fromNottingham City Hospital for hiscomprehensive article about allogeneictransplantation and to Kevin Brownless, apatient from Dorset, for sharing hisexperience of receiving this type of transplant.

This issue also includes a summary of theresults from a Myeloma UK survey sent toover 80 patients which emphasises theimportance of patient choice when decisionsare made regarding the type and location oftreatment.

With National Myeloma Week just passed, I am positive that the efforts of everyoneacross the country helped to make this year'sweek the most successful one yet. Thanks to all who took part. In the next issue ofMyeloma Matters we will update you on whatwent on around the country to raise bothfunds 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 hopeyou get a lot out of this issue and thank youfor your continued support.

With best wishes

Jude WatsonEditor

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L E T T E R S / C O N T E N T S

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 newresearch initiatives this year. Keep up the good work.

Letters

Editor’s letter

I was recently asked if Myeloma Infodays are worthwhile. Don'thesitate, 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 latestinformation and (separate) patient and carer workshops, where wecould discuss our various problems with like-minded people. Theylaid 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 thatyou're not alone with this disease. There are lots of people out therewho want to help and be helped. An Infoday is a worthwhile thing;Myeloma UK is to be congratulated on bringing us all together.

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 asignificant rapport with its supporters in such a competitive world.

Send us your feedbackSend your letters to: Jude Watson, Myeloma UK,

Broughton House, 31 Dunedin Street, Edinburgh EH7 4JG jude@myeloma.org.uk

3 – 4 Newsround5 Special feature6 Myelomascope7 Research8 – 10 Medical matters11 Ask the nurse12 – 14 Living with myeloma15 AL amyloidosis16 – 17 Patient experience18 – 19 Policy and politics20 – 21 Fundraising in action 22 – 23 Myeloma UK news

Contents

Kevin Brownless, patient, Dorset

Edward Mitchell, Birmingham

Anon

I was disappointed to read that Revlimid is not available for myelomapatients in Scotland but will be for patients in England and Wales atsecond relapse. Although I have only recently been diagnosed, it is aworry that if the time comes when my doctor thinks I need Revlimid, I may not get it. Mrs Adams, Inverness

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A team of international researchershas identified a new gene involved in the development of myeloma. Thegene, called UTX, is located on the X chromosome and has beenidentified in 10% of myelomapatients.

Normally, the UTX gene plays a rolein basic gene regulation. It has theability to turn other genes on and off,through altering the structure of something called a 'histone', asub-unit of our genetic material thatplays a big part in gene activity.

In myeloma patients, the UTX gene is expressed in an altered form andbecomes deactivated. Unlike otheraltered genes involved in cancer,UTX is not involved in cell division or cell death. Instead, it alters thefunction of other genes in the cell,allowing them to 'run riot'.

This is the first example of analteration of this type indicated incancer. According to Dr AndyFutreal, co-leader of the CancerGenome Project, it "shows thedepths that cancers will plumb inorder to get themselves ready to go".

Ongoing research into this gene willtake place in a select few locationsaround the world, including theInstitute of Cancer Research (ICR) in London, as part of the myelomagenetics and drug developmentprogramme funded in part byMyeloma UK. The discovery of theUTX gene represents another veryimportant treatment target.

New 'ring-leader' geneidentified in myeloma

Myeloma UK recently attended aRare Disease Day event organisedby Rare Disease UK at the ScottishParliament. Similar events were alsoheld at the Westminster Parliamentand the Welsh Assembly.

Rare Disease Day is observedacross the world on the 28 February. The main aim of theday is to raise awareness of rarediseases such as myeloma and toreinforce their importance as apublic health priority.

The event held at the ScottishParliament had a focus on rarediseases across the EuropeanUnion (EU). There has been agrowing call for European action inthe field of rare diseases and a pushfor national strategies to bedeveloped for rare diseases in eachEU member state.

So far only three EU countries haveimplemented national plans for rare

diseases (Belgium, the CzechRepublic and France).

Rare Disease UK took theopportunity provided by RareDisease Day to reinforce its positionthat a rare disease strategy shouldbe developed in Scotland and eachof the other home nations, in order'to improve care for people with rarediseases and to increase thechance of finding new treatmentsand cures'.

The national strategies proposedwould work to develop a morecoherent research framework ineach nation, improve the diagnosisand treatment of rare diseases andincrease information provision forpatients.

Myeloma UK will be following theprogress made with interest and willkeep you up-to-date on anydevelopments relating to proposalsspecific to the UK.

Rare Disease Day at the Scottish Parliament

A survey has been developed byMyeloma Euronet, the Europeannetwork of myeloma patient groupsof which Myeloma UK is a member,to collect information about'myeloma treatment compliance'among myeloma patients.

'Treatment compliance' is a termused to describe when a patientcorrectly follows treatment advice,which is essential in ensuring thattreatment is effective.

Myeloma Euronet hope that theresults of the survey will encourage

Launch of Europeanmyeloma survey: have your say

patient and medical communitiesacross Europe to recognise theissues and challenges patients facein complying with their treatment.

Please get involved with this valuablesurvey – either contact Jude atMyeloma UK (jude@myeloma.org.uk)to get a survey sent out to you, or fillit in online by visiting:www.myeloma-euronet.org/en/survey/myeloma-patient-compliance.php

The information you provide will be completely confidential.

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Health Select Committee publishes report on access to NHS drugsA report has been published by theHouse of Commons Health SelectCommittee following its recent inquiryinto 'top-up fees'. The inquiry waslaunched to examine the recentpackage of reforms, including co-payments or 'top-up fees', introducedby the Government to improve accessto some NHS drugs.

The package of reforms wasannounced by the Health Secretary,Alan Johnson, in November 2008 and included:

• Allowing patients to pay privately fora drug that is not available on theNHS without having the rest of theirNHS care withdrawn

• Taking a new approach to allowNICE greater flexibility when itassesses whether expensive drugsfor rare and severe diseases shouldbe made available on the NHS

• Introducing a framework for NICE toconsider patient access schemes,put forward by pharmaceuticalcompanies as a way to effectivelyreduce the cost of a drug

• Giving a commitment to greatertransparency and standardisationwithin the exceptional case fundingsystem. This is the system by whichpatients – under exceptionalcircumstances – may obtain localfunding approval for drugs that arenot yet routinely available on theNHS

The Committee expressed its concerns that the Government's pol-icy on co-payments would be very dif-ficult to implement and could have animpact on patient safety. It also ex-pressed its reservations about the re-forms to the NICE process.

Two new clinical guidelines havebeen developed with the support ofeducational grants from MyelomaUK. They contribute to a series ofmyeloma clinical guidelines preparedby the UK Myeloma Forum (UKMF)and endorsed for use by the BritishCommittee for Standards inHaematology.

The guidelines summarise availableevidence and best practice to helpinform healthcare professionals on allaspects of the diagnosis, prognosisand management of myeloma and itsrelated disorders.

Myeloma UK funds two new clinical guidelines

In particular, it felt that the new, moreflexible approach to appraising cer-tain drugs may not be a fair or effi-cient way to make them available.Similarly, it considered that patientaccess schemes may over-burdenthe NHS. In relation to the exceptional casefunding system, the Committee agreed that the system should be standardised and made moretransparent for patients. The Department of Health will shortlyrespond to the outcome of the inquiry, and Myeloma UK will continue to monitor developments.

The first set of guidelines, developedby the UKMF, is a 2009 update onplasmacytoma. The updateincorporates new information onsolitary plasmacytoma of bone andextramedullary plasmacytoma(outside of bone) as well as anadditional section on themanagement of multiple solitaryplasmacytoma.

The second set of guidelines, jointlyprepared by the UKMF and theNordic Myeloma Study Group,focuses on the investigation of newlydetected M-proteins and the

management of MonoclonalGammopathy of UndeterminedSignificance (MGUS).

Myeloma UK keenly supports thedevelopment of evidence-basedclinical guidelines to helpbenchmark, guide and shape aconsistent approach to practiceacross the whole of the UK. Theguidelines can be downloadedfrom both the UKMF and BritishCommittee for Standards inHaematology websites –www.UKMF.org.ukwww.bcshguidelines.com

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Myeloma UK survey emphasises the importance of patient choice

When making treatment decisions itis vital that patients are given a say inthe type and location of theirtreatment, wherever possible andapplicable.

The criteria that patients apply tomaking such decisions are wide-ranging, individual and subject tochange throughout the course of theirmyeloma.

The ability of healthcare professionalsto recognise this can make adramatic difference to a positivetherapeutic partnership. The need todo this has been brought into sharpfocus most recently due to a policyshift, led by the UK Government,towards moving some aspects ofcancer care out of the hospital andinto the home and communitysetting. The hope is that this movewill address capacity issues in cancercentres by minimising inpatient care.To gain support for this initiative, theGovernment has stated that "mostcancer patients want to receive asmuch of their care as possible closeto home".

Contrary to this statement, aMyeloma UK survey has shown that,if given a choice, there is a clear splitin patient preference to receivetreatment at home versus in hospital.

The surveyBone disease is a common feature ofmyeloma that is treated by a group ofdrugs called bisphosphonates.Myeloma UK surveyed over 80patients taking either intravenousbisphosphonates (received athospital) or oral bisphosphonates(taken at home) in order to gaininsight into patient preference forboth the type of treatment and whereit is given.

• Nearly half of patientssurveyed would opt forintravenous bisphosphonatetreatment in hospital overtaking oral tablets at home

• 40% of patients want moreinformation from theirhealthcare professional

• Healthcare professionalsshould aim to provideinformation from a variety ofsources, in different formats,on numerous occasions

• At least 10% of patients donot comply with the drugdosing information

Results snap shot

While the shift from hospital to homesuits many patients, a largeproportion (35%) of respondents toour survey reported a preference toreceive treatment in hospital. In fact,of those patients who hadexperience of both intravenous andoral treatment, there was virtually a50 / 50 divide in patient preference,making a strong case for the need totake patient choice, where oneexists, into consideration whenmaking decisions about the settingof treatment and care.

Patients who prefer to receiveintravenous bisphosphonatetreatment told us that they "feel moresecure" and enjoy the "socialinteraction with hospital staff andother patients". Patients who preferoral at-home treatment do sobecause it is more convenient, lessinvasive and takes less time.

As myeloma patients are usuallytaking numerous drugs, the

convenience of oralbisphosphonates has to be weighedup against the likelihood of thepatient committing to the drug'sdosing schedule. Unsurprisingly, notall patients reported taking their pillsas instructed because "it is notconvenient to wait an hour beforebreakfast" or they "forget to take it atthe right time". 97% of patientsreported that oral bisphosphonateshave an impact on their ability toschedule their day.

Patients receiving bisphosphonatesreported that the more informationthey received about the drug, themore confident they felt about theirtreatment. Although doctors andnurses are providing information onbisphosphonate treatment, more isneeded. Less than 20% of patientsreceived an information sheet to takehome and 40% of patients stated adesire for more and betterinformation, primarily on the pros,cons and long-term effects oftreatment.

SummaryThe setting of care can have asignificant emotional and physicalimpact on patient quality of life. The Government is pushing care outof the hospital and into the home.This move, whilst sensible to dealwith increasing demands on thehealth system, may come at theexpense of patient preference for alarge proportion of patients who, forvarious reasons, prefer to receivetreatment in hospital. Myeloma UKwill feed the results of this survey into appropriate forums to ensurethat, wherever possible, healthcareprofessionals will engage patients in a dialogue to determine theirpreferences.

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MyelomascopeNew drug defibrotide showspromise for myeloma

Defribrotide is a deoxyribonucleic acid(DNA) derivative being tested formyeloma in early phase (first-in-human) clinical studies with theestablished combination ofmelphalan, prednisone andThalidomide Pharmion™ (MPT). It is anticipated that the addition ofdefibrotide to this combination mayenhance the effectiveness of MPT in myeloma patients and help toreduce some of the side-effects of this combination.

Preclinical (in the laboratory) studiesconducted by the Dana-FarberCancer Institute in Boston confirmedthat defibrotide may improveoutcomes for myeloma patients by:

• Enhancing the sensitivity ofmyeloma cells to melphalan and prednisolone

• Decreasing myeloma cell adhesionin the bone marrowmicroenvironment, thereforereducing the damaging effect of myeloma on bone

• Inhibiting the expression ofcytokines that stimulate the survivalof myeloma cells in the bonemarrow microenvironment

Defibrotide has also recently shownpromise when used in conjunctionwith Granulocyte Colony-StimulatingFactor (GCSF) to increase the numberof stem cells available for harvest forpatients undergoing stem celltransplantation. These initial resultssupport the need for further researchinto the potential of defibrotide as ananti-myeloma treatment.

Samariam lexidronam /Velcade™ combination showspositive results for myelomabone disease

A Phase I study designed to assessthe safety, tolerability and efficacy ofa drug called Samariam 153-lexidronam in combination withVelcade (bortezomib), for thetreatment of patients with relapsed orrefractory myeloma, has shownpositive results.

This small study at the Institute ofMyeloma and Bone CancerResearch in California enrolled 24patients and collected measures ofthe usefulness and the side-effectsof the drug on myeloma when usedin combination with Velcade.

Samariam 153-lexidronam belongsto a class of drugs calledradiopharmaceuticals and works to reduce pain associated with bonedisease. It is taken up by areas ofthe bone affected by myeloma andgives off radiation to reduce bonepain.

21% of patients responded to the drug, which appears to be well tolerated with minimal ormanageable side-effects. The side-effects that were experiencedincluded neutropenia (abnormally

low levels of white blood cells) andthrombocytopenia (low platelet levels).The incidence and severity ofperipheral neuropathy was low.Further studies in larger numbers ofpatients are required to test the drug'seffectiveness.

Protein kinases provide goodtargets for the treatment ofmyeloma

Protein kinases modify proteins in thebody by changing their activity orfunction and are the major regulatorsof cellular activity. They have asignificant role to play in thedevelopment of many cancers.Consequently, protein kinases are anongoing target in cancer research anddrug development.

Recently, studies have suggested thatthe elevated expression of the proteinkinase NIK (otherwise known asMAP4KA) activates the NFkBpathway, which regulates the body'simmune response and is implicated inthe development of myeloma.

This discovery has thereforeestablished a new protein kinasetarget for research in myeloma.Studies in the near future will focus onthe protein kinase NIK in order to testa new way to treat myeloma.

Any questions?

Myeloma Infoline0800 980 3332

askthenurse@myeloma.org.uk

www.myeloma.org.uk

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First ever myeloma patient to receive new targeted radiation now in remission The first ever myeloma patient in a UK clinical study toreceive a new, targeted type of radiotherapy is now inremission. Mrs Pauline Pain is doing well after receivingthe treatment which is designed to completely clear thebone marrow of myeloma cells before a stem celltransplant.

Radiotherapy is now rarely used as part of the transplantprocess because of potentially serious side-effects.However, this is a new technique which aims to achievesimilar results with minimal side-effects.

Patients in the study, led by Dr Kim Orchard atSouthampton University Hospital, are treated with 'highlytargeted' radiotherapy that specifically destroys myelomacells in the bone marrow but doesn't affect surroundinghealthy cells.

It is thought that one of the main reasons why stem celltransplants so far have had only partial success inproviding long-term remission is that patients are notcleared of all the myeloma cells before the transplant.

The radiotherapy currently being tested by Dr Orchardcan deliver much higher doses of radiation thanconventional radiotherapy, directly to the myeloma cells in the bone marrow, which will potentially delay the onsetof relapse after a transplant.

Dr Orchard's team is assessing whether this newtreatment can prolong remission for transplant patients.Mrs Pain from the Isle of Wight was the first patientrecruited to the study. It is now over a year since hertransplant 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 willrespond in the same way. Another bonus is that wesuspect that this treatment may also be triggering thepatient's immune system to seek out and destroy anyremaining myeloma cells after the transplant. If this is truethen this approach offers great promise for the treatmentof 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 atSouthampton General Hospital over a two-year period.

Case StudyMy 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 backwouldn't heal after a fall in the garden. I sensedsomething was not right, telling my GP, "I'm not ill but I know I'm not well.” The series of blood tests and abiopsy confirmed that I had myeloma.

In April 2008, after 18 weeks of initial treatment, I started preparation for a stem cell transplant atSouthampton General Hospital under Dr Kim Orchard. Dr Orchard asked if I wanted to be the first patient ona new study, and, after discussing the details with myGP, I accepted.

I had a preliminary, small dose of the radiotherapytreatment 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 bewalking 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. Ihad the transplant two weeks later.

The remarkable aspect of this treatment is that I feltfew ill-effects, and I was able to go home for the twoweeks before my transplant rather than having to stayin hospital. I'm still going backwards and forwards fromthe Isle of Wight to Southampton for check-ups everymonth, but I am feeling incredibly well.

I am able to take part in all the hobbies and activitiesthat I did before my diagnosis.

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patients with a matched siblingdonor. In contrast, unrelated donortransplants are regarded as"developmental" or "generally notrecommended" unless in thecontext of a clinical study.

The major reason for cautionconcerning the use of allogeneictransplantation remains the risk of the procedure. It is well recognisedthat conventional high-intensity pre-treatment for this type oftransplantation in myeloma isassociated with a high risk oftreatment-related mortality, whichhas shown to be as high as 30%.The risks of allogeneic transplantsare greatest in patients over the ageof 50 and in patients who havebeen heavily pre-treated prior toreceiving the transplant. There is,however, evidence of animprovement in survival and areduction in treatment-relatedmortality over more recent years.Nevertheless this remains aprocedure associated withsignificant risks.

Clinical studies The high risk of transplant-relatedmortality associated with allogeneictransplants has lead investigators to undertake clinical studies toevaluate the role of allogeneictransplants using reduced intensitypre-treatment (often called 'mini allo-transplants'). A number of thesestudies have been carried outthroughout Europe including studiesby French and Italian Myeloma studygroups and the European BoneMarrow Transplant (EBMT) group.

The strategy used in these studieshas been to evaluate the role of aninitial autologous transplant followed

Allogeneic transplantationby Prof N H Russell, Consultant Haematologist, Nottingham City Hospital

The role of allogeneic transplantationin myeloma remains controversialand generates frequent debateamongst haematologists who lookafter patients. Although allogeneictransplants for myeloma have beenperformed for over 20 years, theyhave mainly been carried out inyounger patients up to the age of50, and are usually only suitable forthe small minority of youngerpatients who have a brother or sister(sibling) with a matched tissue typewho can act as a donor.

What continues to intriguehaematologists about the use ofallogeneic stem cell transplantationfor myeloma is that there is evidencethat this may offer a chance of curefor some patients.

However, the treatment remainscomplicated and associated withsignificantly greater risks thanautologous stem cell transplantation(using your own cells), whichremains the other major alternativetreatment option.

Indeed, autologous stem celltransplantation has been thestandard of care for patients withmyeloma under the age of 65 yearsfor several years. Furthermore, there are an increasing number ofnon-transplant options for patientswith myeloma such as treatmentcombinations that include Velcadeand Revlimid, which are effective inprolonging the survival of patientswho relapse after autologoustransplantation.

All of these developments havegiven rise to clear evidence of animproving outlook for myelomapatients. Population-based studiesfrom the United States and

Scandinavia have shownimpressive improvements over thelast decade in both five- and 10-year survival particularly for patientsunder the age of 60. This is mostlikely to reflect the impact of recentadvances in treatment and theirwidespread dissemination intoclinical practice.

So where does this leaveallogeneic transplantation?The number of allogeneictransplants performed in the UK isrelatively low. In 2007 just 37 werereported to the British Society ofBone Marrow Transplantation(BSBMT) across all blood cancers,compared with 756 patientsundergoing autologoustransplantation during the sameyear. The BSBMT has recentlyproduced a table of guidance forwhen to consider transplantationfor a variety of diseases includingmyeloma.

Currently for those patientsconsidered "fit for intensivetherapy" an autologous transplantis considered to be a standardtreatment. An allogeneic transplantcould be considered as a "clinicaloption" in selected younger

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a few months later by a mini allo-transplant using cells from a siblingdonor. In these studies this tandemautologous / allogeneic transplanthas been compared with either a single autologous or adouble autologous transplantprocedure. The concept behindthis approach is to try to get thepatient to achieve the best possibleresponse with the autologoustransplant prior to proceeding to amini allo-transplant.

The aim of the mini allo-transplantis to make use of a 'graft versusmyeloma effect' (see Box 1) toeradicate any persisting myelomacells, leading to long-term survivaland, potentially, "cure".

What are the long-term resultsof the mini allo-transplantstudies? In a recent update, two of thestudies performed by the EBMTand by the Italian myeloma studygroup, show statistical evidence ofa reduced risk of relapse emergingat five to six years in the group ofpatients receiving the mini allo-transplant compared with thegroup of patients just receivingautologous transplantation.

This currently translates into asurvival advantage for the tandemtransplant group. In the EMBTgroup, for example, the resultsshow 65% of patients surviving atsix years compared with 50% in the patients receiving autologoustransplantation alone.

However, this apparent advantagecomes at a price as a significantproportion of patients go on todevelop chronic Graft versus HostDisease (GvHD – see Box 2) whichcan be debilitating and requireongoing immuno-suppressivetherapy for some years. In the

Italian study 50% of patientsdeveloped this complication and atfour years 28% of patients were stillon immuno-suppressive treatmentsuch as steroids for the GvHD.

So there clearly are toxicitiesassociated with this approach. Alsoit is important to see if this apparentadvantage continues with longerfollow-up. Some study groups haveattempted to reduce the risk ofGvHD by using specific techniquesto prevent this complication fromoccurring; unfortunately, thisapproach has been associatedwith poorer results.

What about unrelated donortransplants?The previously mentioned studieshave only recruited patients withsibling donors. This means there isless information to guide usconcerning unrelated donortransplants and in general thesemust be regarded asdevelopmental. That said, there aresome circumstances where thisoption might be considered, suchas in a young patient with poor riskfeatures at diagnosis (such ascoexisting plasma cell leukaemia) or in a young patient whosemyeloma is behaving aggressivelyfollowing initial treatment. However,for patients with advancedrefractory myeloma, an unrelateddonor transplant is unlikely to bebeneficial.

So where does this leave us? In Nottingham we have notundertaken a conventional high-intensity allogeneic transplant formyeloma for several years.However, we continue to considersuitable younger patients for thecombination of autologous followedby mini allo-transplant. Generallywe only consider this option in

Patient Experience

Read more about allogeneictransplantation froma patient’s point ofview on page 16

An important part of any allogeneictransplant procedure and a crucialdifference from autologoustransplantation is to exploit a 'graftversus myeloma effect'. Graftversus myeloma is thought to bemediated by special blood cellscalled T cells. T cells in myelomapatients do not function as theyshould, so the T cells from thedonor attack and kill any persistingmyeloma cells in the patientfollowing the transplant.

Therefore allogeneictransplantation relies on twodifferent ways of killing themyeloma cells: firstly, the effects of the high- or reduced-intensitytreatment the patient receives priorto the transplant; secondly, the'graft versus myeloma effect' whichmay operate over several monthsin the post-transplant period and islacking when the patientundergoes an autologoustransplant procedure.

Studies have clearly demonstratedthat the risks of transplant-relatedmortality have fallen to 10-15%with the use of mini allo-transplants, therefore theprocedure is considerably saferthan the conventional high-intensityallogeneic transplant but still notwithout risk.

Graft versus myeloma effect Box 1

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The development of GvHD is adouble-edged sword: severeGvHD can be life-threatening butmild or moderate GvHD isassociated with the advantageous'graft versus myeloma effect'.Therefore, some GvHD isbeneficial, but too much GvHD isa serious complication of thetransplant procedure. The majorcauses of transplant-relatedmortality following allogeneictransplantation in myeloma arepost-transplant infections and thedevelopment of graft versus hostdisease (GvHD).

Graft versus myeloma withoutsevere GvHD is the holy grail of allogeneic transplantation inmyeloma.

Allogeneic stem celltransplant A procedure in which stem cells or bone marrow from acompatible donor (usually asibling) are collected, stored andgiven to the patient after high-dose chemotherapy treatment.

Autologous stem celltransplantA procedure in which a patient's own stem cells are collected, stored and then givenback following high-dose chemotherapy. Patients may havetwo autologous transplants – oneas part of initial treatment and a second at relapse (sometimescalled a double transplant), or they may just have one (single) autologous transplant.

Mini allogeneic transplant Also called mini allo-transplant or reduced intensity conditioningtransplant. A type of allogeneictransplant that uses lower dose of chemotherapy than astandard allogeneic transplantand therefore avoids some of theside-effects and risks associated with higher-dose chemotherapy.

Tandem transplantA planned double transplant procedure. It can be two autologous stem cell transplantsor an autologous followed by amini-allogeneic transplant.

Total body radiationTreatment with X-rays, gammarays or electrons to damage or kill cancerous cells. Rarely, it canbe used over the whole body inpreparation for a transplant.

GvHD can be characterised by selective damage to the liver, skin and mucosa, and the gastrointestinal tract. Skin GvHDcan manifest in a diffuse rash similar to the one pictured above.

patients under the age of 55 yearswith a matched sibling donor.

We have used the same approachfor the mini allo-transplant as theEBMT group, which is based onthat pioneered in Seattle someyears ago and uses low-dose totalbody radiation combined withfludarabine. This type of pre-treatment for a mini allo-transplantis well tolerated by the patient andoften the stay in hospital is limitedto a week or so. We haveobserved chronic GvHD in some of these patients but they haveresponded well to immuno-suppressive therapy.

The final decision aboutproceeding to a mini allo-transplantis difficult and the patient needscareful counselling about the risksand possible benefits of theprocedure. It is often useful forthem to spend time with aMyeloma Clinical Nurse Specialistwho can discuss these risks andact as a patient advocate in thedecision-making process.

Summary Although the role of allogeneictransplant in myeloma is thesubject of much ongoing debateand indeed controversy, there islittle doubt that it has its place,likely a very important one, in thetreatment of myeloma. This isespecially true for the youngerpatient group.

Any questions?

Myeloma Infoline0800 980 3332

askthenurse@myeloma.org.uk

www.myeloma.org.uk

Graft versus Host Disease Box 2 Medical terms

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L I V I N G W I T H M Y E L O M A

Since my transplant two and a half years ago I have been onThalidomide as a maintenancetherapy. My doctor has recentlytold me that she is planning totake me off it. Can you tell mewhy?Thalidomide has been proven to be an effective treatment for use atdifferent stages of myeloma, eitheron its own or in combination withother drugs.

One focus of the national myelomastudy MRC Myeloma IX was todetermine whether there is abeneficial effect for patients of usingThalidomide as a maintenancetherapy (i.e. used continuously untilthe myeloma comes back).

The latest analysis of the resultsemerging from this study hasindicated that maintenanceThalidomide may not result in abetter outcome for patientscompared with when nomaintenance treatment is used. It isimportant to stress that no harmfuleffects of using Thalidomidemaintenance have been reportedfrom the study either.

Given that these recent findingssuggest there are no proven benefitsto remain long-term on Thalidomide,the current recommendation is thatdoctors discuss stoppingmaintenance therapy with theirpatients. There remain, however,clinical circumstances whereThalidomide maintenance is anappropriate treatment option.

More work and analysis is necessaryto give a complete picture of the roleand effects of Thalidomide as amaintenance therapy.

Ask the nurseby Ellen Watters RGN,, Myeloma Information Nurse Specialist, Myeloma UK

Myeloma UK has developed a range of patient information for AL amyloidosis patients. Call theInfoline 0800 980 3332 or visit www.myeloma.org.uk /amyloidosis

AL amyloidosisinformation

I have heard that prescriptioncharges have been scrappedfor cancer patients in England.Can you tell me more aboutthis and what is happening inthe rest of the UnitedKingdom? From 1 April 2009, all patients inEngland receiving treatment forcancer or the effects of cancer havebeen entitled to free prescriptions.

Patients seeking free prescriptionsshould apply for an exemptioncertificate from their GP surgery orhaematology clinic; this certificatecan then be shown to pharmacistsas proof of exemption. Patients canalso claim a refund for anyprescriptions paid for since 1 April.

The certificate lasts for five years and can be applied for again once itexpires.

Prescriptions have been free inWales since 2007; they will be freein Northern Ireland by 2010 andScotland will phase out prescriptioncharges by April 2011 (their cost will be reduced year-on-year untilthat time).

I have had myeloma for threeyears 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 termused for a group of diseases where an abnormal protein, called amyloid, is produced. AL amyloidosis is oneform of amyloidosis where the amyloid is produced as a consequence ofabnormal plasma cell production in the bone marrow.

Amyloid is broken down by the bodyonly very slowly, so it builds up intissues and organs, disrupts theirfunction and causes symptoms.

Each patient has a different pattern ofamyloid deposition, with differentorgans affected. Amyloid can build upin the kidneys, heart, liver, spleen,nerves, or digestive system, and mayaffect two or more organs at the sametime.

AL amyloidosis is a relatively raredisease, with approximately 500 – 600people diagnosed in the UK each year.About 10 – 20% of myeloma patientsalso have AL amyloidosis, or go on todevelop it. Due to similarities betweenmyeloma and AL amyloidosis they are almost always treated the sameway, and, for most patients, myelomaremains the priority in terms oftreatment.

12

L I V I N G W I T H M Y E L O M A

Complementary medicine is based on different practices and ideas, andconsists of many different approachesto both treat and understand theorigin of disease. Patients choose touse a complementary therapy for anumber of reasons including: toimprove general wellbeing andemotional state, to gain some sort ofpersonal control over the impactmyeloma is having on their life and tohelp deal with the symptoms andcomplications of myeloma and itstreatment.

Complementary therapies can beused in addition to and alongsideconventional myeloma treatmentprescribed by your doctor. Theyshould not be confused withalternative therapies, which are those used instead of conventionaltreatment.

Types of complementarytherapies There are many forms ofcomplementary therapies. The most well known and commonly used forms include acupuncture,aromatherapy, homeopathy, massage, medication, music therapy,

Complementary Therapies

reflexology, relaxation, visualisationand yoga. Different therapies canbe used to treat different problems,and the approach used dependsvery much on your particular illness,complaint or preference. Whatworks for one person may not bebeneficial to someone else.

Combining conventional andcomplementary approaches Conventional treatment (i.e. typesof chemotherapy) is the treatmentthat almost all patients withmyeloma will receive. Althoughconventional treatment approachesdo have their limitations, there isusually a great deal of publishedevidence, derived from rigorouslyconducted clinical studies, tosuggest that these treatments workand improve patients' lives.

Generally, such studies have not been conducted withcomplementary therapies.Therefore it is very hard to makeany specific recommendationsabout the use of complementarytherapies and the likely impact theyhave on an individual patient or their myeloma.

by Eve Hallam, Myeloma Information Nurse Specialist, Myeloma UK

Although complementary therapies are increasingly accepted andintegrated into conventional medicine,further research is needed to betterunderstand exactly how they work and the ways in which they interact,positively, negatively or if at all, withconventional treatment.

While complementary therapies may help to alleviate symptoms and side-effects of myeloma and itstreatment, they should be taken only alongside, and not instead of,conventional medical treatment. Always inform your doctor of anycomplementary therapies you intend to take or participate in.

What follows is an overview of some of the more common complementarytherapies:

Acupuncture focuses on improvingoverall wellbeing, rather than atreatment of a specific symptom.Acupuncture is part of traditionalChinese medicine and uses thebalance of the body's own life force to restore wellbeing. Practitioners ofChinese medicine believe that the body has a system of chi (life force)which becomes unbalanced whensomeone is ill. The acupunctureneedles are applied to areas where this flow has been blocked, in order to restore balance and health. During acupuncture the body releasesendorphins (the body's naturalmorphine), which help to reduce pain,relax muscles and increase feelings of wellbeing. The principal aim ofacupuncture is to recover theequilibrium between the physical,emotional and spiritual aspects of the individual.

13

L I V I N G W I T H M Y E L O M A

Meditation is a relaxing mentalexercise that can help to reduceanxiety, stress and pain. Breathingtechniques and concentration areused to relax each part of the body in turn. Meditation is usually carriedout in a quiet room and in a sittingposition for about 15 – 20 minutes.You can learn to guide your ownmeditation and can use music andaromatherapy candles to helprelaxation, relieve tension and aidsleep.

Music Therapy is the use of musicand sound to promote relaxation andto reduce treatment or disease-related anxiety. Although musictherapy does not treat physicalsymptoms, it aims to reduce thesymptoms of disease such as pain,which are often exacerbated byanxiety. Music therapy is usuallyprovided through a one-to-one orgroup session with a trained musictherapist. This will usually lastbetween 30 minutes and an hour.Music therapists have a wide-range oftechniques that they may use during

Aromatherapy is the use ofessential oils (concentrated plantoils) and massage to promoterelaxation, alleviate stress and relievesymptoms of anxiety. The scent andproperties of different oils vary, and avariety of oils is used to producedifferent results. Oils like lavender arerelaxing and soothing, whereaslemon and other citrus oils areinvigorating. It may be best to avoidusing oils on the skin when you arehaving chemotherapy orradiotherapy as the skin canbecome sensitive, but the perfumeof lightly scented candles can beused to help with nausea and to aidrelaxation. You should always betreated by a registeredaromatherapist who is experiencedin treating people with cancer.

Homeopathy is a form of medicinein which patients are treated withsmall quantities of naturalsubstances that trigger symptomsmuch like those that are in need ofbeing 'cured'. The body is thusencouraged to heal itself.Homeopathic remedies come intablet form, liquids or creams. Some remedies are tailor-made for the individual's symptoms, takingtheir whole physical and mentalwellbeing into account. Otherremedies are ready-prepared andcan be bought in chemists andhealth food shops. Remember todiscuss with your doctor anyremedies you plan to take.

Massage is used to relieve musclepain and tension and can be boththerapeutic and relaxing. A trainedmasseuse or masseur will gentlymanipulate problem areas to easetightened muscles and relieve pain.Remember to tell the masseuse /masseur that you have myeloma and that forceful massage coulddamage your bones.

Before you start treatmentwith a complementarytherapy:

• Tell your doctor

• Use a reliable, qualifiedpractitioner who hasexperience with cancerpatients

• Use a practitioner you feelcomfortable with

• Remember, there are no"miracle" cures

• Don't stop yourconventional myelomatreatment

• Do not pay too muchmoney for any therapy

sessions. Individual needs andmusical preferences are discussed inthe initial session, and the techniquesthen used are based on these.

During a session, live or pre-recorded music may be played bythe music therapist. Pre-recordedmusic is sometimes provided for youto listen to between therapysessions, or you may be encouragedto listen to your favourite pieces ofmusic whilst relaxing at home. This isdesigned to induce a relaxed state ofmind. Central to music therapy is thepowerful association that music canhave with different periods of our life;listening to specific types of musiccan promote pleasant thoughts andmemories.

Other techniques used in musictherapy involve composition andsong-writing, lyric analysis andimprovisation. You may be providedwith musical instruments such aspercussion and wind instruments,keyboards, or be encouraged to singin order to create individual sets andpieces of music.

The music therapist may alsoencourage discussion about thechosen music and lyrics, in order toestablish the emotion and meaningbehind them. In this sense, musictherapy is designed to promotecommunication with others and self-confidence. This may be especiallybeneficial to people who find itdifficult to talk about their diseaseand their emotions.

You do not have to have a musicalbackground, or be able to play amusical instrument or sing toparticipate in music therapy. Thepremise behind this type of therapy isnot music learning – although thismay be an added benefit – but toprovide an alternative and enjoyableway of expressing emotions.

14

L I V I N G W I T H M Y E L O M A

Institute forComplementaryMedicine (ICM)

0207 922 7980

www.i-c-m.org.uk

Reflexology is a specialised form of foot massage, based on thetheory that different areas of the footrepresent and are connected withthe body's internal organs. Pressureis applied to different points on thesole of the foot to help relieve painand sickness. This type of massagecan be very relaxing.

Relaxation is the use of breathingexercises to bring about a feeling ofcalmness by concentrating on yourbreathing rhythm and the tighteningand relaxing of muscles. It is usuallydone lying down wearing looseclothing.

Reiki is an ancient form of healingwhich uses the body's own energyor life force to restore a sense ofbalance or calmness, releasetension and reduce pain. The reikihealer will channel energy throughtheir hands to various parts of yourbody. Although he or she will notactually touch you, you may feelsensations of heat, cold, vibrationand tingling on the skin. You are fullydressed while receiving reiki healing.

Visualisation involves the use ofmental imagery while you are in astate of meditation or relaxation. Itcan be used as a relaxation tool or toreduce stress and anxiety. Picturingyourself within a peaceful scene willencourage relaxation. Other forms ofvisualisation are more closely linkedto the symptoms of illness. Somepatients imagine their immunesystem destroying the myelomacells, their blood counts coming up,or visualise themselves becomingstronger.

Yoga is a form of exercise thatinvolves stretching and breathingand is used to unite mind, body andspirit. Light yoga exercises can beuseful in strengthening muscles andbone, while yoga breathing exercisescan be very relaxing.

(Monday – Friday, 10am – 4pm)

“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.”

Summary Using complementary therapies can give you a feeling of control over your myeloma and treatment,enabling you to do somethingpositive and pro-active to manageany symptoms and side-effects.Carers and family members may also find therapies aimed atreducing tension and promotingrelaxation helpful.

If you are thinking of using any ofthese therapies you should look for a qualified, registered therapist whohas experience of treating cancerpatients. The Institute forComplementary Medicine can becontacted for a list of registeredpractitioners in your area (contactdetails are provided in the box to the right).

Many cancer centres offercomplementary therapies, but notalways free of charge, or can giveyou the contact details of servicesavailable locally.

15

A L A M Y L O I D O S I S

By Eve Hallam, Myeloma Information Nurse Specialist, Myeloma UK

Living with AL amyloidosis: Heart and / or kidney involvement

IntroductionThis article focuses on thesymptoms you may expect withheart and / or kidney involvementafter a diagnosis of AL amyloidosis,and what can be done to managethese symptoms. This is the first in aseries of similar articles looking atdifferent aspects of living with AL amyloidosis. Future articles willinclude information about managingthe symptoms caused by amyloid inother organs, including the nervousand digestive systems.

SymptomsAmyloid and the heartIn AL amyloidosis heart problemsusually occur as a result of amyloiddeposits within the heart muscle.This causes the heart to become stiff and affects its ability to pumpeffectively, leading to fatigue,shortness of breath and fluidretention often causing swollenankles.

Amyloid and the kidneysKidney problems usually occur inpeople with AL amyloidosis as adirect result of amyloid deposits inthe filtering system of the kidney, orsometimes by the drugs used totreat AL amyloidosis.

Amyloid deposits in the kidney mostoften cause a condition called'nephrotic syndrome'. This is wherethe damaged kidneys leak healthyblood protein (called albumin) intothe urine. This loss of albumin fromthe bloodstream causes water toleak out from the blood vessels intothe tissues. This causes swelling(oedema) which is most noticeable in the lower legs and feet.

Symptom managementLow salt dietIf your heart or kidneys are affectedyou may be advised to go on a lowsalt (sodium) diet. Salt is made up oftwo elements, sodium and chloride.These are used by the body tomaintain water balance in the bloodand tissues. If you have heart orkidney problems you may already beretaining more fluid than normal. Byreducing your salt intake this canhelp reduce the fluid retention thatcauses a strain on your heart,increases your breathlessness, andleads to swelling in your feet, ankles,legs and abdomen (oedema).

Fluid restrictionYou may have your fluid intakerestricted to less than two litres perday by your doctor. This is also tohelp reduce the fluid retentiondiscussed above. If you find therestriction difficult try sucking on icecubes or boiled sweets, garglingwith ice cold water, or chewing gum.

Daily weightYour doctor may recommend youweigh yourself daily. This helps tomonitor any fluid retention and allows you to manage this at homeby increasing your diuretic (water)tablets as agreed with your doctor.

In order to do this you will need tobuy a set of accurate digital scalesand weigh yourself at the same timeevery day. You should then keep arecord of how much you weigh andnote any increases or decreases.Your doctor will advise you how to manage your diuretic tabletsaccording to any weight gain or loss.

Exercise Gentle forms of exercise such aswalking, swimming, cycling, gentleaqua-aerobics, gentle gym work, yogaand tai chi are good for overall healthand can help reduce fatigue andimprove circulation.

You may have less energy during andafter treatment and feel frustrated thatyou cannot do as much as you couldbefore. Try to do just a small amountof exercise to start with and see howyou feel, both immediately afterwardsand a day or so later. Make sure youonly do what feels comfortable.

Exercise may also be particularlybeneficial after you have finished acourse of chemotherapy treatment.Your doctor will be able to refer you toa physiotherapist who will advise youon an exercise regime. Remember todiscuss any changes to your diet orlevel of exercise with your doctorbefore starting.

Summary If your heart and / or kidneys areaffected by AL amyloidosis you mayexperience some of the symptomsoutlined here. These may be alleviatedthrough a combination of prescribedtreatment and lifestyle changes, andafter treatment for the underlying AL amyloidosis your organ functionmay improve slowly over time.

Myeloma UK has developed a range ofpatient information for AL amyloidosispatients. To order your copy of Livingwith AL amyloidosis – Your EssentialGuide, call the Infoline 0800 9803332. This information is available atwww.myeloma.org.uk / amyloidosis

Further information

16

P A T I E N T E X P E R I E N C E

I was diagnosed with myeloma inNovember 2005. The followingMarch, I had a stem cell transplantusing my own stem cells(autologous). This held up well untilfifteen months later, when myparaprotein levels started to riseagain.

My Consultant at Poole GeneralHospital gave me a choice: haveanother autologous transplant andthen face another problem fifteenmonths down the road, or considerhaving a donor transplant(allogeneic) using somebody else'sstem cells, which could potentiallycure me.

I was referred to anotherConsultant, at SouthamptonGeneral Hospital, who advised that Iwas in the right age bracket (I'mcurrently 52), albeit at the top end,to have a transplant. He told methat I was too old to have totalradiation (I was relieved to hearthat), then he explained theprocedure to me: chemotherapy, towipe out the system, followed bythe transplant. He explained that, inmany ways, it would be similar towhat I had already gone throughbefore, but more arduous.

There was also the 30% mortalityrate to consider. I told him thatwithout the transplant, there was avery good chance it'd be a 100%rate in my case, so I agreed to goahead with it.

As I don't have any brothers orsisters, I was told that my tissuestatus would be analysed on theAntony Nolan Trust bone marrowcomputer system, which searchesworldwide for suitable donors.

My experience of allogeneic transplantationKevin Brownless, myeloma patient, Dorset

I was soon advised that there werefour suitable donors for me, in the UK alone.

Incidentally, with 'allo' transplants,the doctors like to give menyounger women's stem cells as,apparently, there's something in thecells which protects againsttesticular cancer.

In the meantime, I was put on acourse of Thalidomide and

dexamethasone, which got me backinto remission within three months.Towards the end though, I hadterrible diarrhoea and so my PooleHospital Consultant put me on acourse of Velcade injections. Aftereight injections though, my stomachswelled up and, again, I had verybad diarrhoea, so I stopped taking it and had a clear, drug-free monthbefore I went into SouthamptonHospital for the transplant.

(I often wondered why, if I was backin remission, my Consultant hadthen put me on Velcade. Much lateron, I learned that Thalidomide andVelcade work in completely differentways when killing the cancer cells;the latter 'mops up' anythingoverlooked 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 actualtransplant four days later. Having been through it all before, it wasnothing new, other than the cellswere given to me fresh, as opposedto having been frozen first.

However, I was also given a newdrug called cyclosporin (an immune-suppressive drug to reduce the riskof Graft versus Host disease(GVHD), where the stem cells canreject the body) and that caused meto have paranoid psychosis. I wentloopy for a while, imagining all sortsof weird things, such as I was underPolice surveillance, my wife's carhad been stolen and, apparently, I even tried to escape from myhospital bed!

My whole mouth lining disintegratedand my throat swelled up, making it

“My Consultant gave me achoice: have anotherautologous transplant and then face another problem fifteen months

down the road, or considerhaving a donor transplant

(allogeneic) using somebody else’s stem cells, which could

potentially cure me.”

Kevin Brownless

17

P A T I E N T E X P E R I E N C E

very difficult and uncomfortable toswallow. I have since learned withthis is called mucositis, which I don'trecommend. I eventually ended upin intensive care for a few days witha lung infection while they sortedme out, which included putting atube down my throat to feed me.

Thankfully, I don't remember any ofthat. My blood pressure dropped(which it had also done during myprevious transplant) and it took awhile for them to balance it. After sixweeks, I was discharged.

At the time of writing this, over sixmonths have passed since thetransplant and I feel fine. I've put ona lot of weight, thanks to my wife'sexcellent cooking (I lost 4 kilos inhospital) and am back to my normalweight now. When I wasdischarged, there was a long list ofthings I couldn't do or eat / drink.

Here are just a few examples:

• Alcohol

• Nuts

• Spices

• Herbs

• Unpeeled fruit or salad

• Seafood (apart from fish)

• Honey

• Meat from a delicatessen

I also had to avoid caffeine and onlydrink water if it had been boiled first(even though it's filtered twice in myhouse beforehand). Decaffeinated tea was fine though.

I had to avoid all contact withcrowds and I couldn't see my fouryear old grand-daughter for 6months (what with schools beingbreeding grounds for germs).Anyone who wanted to see me hadfirst to be interrogated to make sure

they hadn't got any bugs, as Iwon't have a fully functioningimmune system for 12 monthspost-transplant.

After three months, the restrictionswere lifted a little; for example I cannow mingle with people, drinkcoffee, eat salad and see mygrand-daughter.

Needless to say, there have a smallnumber of problems since thetransplant.

I thought I'd broken a tooth insidemy gum. The fragment eventuallycame out. It turned out that itwasn't a bit of tooth, it was a bit ofjaw: I had necrosis of the jaw,caused by the two shots ofZometa (bisphosphonate) which Ihad had two years before.

I also developed GvHD afterreceiving a top-up shot oflymphocytes (white cells) from mydonor to boost my depletedimmune system. After myConsultant spotted that I looked abit pink on my chest and legs,what followed was two weeks ofhaving to smear steroid Betnovatecream all over my body. My wifehad to don rubber gloves and domy back. I assure you, the noveltysoon wore off!

Lately, I've noticed that my upper right hand teeth and a small area ofcheek above them are numb. This is under investigation; I'm not surewhat it is, maybe nerve damagefrom the necrosis.

The Consultants, however, arepleased with my progress and,apparently, my bone marrow isalready a 98% clone of the donor's (a 34 year old woman – I'd love toknow who she is); it appears I'vereversed the aging process!

I am full of optimism about thefuture.

Explains high-dose therapy andstem cell transplantation, why

they are needed, and thepotential advantages and

disadvantages of treatments. Italso discusses what will happenduring the procedure, as well asoutlining what to expect during

the recovery process

Further information

Myeloma Infoline0800 980 3332

askthenurse@myeloma.org.uk

High-Dose Therapy and Stem Cell

Transplantation Infoguide

Medical Matters

See page 8 for an article about allogeneic transplantation by Prof Nigel Russell, ConsultantHaematologist at Nottingham City Hospital

Finally, I would like to thank myConsultants, Dr Fergus Jack atPoole and Dr Kim Orchard atSouthampton and all of the staff atSouthampton and Poole GeneralHospitals' Oncology departmentsfor their truly amazing work.

18

Exceptional funding for drugs: patientexperience and government policy

P O L I C Y A N D P O L I T I C S

bbyy SSaarraahh RRiittcchhiiee, Policy and Advocacy, Myeloma UK

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.

The experience of patients is at the

heart of the issue and must form the

basis of reform.

The experience of patientsThere 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

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.

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 isoften 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 appropriateexpertise on decision-makingpanels. 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

Accessing Treatments on theNHS: An Information Pack

Myeloma UK has produced anAccess to Treatments Pack forpatients as a guide to theprocesses involved in securingaccess to myeloma treatments.We hope it will empower patientsand their family members, togetherwith their doctors, to gain accessto treatments.

It provides information on:

• The role of the Primary CareTrust (PCT) in deciding whattreatments it funds

• The role of the National Institutefor Clinical Excellence (NICE)

• How exceptional case fundingprocesses work

The pack also provides advice topatients on:

• How to get the right decisionfrom the PCT

• Applying for exceptional fundingand appealing against anegative decision

• Additional action to consider • How Myeloma UK can help

Further information

P O L I C Y A N D P O L I T I C S

will be looking at a wide array ofdifferent applications each time theymeet. The lack of understandingabout myeloma is worryingly clearfrom some PCT refusal lettersMyeloma UK has seen.

To refuse an application withouthaving sought alternative relevantexpertise is not the right way to bemaking decisions on life-extendingcancer drugs.

It is almost impossible to provethat someone is "exceptional".Clinicians must prove to the PCT thattheir patient is 'more exceptional'than other patients with the samecancer. Often this is wronglyinterpreted by PCTs to mean that thepatient must be clinically "unique".This is impossible to fulfil as it isalways imaginable for another patientto be similar.

PCTs refuse applications onimproper grounds. Even thoughthe Department of Health tells PCTsthat they cannot refuse funding forindividual drugs on the grounds ofcost alone, or because there is nosupporting NICE guidance for thedrug, in practice these are oftenused as grounds for refusing anindividual patient's request.

Furthermore, with limited data oncost-effectiveness available somePCTs confuse cost-effectivenesswith the drug's net price. Thisimmediately generates a bias againstpatients who require an expensivedrug, whether or not the drug inquestion is cost-effective.

The Government's proposedreformsThe Government says that new NHSConstitution will help address theproblem by stating that:

"You have the right to expect localdecisions on funding of other drugs

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.

To order your pack or for more information contact:Sarah Ritchie 0131 557 3332 sarahr@myeloma.org.uk www.myeloma.org.uk

19

20

FUNDRAISING IN ACTION

Sub-zero polar race in aid of

Myeloma UK

On 9 January, Myeloma UK Chief Executive Eric Low attendedSt Mary's College in Hull to receivea cheque for over £20,000, raised by staff and students of the College.

The fundraising took place inmemory of Christine Leech whohad myeloma. It was largelyorganised by her husband PhilLeech and Julie Carr, both assistanthead teachers at the College.

The College's fundraising effortsbegan with the Humber Bridge Half-Marathon on 29 June 2008, withover 40 students and staff takingpart and raising £7,500 insponsorship. The fundraisingcontinued with everyone at theCollege getting involved in a widerange of events during the year

including a Year Seven disco, a race night and an event wherestudents were allowed to throw wetsponges at their teachers.

The fundraising target of £20,000was reached just before Christmasat a staff pantomime. Eric Lowcommented: "A huge thank you to all of the staff and pupils of St Mary's College for raising suchan impressive amount for MyelomaUK. The range and diversity offundraising activities have beenremarkable and it's obvious thateverybody at the College has beeninvolved in some way. It has reallybrought the students togethertowards a common goal.'' More information can be found at www.st-marys.hull.sch.uk/MyelomaHalfMarathon.htm

St Mary's College completes a six-month fundraising drive, raising

£20,960 for Myeloma UK

After finding out one of his veryclose friends had myeloma, JulianEvans wanted to do something toshow his support. He decided to dosomething truly unique and signedup to compete in the 'Polar Race', a competitive 350 mile team raceacross the arctic to the magneticnorth pole.

The race started in mid April andthey completed it six weeks later.Julian raced on skis, pulling hissupplies in 120Ib pulks, stopping attwo checkpoints en route to stockup on essentials and he facedtemperatures as low as -50 C.

Julian has so far raised over£10,000 from the challenge andhas just taken part in the Edinburghmarathon as well. Well done fromeveryone at Myeloma UK.

Myeloma UK was one of 24 Scottish-based charities to receive adonation from the Hospital SaturdayFund at an event at Glasgow's CityChambers 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.

Hospital Saturday Fund presents £1,000 cheque to Myeloma UKThe Hospital Saturday Fund is a registered charity which this year celebrates its 60th anniversary.

Chief Executive of the HospitalSaturday Fund, Keith Bradley, said:"We're delighted to put money backinto the communities aroundScotland". Thank you to the HospitalSaturday Fund for their kind support. Go to www.hsf.eu.com for more information. Pictured: Keith Bradley, Lawrence Orr

and Carol Smillie

21

Sunday 10 May saw 300 Myeloma UK supportersflexing 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 assembledbefore Myeloma UK Patron Maureen Lipman andcelebrity ambassadors Dom and Sandi Wood officiallystarted the walk.

The event raised in excess of £70,000, and everypenny will be used to fund our research programme.Thanks to all those who took part in the day andhelped to make it such a success.www.myeloma.org.uk/rtw

Stand up for myelomain Manchesterraises £5,000

When Geoff Woodruff was diagnosed with myeloma in 2008 itcame as a great shock to his family.They decided they wanted to dosomething positive to show theirsupport for Geoff and so the entirefamily of seven decided that theywould take part in the Bupa GreatManchester 10K Run on Sunday 17 May. The whole family completedthe run successfully, and altogetherthey raised over £3,000.

Entire family takes partin the Great ManchesterRun for their dad

On Sunday 24 May, Myeloma UK held a comedy night'Stand up for myeloma' at the Comedy Store inManchester. Over 400 people attended the event, withtop comedians Stephen K Amos, Jack Whitehall, IvanBrackenbury, Alex Boardman and Justin Moorhouseproviding the entertainment for the evening.

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 alongand supported the event, and to those who helped selltickets or provided advertising for the event brochure.

www.myeloma.org.uk/comedy

On 31 May, Mike Derry, (pictured left) an architect from Devon, embarked on his second marathonchallenge in three months. He ranthe Edinburgh Marathon, having alsotackled the gruelling South DevonCoastal Marathon back in February.Mike ran both marathons in aid of hisdad Geoff, who was diagnosed withmyeloma last year. Since announcinghis challenges in January, Mike hasalready raised more than £2,500through sponsorship.

Double marathon challenge for myeloma

FUNDRAISING IN ACTION

Myeloma UK Retail Therapy Walk

raises £70,000

More about the Edinburgh Marathon will

follow in the next issue of Myeloma Matters.

Pictured right 'The Woodruff Runners'

(left to right): Ian, Liz, Al, Carole, Kath,

Jess, James.

22

M Y E L O M A U K N E W S

Myeloma UK awards twoMy working dayWhen I accepted this job, Iremember Eric saying to me, "I wish I had your job". As timegoes by, and the more I learnabout myeloma and Myeloma UK, I completely understand why Eric said this to me.

In January 2009 I started atMyeloma UK, coordinating all ofour Patient to Patient programmes.A key part of this role is workingwith the network of MyelomaSupport Groups across the UK,helping new ones to get off theground and providing assistance to existing groups. I am alsoexcited about future projects wehave planned such as a 'buddy'network. This programme will putpatients in contact with otherpatients all over the UK. We arealso working towards setting up a representative patient panel to help develop and progressMyeloma UK services.

Rebecca Wight, Patient to Patient Programmes, Myeloma UK

I love the fact that no two days arethe same. One day I am directing film clips for Myeloma TV(www.myelomatv.org.uk) andanother I am designing a patientdiary, a tool patients can use inconjunction with their doctors andnurses to track and record theirsymptoms and treatment. Every dayis so busy but all it takes is a phonecall or email from a patient to stopme in my tracks. The feeling ofcommunity between myelomapatients, family and friends makesthis a unique environment to be part of.

I was fortunate to attend the LondonMyeloma Support Group meetingone week into the job. By far thebest part of the day was speaking topatients and their families andfriends: the positive impact belongingto the group had on them wasevident. On a daily basis I communicate by phone and email

with Support Group Leaders and itcontinually astounds me how muchtime and dedication they put intothe running of their groups.

I am really looking forward tomeeting more Support GroupLeaders and fellow group membersat forthcoming Myeloma SupportGroup meetings, Patient and FamilyInfodays and other Myeloma UKevents.

If you have any queries regardingany of the patient programmes atMyeloma UK, please do nothesitate to get in touch.Rebecca on 0131 557 3332 rebecca@myeloma.org.uk

New videos have been added to Myeloma TV and are available to view now atwww.myelomatv.org.uk. The new clips include Dr Paul Richardson from theDana-Farber Cancer Institute in Boston talking about new insights in myeloma. Dr Richardson is one of the world leaders in the research and treatment ofmyeloma 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 theirexperiences of myeloma. They include Neil Atterson who was diagnosed withmyeloma in his mid-thirties, and carer Thea Scott who talks about her role as acarer 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.

New videos added to Myeloma TV – watch now

23

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 NovemberTo register your place contact Kirsty 0131 557 3332 kirsty@myeloma.org.ukor register online www.myeloma.org.uk

Rat Race Urban Adventure

To register your place contact Lorna 0131 557 3332 lorna@myeloma.org.uk

• Edinburgh 18 / 19 July• London 26 / 27 September

• BristolSaturday 19 September

Retail Therapy Walk for Research

For details contact Sara 0131 557 3332 sara@myeloma.org.ukwww.myeloma.org.uk/rtw

Join Team Myeloma UK to take part in this unique team event.

Join a sponsored walk with a difference –

F O R T H C O M I N G E V E N T S

2009 Raffle for Research

WalkingTours Holiday

in Spain

Apple iPodtouch

£250John Lewis

voucher

£100ASOS.com

voucher

with High Point Holidays

£19,000

£18,000

£17,000

£16,000

£15,000£14,000

£13,000

£12,000

£11,000

£10,000£9,000

£8,000

£7,000

£6,000

£5,000£4,000

£3,000

£2,000

£1,000

£20,000

Runners up prizes: 10 lucky runners up will receive

a £20 Oddbins voucher

£1per ticket

To order your raffle books contact Lawrence 0131 557 3332 lawrence@myeloma.org.ukwww.myeloma.org.uk/fr

1st

2nd

3rd

4th

Myeloma Matters editorial and production team Medical Editor: Dr Gordon Cook Director, Blood and Marrow Transplantation Programme St James’s University Hospital, LeedsEditor: Jude Watson Editorial support: Sara MorganDesign: Linda Scott-McFarlane

Myeloma UKChairman: Judy Dewinter Chief Executive: Eric Low Patron: Maureen Lipman CBEBoard 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 Mattersis not intended to take the place of medical care or the advice of a doctor. Your doctor should always beconsulted regarding diagnosis and treatment. No part of this newsletter may be reproduced in any way without prior permission from Myeloma UK

Myeloma UKBroughton House, 31 Dunedin Street, Edinburgh EH7 4JG Tel: 0131 557 3332 Fax: 0131 557 9785Email: myelomauk@myeloma.org.uk Myeloma Infoline: 0800 980 3332www.myeloma.org.uk Charity No. SC 026116National Myeloma Week 21 – 28 June