2008 Booklet Understanding MPD

7/29/2019 2008 Booklet Understanding MPD

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Understanding

Myeloprolierative

Disorders

A guide or patients and amilies


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NOTES


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CONTENTS

PAGE

Introduction 3

TheLeukaemiaFoundation 4

Bonemarrow,stemcellsandbloodcellormation 8

Whataremyeloprolierativedisorders? 14

Whatcausesmyeloprolierativedisorders? 15

Whichdoctor? 16

Polycythaemia(rubra)vera 17

Essentialthrombocythaemia(ET) 26

Idiopathicmyelobrosis 31

Chroniceosinophilicleukaemia/hypereosinophilicsyndrome 35

Chronicneutrophilicleukaemia 36

Systemicmastocytosisormastcelldisease 37

Complementarytherapies 38

Makingtreatmentdecisions 39

Inormationandsupport 42

Useulinternetaddresses 43

Glossaryoterms 44

Requestingmoreinormation 58


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ACkNOwlEdGEmENTSTheLeukaemiaFoundationgrateullyacknowledgesLilianDalyorresearchandauthorshipothisbookletandtheollowinggroupswhohaveassistedinthedevelopmentandrevisionothe

inormation:peoplewhohaveexperiencedamyeloprolierativedisorderasapatientorcarer,especiallyKenYoungandmembersoMPD-Oz,LeukaemiaFoundationsupportservicessta,nursingstaand clinicalhaematologists representing the variousstatesandterritoriesoAustralia.ThecartoonillustrationsweredrawnbyBrettHansen.

TheLeukaemiaFoundationalsograteullyacknowledgesShireAustraliaPtyLtdoritssupportintheproductionothisbooklet

throughanunrestrictededucationalgrant.

December 2008


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INTROdUCTIONThisbooklethasbeenwrittentohelpyouandyouramilytounderstandmoreaboutmyeloprolierative disorders(alsoknownasMPD).

Someoyoumaybeeelinganxiousoralittleoverwhelmediyouorsomeoneyoucareorhasbeendiagnosedwithamyeloprolierativedisorder.Thisisnormal.Perhapsyouhavealreadystartedtreatmentoryouarediscussingdierenttreatmentoptionswithyourdoctorandyouramily.Whateverpointyouareat,wehopethattheinormationcontainedinthisbookletisuseulinansweringsomeoyourquestions.Itmayraiseotherquestions,whichyoushoulddiscusswithyourdoctor,orspecialistnurse.

Youmaynoteellikereadingthisbookletromcovertocover.Itmightbemoreuseultolookatthelistocontentsandreadthepartsthatyouthinkwillbeomostuseataparticularpointintime.

Wehaveusedsomemedicalwordsandterms,whichyoumaynotbeamiliarwith.Thesearehighlightedin italics.Theirmeaningisexplainedinthebookletand/orintheglossaryotermsatthebackothebooklet.

Insomepartsothebookletwehaveprovidedadditionalinormationyoumaywishtoreadonselectedtopics.Thisinormationispresentedintheshadedboxes.Someoyoumayrequiremoreinormationthaniscontainedinthisbooklet;wehaveincludedsomeInternetaddressesthatyoumightnduseul.Inaddition,manyoyouwillreceivewritteninormationromthedoctorsandnursesatyourtreatinghospital.

Itisnottheintentionothisbooklettorecommendanyparticularormotreatmenttoyou.Youneedtodiscussyourparticularcircumstancesatalltimeswithyourtreatingdoctor.

Finally,wehopethatyoundthisbookletuseulandwewouldappreciateanyeedbackromyousothatwecancontinuetoserveyouandyouramiliesbetterintheuture.


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THE lEUkAEmIA FOUNdATIONThe Leukaemia Foundation is the only national not-or-protorganisationdedicatedtothecareandcureopatientsandamilieslivingwithleukaemias,lymphomas,myelomaandrelatedblooddisorders.Since1975,theFoundationhasbeencommittedtoimprovingsurvivalorpatientsandprovidingmuchneededsupport.The Foundation does not receive direct ongoing governmentunding,relyinginsteadonthecontinuedandgeneroussupportoindividualsandcorporatesupporterstodevelopandexpanditsservices.

TheFoundationprovidesarangeo reesupportservicestopatientsandtheircarers,amilyandriends.Thissupportmaybe

oeredoverthetelephone,acetoaceathome,hospitalorattheFoundationsoceoraccommodationcentres,dependingonthelocationandindividualneeds.Supportmayincludeprovidinginormation, patient education seminars and programs thatprovideaorumorpeersupportandconsumerrepresentation,practical assistance, accommodation, transport and emotionalsupport/counselling.

TheLeukaemiaFoundationundsleadingresearchintobettertreatmentsandcuresorleukaemias,lymphomas,myelomaandrelatedblooddisorders.ThroughitsNationalResearchProgram,theFoundationhasestablishedthePwCFoundationLeukaemiaandLymphomaTissueBankandtheLeukaemiaFoundationResearchLaboratoryattheQueenslandInstituteorMedicalResearch.Inaddition,theFoundationundsresearchgrants,scholarshipsandellowshipsortalentedresearchersandhealthproessionals.


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Foundation sta provide patients and their amilies with inormation and support acrossAustralia

TheLeukaemiaFoundationhasateamohighlytrainedandcaringSupportServicesstawithqualicationsand/orexperienceinnursingorallied health thatworkacross the country.They canoerindividualsupportandcaretoyouandyouramilywhenitisneeded.

SupportServicesmayinclude:

Inoration

TheLeukaemia Foundation has arange obooklets, actsheetsandresourcessuchasthisonethatareavailablereeocharge.

Thesecanbeorderedviatheormatthebackothisbookletordownloadedromthewebsite.Translatedversions(inlanguagesotherthanEnglish)osomebookletsandactsheetsarealsoavailableromourwebsite.

Eucation & Support progras

TheLeukaemiaFoundation oers youand youramily disease-specicandgeneraleducationandsupportprogramsthroughout

Australia.Theseprogramsaredesignedtoempoweryouwithinormationaboutvariousaspectsodiagnosisandtreatmentandhowtosupportyourgeneralhealthandwellbeing.

Support Services


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Eotiona support/counseing

Adiagnosisoabloodcancer/disordercanhaveadramaticimpactonapersonslie.Attimesitcanbediculttocopewiththeemotionalstress involved.TheLeukaemiaFoundationsSupportServicesstacanprovideyouandyouramilywithmuchneeded

supportduringthistime.TheymayreeryouoralovedonetoaspecialisthealthproessionalegPsychologistirequired.

Onine discussion Foru

TheFoundationhasestablishedanon-lineinormationandsupportgrouporpeoplelivingwithleukaemia,lymphoma,myeloma,orarelatedblooddisorder.Registrationisreeandparticipantscanremainanonymous,seewww.talkbloodcancer.com

Accooation

Somepatientsandcarersneedtorelocateortreatmentandmayneedhelpwithaccommodation.TheLeukaemiaFoundationstacanhelpyoutondsuitableaccommodationclosetoyourhospitalortreatmentcentre.Inmanyareas,theFoundationsullyurnishedsel-containedunitsandhousescanprovideahomeawayrom

homeoryouandyouramily.

Transport

TheFoundationalsoassistswithtransportingpatientsandcarerstoandromhospitalortreatment.Courtesycarsandotherservicesareavailableinmanyareasthroughoutthecountry.

Practica Assistance

TheurgencyandlengthydurationomedicaltreatmentcanaectyouandyouramilysnormalwayolieandtheremaybepracticalthingstheFoundationcandotohelp.Inspecialcircumstances,theLeukaemiaFoundationprovidesnancialsupportorpatientswhoareexperiencingnancialdicultiesorhardshipsasaresultotheirillnessoritstreatment.Thisassistanceisassessedonanindividualbasis.


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Young Auts

AwebsiteoryoungadultshasbeendevelopedcalledRevive.Thissitehasinormationspecicallydesignedoryoungadultsandcontainsadiscussionorumtoallowpatienttopatientinteractionandsupport.Thesiteiswww.teamrevive.com

Contacting us

TheLeukaemiaFoundationprovidesservicesandsupportineveryAustralian state and territory. Everypersons experienceolivingwiththesebloodcancersanddisordersisdierent.Livingwithleukaemias,lymphomas,myelomaandrelatedblooddisordersisnoteasy,butyoudonthavetodoitalone.Pleasecall800 0 0(Freecall)tospeaktoalocalsupportservicesta

memberortondoutmoreabouttheservicesoeredbytheFoundation.Alternatively,contactusviaemailbysendingamessagetoino@leukaemia.org.auorvisitwww.leukaemia.org.au


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8BONE mARROw, STEm CEllS ANdBlOOd CEll FORmATION

Bone arroBone marrowisthespongytissuethatllsthecavitiesinsideyourbones.Mostoyourbloodcellsaremadeinyourbonemarrow.Theprocessbywhichbloodcellsaremadeiscalledhaemopoiesis.Therearethreemaintypesobloodcells;red cells, white cellsandplatelets.

Asaninant,haemopoiesistakesplaceatthecentreoallbones.

Inlaterlie,itislimitedtothehips,ribsandbreastbone(sternum).Someoyoumayhavehadabonemarrowbiopsytakenromtheboneatthebackoyourhip(theiliaccrest)orthebreastbone.

Youmightliketothinkothebonemarrowasthebloodcellactory.Themainworkersattheactoryarethebloodstem cells.Theyarerelativelysmallinnumberbutareable,whenstimulated,toreproducevitalnumbersoredcells,whitecellsandplatelets.Allbloodcellsneedtobereplacedbecausetheyhavelimitedlie

spans.

Therearetwomainamiliesostemcells,whichdevelopintothevarioustypeobloodcells.

Bone marrow


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Myeloid (my-loid) stem cells develop intored cells, white cells (neutrophils, eosinophils,

basophils and monocytes) and platelets.

Lymphoid (lim-oid) stem cells develop into twoother types o white cells called T-lymphocytes and

B-lymphocytes.

Groth actors an cytoines

Allnormalbloodcellshavealimitedsurvivalincirculationandneedtobereplacedonacontinualbasis.Thismeansthatthe

bonemarrowremainsaveryactivetissue throughoutyourlie.Naturalchemicalsinyourbloodcalledgrowth actorsorcytokinescontroltheprocessobloodcellormation.Dierentgrowthactorsstimulatethebloodstemcellsinthebonemarrowtoproducedierenttypesobloodcells.

Thesedayssomegrowth actorscanbemadeinthe laboratory(synthesised)andareavailableoruseinpeoplewithblooddisorders.Forexample,granulocyte-colonystimulatingactor(G-

CSF)stimulatestheproductionowhitecellscalledneutrophils,whileerythropoietin(EPO)stimulatestheproductionoredcells.Unortunately,drugstostimulateplateletproductionhavebeenlesssuccessul,butresearchiscontinuinginthisarea.

Boo Ste Ces

myeoi Ste Ce line lyphoi Ste Ce line

Re Ces white Ces Pateets B-yphocytes T-yphocytes

Neutrophis, Eosinophis, Pasa CesBasophis, monocytes


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Anaeia

Anaemiaisaconditioncausedbyareductioninthenumberoredcells,whichinturnresultsinalowhaemoglobin.Measuringeitherthehaematocritorthehaemoglobinwillprovideinormationregardingthedegreeoanaemia.

Iyouareanaemicyouwilleelrundownandweak.Youmaybepaleandshortobreathoryoumaytireeasilybecauseyourbodyisnotgettingenoughoxygen.Inthissituationaredcelltransusionmaybegiventorestoretheredcellnumbersandthereorethehaemoglobintomorenormallevels.

white ces

Whitecells,alsoknownasleukocytes,ghtinection.Therearedierenttypesowhitecellsthatghtinectiontogetherandindierentways.

Neutrophils kill bacteria and ungi.

Eosinophils kill parasites.

Basophils work with neutrophils to fght inection.

Monocytes work with neutrophils and lymphocytesto fght inection; they also help withantibody production and act as scavengersto remove dead tissue. These cells areknown as monocytes when they areound in the blood and macrophageswhen they migrate into body tissues tohelp fght inection.

T-Lymphocytes kill viruses, parasites and cancer cells;produce cytokines.

B-Lymphocytes mak e an t i bod ie s wh ich t a r ge tmicroorganisms.

Whenyourwhitecellcountdropsbelownormalyouareatriskoinection.

The normal adult white cell count varies between3.7 and 11 (3.7 - 11 x 109/L)


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Neutropenia

Neutropeniaisthetermusedtodescribealowerthannormalneutrophilcount.Iyouhaveaneutrophilcountolessthan1(1x109/L)youareconsideredtobeneutropenicandat riskodevelopingrequentandsometimessevereinections.

The normal adult neutrophil count varies between2.0 and 7.5 (2.0 - 7.5 x 109/L)

Pateets

Plateletsaredisc-shapedcellularragmentsthatcirculateinthe

bloodandplayanimportantroleinclotormation.Theyhelptopreventbleeding.Iabloodvesselisdamaged(e.g.byacut)theplateletsgatheratthesiteoinjury,sticktogetherandormaplugtohelpstopthebleeding.

The normal adult platelet count varies between 150and 400 (150 - 400 x 109/L)

Throbocytopenia

Thrombocytopeniaisthetermusedtodescribeareductionintheplateletcounttobelownormal.Iyourplateletcountdropsbelow20(20x109/L),youareatriskospontaneouslybleedingandtendtobruiseeasily.Platelettransusionsaresometimesgiventobringtheplateletcountbacktoasaelevel.

Thenormalbloodcountsprovidedheremaydierslightlyromtheonesusedatyourtreatmentcentre.Youcanaskoracopyoyourbloodresults,whichshouldincludethenormalvaluesoreachcelltype.

Chiren

Inchildren,somenormalbloodcellcountsvarywithage.IyourchildisbeingtreatedoranMPDyoucanaskyourdoctoror

nurseoracopyotheirbloodresults,whichshouldincludethenormalvaluesoreachbloodtypeoramaleoremalechildothesameage.


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Inchildren,somenormalbloodcellcountsvarywithage(seetablebelow).

month

year

years

years

years

years

Haemoglobin

(g/L)

102-130 104-132 107-136 110-139 113-143 115-165F

130-180M

Whitecellcount(x109/L)

6.4-12.1 5.4-13.6 4.9-12.8 4.7-12.3 4.7-12.2 3.5-11

Platelets(x109/L)

270-645 205-553 214-483 205-457 187-415 150-450

Neutrophils(x109/L)

0.8-4.9 1.1-6.0 1.7-6.7 1.8-7.7 1.8-7.6 1.7-7.0


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wHAT ARE mYElOPROlIFERATIVEdISORdERS?MyeloistheGreekwordormarrowandprolierativeisanother

wordorgrowingorreproducing. Myeloprolierative disorders

thereoreareagroupodisordersinwhichthebonemarrow

stemcellsgrowandreproduceabnormally.Inmyeloprolierative

disordersabnormalstemcellsproduceexcessnumbersooneor

moretypesobloodcells(redcells,whitecellsand/orplatelets).

These abnormal cellscannot unctionproperly and can cause

serioushealthproblemsunlessproperlytreatedandcontrolled.

It is important toremember, asyou read through this booklet,that myeloprolierative disorders arechronicdiseasesthat,in

mostcases,remainstableormanyyearsandprogressgradually

overtime.Thesymptomsandcomplicationsomyeloprolierative

disordersdescribedinthisbookletdonotoccurineveryone,and

maynotoccurormanyyears.

Types o yeoproierative isorers

Myeloprolierativedisordersareusuallydescribedaccordingtothe

typeobloodcellwhichismostaected.Thereare4maintypes

omyeloprolierativedisordersthattogetherrepresentaround95

percentoallcases:

1.ChronicmyeloidleukaemiareerCMLbooklet

2. Polycythaemiavera(PV)toomanyredcells

3. Essentialthrombocythaemia(ET)toomanyplatelets

4. Idiopathicmyelobrosisbonemarrowtissueis

replacedbybrousscar-liketissue.Thisdisruptsnormal

bloodcellproduction.

Myeloprolierative disordersare closely related diseases, so its

not uncommon or people to have eatures o more than one

myeloprolierative disorder when they are rst diagnosed, or

duringthecourseotheirillness.Insomecases,onedisordermay


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transormovertimetoanother,ortoatypeoleukaemiacalled

acutemyeloidleukaemia.

Lesscommontypesomyeloprolierativedisordersinclude:

Chronic neutrophilic leukaemia (CNL) too many

neutrophils(atypeowhitecell)inbloodandbonemarrow

Chroniceosinophilicleukaemia(CEL)/hypereosinophilic

syndrometoomanyeosinophils(anothertypeowhite

cell)inbloodandbonemarrow

Chronicmyelomonocyticleukaemia(CMML)toomany

monocytes (a type o white cell) in blood and bone

marrow

Systemicmastocytosistoomanybasophils (atypeo

whitecell)inbloodandbonemarrowandmastcells

(relatedtobasophils)inskinandothertissues

Myeloprolierativediseaseunclassiable

wHAT CAUSES mYElOPROlIFERATIVEdISORdERS?The exact cause o myeloprolierative disorders remains

unknownbuttherearelikelytobeanumberoactorsinvolved.

Myeloprolierativedisordersaresometimesdescribedasbeing

clonalbloodstemcelldisorders.Thismeansthattheyresult

romachange,ormutation,intheDNAoasinglebloodstemcell.Thischange(orchanges)resultsinabnormalbloodcell

developmentandinthiscasetheoverproductionobloodcells.

Inmyeloprolierativedisorderstheoriginalmutationispreserved

whentheaectedstemcelldivides(prolierates)andproducesa

clone;agroupoidenticalstemcellsallwiththesamedeect.

Mutationsindividingcellsoccurallthetimeandhealthycellshave

sophisticatedmechanismswithinthemtostoptheseabnormalitiespersisting.Butthelongerwelive,themorechancewehaveo

acquiringmutationsthatmanagetoescapethesesae-guards.Thats


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whymyeloprolierativedisorders,likemostleukaemiasandother

cancers,becomemorecommonaswegetolder.

Amutationoaparticulargene(asegmentoDNAthatmakes

proteins)knownasJanuskinase2(JAK2)isoundinalarge

proportionopeoplewithmyeloprolierativedisorders.Theexactmeaningothismutationremainsunclearbutitappearstoplaya

roleintheoverproductionobloodcellsseeninthesedisorders.

ThediscoveryoamutationintheJAK2geneisimportantbecause

itislikelytohaveasignicantimpactonthewaymyeloprolierative

disordersarediagnosedandtreatedintheuture.

Finally,myeloprolierativedisordersarenotcontagious;youcannot

catchthedisorderbybeingincontactwithsomeonewhohasone.Mostpeoplewithamyeloprolierativedisorderhavenoamily

historyothedisease.

wHICH dOCTOR?

IyourGPsuspectsthatyoumighthaveanMPD,youwillbe

reerredtoanotherspecialistdoctorcalledahaematologistor

urther tests and treatment. A haematologist is a doctor who

specialisesinthecareopeoplewithdiseasesotheblood,bone

marrowandimmunesystem.


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POlYCYTHAEmIA (RUBRA) VERAPolycythaemia(rubra)vera,alsoknownasprimarypolycythaemiavera,isadisorderinwhichtoomanyredcellsareproducedinthebonemarrow,withoutanyidentiablecause.Thesecellsaccumulateinthebonemarrowandinthebloodstreamwheretheyincreasethebloodvolumeandcausethebloodtobecomethicker,ormoreviscousthannormal.Inmanypeoplewithpolycythaemiavera,toomanyplateletsandwhitecellsarealsoproduced.

Polycythaemiaveraisararechronicdiseasediagnosedinanestimated2to3per100,000population.Althoughitcanoccuratanyage,polycythaemiaverausuallyaectsolderpeople,withmostpatientsdiagnosedovertheageo55years.Polycythaemia

veraisrareinchildrenandyoungadults.Itoccursmorecommonlyinmalesthaninemales.

what is seconary or reactive poycythaeia?

Insecondaryorreactivepolycythaemia,redcellproductionisincreasedinresponsetoexcessamountsoerythropoietin(aredcellgrowthactor)circulatinginthebloodstream.Highlevelsoerythropoietincanbearesponsetolowerthannormallevelsooxygenintheblood(orexampleathighaltitudes,inheavysmokersandinpeoplewithheartorlungdisease).Thisisauseulcompensatorymechanismthathelpsthebodytoproducemoreredcellsandhaemoglobintotransportmoreoxygenaroundthebody.Erythropoietinlevels,andthereoreredcellproductionmayalsobeincreasedabnormallyinsometypesokidneydiseaseandinsometypesocancer.

In a condition known as relative, apparent or spuriouspolycythaemia,thevolumeoplasma(theliquidportionotheblood)isreduced,usuallyasaresultodehydration,vomitingordiuretic(fuidloss)therapy.Thisincreasestheconcentrationoredcellsinthebloodbuttheactualredcellmass(thetotalnumberoredcells)remainsnormal.


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8SYmPTOmS ANd COmPlICATIONS OFPOlYCYTHAEmIA VERA

Manypeoplehavenosymptomswhentheyarerstdiagnosedwithpolycythaemiaveraandthediseaseispickedupaccidentallyduringaroutinebloodtestorphysicalexamination.Inothercases,peoplegotoseetheirGPbecausetheyhavesometroublingsymptomsotheirdisease.

Whensymptomsdooccur,theydevelopgraduallyovertime.Theyaremainlyduetotheincreasedthickness(hyperviscosity)andabnormallyhighnumbersobloodcellsinthecirculatingblood.Commonsymptomsinclude:headaches,blurredvision,atigue,weakness,dizziness,itchiness(pruritis),especiallyaterahotbath,

andnightsweats.Enlargementothespleen( splenomegaly)isalsocommonandoccursinaround75percentocases.Symptomsincludeeelingsodiscomort,painorullnessintheupperlet-sideotheabdomen.Anenlargedspleenmayalsocausepressureonthestomachcausingaeelingoullness,indigestionandalossoappetite.Insomecasesthelivermayalsobeenlarged.Thisiscalledhepatomegaly.

Somepeopleexperiencegout,whichusuallypresentsasapainulinfammationothebigtoeoroot.Thiscanresultromabuildupouricacid,abyproductotheincreasedproductionandbreakdownobloodcells.Someindividualsmaydeveloperythromelalgia,arareconditionthatprimarilyaectstheeetand,lesscommonly,thehands.Itischaracterisedbyintense,burningpainoaectedextremities,andincreasedskintemperaturethatmaybeepisodicoralmostcontinuousinnature.

Inmanycases,peoplewithpolycythaemiaverahavearuddy(red)complexion,andareddeningothepalmsothehandandsolesotheeet,earlobes,mucousmembranesandtheeyes.Thisisduetothehighnumbersoredcellinthecirculation.Araisedbloodpressure(hypertension)isalsocommon.

Boo cots (throbosis) an beeing

Asthebloodisthickerthannormalitcannotfowaseasily,

especiallythroughthesmallerbloodvessels.Iletuntreated,thisincreases the risko thrombosis, the ormation o a blood clotwithinabloodvessel.Bloodclotscanorminvariouspartsothebodyincludingthedeepandsupercialveinsothelegs,inthe


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heart(causingamyocardialinarctionorheartattack)andinthebrain(causingastroke).Bloodclotsareacommoncomplicationopolycythaemiaveraandoccurinaround30percentopeople,evenbeoretheyarediagnosed.Olderpeopleandthosewithahistoryoapreviousbloodclotareatincreasedrisk.Amajoraim

otreatmentinpolycythaemiaveraistomaintainanormalbloodcountandreduceyourriskothrombosis.

Bleedingandeasybruisingcanalsooccur.Thisisusuallyminorandoccursinaroundonequarteroallpatients.Occasionallybleedingintothegutcanbeprolongedorsevere.

HOw IS POlYCYTHAEmIA VERA dIAGNOSEd?

Polycythaemiaveraisdiagnosedusingacombinationolaboratorytestsandaphysicalexamination.

Fu boo count

Peoplewith polycythaemiaverahaveahighredcellcount,haemoglobinlevelandhaematocrit(>52%inmenor>48%inemales)duetotheexcessiveproductionoredcells.Araisedwhitecellcount(especiallyaraisedneutrophilcount)andaraisedplateletcountarealsocommonndings.

Theredcellmassisthetotalnumberoredcellscirculatinginyourblood.Polycythaemiaveraisdiagnosedwhentheredcellmassis25%greaterthantheaveragenormalexpectedvalue.Otherndingsthathelpconrmthediagnosisopolycythaemiaveraincludeanenlargedspleen(splenomegalytheenlargedspleencanpoolredbloodcellsthusshowingaalsebloodreadingintheveins)and/orthepresenceotheJAK2mutationorother

cytogeneticabnormalitiesinyourbloodorbonemarrowcells.


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0

measuring your re ce ass

The blood test that measures your red cell mass may take acouple o hours to complete. It involves taking a sample o yourblood rom a vein in your hand or arm, mixing it with a special

substance called an isotope, and reinjecting it back into yourbloodstream. Ater this more blood tests are taken over the nexthour to measure your red cell mass and compare it to expectednormal values.

Bone arro exaination

Inpolycythaemiaverathebonemarrowisotenveryactivewith

abnormallyhighnumbersonormalcells.Ironstoresmaybedepleted since iron is being used to make more andmore redcells.

Bone arro aspirate an biopsy

A procedure that involves removing a small core o bone marrowor examination in the laboratory. The biopsy (or trephine) istaken under local or general anaesthetic, rom the back o thehip. A sample o bone marrow and sot inner bone is withdrawnor testing under a microscope

Other possibe boo tests

serumvitaminB-12levels

uricacidlevels

erythropoietinlevels

coagulationstudies(toseeiyourbloodisclottingnormally)

bloodoxygenlevels

Other possibe tests

ChestX-raytoruleoutlungdisease

Abdominalultrasoundand/ orCTscantoruleoutkidneydiseaseandmeasurespleen/liversize


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JAK2mutationtest-sensitivemethodsdemonstratethecommon

V617Fmutationinover95%opatientswithpolycythaemiavera.AdditionalmutationselsewhereintheJAK2geneappeartoaccountortherest.

HOw IS POlYCYTHAEmIA VERA TREATEd?Thegoalotreatmentorpolycythaemiaveraistoreducethenumberocellsinyourbloodandhelpyoutomaintainanormalbloodcount.Thishelpscontrolanysymptomsoyourdiseaseandreducestheriskocomplicationsduetobloodclotting,orbleeding.Thetreatment,orcombinationotreatmentschosenoryouwilldependonseveralactorsincludingthedurationandseverityoyourdisorder,whetherornotyouhaveahistoryobloodclots,

yourageandyourgeneralhealth.

Venesection

Venesection(orphlebotomy)isaprocedureinwhichacontrolledamountobloodisremovedromyourbloodstream.Thisprocedureiscommonlyusedwhenpeoplearerstdiagnosedwithpolycythaemiaverabecauseitcanhelptorapidlyreduceahighredcellcount.Inaprocesssimilartoablooddonation,450mlsto

500mlsoyourbloodisremoved,usuallyromalargeveininthearm,insidetheelbowbend.Thisisusuallydoneintheoutpatientsdepartmentothehospital.Ittakesabout30minutestocomplete.Youwillneedtohaveabloodtestbeoretocheckyourbloodcount,andyoumustmakesureyoudrinkplentyowaterbeoreandatertheprocedure.

Thisproceduremayneedtoberepeatedrequentlyatrst,usuallyeveryewdays,untilyourhaematocritisreducedtothedesiredlevel.Aterthis,youmayneedtohavetheprocedurerepeatedperiodically,orexampleatmonthlyintervals,tohelpmaintainanormalbloodcount.

Formanypeople,particularlyyoungerpatientsandthosewithmilddisease,regularvenesection(everyewmonths)maybeallthatisneededtocontroltheirdiseaseormanyyears.

Manypeoplewithpolycythaemiaveraalsoneedothertreatmentsinadditionto,orinsteadovenesection,tohelpcontroltheirbloodcount.


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myeosuppressive drugs

Myelosuppressive(bonemarrowsuppressing)drugsorchemotherapyarecommonlyusedtoreducebloodcellproductioninthebonemarrow.Thesedrugsarecommonlyusedorpeoplewithanextremelyhighplateletcount,complicationsduetobloodclotting

orbleeding,orsymptomsoanenlargedspleen.Theyarealsousedorsomepeoplewhoareunabletotoleratevenesectionorwhosediseaseisnolongerrespondingtovenesection.

Themostcommonlyusedmyelosuppressiveagentisachemotherapydrugcalledhydroxyurea.Itisparticularlyuseulincontrollingahighplateletcount(thrombocytosis)inolderpatientsandthereorereducingtheriskothrombosis.Hydroxyureaistakenintheorm

oacapsuleathomeeveryday.Ashydroxyureaisachemotherapydrug,itisknowntoaectertilityandshouldbeavoidedduringpregnancyoritcancauseharmordeathtotheoetus.Ithiscouldbeanissueoryou,youshouldaskyourhaematologistaboutyouroptions.

Otherlesscommonlyusedchemotherapydrugisbusulphan.Thisdrugisalsogivenintabletorm.

Chemotherapytakenincapsuleormistoleratedwellbymostpeopleandsideeectstendtobeewandmild.Asthesedrugsworkbysuppressingbloodormation,periodicbloodtestsshouldbeperormedwhentakingthesedrugstomonitorthebloodcountandtoguardagainstseverereductionsinthewhitecellorplateletcounts.Thereisaverysmallriskodevelopingleukaemialateron,inpeoplewhoreceivechemotherapyorprolongedperiodsotime.Thisriskisvery small or people receiving hydroxyureaand

mustbeweighedagainstthepotentiallyseriouscomplicationsouncontrolleddisease(thrombosis).Discusswithyourdoctorithisisaconcerntoyou.

Intereron

Intereronisasubstanceproducednaturallybythebodysimmunesystem.Itplaysanimportantroleinghtingdisease.Inpolycythaemiavera,intereronissometimesprescribedoryounger

patientstohelpcontroltheproductionobloodcells.Intereronisusuallygiventhreetimesaweekasaninjectionundertheskin(subcutaneousinjection)usingaverysmallneedle.Youoraamily


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member(orriend)willbetaughthowtodothisathome.Aweeklyinjectionisnowavailableandisbecomingmorewidelyused.

Side eects o intereron can be unpleasant but they can beminimisedbystartingwithasmalldose,andbuildinguptotheulldoseoverseveralweeks.Themainside-eectsarefu-like

symptomssuchaschills,evers,achesandpainsandweakness.Yourdoctorornursewillexplainanysideeectsyoumightexperiencewhileyouarehavingthesetreatmentsandhowtheycanbemanaged.

Other Treatents

Aspirin

Manypeopleareprescribedsmalldailydosesoaspirin,whichhavebeenshowntosignicantlyreducetheriskothrombosisinpeoplewithpolycythaemiavera.

Aspirinworksbypreventingyourplateletsromclumpingtogethertoormharmulbloodclotsindierentpartsoyourbody.Aspirincanirritatetheliningothestomachwhichcanresultinpainordiscomortinthestomach,causingnausea,heartburnorlossoappetite.Takingyouraspirinwithoodormilkmayhelpprevent

this.Inaddition,manypeopleareprescribedenteric-coatedaspirinthatallowsthedrugtopassthroughthestomachandintotheintestinebeorebeingdissolved.Thishelpstoreducetheriskostomachupset.

Aspirinistakenathomeintabletorm.Druginteractionscanoccur,soitisimportanttoavoidtakingothermedicationswhileyouareonaspirin,unlessyouareadvisedtodosobyyourdoctor.

AnagrelidehydrochlorideAnagrelidehydrochloride(Agrylin )isadrugusedtoreducehighplateletcountsinpeoplewithpolycythaemiaveraandessentialthrombocythaemia.Anagrelideaectsplatelet-producingcellsinthebonemarrowcalledmegakaryocytes,slowingdownplateletproductionandthereorereducingthenumberoplateletsinthecirculatingblood.Thiscanhelptoreducesymptomsandtheriskoclottingcomplicationsintheuture.Althoughanagrelidelowersplateletcountstomorenormallevels,itdoesnotaectthebodysnaturalprocesstoormaclotwhenneeded.Anagrelideistakenincapsuleormbymouth.Itcanbetakenwithorwithoutood.


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Thecapsulestrengthandthenumberotimesadayyouneedtotakeanagrelidewilldependonyourplateletcount,yourresponsetotreatmentandhowwellyoutoleratethedrug.

Your doctor willkeeptrackoyourresponsetoanagrelideandadjustyourdoseasneededtomaintainyourplateletcountatthe

desiredlevel.Sideeectsaregenerallymildtomoderateandmaydecreasewithcontinuedtherapy.Themostcommonlyreportedsideeectsincludeheadaches,astororceulheartbeat(palpitations),diarrhoea,weakness,fuidretention,nausea,dizziness,abdominalpainandshortnessobreath.Youshouldreportanysideeectsyouareexperiencingtoyourdoctorasmanyothemcanbetreatedtoreduceanydiscomorttoyou.Youneedtocontactyourdoctorimmediatelyiyouexperiencetheollowingsymptoms:shortness

obreathordicultybreathing,swollenankles,astorirregularheartbeat,and/orchestpain.

Youshouldnotstoptakingthisoranyothermedicationorpolycythaemiaveraunlessinstructedbyyourdoctor.Stoppingthesemedicationssuddenlycanbeharmul.

Radioactive phosphorus (32P)

Radioactivephosphorus(32P)isaradioisotopewhichmaybeusedorlong-lastingcontrolobloodcountsinolderpeople.Oneortwodoseso32Pareusuallygiven,byinjectionintoaveininthehandorarm,inthenuclearmedicinedepartmentothehospital.Thissubstanceistakenupandconcentratedinbonemarrowwhereitsuppressestheoveractivebonemarrowandhelpstocontrolbloodcounts.

Inadditiontothe treatmentsdescribedabove, yourdoctor will

advise you onways tostay healthy and reduce any lie-styleactorsthatmightincreaseyourriskothrombosis.Forexampleyoumaybeadvisedtostopsmoking,and/ortakeaseriesostepstomaintainahealthyweightrangeandbloodpressure.

PROGNOSIS

Aprognosisisanestimateothelikelycourseoadisease.Itprovidessomeguideregardingthechancesocuringthediseaseorcontrollingitoragiventime.

Thenaturalcourseopolycythaemiaveracanvaryconsiderablybetweenindividuals.Inmanypatients,withtreatment,thedisease


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remainsstableorlongperiodsotime,otenmanyyears.Inaroundonethirdoallcases,polycythaemiaveratransormsovertimeintoanothertypeomyeloprolierativedisordercalledmyelobrosis,andlesscommonly,inupto10percentocasesintoacutemyeloidleukaemia.

Insomepeople,polycythaemiaprogressesovertimedespitetreatment.Thespleenmaybecomeincreasinglyenlarged.Anaemiaand thrombocytopenia (low numbers o circulating platelets)is common as the bone marrow is no longer able to produceadequatenumbersoredcellsorplatelets.Inaddition,abnormalimmaturebloodcells,knownasblastcellsmaystarttoappearintheblood.

Treatmentduringthistimeissupportiveandinvolvesmakingeveryeort toimprovethe patientsqualityolie,by relievinganysymptomstheymighthaveandbypreventingandtreatinganycomplicationsthatariseromtheirdiseaseoritstreatment.Thismayinvolvebloodtransusionsirequired,painrelieandcareulmyelosuppression.

Inselectedcases,surgicalremovalothespleen,orlowdoseradiationtothespleenmayberequiredtorelievesymptoms.

Yourdoctoristhebestpersontogiveyouanaccurateprognosisregardingyourdiseaseasheorshehasallthenecessaryinormationtomakethisassessment.


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ESSENTIAl THROmBOCYTHAEmIA (ET)Essentialthrombocythaemia(ET)isadisorderinwhichtoomanyplateletsareproducedinthebonemarrow.Plateletsarenormallyneededinthebodytocontrolbleeding.However,excessnumbersoplateletscanleadtoabnormalbloodclottingwhichcanblockthefowobloodinthebloodvessels.

Thereareanumberoconditionsthatcancauseariseinthenumberoplateletsinthecirculatingblood(thrombocytosis).Theseincludebleeding,inectionandsometypesocancer.Inessentialthrombocythaemiahowever,thebloodplateletcountispersistentlyelevatedasaresultoincreasedbonemarrowproductionoplatelets,intheabsenceoanyidentiablecause.

Like polycythaemia vera, essential thrombocythaemia is ararechronicdiseasediagnosedinanestimated3per100,000population.Althoughitcanoccuratanyage,even(rarely)inchildren,essentialthrombocythaemiausuallyaectsolderpeople,withmostpatientsdiagnosedbetweentheageso50and70years.Itoccursequallyinbothmalesandemales.

SYmPTOmS ANd COmPlICATIONS OFESSENTIAl THROmBOCYTHAEmIA

Manypeoplehavenosymptomswhentheyarerstdiagnosedwithessentialthrombocythaemiaandtheirdiseaseispickedupaccidentallyduringaroutinebloodtest.Howeverisymptomsdooccurtheygenerallyincludetinglingorburninginthehandsandeet,headache,visualproblems,weaknessanddizziness.Thesesymptomsandothersresultromexcessivenumbersoplatelets

causingblockagesinsmallorlargebloodvesselsindierentpartsothebody.

Anenlargedspleeniscommonandoccursinaround30percentocases.Symptomsincludeeelingsodiscomort,painorullnessintheupperlet-sideotheabdomen.Anenlargedspleenmayalsocausepressureonthestomachcausingaeelingoullness,indigestionandalossoappetite.Insomecasesthelivermayalso

beenlarged(hepatomegaly).Othersymptomsincludeweightlossandgeneraliseditching.


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Boo cots (throbosis) an beeing

Thrombosisisamajorcomplicationoessentialthrombocythaemia.Olderpatients andthosewith ahigh platelet count, or apriorhistoryothrombosis,maybeatincreasedrisk.Amajoraimotreatmentinessentialthrombocythaemiaistoreduceyourplatelet

count,andthereoreyourriskothrombosis.

Bloodclotscanoccurinlargeorsmallarteriesintereringwiththebloodandthereoreoxygensupplytovariousorgansortissues.Blockagesinthesmallerbloodvessels(microvasculature)inthetoesandngerscancauserednessotheskinandburningandthrobbingpains.Thesepainsareotenmadeworsebyheatorexerciseandrelievedbycoolingandelevatingtheaectedarea.

Thesesymptomsareotendramaticallyimprovedusingsmalldailydosesoaspirin,and/orreducingthepatientsplateletcount.

Blockagesinthearteriessupplyingtheheart(causingamyocardialinarctionorheartattack),kidneysorbrain(causingastroke)canbeseriousandcanleadtosignicanttissuedamageorischaemia (tissuedeath).Bloodclotscanalsodevelopintheveinsothelegs(causingdeepveinthrombosis),andlesscommonly,thespleenandliveroccludingthebloodfowandcausingpainintheseareas.

Abloodclotthatbreaksothewallotheveinandtravelsinthebloodstreamisknownasanembolism.Whenabloodclottravelstothelungsitisknownasapulmonaryembolismandcancausebreathingproblems.

Less commonly, people experience symptoms o abnormalbleedingincludingbruisingornoapparentreason,orexaggeratedorprolongedbleedingollowingminorcutsorinjury.Somepeople

noticerequentorseverenosebleedsorbleedinggumsandsomewomenmayhaveunusuallyheavymenstrualperiods.

Inpregnancy,uncontrolledessentialthrombocythaemiacanreducethebloodsupplytotheplacentaoretus.Thiscancauseproblemswithetalgrowthandmayinsomecasesleadtomiscarriage.

HOw IS ESSENTIAl THROmBOCYTHAEmIAdIAGNOSEd?

Thediagnosisoessentialthrombocythaemiaisonlymadewhenothercausesoaraisedplateletcounthavebeenexcluded.


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8Fu boo count

Apersistentlyraisedplateletcountisthemostcommonsignoessentialthrombocythaemia.Theplateletcountcanrangeromslightlyhigherthannormaltomanytimeshigherthannormal.Underthemicroscopetheplateletsmaybeabnormallylargeand

paleblue-stained.Fragmentsomegakaryocytes,thecellsromwhichplateletsarereleased,mayalsobeseeninthebloodlm.Aroundathirdopeoplewithessentialthrombocythaemiawillalsohaveamildlyraisedredcelland/orwhitecellcount.

Itheresultsoyourbloodtestsuggestthatyoumayhaveessentialthrombocythaemia,urtherinvestigationandtestsincludingabonemarrowexaminationmayberequiredtohelpconrmthe

diagnosisandruleoutothersecondaryorreactivecausesoaraisedplateletcount.


Inessentialthrombocythaemiathebonemarrowisusuallyoundtobeoveractive,similartopolycythaemiavera.Anexcessnumberoabnormalmegakaryocytesisacommonnding.Cytogeneticandmolecularanalysisobloodandbonemarrowcellsmaybe

carriedouttohelpconrmthediagnosis.AmutationintheJAK2geneisoundinasignicantproportion(50-60%)opeoplewithET.Mutationsinthec-MPLgene(whichproducesaproteinthatrespondstoagrowth actor thatstimulatesplateletproduction)accountorapproximately10%ocases.

Otherbloodtestsmaybedonetocheckyourgeneralhealthandhowwellyourkidneys,liverandothervitalorgansareunctioning.

HOw IS ESSENTIAl THROmBOCYTHAEmIATREATEd?

Thegoalotreatmentorpeoplewithessentialthrombocythaemiaistopreventcomplicationslikeabnormalbleedingandbruisingandinsomecasesreducingthenumberoplateletsintheblood.Youmaynothaveanysymptomsoessentialthrombocythaemiawhen

youarerstdiagnosedandthereoremaynotrequireanytreatmentorsometime.Insteadyourdoctormayrecommendawatchandwaitstrategywhichinvolvesregularcheck-upsandbloodcountstocareullymonitoryourhealth.Inadditionheorshewilladvise


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youonthestepsyoucantaketostayhealthyandreduceanyliestyle-relatedriskactorsyoumayhavethatincreaseyourchancesodevelopingabloodclot.Youmaybeadvisedorexampleonwaystohelpyoustopsmoking,and/ormaintainahealthyweightrangeandbloodpressure.

Forthemajorityopeople,essentialthrombocythaemiawillrequiresomeormotreatmenttoreducetheirplateletcountandthereoretheirriskothrombosis.Thetreatmentchosenoryouwilldependonanumberoactorsthatinfuenceyourparticularriskocomplicationsduetothrombosisorbleeding.Theseincludeyourage,plateletcountandwhetherornotyouhavehadanypreviousepisodesobloodclotsorbleedinginthepast.Ahistoryosmokingorhighbloodpressurecanaectyourriskothrombosis.These

actorsandothersaretakenintoaccountwhenplanningthemostappropriatetreatmentoryourdisease.

Forpeopleathigh-risothrombosis,achemotherapydrugcalledhydroxyurea(seepolycythaemiavera)withorwithoutlow-doseaspirin isotenused asrst-line treatment.Hydroxyureaworksbysuppressingtheunctionoyourbonemarrowandtherebycontrollingplateletproduction,whileaspirinpreventsyourplatelets

romaggregatingandormingharmulclotsinyourbody.Anagrelide hydrochloride (Agrylin) andintereron(seepolycythaemiavera)mayalsobeused.Anagrelideslowsdownplatelet production in your bone marrow, thereby helping toreducesymptomsandyourriskothrombosis.Intereronworksbysuppressingtheabnormalmegakaryocytecloneinyourbonemarrowtherebyreducingtheoverproductionoplatelets.

Thoseato-rismaybesimplytreatedusinglow-doseaspirinoranequivalentdrugalone.Theyusuallyhaveaverygoodoutlookwithnodierencetothegeneralpopulation.

Yourdoctorwillbeabletodiscusswithyouallothetreatmentoptionssuitableoryou.

Pateetpheresis

Iyourplateletcountisveryhighandyouhavesymptomsoclotting

orbleeding,yourplateletcountwillneedtobereducedquicklytopreventurthercomplications.Intheseemergencysituations,excessplateletscanberemovedromyourbloodstreamusingaprocedureknownasplateletpheresis.Duringthisprocedureallyourblood


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0ispassedthroughaspecialmachinecalledacellseparator.Thebloodisdrawnromacannula(plasticneedle)placedinaveininonearm.Themachinespinsthebloodveryquicklyandremovestheexcessplatelets.Thisisacontinuousprocess.Whileplateletsarebeingremovedtherestoyourbloodisbeingreturnedtoyou

viaanothercannula,placedinyourotherarm.Iyourveinsarenotsuitableorthisprocedure,aspecialwideboredoublelumencentralvenous cathetermightbeusedinstead.This lineallowsbloodtobedrawnromoneothebiggerveinsinyourbody.

Plateletpheresisisusuallycarriedoutinhospital.Itusuallytakesabouttwohourstocomplete.

PROGNOSIS

Essentialthrombocythaemiaisregardedasanincurablediseasebutinmanypeoplewithtreatment,thediseaseremainsstableorlongperiodsotime,oten10-20yearsormore.Inthelongerterm,asmallnumberopeoplewithessentialthrombocythaemiamaydevelopmyelobrosis.Theriskotransormingtoacutemyeloidleukaemiaisrelativelylow(12percent).

Yourdoctoristhebestpersontogiveyouanaccurateprognosis

regardingyourdiseaseasheorshehasallthenecessaryinormationtomakethisassessment.


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IdIOPATHIC mYElOFIBROSISIdiopathic myeloibrosis (also called chronic idiopathicmyelobrosis,agnogenicmyeloidmetaplasia)isadisorderinwhichnormal bonemarrowtissue isgraduallyreplacedwithabrousscar-likematerial.Overtime,thisleadstoprogressivebone

marrowailure.

Undernormalconditions,thebonemarrowprovidesanenetworkoreticulinbresonwhichthestemcellscandivideandgrow.Specialisedcellsinthebonemarrowknownasbroblastsmakethesebres.Inidiopathicmyelobrosis,chemicalsreleasedbyhighnumbersoplateletsandabnormalmegakaryocytes(plateletormingcells)over-stimulatethebroblasts.Thisresultsintheovergrowthothickcoarsebresinthebonemarrow,whichgraduallyreplace

normalbonemarrowtissue.Overtimethisdestroysthenormalbonemarrowenvironment,preventingtheproductionoadequatenumbersoredcells,whitecellsandplatelets.Thisresultsinanaemia,lowplateletcountsandtheproductionobloodcellsinareasoutsidethebonemarroworexampleinthespleenandliver,whichbecomeenlargedasaresult.

Idiopathicmyelobrosisisararechronicdisorderdiagnosedinanestimated1per100,000population.Itcanoccuratanyagebutisusuallydiagnosedlaterinlie,betweentheageso60and70years.Thecauseoidiopathicmyelobrosisremainslargelyunknown.Long-termexposuretohighlevelsobenzeneorveryhighdosesoionisingradiationmayincreasetheriskoidiopathicmyelobrosisinasmallnumberocases.Aroundonethirdopeoplewithmyelobrosishavebeenpreviouslydiagnosedwithpolycythaemiaoressentialthrombocythaemia.

SYmPTOmS ANd COmPlICATIONS OFIdIOPATHIC mYElOFIBROSIS

Around20percentopeoplehavenosymptomsoidiopathicmyelobrosiswhentheyarerstdiagnosedandthedisorderispickedupaccidentallyasaresultoaroutinebloodtest.Forothers,symptomsdevelopgraduallyovertime.Symptomsoanaemiaarecommonandincludeunexplainedtiredness,weakness,shortness

obreathandpalpitations.Othernonspecicsymptomsincludeever,unintendedweightloss,pruritis(generaliseditching)andexcesssweating,especiallyatnight.


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Virtuallyallpatientswithidiopathicmyelobrosishaveanenlargedspleen(splenomegaly)whentheyarerstdiagnosed.Inaroundathirdocasesthespleenisveryenlarged.Commonsymptomsincludeeelingsodiscomort,painorullnessintheupperlet-sideotheabdomen.Anenlargedspleenmayalsocausepressure

onyourstomachcausingaeelingoullness,indigestionandalossoappetite.Abdominaldiscomortcanalsoresultromanenlargedliver(hepatomegaly),whichoccursinaroundtwo-thirdsocases.

Otherlesscommonsymptomsincludeboneandjointpain,andbleedingproblems.

HOw IS IdIOPATHIC mYElOFIBROSIS

dIAGNOSEd?Idiopathicmyelobrosisisdiagnosedusingacombinationoa physical examination showing the presence o an enlargedspleen,bloodtestsandabonemarrowexamination.Idiopathicmyelobrosis isonly diagnosed whenother causes omarrowbrosis(includingleukaemia,lymphoma,othertypesocancerthathavespreadtothebonemarrow)havebeenruledout.

Fu boo count

People with idiopathic myelobrosis commonly present withvaryingdegreesoanaemia.Whenexaminedunderthemicroscopetheredcellsareotendescribedasbeingteardrop-shaped.Higherthannormalnumbersowhitecellsandplateletsmaybeoundintheearlystagesothisdisorder,butlowwhitecellandplateletcountsarecommoninmoreadvanceddisease.


Itisrequentlyimpossibletoobtainanysamplesobonemarrowfuidusinganeedleandsyringe(bonemarrowaspiration)duetomarrowbrosis.Thisisknownasadrytap.Thebonemarrowtrephinebiopsytypicallyshowsabnormalbrosisothemarrowcavity.

Cytogeneticandmolecularanalysisobloodandbonemarrow

cellsisalsocarriedouttohelpconrmthediagnosisandamutationinJAK2isoundinasignicantnumberopeoplewithidiopathicmyelobrosis.


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HOw IS IdIOPATHIC mYElOFIBROSISTREATEd?

Somepeoplehavenosymptomswhentheyarerstdiagnosedwithidiopathicmyelobrosisanddonotrequiretreatmentstraightaway,apartromregularcheck-upswiththeirdoctortocareullymonitortheirdisease.

Forotherstreatmentislargelysupportiveandisaimedatpreventingcomplicationsduetolowbloodcountsandanenlargedspleen(splenomegaly).Thisinvolvesmakingeveryeorttoimproveyourqualityolie,byrelievinganysymptomsoanaemiaoranenlargedspleen,andpreventingandtreatinganycomplicationsthatmightariseromyourdiseaseoritstreatment.Thismayincludeperiodic

bloodtransusionsandtakingantibioticstopreventandtreatanyinections.

A chemotherapy drug such ashydroxyurea (see polycythaemiavera),orlow-dosesoadrugcalledthalidomidemaybeusedtoreduceanenlargedspleen.Insomecases,thesurgicalremovalothespleen(splenectomy)maybeconsidered,especiallywhenyourspleenhasenlargedsomuchthatitiscausingseveresymptoms.Asplenectomymayalsobeconsiderediyouhaveanincreasedneedorbloodtransusions.Thissometimeshappensbecausethespleenisdestroyingbloodcells,particularlyplatelets,ataveryastrate.Smalldosesoradiationtothespleencanalsobegiventoreduceitssize.Thisusuallyprovidestemporaryrelieorabout3to6months.

Someyoungerpatientswhohaveasuitablymatcheddonormaybeoeredanallogeneic(donor)stem cell transplant.Thisisamedical

procedurethatoerstheonlychanceocureorlong-termsurvivalinpatientswithidiopathicmyelobrosis.Itinvolvestheuseoveryhighdosesochemotherapy,withorwithoutradiotherapy,ollowedbyinusionobloodstemcells,whichhavebeendonatedbyasuitablymatcheddonor.Stemcelltransplantscarrysignicantrisksandareonlysuitableorasmallminorityoyoungerpatients(usuallyunder60yearsoage).


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Boo an Pateet transusions

I symptoms o anaemia are interering with your normal dailyactivities, your doctor may recommend that you have a red bloodcell transusion. Platelet transusions are sometimes given to

prevent or treat bleeding (or example a persistent nose bleed)when the platelet count is below a critical level.

You do not need to be admitted to hospital or a red blood cellor platelet transusion. They are usually given in the outpatientdepartment. Transusions these days are relatively sae and theydont usually cause any serious complications. Neverthelessyou will be careully monitored throughout the transusion. Inthe meantime, remember to call the nurse i you are eeling hot,

cold and shivery or in any way unwell during the transusion, asthis might indicate that you are having a reaction. Steps can betaken to minimise these symptoms and ensure that they donthappen again.

PROGNOSIS

Idiopathic myelobrosis is generally regarded as an incurable

diseasebutwithtreatmentmanypeoplecanremaincomortableandsymptom-reeorsometime.

Thenaturalcourseothediseasecanvaryconsiderablybetweenindividuals. In some people their disease remains stable orlongperiodsandtheyarereetoliveanormalliewithminimalinterruptionsromtheirdiseaseoritstreatment.Forothers,idiopathicmyelobrosisprogressesmorequicklyandpeoplerequiretreatmenttohelprelievesymptomsotheirdisease.Transormationtoatypeoleukaemiacalledacutemyeloidleukaemiaoccursinbetween10and20percentocases.

Yourdoctoristhebestpersontogiveyouanaccurateprognosisregardingyourdiseaseasheorshehasallthenecessaryinormationtomakethisassessment.


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CHRONIC EOSINOPHIlIClEUkAEmIA/HYPEREOSINOPHIlICSYNdROmE

Chroniceosinophilicleukaemiaisararemyeloprolierativedisorderinwhichtoomanyeosinophils(atypeowhitebloodcell)aremadeinthebonemarrow.Thesecellsspilloutothebonemarrowandaccumulateinthebloodandothertissuesaroundthebody.Thisdisorderisdiagnosedbyaullbloodcountshowingpersistentlyelevatednumbersoeosinophils.ManypatientscarrytheFIP1L1-PDGFRalphamutation.

Somepeoplewithchroniceosinophilicleukaemiadonthaveany

symptomsandthediseaseispickedupaccidentallyduringaroutinebloodtest.Othersmaygototheirdoctorbecausetheyhaveoneormoreoarangeosymptomsincludingever,atigue,cough,musclepains,pruritis(generaliseditching)anddiarrhoea.

Chroniceosinophilicleukaemiaisararediseaseanditsnaturalcoursecanvaryconsiderablybetweenindividuals.Thediseasemayremainstableormanyyears,evendecades,oritmayquickly

progressandtransormtoacuteleukaemia.Forthisreason,themostappropriatetreatmentoreachpatientisdecidedonanindividualbasis.Treatmentmayincludecorticosteroids,chemotherapydrugssuchashydroxyurea,andintererontherapy.Somepatientsmayrespondtoanewerdrugcalledimatinibmesylate(GLIVEC),mostotenusedinthetreatmentoanothertypeoMPDcalledchronicmyeloidleukaemia.Astemcelltransplantmaybeconsideredinselectedcases.


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CHRONICNEUTROPHIlIC lEUkAEmIAChronicneutrophilicleukaemiaisanotherraremyeloprolierativedisorder in which too many neutrophils are made in the bone

marrow.Thesecellsspilloutintothecirculatingbloodandtendtoaccumulateintheliverandspleen,whichbecomeenlargedasaresult.

Chronic neutrophilic leukaemia isusually a slowly progressingdisease,closelyrelatedtoanothertypeoleukaemiacalledchronicmyeloid leukaemia.Itsnaturalcoursecanvaryconsiderablybetweenindividualswithsurvivaltimesrangingrom6monthstoover20years.Treatmentoptionsmayincludetheuseochemotherapydrugssuchasbusulphan orhydroxyurea, whicharegivenintabletorcapsuleorm.Thesedrugsareusedtocontrolthehighwhitecellcount.


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SYSTEmIC mASTOCYTOSIS OR mASTCEll dISEASESystemicmastocytosisormastcelldiseaseisadisorderthatresultsromtheoverproductionomastcells(atypeowhitebloodcell),

inthebonemarrow.Thesecellsaccumulateintheblood,lymphnodes(glands),skinandotherbodytissues.Excessnumbersomastcellsreleaselargeamountsohistamineandothersubstanceswhichcancauseallergictypereactionsinaectedtissues.Forexample,whenthesesubstancescollectintheskintheytendtocauseanitchyrash.Otherallergictypesymptomsmayincludeabdominalpainanddicultybreathing.Over90%opatientswithsystemicmastocytosiscarryamutationinthec-KITgene.

Medicationsknownas antihistaminesareusedtopreventandreduceallergicreactions.Treatmentdecisionstendtobemadeonanindividualbasisandmayincludechemotherapyintabletormand/orintererontohelpcontroltheoverproductionomastcellsinthebonemarrow.Researchindicatesnewtyrosinekinaseinhibitorsmayalsobeuseulasatreatmenttherapy.


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8COmPlEmENTARY THERAPIESComplementary therapies are not considered standardmedicaltherapies.Manypeoplehoweverndthattheyarehelpulincopingwiththeirtreatmentandrecoveryromdisease.Therearemanydierenttypesocomplementarytherapies.Theseincludeyoga,exercise,meditation,prayer,acupuncture,relaxationandherbalandvitaminsupplements.

Complementarytherapiesshouldcomplementorassistwithrecommendedmedicaltreatmentormyeloprolierativedisorders.TheyshouldnotbeusedinsteadasanalternativetomedicaltreatmentorMPD.Itisimportanttorealisethatnocomplementaryoralternativetreatmentalonehasproventobeeectiveagainst

MPD.It is aso iportant to et your octor or nurse no i youare using any copeentary or aternative treatents, in casethey interere ith the eectiveness o cheotherapy or othertreatents you ay be having.

NUTRITIONAhealthyandnutritiousdietisimportantinhelpingyourbodyto

copewithyourdiseaseandtreatment.Talktoyourdoctorornurseiyouhaveanyquestionsaboutyourdietoriyouareconsideringmakinganyradicalchangestothewayyoueat.Youmaywishtoseeanutritionistordieticianwhocanadviseyouonplanningabalancedandnutritiousdiet*.

Iyouarethinkingaboutusingherbsorvitaminsitisveryimportanttotalkthisoverwithyourdoctorrst.Someothesesubstancescanintererewiththeeectivenessochemotherapyorothertreatmentsyouarehaving.

*There is a separate Leukaemia Foundation booklet called 'Eating Well:a practical guide or people living with leukaemias, lymphomas andmyeloma', that provides more detail.


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mAkING TREATmENT dECISIONS

Manypeopleeeloverwhelmedwhentheyarediagnosedwithamyeloprolierativedisorder.Inaddition to this, waiting or testresultsandthenhavingtomakedecisionsaboutproceedingwiththerecommendedtreatmentcanbeverystressul.Somepeopledonoteelthattheyhaveenoughinormationtomakedecisionswhileotherseeloverwhelmedbytheamountoinormationtheyaregiven,orthattheyarebeingrushedintomakinga

decision.Itisimportantthatyoueelyouhaveenoughinormationaboutyourillnessandallothetreatmentoptionsavailable,sothatyoucanmakeyour own decisionsaboutwhichtreatmenttohave.

Beoregoingtoseeyourdoctormakealistothequestionsyouwanttoask.Itishandytokeepanotebookorsomepaperanda

penhandyasmanyquestionsarethoughtointheearlyhoursothemorning.

Sometimesitishardtoremembereverythingthedoctorhassaid.Ithelpstobringaamilymemberorariendalongwhocanwritedowntheanswerstoyourquestions,promptyoutoaskothers,beanextrasetoearsorsimplybetheretosupportyou.

Yourtreatingdoctor(haematologist)will

spendtimediscussingwithyouandyouramilywhatheorsheeelsisthebestoptionoryou.Feelreetoaskasmanyquestionsasyouneedto,atanystage.You are involved in making importantdecisions regarding your wellbeing.Youshouldeelthatyouhaveenoughinormationtodothisandthatthe

decisionsmadeareinyourbestinterests.Remember,youcanalwaysrequestasecondopinioniyoueelthatthisisnecessary.


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0Cinica Trias

Thesetrials(alsocalledresearchstudies)testnewtreatmentsorexistingtreatmentsgiveninnewwaystoseeitheyworkbetter.Clinicaltrialsareimportantbecausetheyprovidevitalinormationabouthowtoimprovetreatmentbyachievingbetterresultswith

ewersideeects.Cinica trias oten give peope access to netherapies not yet une by governents.

Iyouareconsideringtakingpartinaclinicaltrialmakesurethatyouunderstandthereasonsorthetrialandwhatitinvolvesoryou.Youalsoneedtounderstandthebenetsandrisksothetrialbeoreyoucangiveinormedconsent.Talktoyourdoctorastheycanguideyouinmakingthebestdecisionoryou*.

Inore consentGivinganinormedconsentmeansthatyoucanunderstandandaccepttherisksandbenetsoaproposedprocedureortreatment.Itmeansthatyouarehappythatyouhaveadequateinormationtomakesuchadecision.

Yourinormedconsentisalsorequirediyouagreetotakepartinaclinicaltrialoriinormationisbeingcollectedaboutyouor

someaspectoyourcare(datacollection).

Iyouhaveanydoubtsorquestionsregardinganyproposedprocedureortreatmentpleasedonothesitatetotalktothedoctorornurseagain.

Ho can I hep ith boo cancer research?

The Australasian Leukaemia and Lymphoma Group (clinical

trialsresearchgroup)hasestablishedanationalLeukaemiaandLymphomaTissueBankatthePrincessAlexandraHospitalinBrisbane.TheTissueBankisatemperaturecontrolledacilityorstoringclinicaltissuesamplestobeusedinapprovedresearchintoleukaemia,lymphoma,myelomaandrelatedblooddisorders.Current research ocuses onunderstanding the developmentocancers, why dierent patients respond dierently to currenttreatments and more eective therapies,especially thosebeing

*You can also reer to the inormation sheets about clinical trials on ourwebsite. There are also questions that you can ask your doctor. Seewww.leukaemia.org.au


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assessedinclinicaltrials.Theclinicaltissuesamplesusedorthisresearchcomerombloodandbonemarrowsamplesrompatients routinetestingand romsamplestakenor monitoringduringclinicaltrials.

Inordertodonateyourbloodand/orbonemarrowsamplesto

theTissueBankyouwillneedtosignaconsent oratthetimeoyourdiagnosis.Thiscanbeobtainedromyourclinician.Beassured,donatingdoesnotinvolveanyadditionalprocedures,itsimplyinvolvessavingandstoringintheTissueBankanyexcessbloodorbonemarrowextractedduringyourroutinetests.Samplesarealsowelcomedromrelapsedpatientsatre-diagnosis.

Thedonationoyourtissuesampleisaninvaluablewaytosupportbloodcancerresearchandcouldbringusclosertonding acure.Tissues rombloodcancer patientsare preciousmaterialsorresearchersbecausethesecancersarerelativelyrareandarevitalorndingcures.ForurtherinormationonthePwCFoundationLeukaemiaandLymphomaTissueBankgotohttp://www.leukaemia.org.au/web/research/tissuebank.php


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INFORmATION ANd SUPPORT*Peoplecopewithadiagnosisoamyeloprolierativedisorderindierentways,andthereisnorightorwrongorstandardreaction.Forsomepeoplethediagnosiscantriggeranynumberoemotionalresponsesrangingromdenialtodevastation.Itisnotuncommon

toeelangry,helplessandconused.Naturallypeopleearortheirownlivesorthatolovedones.

Itisworthrememberingthatinormationcanotenhelptotakeawaytheearotheunknown.Itisbestorpatientsandamiliestospeakdirectlytotheirdoctorregardinganyquestionstheymighthaveabouttheirdiseaseortreatment.Itcanalsobehelpultotalktootherhealthproessionalsincludingsocialworkersornurseswhohavebeenspeciallyeducatedtotakecareopeoplewithblood

andbonemarrowdiseases.Somepeoplendituseultotalkwithotherpatientsandamilymemberswhounderstandthecomplexityoeelingsandthekindsoissuesthatcomeuporpeoplelivingwithanillnessothisnature.

Myeloprolierative Disorders Australia www.mpd-oz.orgis a website that provides inormation to people withmyeloprolierative disorders and their amilies. Anotheruseulsiteorinormationonmyeloprolierativedisordersishttp://www.mdpino.org

I you have a psychoogica or psychiatric conition pease inoryour octor an ont hesitate to reuest aitiona support roa enta heath proessiona.

Manypeopleareconcernedaboutthesocialandnancialimpactothediagnosisandtreatmentontheiramilies.Normalamilyroutinesareotendisruptedandothermembersotheamilymaysuddenlyhavetoulllrolestheyarenotamiliarwith,or

examplecooking,cleaning,doingthebankingandtakingcareochildren.

Thereareavarietyoprogramsdesignedtohelpeasetheemotionalandnancialstraincreatedbybloodcancersandrelateddisorders.TheLeukaemiaFoundationistheretoprovideyouandyouramilywithinormationandsupporttohelpyoucopeduringthistime.ContactdetailsoryourstateoceotheLeukaemiaFoundationareprovidedonthebackothisbooklet.

*There is a separate Leukaemia Foundation booklet called Living withLeukaemias, Lymphomas, Myeloma & Related Disorders. This bookletaddresses the impact o the diagnosis, amily matters, support,survivorship, and other general issues around treatment.


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USEFUl INTERNET AddRESSES

AmericanCancerSocietywww.cancer.org

ArrowFoundation

www.arrow.org.auAustralianBoneMarrowDonorRegistrywww.abmdr.org.au

CancerBACUP(AUKcancerinormationsite)www.cancerbacup.org.uk

CancerCounciloAustraliawww.cancercouncil.com.au

CentreorGrieandLosswww.grie.org.au

LeukaemiaFoundationwww.leukaemia.org.au

LeukaemiaFoundationOn-lineSupportgroupwww.talkbloodcancer.com

LeukaemiaFoundationsNetworkorYoungAdults

www.teamrevive.comLeukemia&LymphomaSocietyoAmerica www.leukemia-lymphoma.org

LeukaemiaResearchFund(UK)www.lr.org.uk

LookGoodFeelBetterprogramwww.lgb.org.au

MyeloprolierativeDisordersAustraliawww.mpd-oz.org

MPDFoundationwww.mpdoundation.org

NationalCancerInstitute(USA)www.cancer.gov/cancerino

TheMyeloprolierativeDisordersResearchConsortiumhttp://www.mpd-rc.org/home.php

Up-To-Date(USA)http://www.mdpino.org/


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GlOSSARY OF TERmS

Acute euaeias

Rapidlyprogressingcancersothebloodandbonemarrow,usuallyosuddenonsetandcharacterisedbyuncontrolledgrowtho

immaturebloodcellswhichcrowdthebonemarrowandspilloutintothebloodstream.

Acute yeoi euaeia (Aml)

Arapidlyprogressingcancerothebloodandbonemarrow.AMLaectsdevelopingbloodcellsonthemyeloidcell line,usuallywhitebloodcells.Itismorecommoninadultsthaninchildren.

Aogeneic ste ce transpant

Thetransplantobloodstemcellsromonepersontoanother.Thedonorisusuallyasisterorbrotheroranunrelatedvolunteerdonor.

Aopecia

Hairloss.Thisisaside-eectosomekindsochemotherapyandradiotherapy.Itisusuallytemporary.

Anaeia

Areductioninhaemoglobinintheblood.Haemoglobinnormallycarriesoxygentoallthebodystissues.Anaemiacausestiredness,palenessandsometimesshortnessobreath.

Antibiotic

Adrugusedtopreventortreatbacterialinections.

Antiboies

Naturallyproducedsubstancesintheblood,madebywhitebloodcellscalledB-lymphocytesorB-cells.Antibodiestargetantigensonothersubstancessuchasbacteria,virusesandsomecancercellsandcausetheirdestruction.

Antieetic

Adrugwhichpreventsorreduceseelingsosickness(nausea)andvomiting.

Anti-ungaAdrugusedtopreventortreatungalinections.


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Antigen

Asubstance,usuallyonthesuraceoaoreignbodysuchasavirusorbacteriathatstimulatesthe cellsothebodys immunesystemtoreactagainstitbyproducingantibodies.

Antihistaine

Adrugusedtopreventorreduceallergicreactions.

Anti-vira

Adrugusedtopreventortreatviralinections.

Bast ces

Immaturebloodcellsnormallyoundinthebonemarrow.Blastcellsnormallyconstituteupto5percentoallbonemarrowcells.

Thesecellsdivideandreplenishallthenormalbloodcellsinthemarrowandcirculatingblood.Acuteleukaemiaischaracterisedbyanaccumulationoabnormalblastcellsthattakeoverthemarrowandspilloutintothebloodstream.

Boo ces

Therearethreemaintypesocells.Redbloodcellscarryoxygen,whitebloodcellsightinection,andplateletshelppreventbleeding.Normalnumbersoeachcelltypemustbemaintainedorthebodytoremainhealthy.

Boo count

Alsocalledaullbloodcount(FBC).Aroutinebloodtestthatmeasuresthenumberandtypeocellscirculatingintheblood.

Boo ste ces

Primitiveblood-ormingcellsthatnormallyliveinthebonemarrow.

Theydivideandmatureintoallthedierenttypesobloodcells(red cells, white cells and platelets), including the cells o ourimmunesystem.

B-yphocyte (B-ce)

Atypeowhitecellnormallyinvolvedintheproductionoantibodiestocombatinection.

Bone arro

The tissue oundat thecentre o manyfator bigbones othebody.Activeorredbonemarrowcontainsstemcellsromwhichallbloodcellsaremadeandintheadultthisisoundmainlyin


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thebonesmakinguptheaxialskeletonhips,ribs,spine,skullandbreastbone (sternum).Theotherbonescontaininactiveor(yellow)attymarrow,which,asitsnamesuggests,consistsmostlyoatcells.

Bone arro aspirate

Aprocedurethatinvolvesremovingasmallsampleobonemarrowfuidorexaminationinthelaboratory.Thefuidisdrawn,underlocalorgeneralanaesthetic,usuallyromthebackothehip,oroccasionallyromthebreastbone.

Bone arro biopsy

Aprocedurethatinvolvesremovingasmallcoreobonemarroworexaminationinthelaboratory.Thebiopsy(ortrephine)istaken

underlocalorgeneralanaesthetic,romthebackothehip.Itisusuallydoneatthesametimeasthebonemarrowaspirate.

Bone arro transpant

Seestemcelltransplant.

Cancer

Amalignantdiseasecharacterisedbyuncontrolledgrowth,division,accumulation,andinvasionintoothertissuesoabnormalcells

romtheoriginalsitewherethecancerstarted.Cancercellscangrowandmultiplytotheextentthattheyeventuallyormalumporswelling.Thisisamassocancercellsknownasatumour.Notalltumoursareduetocancer;inwhichcasetheyarereerredtoasnon-malignantorbenigntumours.

Cannua

Aplastictubewhichcanbeinsertedintoaveintoallowfuidto

enterthebloodstream.Centra venous catheter (CVC)

Alsoknownasacentralvenousaccessdevice(CVAD).Alinetubepassedthroughthelargeveinsotheneck,chestorgroinandintothecentralbloodcirculation.Itcanbeusedortakingsamplesoblood,givingintravenousfuids,blood,chemotherapyandotherdrugswithouttheneedorrepeatedneedles.

CheotherapySingledrugsorcombinationsodrugswhichmaybeusedtokillandpreventthegrowthanddivisionocancercells.Although


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aimedatcancercells,chemotherapycanalsoaectrapidlydividingnormalcellsandthisisresponsibleorsomecommonside-eectsincludinghairlossandasoremouth.Nauseaandvomitingarealsocommon,butnowadayslargelypreventablewithmodernanti-nauseamedication.Mostside-eectsoaretemporaryand

reversible.Chroosoes

ChromosomesaremadeupocoilsoDNA(deoxyribonucleicacid).DNAcarriesallthegeneticinormationorthebodyinsequencesknownasgenes.Thereareapproximately40,000geneson23dierentchromosomes.Thechromosomesarecontainedwithinthenucleusoacell.

Chronic euaeiasAgroupocancersthataectthebloodandbonemarrow.Chronicleukaemiasusuallydevelopgraduallyandslowlyprogress,particularlyintheearlystagesodisease.Theleukaemiaiscalledchronicbecauseittheleukaemiccellsaremorematurethanthoseoundinacute leukaemia. Chronic leukaemias are sometimesdiagnosedbychance,duringaroutinebloodtest.

Chronic yeoi euaeia (Cml)

Atypeoleukaemiawhichisaninitiallyslowgrowing(indolent)diseasewherethebonemarrowproducestoomanywhitecells.Overtime,CMLusuallytransormsintoacuteleukaemia,amoreaggressivetypeodiseasewherethebonemarrowproduceslargenumbersoabnormalimmaturegranulocytes,knownasblast cellsorleukaemic blasts.CMLisalsocalledchronic myelogenousorchronic granulocytic leukaemia(CGL).

ConeApopulationogeneticallyidentical cellsarisingromasingleparentcell.

Cotting actors

Agrouponaturallyoccurringsubstancesoundintheblood(actorsItoXIII)which,whenactivated,interacttohelpbloodclotandpreventbleeding.


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8Coaguation

Clottingotheblood.Acomplexprocessinvolvingtheinteractionaseriesobiochemicalcomponentsandbloodcellsknownasplatelets.

Coputerise axia toography (CT scan or CAT scan)

Aspecialisedx-rayorimagingtechniquethatproducesaseriesodetailedthreedimensional(3D)imagesocrosssectionsothebody.

Corticosterois (sterois)

A group o man-made hormones including prednisone,prednisolone,methylprednisolone anddexamethasone used inthetreatmentocertainbloodandbonemarrowcancers.As

wellashavinganti-cancereects,corticosteroidsalsohaveanti-infammatoryandimmunosuppressive(anti-rejection)eects.

Cure

Thismeansthatthereisnoevidenceodiseaseandnosignoitreappearing,evenatermanyyears.

Cytogenetic tests

Thestudyothegeneticmake-upothecells,inotherwords,thestructureandnumberochromosomespresent.Cytogenetictestsarecommonlycarriedoutonsamplesobloodandbonemarrowtodetectchromosomalabnormalitiesassociatedwithdisease.Thisinormationhelpsinthediagnosisandselectionothemostappropriatetreatment.

Cytoines

Seegrowthactors.

Cytopenia

Areductioninthenumberobloodcellscirculatinginthebloodstream.

disease progression

Thismeansthatthediseaseisgettingworsedespitetreatment.

Echocariogra

Aspecialultrasoundscanotheheart.

Eectrocariogra (ECG)

Electricaltraceotheheart.


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Essentia throbocytheia

A condition caused by abnormal bone marrow growth(myeloprolierativedisease).Itischaracterisedbytheproductionolargenumbersoplatelets.Symptomsincludebleeding,bloodclotsandenlargementothespleen.

Groth actorsAcomplexamilyoproteinsproducedbythebodytocontrolthegrowth,divisionandmaturationobloodcellsbythebonemarrow.Somearenowavailableasdrugsasaresultogeneticengineeringandmaybeusedtostimulatenormalbloodcellproductionollowingchemotherapyorbonemarroworperipheralbloodcelltransplantation.ForexampleG-CSF(granulocytecolonystimulatingactor).

Haeogobin

Theiron containing pigment inred bloodcells, which carriesoxygentoallthebodystissues.

Haeopoiesis

Theormationobloodcells.

Haeatoogist

Adoctorwhospecialisesinthediagnosisandtreatmentodiseasesotheblood,bonemarrowandimmunesystem.

Iiopathic yeofbrosis

Atypeomyeloprolierativedisorderinwhichbonemarrowtissueisreplacedwithabnormalbroustissueandisunabletoproduceadequatenumbersobloodcells.

Iune systeThebodysdeensesystemagainstinectionanddisease.

Iunocoproise

Whentheunctionotheimmunesystemisreduced

Iunophenotyping

Specialisedlaboratorytestsusedtodetectmarkersonthesuraceocells.Thesemarkersidentiytheoriginothecell.

Iunosuppression

Theuseodrugstoreducetheunctionotheimmunesystem.


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0leuaeia

Acancerothebloodandbonemarrowcharacterisedbythewidespread,uncontrolledproductionolargenumbersoabnormaland/orimmaturebloodcells.Thesecellstakeoverthebonemarrowotencausingaallinbloodcounts.Itheyspilloutinto

thebloodstreamhowevertheycancauseveryhighabnormalwhitecellcounts.

leuaeic basts

Abnormalimmaturebloodcellsthatmultiplyinanuncontrolledmanner,crowdingoutthebonemarrowandpreventingitromproducingnormalbloodcells.Theseabnormalcellsalsospilloutintothebloodstreamandcanaccumulateinotherorgans.

lyph noes or gansStructuresoundthroughoutthebody,orexampleintheneck,groin,armpit,chestandabdomen,whichcontainbothmatureandimmaturelymphocytes.Therearemillionsoverysmalllymphglandsinallorgansothebody.

lyphocytes

Specialisedwhitecellsthathelpdeendthebodyagainstdisease

andinection.Therearetwotypesolymphocytes:B-lymphocytesandT-lymphocytes.TheyarealsocalledB-cellsandT-cells.

lyphoi

Term usedto describe a pathway o maturation oblood cellsinthebonemarrow.Whitebloodcells(B-lymphocytesandT-lymphocytes)arederivedromthelymphoidstemcellline.

matche (Vounteer) unreate onor (mUd) transpant

Anallogeneicstemcelltransplantwherethedonorisunrelatedtothepatient,butwithasimilarlymatchedtissuetype.Alsocalledvoluntaryunrelateddonor(VUD)transplant.

maignancy

Atermappliedtotumourscharacterisedbyuncontrolledgrowthanddivisionocells(seecancer).

mucositis

Aninfammationotheliningothemouth,throatorgut.


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mutation

AchangeintheDNAcodeoacell,causedorexamplebyexposuretohazardouschemicalsorcopyingerrorsduringcelldivision.Imutationsaectnormalcellunctionthiscanleadtothedevelopmentodiseaseduetothelossonormalunctionor

thedevelopmentoabnormalunctionsothatcell.myeoi

Termusedtodescribeapathwayomaturationobloodcellsinthebonemarrow.Redcells,whitecells(neutrophils,eosinophils,basophilsandmonocytes)andplateletsarederivedromthemyeloidstemcellline.

myeoyspastic isorers

Alsoknownasmyelodysplasticsyndromes(MDS).Theseareagroupoblooddiseasesthataectnormalbloodcellproductioninthebonemarrow.InMDS,thebonemarrowproducestooewredcells,whitecellsandplatelets,andanexcessoimmaturebloodcellsknownasblastcells.

myeofbrosis

A disorder in which the bone marrow becomes replaced by

brous tissue and isunable to produce adequate numbers obloodcells.

myeoproierative isorers

Agroupodisorderscharacterisedbytheover-productionobloodcellsbythebonemarrow.Oneormoreothecellamilies-red,white,plateletsorsupporttissue,maybeinvolvedandtreatmentvariesdependingonthetypeandseverityothedisease.Includes

chronic myeloidleukaemia, polycythemia rubra vera, essentialthrombocythemiaandidiopathicmyelobrosis.

Neutropenia

Areductioninthenumberocirculatingneutrophils,animportanttypeowhitecell.Neutropeniaisassociatedwithanincreasedriskoinection.

Neutrophis

Neutrophilsarethemostcommontypeowhitecell.Theyareneeded tomountaneectiveghtagainst inection,especiallybacteriaandungi.


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Pathoogist

Adoctorwhospecialisesinthelaboratorydiagnosisodiseaseandhowdiseaseisaectingtheorgansothebody.

Petechiae

Redorpurplefatpinheadsizedspotsontheskin,especiallyonthelegs.Theyarecausedbytinybleedsundertheskin,usuallyasaresultoasevereshortageoplatelets.

PICC ine

Peripherallyinsertedcentralvenouscatheter(seecentralvenouscatheter).Itisinsertedinthemiddleotheorearm.PICCsaresometimesusedorpeoplehavingchemotherapy.

PateetsTinydisc-like ragmentsthatcirculate inthebloodandplayanimportantroleinclotormation.

Prognosis

Anestimateothelikelycourseoadisease.

Purpura

Purplespotsontheskin,otenaccompaniedbybleedingrom

thegums.Itiscausedbyashortageoplateletsaswellasragileskin.

Raiotherapy (raiation therapy)

Theuseohighenergyx-raystokillcancercellsandshrinktumours.

Reapse

Thereturnotheoriginaldisease.

Resistant or reractory isease

Thismeansthatthediseaseisnotrespondingtotreatment.

Reission

Whenthereisnoevidenceodiseasedetectableinthebody.Thisisnotthesameasacureasrelapsemaystilloccur.

Speen

Anorganthataccumulateslymphocytes,actsasareservoirorredcellsoremergencies,anddestroysbloodcellsattheendotheirliespan.Thespleenisoundhighintheabdomenonthelet-hand


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side.Itcannotnormallybeeltonexaminationunlessitisenlarged.Itisotenenlargedindiseasesothebloodorbonemarrowthisisknownashypersplenismorsplenomegaly.

Spenoegay

Anothertermusedtodescribeanenlargedspleen.

Stabe isease

Whenthediseaseisstableitisnotgettinganyworseoranybetterwithtreatment.

Stanar therapy

Themosteectiveandsaesttherapycurrentlybeingused.

Ste ces

Stemcellsareprimitivebloodcellsthatcangiverisetomorethanonecelltype.Therearemanydierenttypesostemcellsinthebody.Bonemarrow(blood)stemcellshavetheabilitytogrowandproduceallthedierentbloodcellsincludingredcells,whitecellsandplatelets.

Ste ce transpant

Generalnamegiventobone marrowandperipheral blood stemcell transplants.Thesetreatmentsareusedtosupporttheuseo

high-dosechemotherapyand/orradiotherapyinthetreatmentoawiderangeocancersincludingleukaemia,lymphoma,myelomaandotherseriousdiseases.

Throbocytopenia

Areductioninthenumberocirculatingplatelets.Thrombocytopeniaisassociatedwithanincreasedriskobleedingandbruising.

T-yphocyte

Atypeowhitecellinvolvedincontrollingimmunereactions.Utrasoun

Picturesothebodysinternalorgansbuiltupromtheinterpretationorefectedsoundwaves.

white ces

Specialisedbloodcellsotheimmunesystemthatprotectthebodyagainstinection.Therearevemaintypesowhitecells:neutrophils,

eosinophils,basophils,monocytesandlymphocytes.


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A BEqUESTYour panne git to the leuaeia Founation

Awonderulwaytomakeasignicantgitisthroughabequestinyourwill.Atermakingdueallowanceorlovedones,abequestoaspecicamountoraproportionotheresidueoyourestate,isawayoleaving

arealandlastinglegacytotheuture.YourbequesttotheLeukaemiaFoundationwillbeusedtosupportourmissiontocareorpatients,carersandamiliesandhelpusachieveourvisiontondacureorleukaemias,lymphomas,myelomaandrelatedblooddisorders.

woring your beuest to the leuaeia Founation

YoumaychoosetomakeageneralbequestandallowtheLeukaemia

Foundationtodecidehowyourbequestwillbeused,oryoumaypreertomakethatdecisionyoursele.g.directyourbequesttopatientsupportorresearch.Yourlegaladvisercanprovideurtherinormationonthedierenttypesobequests,andontheappropriatewordingorabequest.

As a guie, the ooing oring ay be useu:

Igiveandbequeathreeoallduties(herestatetheamount/percentageorshare/residueorassetstobegited)totheLeukaemiaFoundationo

(hereinserttheaddress)absolutely- orthegeneralcharitablepurposesothesaidFoundation(thisisthe

LeukaemiaFoundationspreerredoption);or

orthepurposeopatientandamilysupport;or

orthepurposeoresearchintothecause,cureortreatmento

leukaemia,lymphoma,myelomaandrelatedblooddisorders

andIdirectthatareceiptotheproperocerorthetimebeingothe

LeukaemiaFoundationshallbeagoodandsucientdischargetomytrustee/s.

Pleaseseethenextpageortheresponseorm.


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Response For

IhavealreadymadeabequesttotheLeukaemiaFoundationinmywill

Iamconsidering/itismyintentiontomake(pleasecircle)a

bequesttotheLeukaemiaFoundationIwouldlikemoreinormationaboutmakingabequestand/orwheretodirectmybequest

IwouldliketospeaktothePlannedGivingManageraboutappropriaterecognitionormybequest

Iwouldliketoreceiveinvitationstounctions

Dr/Mr/Mrs/Ms/Miss:....................................................................

Address:......................................................................................

.................................................................. Postcode...................

Telephone: (h)............................................................................

(w)..........................................................................

Email: ..........................................................................

Pease return this or to the:

PlannedGivingManager, TheLeukaemiaFoundation, GPOBox9954, inyourCapitalCity (markedPrivate&Condential)

IyouareinterestedinleavingabequesttotheLeukaemiaFoundationinyourwillandyouwouldlikeurtherinormation,withoutanyobligation,instrictestcondence,pleasecontactthePlannedGivingManagerinyourstateonFreecall1800620420.

q

q

q

q

q

"


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"


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maing a donation

TheLeukaemiaFoundationistheonlynationalnot-or-protorganisationdedicatedtothecareandcureopatientsandamilieslivingwithleukaemias,lymphomas,myelomaandrelatedblooddisorders.

Youcanhelpbymakingadonation.Pleasellouttheormbeloworvisitwww.leukaemia.org.automakeyourgitonline.

Dr/Mr/Mrs/Ms/Miss:...................................................................

Address:.....................................................................................

.................................................................. Postcode..................

Telephone: (h)............................................................................

(w)..........................................................................Email: .......................................................................... Pleaseacceptmytaxdeductibledonationor$.........................

Mycheque,madepayabletotheLeukaemiaFoundation,isenclosed,orpleasecharge$.................tomycreditcard:

qBankcardqVisaqMastercardqAmexqDiners

________/________/________/________

Cardholdersname:....................................................................

Cardholderssignature:...............................................................

Expirydate:......./.......

ContactTelephonenumber:..................................

Pleasesendto: TheLeukaemiaFoundation GPOBox9954 inyourCapitalCity.

"


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8

"

Please send me a copy of the following information booklets:

q LivingwithLeukaemias,Lymphomas,Myeloma&RelatedBloodDisorders:Inormation&Support

q UnderstandingLeukaemias,Lymphomas,MyelomaandRelatedBloodDisorders

q UnderstandingAcuteMyeloidLeukaemia

q UnderstandingAcuteLymphoblasticLeukaemiainAdults

q UnderstandingAcuteLymphoblasticLeukaemiainChildren

q UnderstandingChronicLymphocyticLeukaemia

q UnderstandingChronicMyeloidLeukaemia

q UnderstandingLymphomas(Non-HodgkinsLymphomasorB-cellandT-celllymphomas)

q UnderstandingHodgkinLymphoma

q UnderstandingAllogeneicTransplants

q UnderstandingAutologousTransplants

q UnderstandingMyelodysplasticSyndromes

q UnderstandingMyeloma

q UnderstandingMyeloprolierativeDisorders

q YoungAdultswithaBloodCancer

q EatingWell:Apracticalguideorpeoplelivingwithleukaemias,lymphomasandmyeloma

Or information about:q TheLeukaemiaFoundationsSupportServices

q Workplacegiving

q Regulardeductionscheme

q Nationalundraisingcampaigns

q Communityundraisingopportunities

q Volunteering

q ReceivingtheFoundationsnewsletters

Name:...................................................................................................StreetorPostalAddress:.........................................................................

Suburb...................................................................................................

State/Postcode.......................................................................................

Email:...................................................... Tel:(....)................................

Pleasesendto:LeukaemiaFoundation,GPOBox9954,InYourCapitalCity

orFreecall1800620420

oremail:ino@leukaemia.org.auFurtherinormationisavailableontheLeukaemiaFoundationswebsite.euaeia.org.au


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NOTES


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0NOTES


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NOTES

First produced and printed December 2008


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This inormation booklet is produced

by the Leukaemia Foundation and is one in a series on leukaemias,lymphomas, myeloma and related blood disorders.

Some booklets are also available in other languages. Copies o thisbooklet and the other booklets can be obtained rom theLeukaemia Foundation in your state by contacting us on

Freecall: 1800 620 420Email: [email protected]

Website: www.leukaemia.org.au

The Leukaemia Foundation is a non-proft organisation thatdepends on donations and support rom the community.

Documents

2008 Booklet Understanding MPD