骨髓增生异常综合征诊治-英文2011.ppt

Myelodysplasticsyndromes Apracticalapproachtodiagnosisandtreatment Hong lingHao M D DepartmentofHematology HebeiGeneralHospital Introduction MDSareaheterogeneousgroupofdisordersofbloodcellproductioninthebonemarrowthatcantransformintoacutemyeloidleukemia AML Theyarediagnosedmostoftenintheelderly Theconsequentcytopeniasresultininfectionsandbleedingcomplications Therangeofsymptomsdependsonthebonemarrowcelltypeaffected Theyhavesymptomsstemmingfromanemiaorthrombocytopenia orhaverecurrentinfections SubtypesofMDShavedifferentpathologicandclinicalpresentationsanddifferentprognoses Theyareoftencategorizedaslower riskorhigher risk dependingonthelikelihoodoftransformingtoAML Lower riskMDSsurviveamedianof3to7years Higher risktypesarepathobiologicallysimilartoAMLinolderadults andpatientseitherdevelopAMLordieofcomplicationsofMDS onaveragewithin1 5years Etiology Geneticandenvironmentalfactorsprobablybothplayarole Environmentalfactors smoking ionizingradiation exposuretobenzene hairdyes pesticides Geneticfactors Downsyndrome Fanconianemia andBloomsyndromeareassociatedwithMDS MDSrarelyruninfamilies Diagnosis Laboratorytests Completebloodcellcount CBC EvaluationofanemiaTestsfornutrientdeficienciesFecaloccultbloodtestingLiverfunctiontestsRenalfunctiontests ThyroidfunctiontestsReticulocytecountEPOlevel Screeningforrelevantinfections HIV Hepatitis ParvovirusB19 Reviewofdrugs MTX Depakote Luminal Retrovir Evaluationofothercytopenias Abdominalultrasonography forsplenomegalyCoombsantiglobulintestsLDHlevelAntinuclearantibodytiterRheumatoidfactorlevel Iftestsarenegative PeripheralbloodsmearBonemarrowaspirationandbiopsySpecializedtests 1 Antiplateletantibodies2 Proteinelectrophoresis3 Fluorescenceinsituhybridization FISHEachhasitsprognosticandtherapeuticimplications ClassificationSystemsforMDS MDShavetwomainclassificationsystems theFABandtheWHO TheWHOclassificationiswidelyacceptedbecauseitincorporatesmorphologicandcytogeneticfactorsandcorrelateswithprognosis FABclassificationforMDS WHOclassificationforMDS 接上一页 ScoringsystemsforMDS Thesesystemsarebasedon Thenumberofmyeloblastsinthebonemarrow Thehigherthecount theworsetheprognosis Thenumberordegreeofcytopenias Cytogeneticabnormalities Themostwidelyusedprognosticsystems IPSS InternationalPrognosticScoringSystemWPSS WHOClassification basedPrognosticScoringSystem Blood Vol89 No6 March15 1997 pp2079 2088 CancerControl2008 15 suppl4 4 13 TreatmentofMDS 1 SupportivecareIncludestransfusionofbloodproductstominimizecomplicationsofcytopeniasandtoimprovequalityoflife aswellasantibioticstotreatactiveinfections Redbloodcellstypicallyaregivenwhenthehemoglobinlevelfallsbelow8 5g dL Plateletsaregivenwhentheplateletcountisbelow10 109 L intheabsenceofsymptoms Ironchelation Itisdebatable Bloodproducttransfusionscanleadtoironoverload particularlywithalifetimeadministrationofmorethan20units Thisinterventionshouldbereservedforpatientswithlower riskdiseasewhoareexpectedtosurvivemorethan1year Antibiotics Theriskofinfectionissignificantlyincreasedwhentheneutrophilcountisbelow0 5 109 L Fever temperature 38 C inneutropenicpatientsisanemergency requiringimmediateinitiationofbroad spectrumantibiotics ProphylacticantibioticshavenoprovenroleinMDSpatientswithneutropenia 2 Treatmentoflower riskdisease 1 Erythropoiesis stimulatingagents Epo 100IU Landhaveminimaltransfusionneeds Additionofacolony stimulatingfactorcanbeconsideredforpatientswithneutropenia Responseismeasuredasanimprovementinhemoglobinorasindependencefromtransfusions 2 Othertreatments IfESAtreatmentisineffective Immunosuppressivetherapy ATGLenalidomideforMDSwith5q candecreasetheneedforbloodtransfusioninapproximately2 3ofthesepatients AzacitidineordecitabineStemcelltransplantationExperimentaltreatments clinicaltrials 3 Treatmentofhigher riskdisease About25 ofpatientswithnewlydiagnosedMDSand15 to20 ofpatientswithestablishedMDShavehigher riskdisease Regardlessofbloodcounts giventhehighlikelihoodoftransformationtoAMLordeathwithin1 5years Thetreatmentoptionsforhigher riskdiseaseinclude Methyltransferaseinhibitorssuchasazacitidineanddecitabine Cytotoxicchemotherapy similartotreatmentofacutemyeloidleukemia Bonemarrow hematopoieticstemcelltransplantation HSCT Experimentaltreatmentsinclinicaltrials StemcelltransplantationcuresMDS HematopoieticstemcelltransplantationistheonlycurativetreatmentforMDS However itisperformedinfewerthan5 ofpatients becausetherateoftransplant relateddeathishigh Thebalancebetweenrisksandbenefitsofthistreatmentsmustbejustifiable Take homePoints MDSarecomplexandheterogeneous sotreatmentoptionsrangefromsupportivecareto