儿童慢性髓细胞性白血病治疗策略与药物选择.pdfVIP

HowItreatchronicmyeloidleukemiainchildren

andadocents

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NobukoHijiyaandMeinolfSuttorp

12

DepartmentofPediatrics,ColumbiaUniversityMedicalCenter,NewYork,NY;andMedicalFaculty,PediatricHematologyandOncology,TechnicalUniversity

Dresden,Dresden,Germany

Evidence-basedrecommendationshavebeenestablishedfortreatmentofchronicmyeloidleukemia(CML)inadults

treatedwithtyrosinekinaseinhibitors(TKIs),buttherarityofthisleukemiainchildrenandadocentsmakesit

challengingtodevelopsimilarrecommendationsinpediatrics.Inadditiontoimatinib,whichwasapprovedforpediatric

CMLin2003,thesecond-generationTKIsdasatinibandnilotinibwererecentlyapprovedforuseinchildren,expanding

thetherapeuticoptionsandpushingallogeneicstemcelltransntationtoathird-linetreatmentofmostpediatric

cases.Yet,withoutsufficientdataonefficacyandsafetyspecifictopediatricpatients,theselectionofaTKIcontinuesto

relyonclinicalexperienceinadults.Here,wepresent4casescenarioshighlightingcommonyetchallengingissues

encounteredinthetreatmentofpediatricCML(suboptimalresponse,poortreatmentadherence,growthretardation,and

presentationinadvancedphases).Limitedexperiencewithveryyoungchildren,thetransitionofteenagerstoadult

medicine,andthegoalofachievingtreatment-freeremissionforthisrareleukemiaareadditionalsignificantobstac

thatrequirefurtherclinicalinvestiionthroughinternationalcollaboration.(Blood.2019;133(22