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RESEARCH PRODUCT
Outcome of lower-risk patients with myelodysplastic syndromes without 5q deletion after failure of erythropoiesis-stimulating agents
Jennifer KaiversUlrich GermingRosa SapenaGiovanni CamettiMikkael A. SekeresKatharina GötzeMaria Diez CampeloAgnès Guerci-breslerFabrizio PaneStéphane ChezeFrançois DreyfusTeresa BernalEnrico BalleariDavid P. SteensmaRami S. KomrokjiPierre FenauxChristian RoseGuillermo SanzValeria SantiniCatharina Müller-thomasMarisa CalabuigAspasia StamatoullasNorbert VeyDario FerreroAlessandro SannaJean Francois HamelJaime FensterlDominique BordessouleIoannis KotsianidisSophie ParkAndrea TomaCharikleia KelaidiGail J. RobozFernando RamosGianluca GaidanoSylvain ThepotOdile Beyne-rauzyPascale Cony-makhoulEric WattelDaniela Gioiasubject
MaleCancer Research0302 clinical medicineRecurrenceRisk Factorshemic and lymphatic diseasesHydroxyureaCumulative incidenceTreatment FailureEnzyme InhibitorsLenalidomideAged 80 and overCytarabineAnemiaMiddle AgedThalidomideMelodysplastic syndromeSurvival RateLeukemia Myeloid AcuteOncologyInternational Prognostic Scoring System030220 oncology & carcinogenesisRetreatmentAzacitidineCyclosporineDisease ProgressionChromosomes Human Pair 5FemaleChromosome DeletionErythrocyte Transfusionmedicine.drugmedicine.medical_specialtyMelodysplastic syndrome erytropoiesis stimulating agents 5q-erytropoiesis stimulating agentsDecitabineAntineoplastic AgentsTretinoinDecitabineLower risk5q-Arsenic03 medical and health sciencesInternal medicinemedicineHumansImmunologic FactorsSurvival rateAgedAntilymphocyte SerumRetrospective StudiesLenalidomidebusiness.industryValproic AcidMyelodysplastic syndromesRetrospective cohort studymedicine.diseaseMyelodysplastic SyndromesHematinicsPhysical therapybusiness030215 immunologydescription
Purpose Most anemic patients with non-deleted 5q lower-risk myelodysplastic syndromes (MDS) are treated with erythropoiesis-stimulating agents (ESAs), with a response rate of approximately 50%. Second-line treatments, including hypomethylating agents (HMAs), lenalidomide (LEN), and investigational drugs, may be used after ESA failure in some countries, but their effect on disease progression and overall survival (OS) is unknown. Here, we analyzed outcome after ESA failure and the effect of second-line treatments. Patients and Methods We examined an international retrospective cohort of 1,698 patients with non-del(5q) lower-risk MDS treated with ESAs. Results Erythroid response to ESAs was 61.5%, and median response duration was 17 months. Of 1,147 patients experiencing ESA failure, 653 experienced primary failure and 494 experienced relapse after a response. Primary failure of ESAs was associated with a higher risk of acute myeloid leukemia (AML) progression, which did not translate into an OS difference. Of 450 patients (39%) who received second-line treatment, 194 received HMAs, 148 received LEN, and 108 received other treatments (MISC), whereas 697 received RBC transfusions only. Five-year AML cumulative incidence was 20.3%, 20.3%, and 11.3% for those receiving HMAs, LEN, and MISC, respectively ( P = .05). Five-year OS for patients receiving HMA, LEN, and MISC was 36.5%, 41.7%, and 51%, respectively ( P = .21). In a multivariable analysis adjusted for age, sex, revised International Prognostic Scoring System score, and progression at ESA failure, there was no significant OS difference among the three groups. Conclusion In this large, multicenter, retrospective cohort of patients with non-del(5q) lower-risk MDS treated with ESAs, none of the most commonly used second-line treatments (HMA and LEN) significantly improved OS. Early failure of ESAs was associated with a higher risk of AML progression.
| year | journal | country | edition | language |
|---|---|---|---|---|
| 2017-01-01 |