2023
Long-term utilization and benefit of luspatercept in transfusion-dependent, erythropoiesis-stimulating agent-refractory or -intolerant patients with lower-risk myelodysplastic syndromes with ring sideroblasts
Platzbecker U, Santini V, Komrokji R, Zeidan A, Garcia-Manero G, Buckstein R, Miteva D, Keeperman K, Holot N, Nadal J, Lai Y, Vodala S, Rosettani B, Giuseppi A, Yucel A, Fenaux P. Long-term utilization and benefit of luspatercept in transfusion-dependent, erythropoiesis-stimulating agent-refractory or -intolerant patients with lower-risk myelodysplastic syndromes with ring sideroblasts. Leukemia 2023, 37: 2314-2318. PMID: 37752285, PMCID: PMC10624606, DOI: 10.1038/s41375-023-02031-7.Peer-Reviewed Original ResearchEfficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial
Platzbecker U, Della Porta M, Santini V, Zeidan A, Komrokji R, Shortt J, Valcarcel D, Jonasova A, Dimicoli-Salazar S, Tiong I, Lin C, Li J, Zhang J, Giuseppi A, Kreitz S, Pozharskaya V, Keeperman K, Rose S, Shetty J, Hayati S, Vodala S, Prebet T, Degulys A, Paolini S, Cluzeau T, Fenaux P, Garcia-Manero G. Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial. The Lancet 2023, 402: 373-385. PMID: 37311468, DOI: 10.1016/s0140-6736(23)00874-7.Peer-Reviewed Original ResearchConceptsLower-risk myelodysplastic syndromesRed blood cell transfusion independenceEpoetin alfa groupErythropoiesis-stimulating agentsEpoetin alfaMyelodysplastic syndromeInterim analysisPrimary endpointAdverse eventsAlfa groupTransfusion independenceLower riskBody weightTreatment-emergent adverse eventsTreatment-related adverse eventsRed blood cell transfusionDurable clinical efficacyMean hemoglobin increaseMedian treatment exposureBlood cell transfusionCOVID-19 pneumoniaSubgroup of patientsWeeks of treatmentTreatment of anemiaAcute myeloid leukemia
2022
Neutrophil and platelet increases with luspatercept in lower-risk MDS: secondary endpoints from the MEDALIST trial
Garcia-Manero G, Mufti GJ, Fenaux P, Buckstein R, Santini V, Díez-Campelo M, Finelli C, Ilhan O, Sekeres MA, Zeidan AM, Ito R, Zhang J, Rampersad A, Sinsimer D, Backstrom JT, Platzbecker U, Komrokji RS. Neutrophil and platelet increases with luspatercept in lower-risk MDS: secondary endpoints from the MEDALIST trial. Blood 2022, 139: 624-629. PMID: 34758066, PMCID: PMC8796653, DOI: 10.1182/blood.2021012589.Peer-Reviewed Original Research
2020
Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes
Fenaux P, Platzbecker U, Mufti GJ, Garcia-Manero G, Buckstein R, Santini V, Díez-Campelo M, Finelli C, Cazzola M, Ilhan O, Sekeres MA, Falantes JF, Arrizabalaga B, Salvi F, Giai V, Vyas P, Bowen D, Selleslag D, DeZern AE, Jurcic JG, Germing U, Götze KS, Quesnel B, Beyne-Rauzy O, Cluzeau T, Voso MT, Mazure D, Vellenga E, Greenberg PL, Hellström-Lindberg E, Zeidan AM, Adès L, Verma A, Savona MR, Laadem A, Benzohra A, Zhang J, Rampersad A, Dunshee DR, Linde PG, Sherman ML, Komrokji RS, List AF. Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes. New England Journal Of Medicine 2020, 382: 140-151. PMID: 31914241, DOI: 10.1056/nejmoa1908892.Peer-Reviewed Original ResearchConceptsLower-risk myelodysplastic syndromesRed cell transfusionKey secondary end pointRegular red cell transfusionsSecondary end pointsMyelodysplastic syndromeTransfusion independenceAdverse eventsWeek 1Placebo groupEnd pointRing sideroblastsErythropoiesis-stimulating agent therapyIntermediate-risk myelodysplastic syndromesPrimary end pointPhase 2 studyPhase 3 trialErythropoiesis-stimulating agentsSeverity of anemiaGrowth factor βBaseline characteristicsAgent therapyLuspaterceptPatientsSyndrome
2017
US Food and Drug Administration Black Box Warning and Its Impact on the Use of Erythropoiesis-Stimulating Agents: Thinking Outside the Box
Zeidan AM, Gore SD, Davidoff AJ. US Food and Drug Administration Black Box Warning and Its Impact on the Use of Erythropoiesis-Stimulating Agents: Thinking Outside the Box. Journal Of Clinical Oncology 2017, 35: jco.2017.74.310. PMID: 28787256, DOI: 10.1200/jco.2017.74.3104.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsManagement of myelofibrosis: JAK inhibition and beyond
Stahl M, Zeidan AM. Management of myelofibrosis: JAK inhibition and beyond. Expert Review Of Hematology 2017, 10: 459-477. PMID: 28395559, DOI: 10.1080/17474086.2017.1317590.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsBone marrow fibrosisMarrow fibrosisAbnormal cytokine expressionClinical trial evidenceJAK2 V617F allele burdenErythropoiesis stimulating agentsNovel therapeutic optionsAnti-fibrosis agentsV617F allele burdenJAK2 inhibitor ruxolitinibNovel therapeutic agentsJAK2 V617F mutationConstitutional symptomsCytoreductive drugsSymptom burdenImmunomodulatory drugsSurvival improvementJAK1/2 inhibitorMF patientsTherapeutic optionsTrial evidenceCytokine expressionExtramedullary hematopoiesisPrognostic assessmentRuxolitinib monotherapy
2016
Myelodysplastic Syndromes, Version 2.2017, NCCN Clinical Practice Guidelines in Oncology.
Greenberg PL, Stone RM, Al-Kali A, Barta SK, Bejar R, Bennett JM, Carraway H, De Castro CM, Deeg HJ, DeZern AE, Fathi AT, Frankfurt O, Gaensler K, Garcia-Manero G, Griffiths EA, Head D, Horsfall R, Johnson RA, Juckett M, Klimek VM, Komrokji R, Kujawski LA, Maness LJ, O'Donnell MR, Pollyea DA, Shami PJ, Stein BL, Walker AR, Westervelt P, Zeidan A, Shead DA, Smith C. Myelodysplastic Syndromes, Version 2.2017, NCCN Clinical Practice Guidelines in Oncology. Journal Of The National Comprehensive Cancer Network 2016, 15: 60-87. PMID: 28040720, DOI: 10.6004/jnccn.2017.0007.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsMyelodysplastic syndromeNCCN Clinical Practice GuidelinesHigh-intensity therapyPresence of cytopeniasLow-intensity therapyClinical practice guidelinesVariable disease courseTreatment of anemiaBetter treatment guidanceSupportive careDisease courseTherapeutic optionsPractice guidelinesDiagnostic criteriaTreatment approachesMyeloid disordersHeterogenous groupMolecular abnormalitiesTreatment guidanceDiagnostic classificationPatientsSyndromeTherapyTreatmentGuidelines
2015
Variations in erythropoiesis-stimulating agent administration in transfusion-dependent myelodysplastic syndromes impact response
Duong VH, Baer MR, Hendrick F, Weiss SR, Sato M, Zeidan AM, Gore SD, Davidoff AJ. Variations in erythropoiesis-stimulating agent administration in transfusion-dependent myelodysplastic syndromes impact response. Leukemia Research 2015, 39: 586-591. PMID: 25869077, PMCID: PMC4441842, DOI: 10.1016/j.leukres.2015.03.013.Peer-Reviewed Original ResearchConceptsErythropoiesis-stimulating agentsUse of darbepoetinTransfusion independenceESA initiationTransfusion dependenceMyelodysplastic syndromeESA useTransfusion-dependent MDS patientsRed blood cell transfusionNumber of weeksCox proportional hazards modelErythropoiesis-stimulating agent administrationEnd Results-MedicareWeeks of therapyBlood cell transfusionWeeks of treatmentPopulation-based studyKaplan-Meier statisticsProportional hazards modelAssociation of timingHigh response rateCell transfusionEarly administrationSurveillance EpidemiologyESA administrationPatient Cost Sharing and Receipt of Erythropoiesis-Stimulating Agents Through Medicare Part D
Davidoff AJ, Hendrick FB, Zeidan AM, Baer MR, Stuart BC, Shenolikar RA, Gore SD. Patient Cost Sharing and Receipt of Erythropoiesis-Stimulating Agents Through Medicare Part D. JCO Oncology Practice 2015, 11: e190-e198. PMID: 25563701, PMCID: PMC4371123, DOI: 10.1200/jop.2014.001527.Peer-Reviewed Original ResearchConceptsErythropoiesis-stimulating agentsLow-income subsidyMyelodysplastic syndromePhysician's officePart DOOP paymentsErythropoiesis stimulating agentsPart D claimsMedicare Part APatient cost sharingMedicare Part DESA useAnemia managementD claimsInjected medicationsDiagnosis codesMultivariable modelHome injectionMedicare beneficiariesPart BPatientsPocket costsPatient accessPrescription benefitsCost sharing