2021
Analysis of Duration of Response, Exposure-Adjusted Safety and Progression to Acute Myeloid Leukemia (AML) for Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS) Receiving Luspatercept in the MEDALIST Study
Platzbecker U, Santini V, Komrokji R, Zeidan A, Garcia-Manero G, Buckstein R, Rose S, Fabre S, Miteva D, Zhang J, Yucel A, Hughes C, Fenaux P. Analysis of Duration of Response, Exposure-Adjusted Safety and Progression to Acute Myeloid Leukemia (AML) for Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS) Receiving Luspatercept in the MEDALIST Study. Blood 2021, 138: 1524. DOI: 10.1182/blood-2021-145723.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesTreatment-emergent adverse eventsClinical Trials CommitteeAcute myeloid leukemiaErythropoiesis-stimulating agentsExposure-adjusted incidence ratesRBC-TITrials CommitteeEntire treatment periodDuration of treatmentRing sideroblastsAML progressionCurrent equity holderMedian durationTreatment optionsIncidence rateTreatment periodWk 1Only treatment-emergent adverse eventClass erythroid maturation agentMDS/myeloproliferative neoplasmAdvisory CommitteeRegular RBC transfusionsTolerable safety profileSpeakers bureau
2020
Efficacy and Safety of Luspatercept Treatment in Patients with Myelodysplastic Syndrome/Myeloproliferative Neoplasm with Ring Sideroblasts and Thrombocytosis (MDS/MPN-RS-T): A Retrospective Analysis from the Medalist Study
Komrokji R, Platzbecker U, Fenaux P, Garcia-Manero G, Mufti G, Santini V, Diez-Campelo M, Finelli C, Jurcic J, Greenberg P, Sekeres M, Zeidan A, DeZern A, Savona M, Shetty J, Ito R, Zhang G, Ha X, Sinsimer D, Backstrom J, Verma A. Efficacy and Safety of Luspatercept Treatment in Patients with Myelodysplastic Syndrome/Myeloproliferative Neoplasm with Ring Sideroblasts and Thrombocytosis (MDS/MPN-RS-T): A Retrospective Analysis from the Medalist Study. Blood 2020, 136: 13-15. DOI: 10.1182/blood-2020-137232.Peer-Reviewed Original ResearchMDS/MPN-RSCurrent equity holderMedian leukocyte countMedian platelet countPlacebo armRing sideroblastsSpeakers bureauRBC-TIPrimary endpointClinical benefitPlatelet countTreatment optionsLeukocyte countRetrospective analysisMyelodysplastic syndrome/myeloproliferative neoplasmClass erythroid maturation agentMean serum ferritin levelWk 1Boehringer IngelheimAdvisory CommitteeDaiichi SankyoClinical benefit responseMean Hb increaseRBC transfusion independenceRegular RBC transfusionsLonger-term RBC transfusion reduction in the phase III MEDALIST study of luspatercept in patients (pts) with lower-risk MDS with ring sideroblasts (RS).
Komrokji R, Sekeres M, Zeidan A, Fenaux P, List A, Dezern A, Greenberg P, Savona M, Jurcic J, Verma A, Mufti G, Buckstein R, Santini V, Laadem A, Ito R, Zhang J, Louis C, Linde P, Garcia-Manero G. Longer-term RBC transfusion reduction in the phase III MEDALIST study of luspatercept in patients (pts) with lower-risk MDS with ring sideroblasts (RS). Journal Of Clinical Oncology 2020, 38: 7518-7518. DOI: 10.1200/jco.2020.38.15_suppl.7518.Peer-Reviewed Original ResearchLow-risk MDSPBO armRing sideroblastsWeek 1Transfusion visitRBC transfusionSerum ferritinWeek 33Week 9Mean numberClass erythroid maturation agentRBC unitsBurden reductionPhase 3 trialLower-risk MDSErythroid maturation agentTransfusion reductionMaturation agentsTransfusion eventsClinical efficacyL vsLuspaterceptResponse episodesWeeksTransfusion
2019
Assessment of Longer-Term Efficacy and Safety in the Phase 3, Randomized, Double-Blind, Placebo-Controlled MEDALIST Trial of Luspatercept to Treat Anemia in Patients (Pts) with Revised International Prognostic Scoring System (IPSS-R) Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) with Ring Sideroblasts (RS) Who Require Red Blood Cell (RBC) Transfusions
Fenaux P, Mufti G, Buckstein R, Santini V, Díez-Campelo M, Finelli C, Cazzola M, Ilhan O, Sekeres M, Komrokji R, List A, Zeidan A, Verma A, Laadem A, Ito R, Zhang J, Rampersad A, Sinsimer D, Linde P, Garcia-Manero G, Platzbecker U. Assessment of Longer-Term Efficacy and Safety in the Phase 3, Randomized, Double-Blind, Placebo-Controlled MEDALIST Trial of Luspatercept to Treat Anemia in Patients (Pts) with Revised International Prognostic Scoring System (IPSS-R) Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) with Ring Sideroblasts (RS) Who Require Red Blood Cell (RBC) Transfusions. Blood 2019, 134: 841. DOI: 10.1182/blood-2019-123064.Peer-Reviewed Original ResearchErythropoiesis-stimulating agentsIntermediate-risk myelodysplastic syndromesRBC-TIClinical benefitMyelodysplastic syndromeData cutoffHighest dose levelTransfusion burdenCelgene CorporationRing sideroblastsSpeakers bureauMedian durationDose levelsInternational Working Group 2006 criteriaClass erythroid maturation agentGrade 1Red blood cell transfusionInternational Prognostic Scoring SystemLower-risk myelodysplastic syndromesAdvisory CommitteeLow-risk MDSRBC transfusion independenceRegular RBC transfusionsMedian treatment durationBlood cell transfusion
2018
The Medalist Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept to Treat Anemia in Patients with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) with Ring Sideroblasts (RS) Who Require Red Blood Cell (RBC) Transfusions
Fenaux P, Platzbecker U, Mufti G, Garcia-Manero G, Buckstein R, Santini V, Díez-Campelo M, Finelli C, Cazzola M, Ilhan O, Sekeres M, Falantes J, Arrizabalaga B, Salvi F, Giai V, Vyas P, Bowen D, Selleslag D, DeZern A, Jurcic J, Germing U, Götze K, Quesnel B, Beyne-Rauzy O, Cluzeau T, Voso M, Mazure D, Vellenga E, Greenberg P, Hellström-Lindberg E, Zeidan A, Laadem A, Benzohra A, Zhang J, Rampersad A, Linde P, Sherman M, Komrokji R, List A. The Medalist Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept to Treat Anemia in Patients with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) with Ring Sideroblasts (RS) Who Require Red Blood Cell (RBC) Transfusions. Blood 2018, 132: 1. DOI: 10.1182/blood-2018-99-110805.Peer-Reviewed Original ResearchIntermediate-risk myelodysplastic syndromesErythropoiesis-stimulating agentsIU/LKey secondary endpointMyelodysplastic syndromeRBC-TIRBC transfusionRing sideroblastsSpeakers bureauSecondary endpointsPrimary endpointWeek 1Class erythroid maturation agentRed blood cell transfusionInternational Prognostic Scoring SystemLower-risk myelodysplastic syndromesAdvisory CommitteeAbsence of transfusionIWG 2006 criteriaMean hemoglobin increaseRBC transfusion dependenceRBC transfusion independencePatient baseline characteristicsPlacebo-controlled studyBlood cell transfusion