2023
Improved benefit of continuing luspatercept therapy: sub-analysis of patients with lower-risk MDS in the MEDALIST study
Germing U, Fenaux P, Platzbecker U, Buckstein R, Santini V, Díez-Campelo M, Yucel A, Tang D, Fabre S, Zhang G, Zoffoli R, Ha X, Miteva D, Hughes C, Komrokji R, Zeidan A, Garcia-Manero G. Improved benefit of continuing luspatercept therapy: sub-analysis of patients with lower-risk MDS in the MEDALIST study. Annals Of Hematology 2023, 102: 311-321. PMID: 36635381, PMCID: PMC9889415, DOI: 10.1007/s00277-022-05071-8.Peer-Reviewed Original ResearchConceptsRBC transfusion burdenTransfusion burdenWeek 25Transfusion unitsRBC-TIInitial nonrespondersHemoglobin levelsErythroid responseClinical benefitRed blood cell transfusion independenceLower-risk myelodysplastic syndromesPlacebo-treated patientsSerum ferritin levelsAdditional clinical benefitWeeks of treatmentLower-risk MDSTransfusion independenceFerritin levelsSerum ferritinMyelodysplastic syndromeRing sideroblastsPatientsClinical practiceLuspaterceptMaximum dose
2022
Longer-term benefit of luspatercept in transfusion-dependent lower-risk myelodysplastic syndromes with ring sideroblasts
Zeidan AM, Platzbecker U, Garcia-Manero G, Sekeres MA, Fenaux P, DeZern AE, Greenberg PL, Savona MR, Jurcic JG, Verma A, Mufti G, Buckstein R, Santini V, Shetty JK, Ito R, Zhang J, Zhang G, Ha X, Backstrom JT, Komrokji RS. Longer-term benefit of luspatercept in transfusion-dependent lower-risk myelodysplastic syndromes with ring sideroblasts. Blood 2022, 140: 2170-2174. PMID: 35797468, PMCID: PMC10653038, DOI: 10.1182/blood.2022016171.Peer-Reviewed Original ResearchMeSH KeywordsActivin Receptors, Type IIHumansImmunoglobulin Fc FragmentsMyelodysplastic SyndromesRecombinant Fusion ProteinsConceptsLower-risk myelodysplastic syndromesLow-risk myelodysplasiaErythropoiesis-stimulating agentsEligible patientsTransfusion independenceMyelodysplastic syndromeLong-term benefitsLuspaterceptMedian durabilityRing sideroblastsPatientsIncremental benefitPlaceboSignificant minorityTherapyTransfusionMyelodysplasiaSideroblastsSyndromeTrialsWeeksMonthsA phase I/II multicenter, open-label, dose escalation and randomized trial of BI 836858 in patients with low- or intermediate-1-risk myelodysplastic syndrome.
Komrokji RS, Carraway HE, Germing U, Wermke M, Zeidan AM, Fu E, Rüter B, Burkard U, Osswald A, Foran JM. A phase I/II multicenter, open-label, dose escalation and randomized trial of BI 836858 in patients with low- or intermediate-1-risk myelodysplastic syndrome. Haematologica 2022, 107: 2742-2747. PMID: 35734924, PMCID: PMC9614517, DOI: 10.3324/haematol.2021.280500.Peer-Reviewed Original ResearchNeutrophil 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