2023
Long-Term Evaluation of Luspatercept in Erythropoiesis-Stimulating Agent (ESA)-Intolerant/Refractory Patients (pts) with Lower-Risk Myelodysplastic Syndromes (LR-MDS) in the Phase 3 MEDALIST Study
Santini V, Komrokji R, Garcia-Manero G, Buckstein R, Oliva E, Keeperman K, Rose S, Giuseppi A, Vilmont V, Lai Y, Miteva D, Aggarwal B, Platzbecker U, Fenaux P, Zeidan A. Long-Term Evaluation of Luspatercept in Erythropoiesis-Stimulating Agent (ESA)-Intolerant/Refractory Patients (pts) with Lower-Risk Myelodysplastic Syndromes (LR-MDS) in the Phase 3 MEDALIST Study. Blood 2023, 142: 915. DOI: 10.1182/blood-2023-178546.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesTreatment-emergent adverse eventsLong-term efficacyEntire treatment periodExposure-adjusted incidence ratesMedian cumulative durationRBC-TITreatment periodCumulative durationData cutoffTreatment-related treatment-emergent adverse eventsCommon treatment-emergent adverse eventsRed blood cell transfusion independencePrevious short-term reportsLong-term safety profileAcute myeloid leukemia progressionRegular RBC transfusionsPermanent treatment discontinuationKaplan-Meier analysisErythropoiesis stimulating agentsShort-term reportsMyeloid leukemia progressionEligible ptsHR-MDSRefractory patients
2022
Phase 1 study of anti-CD47 monoclonal antibody CC-90002 in patients with relapsed/refractory acute myeloid leukemia and high-risk myelodysplastic syndromes
Zeidan AM, DeAngelo DJ, Palmer J, Seet CS, Tallman MS, Wei X, Raymon H, Sriraman P, Kopytek S, Bewersdorf JP, Burgess MR, Hege K, Stock W. Phase 1 study of anti-CD47 monoclonal antibody CC-90002 in patients with relapsed/refractory acute myeloid leukemia and high-risk myelodysplastic syndromes. Annals Of Hematology 2022, 101: 557-569. PMID: 34981142, PMCID: PMC9414073, DOI: 10.1007/s00277-021-04734-2.Peer-Reviewed Original ResearchConceptsAnti-drug antibodiesAcute myeloid leukemiaDose-limiting toxicityRefractory acute myeloid leukemiaHigh-risk myelodysplastic syndromeMyelodysplastic syndromeMyeloid leukemiaCommon treatment-emergent adverse eventsTreatment-emergent adverse eventsADA-positive patientsPhase 2 dosePresence/frequencyUnexpected safety findingsPhase 1 studyAnti-CD47 antibodyCD47-SIRPα interactionMacrophage-mediated killingHematological cancer cell linesFebrile neutropeniaMonotherapy activityCancer cell linesPrimary endpointSecondary endpointsAdverse eventsObjective response
2020
Clinical Efficacy and Safety of Oral Decitabine/Cedazuridine in 133 Patients with Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML)
Savona M, McCloskey J, Griffiths E, Yee K, Al-Kali A, Zeidan A, Deeg H, Patel P, Sabloff M, Keating M, Dao K, Zhu N, Gabrail N, Fazal S, Maly J, Odenike O, Kantarjian H, DeZern A, O'Connell C, Roboz G, Busque L, Wells R, Amin H, Randhawa J, Leber B, Hao Y, Keer H, Azab M, Garcia-Manero G. Clinical Efficacy and Safety of Oral Decitabine/Cedazuridine in 133 Patients with Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML). Blood 2020, 136: 37-38. PMCID: PMC8330281, DOI: 10.1182/blood-2020-133855.Peer-Reviewed Original ResearchChronic myelomonocytic leukemiaRed blood cellsMyelodysplastic syndromeComplete responseHematological improvementAdverse eventsAstex PharmaceuticalsHypomethylating agentConsecutive weeksSpeakers bureauMedian durationClinical efficacyDaiichi SankyoDNA methyltransferase inhibitorCommon treatment-emergent adverse eventsAllogeneic hematopoietic cell transplantTreatment-emergent adverse eventsTreatment of MDSBoehringer IngelheimAdvisory CommitteeRandomized cross-over studySeattle GeneticsDose combination drugsOral hypomethylating agentOverall objective responsePhase 1 dose escalation trial of volasertib in combination with decitabine in patients with acute myeloid leukemia
Cortes J, Podoltsev N, Kantarjian H, Borthakur G, Zeidan AM, Stahl M, Taube T, Fagan N, Rajeswari S, Uy GL. Phase 1 dose escalation trial of volasertib in combination with decitabine in patients with acute myeloid leukemia. International Journal Of Hematology 2020, 113: 92-99. PMID: 32951163, DOI: 10.1007/s12185-020-02994-8.Peer-Reviewed Original ResearchMeSH KeywordsAgedAntineoplastic Combined Chemotherapy ProtocolsCell Cycle ProteinsDecitabineDose-Response Relationship, DrugFebrile NeutropeniaFeeding and Eating DisordersFemaleGene ExpressionHumansLeukemia, Myeloid, AcuteMaleMolecular Targeted TherapyProtein Serine-Threonine KinasesProto-Oncogene ProteinsPteridinesTreatment OutcomeConceptsAcute myeloid leukemiaMyeloid leukemiaCommon treatment-emergent adverse eventsPhase 1 dose-escalation trialTreatment-emergent adverse eventsMTD of volasertibObjective response rateAdverse event profileDose-escalation trialPhase 1 trialAnti-leukemic activityPolo-like kinase 1Febrile neutropeniaEscalation trialAdverse eventsCell cycle kinase inhibitorsAML patientsEvent profilePoor prognosisResponse ratePatientsVolasertibDecitabineKinase inhibitorsNumerous cancers