2024
Oral decitabine–cedazuridine versus intravenous decitabine for myelodysplastic syndromes and chronic myelomonocytic leukaemia (ASCERTAIN): a registrational, randomised, crossover, pharmacokinetics, phase 3 study
Garcia-Manero G, McCloskey J, Griffiths E, Yee K, Zeidan A, Al-Kali A, Deeg H, Patel P, Sabloff M, Keating M, Zhu N, Gabrail N, Fazal S, Maly J, Odenike O, Kantarjian H, DeZern A, O'Connell C, Roboz G, Busque L, Buckstein R, Amin H, Randhawa J, Leber B, Shastri A, Dao K, Oganesian A, Hao Y, Keer H, Azab M, Savona M. Oral decitabine–cedazuridine versus intravenous decitabine for myelodysplastic syndromes and chronic myelomonocytic leukaemia (ASCERTAIN): a registrational, randomised, crossover, pharmacokinetics, phase 3 study. The Lancet Haematology 2024, 11: e15-e26. PMID: 38135371, DOI: 10.1016/s2352-3026(23)00338-1.Peer-Reviewed Original ResearchConceptsChronic myelomonocytic leukemiaIntravenous decitabineMyelodysplastic syndromeMyelomonocytic leukemiaOral therapyPrimary endpointAdverse eventsEastern Cooperative Oncology Group performance status 0Treatment cyclesCycle 1Full treatment dosePerformance status 0Treatment-related deathsFrequent adverse eventsSerious adverse eventsPhase 3 studyPhase 3 trialPotential treatment benefitsCommunity-based clinicsAcute myeloid leukemiaNext treatment cycleTreatment of individualsOral decitabineStatus 0Treatment discontinuation
2023
Characterization and Management of Cytopenias after Imetelstat Treatment in the IMerge Phase 3 Trial of Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS)
Zeidan A, Savona M, Madanat Y, Fenaux P, Komrokji R, Jonášová A, Illmer T, Sun L, Berry T, Feller F, Navada S, Santini V, Platzbecker U. Characterization and Management of Cytopenias after Imetelstat Treatment in the IMerge Phase 3 Trial of Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS). Blood 2023, 142: 6478. DOI: 10.1182/blood-2023-180962.Peer-Reviewed Original ResearchTreatment-emergent adverse eventsLower-risk myelodysplastic syndromesPhase 3 trialManagement of cytopeniasGrade 3Placebo groupDose adjustmentMedian timeTreatment delayDose reductionInternational Prognostic Scoring System risk groupsHematologic treatment-emergent adverse eventsRed blood cell transfusion dependencyImetelstat treatmentExperienced grade 3RBC transfusion burdenRBC transfusion independenceTreatment cycles 1Grade 4 neutropeniaGrade 4 thrombocytopeniaPrimary end pointGrowth factor supportErythropoiesis-stimulating agentsCycle 1High telomerase activity
2020
AML-207: Glasdegib in Combination with Azacitidine in Patients with Untreated Higher-Risk Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia: Effects on Marrow Recovery and Transfusion Independence
Zeidan A, Schuster M, Joris M, Krauter J, Maertens J, Gyan E, Kovacsovics T, Verma A, Vyas P, Wang E, Ma W, Zeremski M, Kudla A, Chan G, Sekeres M. AML-207: Glasdegib in Combination with Azacitidine in Patients with Untreated Higher-Risk Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia: Effects on Marrow Recovery and Transfusion Independence. Clinical Lymphoma Myeloma & Leukemia 2020, 20: s191-s192. DOI: 10.1016/s2152-2650(20)30733-3.Peer-Reviewed Original ResearchHigh-risk myelodysplastic syndromeAbsolute neutrophil countChronic myelomonocytic leukemiaAcute myeloid leukemiaMyelodysplastic syndromeEvaluable patientsIntensive chemotherapyMedian durationTransfusion independenceMarrow recoveryHematopoietic recoveryMyeloid leukemiaMyelomonocytic leukemiaEarly marrow recoveryEarly platelet recoveryUp-front treatmentManageable safety profileOpen-label studyRate of transfusionMain outcome measuresEarly hematopoietic recoveryCycle 1Cycle 2Dose delaysPartial remission