2024
Results from a Phase 1 Open-Label Dose Escalation and Expansion Trial of Oral Azacitidine + Cedazuridine (ASTX030) in Patients with Myelodysplastic Syndromes (MDS) and MDS/Myeloproliferative Neoplasms (MPN)
Garcia-Manero G, McCloskey J, Scott B, Griffiths E, Kiner-Strachan B, Brunner A, Zeidan A, Traer E, Madanat Y, Meyer J, Erba H, Baratam P, Borate U, Sano Y, Oganesian A, Zhu L, Keer H, Savona M. Results from a Phase 1 Open-Label Dose Escalation and Expansion Trial of Oral Azacitidine + Cedazuridine (ASTX030) in Patients with Myelodysplastic Syndromes (MDS) and MDS/Myeloproliferative Neoplasms (MPN). Blood 2024, 144: 662. DOI: 10.1182/blood-2024-203569.Peer-Reviewed Original ResearchDose-limiting toxicityPhase 1 trialDose-expansion partMDS/MPN overlap syndromesMyelodysplastic syndromeAdverse eventsDose combinationMDS/myeloproliferative neoplasmImmediate-releaseOverlap syndromeAUC exposureOpen-label phase 1 trialDelayed-releaseCytidine deaminaseProlonged grade 4 neutropeniaIncreased absolute bioavailabilityMarrow complete responsePhase 2 doseGrade 4 neutropeniaDose-escalation partErythropoiesis-stimulating agentsAcute myeloid leukemiaTreatment of patientsClinical efficacy assessmentDNA methyltransferase inhibitor
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