2024
Immune Landscape and Outcomes of Patients with RNA Splicing Factor-Mutant Acute Myeloid Leukemia and Myelodysplastic Syndromes Treated with Azacitidine +/- the Anti-PD-L1 Antibody Durvalumab
Bewersdorf J, Hasle V, Shallis R, Thompson E, De Menezes D, Rose S, Boss I, Mendez L, Podoltsev N, Stahl M, Kewan T, Halene S, Haferlach T, Fox B, Zeidan A. Immune Landscape and Outcomes of Patients with RNA Splicing Factor-Mutant Acute Myeloid Leukemia and Myelodysplastic Syndromes Treated with Azacitidine +/- the Anti-PD-L1 Antibody Durvalumab. Blood 2024, 144: 4585. DOI: 10.1182/blood-2024-194929.Peer-Reviewed Original ResearchAcute myeloid leukemiaAnti-PD-L1 antibody durvalumabOverall response rateMyelodysplastic syndromeComplete responseBM aspiratesMyeloid leukemiaInternational Working GroupBone marrowMyelodysplastic syndromes treated with azacitidineAcute myeloid leukemia ptsWild-type acute myeloid leukemiaSecondary acute myeloid leukemiaResponse criteriaAnti-PD-L1Immune checkpoint inhibitorsTreated with azacitidineOutcomes of patientsAny-cause deathGeneration of neoantigensVariant allele frequencySusceptible to treatmentMarrow CRAdverse cytogeneticsCheckpoint inhibitors
2018
Long Term Results of a Randomized Phase 2 Dose-Response Study of Guadecitabine, a Novel Subcutaneous (SC) Hypomethylating Agent (HMA), in 102 Patients with Intermediate or High Risk Myelodysplastic Syndromes (MDS) or Chronic Myelomonocytic Leukemia (CMML)
Garcia-Manero G, Ritchie E, Walsh K, Savona M, Kantarjian H, Kropf P, O'Connell C, Tibes R, Daver N, Jabbour E, Lunin S, Rosenblat T, Yee K, Stock W, Griffiths E, Mace J, Podoltsev N, Berdeja J, Issa J, Naim S, Hao Y, Azab M, Roboz G. Long Term Results of a Randomized Phase 2 Dose-Response Study of Guadecitabine, a Novel Subcutaneous (SC) Hypomethylating Agent (HMA), in 102 Patients with Intermediate or High Risk Myelodysplastic Syndromes (MDS) or Chronic Myelomonocytic Leukemia (CMML). Blood 2018, 132: 231. DOI: 10.1182/blood-2018-99-110465.Peer-Reviewed Original ResearchMedian overall survivalOverall survivalDose groupMyelodysplastic syndromeTreatment-naïveChronic myelomonocytic leukemiaCMML patientsBristol-Myers SquibbHypomethylating agentAdverse eventsAstex PharmaceuticalsM2 groupMDS patientsMD cohortJazz PharmaceuticalsMarrow CRBM blastsHematological improvementMost patientsSpeakers bureauDaiichi SankyoHMA treatmentJanssen PharmaceuticalsTP53 mutationsCelgene Corporation
2014
First Clinical Results of a Randomized Phase 2 Dose-Response Study of SGI-110, a Novel Subcutaneous (SC) Hypomethylating Agent (HMA), in 102 Patients with Intermediate (Int) or High Risk (HR) Myelodysplastic Syndromes (MDS) or Chronic Myelomonocytic Leukemia (CMML)
Garcia-Manero G, Ritchie E, Walsh K, Savona M, Kropf P, O’Connell C, Tibes R, Daver N, Jabbour E, Lunin S, Rosenblat T, Yee K, Stock W, Griffiths E, Mace J, Podoltsev N, Berdeja J, Issa J, Chung W, Naim S, Taverna P, Hao Y, Azab M, Kantarjian H, Roboz G. First Clinical Results of a Randomized Phase 2 Dose-Response Study of SGI-110, a Novel Subcutaneous (SC) Hypomethylating Agent (HMA), in 102 Patients with Intermediate (Int) or High Risk (HR) Myelodysplastic Syndromes (MDS) or Chronic Myelomonocytic Leukemia (CMML). Blood 2014, 124: 529. DOI: 10.1182/blood.v124.21.529.529.Peer-Reviewed Original ResearchTreatment-naïve patientsMyelodysplastic syndromeChronic myelomonocytic leukemiaTreatment armsCMML patientsHypomethylating agentClinical responseAdverse eventsAstex PharmaceuticalsSGI-110Marrow CRNaïve patientsTransfusion independenceHematological improvementMDS patientsHigh-risk myelodysplastic syndromePhase 1 clinical trialECOG PS 2Treatment-naïve groupRisk myelodysplastic syndromesBaseline patient characteristicsFirst clinical resultsDose-response studySignificant differencesMedian follow