2023
Sabatolimab in Combination with Hypomethylating Agents (HMAs) Was Safe in Patients (Pts) with Intermediate-, High-, or Very-High-Risk Myelodysplastic Syndrome (MDS)
Garcia-Manero G, Lyons R, Nandal S, Ashraf M, Thellaboina R, Ruckel-Kumar J, Menssen H, Zeidan A. Sabatolimab in Combination with Hypomethylating Agents (HMAs) Was Safe in Patients (Pts) with Intermediate-, High-, or Very-High-Risk Myelodysplastic Syndrome (MDS). Blood 2023, 142: 4606. DOI: 10.1182/blood-2023-186490.Peer-Reviewed Original ResearchHigh-risk myelodysplastic syndromeAdverse eventsHematologic improvementPartial remissionMyelodysplastic syndromeHypomethylating agentMarrow CRInterim analysisStable diseaseData cutoffLast doseInternational Prognostic Scoring System criteriaResponse rateCount decreaseCycle 1 day 1Second-line treatment optionExtension phaseHematologic adverse eventsNeutrophil count decreaseOral hypomethylating agentPhase Ib studySerious adverse eventsFatal adverse eventsMonths of treatmentSingle-arm study
2021
Evaluating Complete Remission with Incomplete Hematologic Recovery (CRh) As a Response Criterion in Myelodysplastic Syndromes (MDS)
Brunner A, Gavralidis A, Ali N, Komrokji R, Zeidan A, Sallman D. Evaluating Complete Remission with Incomplete Hematologic Recovery (CRh) As a Response Criterion in Myelodysplastic Syndromes (MDS). Blood 2021, 138: 1522. DOI: 10.1182/blood-2021-151815.Peer-Reviewed Original ResearchClinical Trials CommitteeStable diseaseComplete remissionHMA therapyBone marrow blastsHematologic improvementProgressive diseaseMyelodysplastic syndromePartial remissionTrials CommitteeResponse criteriaMarrow blastsOverall survivalSpeakers bureauHigh-risk myelodysplastic syndromeAdvisory CommitteeIncomplete hematologic recoveryIWG 2006 criteriaMethod of KaplanRisk myelodysplastic syndromesOngoing prospective studyMoffitt Cancer CenterBest overall responsePrediction of OSMassachusetts General Hospital
2019
A Phase 1b Study Evaluating the Safety and Efficacy of Venetoclax As Monotherapy or in Combination with Azacitidine for the Treatment of Relapsed/Refractory Myelodysplastic Syndrome
Zeidan A, Pollyea D, Garcia J, Brunner A, Roncolato F, Borate U, Odenike O, Bajel A, Watson A, Götze K, Nolte F, Tan P, Hong W, Dunbar M, Zhou Y, Gressick L, Ainsworth W, Harb J, Salem A, Hayslip J, Swords R, Garcia-Manero G. A Phase 1b Study Evaluating the Safety and Efficacy of Venetoclax As Monotherapy or in Combination with Azacitidine for the Treatment of Relapsed/Refractory Myelodysplastic Syndrome. Blood 2019, 134: 565. DOI: 10.1182/blood-2019-124994.Peer-Reviewed Original ResearchStock/stock optionsTreatment-emergent adverse eventsProgression-free survivalMedian progression-free survivalAbbVie IncOverall survivalFebrile neutropeniaStable diseaseMarrow blastsMyelodysplastic syndromeBcl-2 inhibitorsAstra ZenecaFrequent treatment-emergent adverse eventsInternational Working Group 2006 criteriaSerious treatment-emergent adverse eventsAllogeneic stem cell transplantationAdvisory CommitteeDaiichi SankyoCycles of AZACycles of decitabineECOG performance statusPhase 1b studyPhase 2 dosePredominant grade 3Refractory myelodysplastic syndrome
2018
A Multi-center Phase I Trial of Ipilimumab in Patients with Myelodysplastic Syndromes following Hypomethylating Agent Failure
Zeidan AM, Knaus HA, Robinson TM, Towlerton AMH, Warren EH, Zeidner JF, Blackford AL, Duffield AS, Rizzieri D, Frattini MG, Levy YM, Schroeder MA, Ferguson A, Sheldon KE, DeZern AE, Gojo I, Gore SD, Streicher H, Luznik L, Smith BD. A Multi-center Phase I Trial of Ipilimumab in Patients with Myelodysplastic Syndromes following Hypomethylating Agent Failure. Clinical Cancer Research 2018, 24: 3519-3527. PMID: 29716921, PMCID: PMC6680246, DOI: 10.1158/1078-0432.ccr-17-3763.Peer-Reviewed Original ResearchConceptsImmune-related adverse eventsMarrow complete responseMyelodysplastic syndromeGrade 2T cellsHigher immune-related adverse eventsHigh-risk myelodysplastic syndromeT-cell receptor sequencingRisk myelodysplastic syndromesInvestigator-initiated trialClin Cancer ResDrug discontinuationHMA failureStable diseaseSystemic steroidsAdverse eventsMedian survivalComplete responseDismal survivalAdditional patientsClinical benefitTreatment optionsExcessive toxicityPatientsDose levels
2017
A phase I trial of ipilimumab (ipi) in patients (pts) with myelodysplastic syndromes (MDS) after hypomethylating agent (HMAs) failure.
Zeidan A, Knaus H, Robinson T, Zeidner J, Blackford A, Rizzieri D, Frattini M, Levy M, Schroeder M, Ferguson A, Sheldon K, Dezern A, Gojo I, Gore S, Streicher H, Luznik L, Duffield A, Smith B. A phase I trial of ipilimumab (ipi) in patients (pts) with myelodysplastic syndromes (MDS) after hypomethylating agent (HMAs) failure. Journal Of Clinical Oncology 2017, 35: 7010-7010. DOI: 10.1200/jco.2017.35.15_suppl.7010.Peer-Reviewed Original ResearchImmune-related adverse eventsOverall survivalMyelodysplastic syndromeHMA failureDose levelsMarrow complete responsePhase 1b studyCTLA-4 blockadeMedian overall survivalImmune checkpoint blockadeKaplan-Meier methodPhase I trialPoor overall survivalExpression of ICOST cell activationDrug discontinuationHR-MDSStable diseaseSystemic steroidsAdverse eventsCheckpoint blockadeClinical responseMaintenance doseObjective responseAllogeneic transplantation
2016
The Use of Hypomethylating Agents (HMAs) in Patients with Relapsed and Refractory Acute Myeloid Leukemia (RR-AML): Clinical Outcomes and Their Predictors in a Large International Patient Cohort
Stahl M, Podoltsev N, DeVeaux M, Perreault S, Itzykson R, Ritchie E, Sekeres M, Fathi A, Komrokji R, Bhatt V, Al-Kali A, Cluzeau T, Santini V, Brunner A, Roboz G, Fenaux P, Litzow M, Vey N, Verma V, Germing U, Fernández P, Zelterman D, Kim T, Prebet T, Gore S, Zeidan A. The Use of Hypomethylating Agents (HMAs) in Patients with Relapsed and Refractory Acute Myeloid Leukemia (RR-AML): Clinical Outcomes and Their Predictors in a Large International Patient Cohort. Blood 2016, 128: 1063. DOI: 10.1182/blood.v128.22.1063.1063.Peer-Reviewed Original ResearchMultivariable logistic regression modelRR-AMLMinority of patientsOverall survivalPrior linesHypomethylating agentHMA therapyMedian numberLogistic regression modelsChromosome 7 abnormalitiesStable diseaseBlast percentageHematologic improvementProgressive diseaseHazard ratioClinical outcomesSpeakers bureauMultivariable Cox proportional hazards modelsPeripheral blood blast percentageRefractory acute myeloid leukemiaAllogeneic stem cell transplantationInternational Working Group criteriaWhite blood cell countCox proportional hazards modelBM blast percentage
2015
Stabilization of Myelodysplastic Syndromes (MDS) Following Hypomethylating Agent (HMAs) Failure Using the Immune Checkpoint Inhibitor Ipilimumab: A Phase I Trial
Zeidan A, Zeidner J, Duffield A, Knaus H, Ferguson A, Sheldon K, DeZern A, Gojo I, Gore S, Streicher H, Luznik L, Smith B. Stabilization of Myelodysplastic Syndromes (MDS) Following Hypomethylating Agent (HMAs) Failure Using the Immune Checkpoint Inhibitor Ipilimumab: A Phase I Trial. Blood 2015, 126: 1666. DOI: 10.1182/blood.v126.23.1666.1666.Peer-Reviewed Original ResearchOverall survivalStable diseaseEnd of inductionMyelodysplastic syndromeFirst doseIpilimumab useMaintenance doseAnti-cytotoxic T-lymphocyte-associated protein 4 antibodyDose levelsInternational Working Group 2006 criteriaMedian white blood cell countImmune checkpoint inhibition therapyImmune checkpoint inhibitor ipilimumabMedian absolute neutrophil countAllogeneic stem cell transplantationWhite blood cell countT cell receptor repertoireBM blast percentageCheckpoint inhibitor ipilimumabDose-escalation partGrade 3 irAEsOngoing stable diseasePhase 1b studyProlonged disease stabilizationTolerability of ipilimumab
2014
The Prognostic Utility of the Current Risk Models in Predicting Outcomes of Patients (pts) with Higher-Risk Myelodysplastic Syndromes (HR-MDS) Treated with Hypomethylating Agents (HMA)
Zeidan A, Sekeres M, Garcia-Manero G, Barnard J, Al Ali N, Zimmerman C, Roboz G, Steensma D, DeZern A, Jabbour E, Kantarjian H, Zell K, Wang Q, Gore S, Nazha A, Maciejewski J, List A, Komrokji R. The Prognostic Utility of the Current Risk Models in Predicting Outcomes of Patients (pts) with Higher-Risk Myelodysplastic Syndromes (HR-MDS) Treated with Hypomethylating Agents (HMA). Blood 2014, 124: 1935. DOI: 10.1182/blood.v124.21.1935.1935.Peer-Reviewed Original ResearchMD Anderson Prognostic Scoring SystemHigh-risk myelodysplastic syndromeInternational Prognostic Scoring SystemMedian overall survivalHematopoietic cell transplantationPrognostic scoring systemOverall response rateOverall survivalHMA therapyHypomethylating agentComplete responseScoring systemStable diseaseHematologic improvementPartial responseProgressive diseaseConfidence intervalsInternational Working Group 2006 criteriaMDS Clinical Research ConsortiumPrognostic modelRevised International Prognostic Scoring SystemRisk categoriesBoehringer Ingelheim CorpCycles of therapyMarrow complete response
2013
Management of High-Risk Myelodysplastic Syndrome
Zeidan A, Gore S. Management of High-Risk Myelodysplastic Syndrome. Hematologic Malignancies 2013, 189-210. DOI: 10.1007/978-3-642-36229-3_12.Peer-Reviewed Original ResearchHigh-risk myelodysplastic syndromeHR-MDSDNMTi therapyDNA methyltransferase inhibitorEmergence of resistanceTherapeutic optionsMyelodysplastic syndromeNovel agentsAllogeneic hematopoietic stem cell transplantationCare first-line therapyHematopoietic stem cell transplantationDuration of therapyFirst-line therapyComplex pathogenetic mechanismsStem cell transplantationLimited therapeutic optionsGood responseMechanism of actionAzacitidine therapyStable diseaseHematologic improvementIntensive chemotherapyMaintenance therapyUntreated patientsMedian survival