2023
Clinical Outcomes in Patients With Refractory Anemia With Excess Blasts (RAEB) Who Receive Hypomethylating Agents (HMAs)
Zeidan A, Mearns E, Ng C, Shah A, Lamarre N, Yellow-Duke A, Alrawashdh N, Yang B, Cheng W, Bui C, Svensson A. Clinical Outcomes in Patients With Refractory Anemia With Excess Blasts (RAEB) Who Receive Hypomethylating Agents (HMAs). Clinical Lymphoma Myeloma & Leukemia 2023, 24: 177-186. PMID: 37996264, DOI: 10.1016/j.clml.2023.10.010.Peer-Reviewed Original ResearchEvent-free survivalAcute myeloid leukemiaMedian overall survivalOverall survivalHypomethylating agentExcess blastsRefractory anemiaReal-world settingMedian event-free survivalFirst-line therapyHematopoietic cell transplantationEligible patientsClinical outcomesCancer RegistryCell transplantationClinical benefitMedicare databaseClinical effectivenessAML progressionClinical trialsPatient outcomesMyeloid leukemiaPatientsOverall populationSignificant differences
2022
MDS-476 Sabatolimab (MBG453) Combination Treatment Regimens for Patients With Higher-Risk Myelodysplastic Syndromes (HR-MDS): The Myelodysplastic Syndromes Studies in the STIMULUS Immuno-Myeloid Clinical Trial Program
Zeidan A, Al-Kali A, Borate U, Cluzeau T, DeZern A, Esteve J, Giagounidis A, Kobata K, Lyons R, Platzbecker U, Sallman D, Santini V, Sanz G, Sekeres M, Wei A, Xiao Z, Van Hoef M, Nourry-Boulot C, Sadek I, Ma F, Iordan A, Sabo J, Garcia-Manero G. MDS-476 Sabatolimab (MBG453) Combination Treatment Regimens for Patients With Higher-Risk Myelodysplastic Syndromes (HR-MDS): The Myelodysplastic Syndromes Studies in the STIMULUS Immuno-Myeloid Clinical Trial Program. Clinical Lymphoma Myeloma & Leukemia 2022, 22: s317. DOI: 10.1016/s2152-2650(22)01420-3.Peer-Reviewed Original ResearchHematopoietic stem cell transplantLeukemia-free survivalPhase II trialHigh-risk myelodysplastic syndromeHR-MDS patientsClinical trial programII trialLeukemic stem cellsCombination therapyTim-3Trial programLeukemic cellsTransfusion-free intervalEvent-free survivalPhase III trialsStem cell transplantOverall response rateEarly phase trialsNovartis Pharmaceuticals CorporationImprovement of fatigueExpansion cohortIntensive chemotherapyPrimary endpointDurable responsesIII trialsHealth-related quality of life (HRQoL) with enasidenib versus conventional care regimens in older patients with late-stage mutant-IDH2 relapsed or refractory acute myeloid leukemia (R/R AML).
Dinardo C, Montesinos P, Schuh A, Papayannidis C, Vyas P, Wei A, Zeidan A, Chen C, Lord-Bessen J, Yu P, Shi L, Guo S, Bluemmert I, Yu X, Hasan M, Regueira P, De Botton S. Health-related quality of life (HRQoL) with enasidenib versus conventional care regimens in older patients with late-stage mutant-IDH2 relapsed or refractory acute myeloid leukemia (R/R AML). Journal Of Clinical Oncology 2022, 40: 7032-7032. DOI: 10.1200/jco.2022.40.16_suppl.7032.Peer-Reviewed Original ResearchConventional care regimensEvent-free survivalEQ-5DR AMLCare regimensGlobal health status/QoLRefractory acute myeloid leukemiaVisual analog scale scoreWorse event-free survivalEORTC QLQ-C30 questionnaireSecondary trial endpointsComplete remission rateAnalog scale scoreHealth-related qualityMean HRQoL scoresQLQ-C30 questionnaireClinical efficacy measuresUtility indexMean EQ-5DAcute myeloid leukemiaQLQ-C30 domainsYears of ageLow response rateHRQOL endpointsOlder pts
2021
Outcomes for Patients with Late-Stage Mutant-IDH2 (m IDH2) Relapsed/Refractory Acute Myeloid Leukemia (R/R AML) Treated with Enasidenib Vs Other Lower-Intensity Therapies in the Randomized, Phase 3 IDHentify Trial
DiNardo C, Montesinos P, Schuh A, Papayannidis C, Vyas P, Wei A, Zeidan A, Bluemmert I, Yu X, Hasan M, Martin-Regueira P, de Botton S. Outcomes for Patients with Late-Stage Mutant-IDH2 (m IDH2) Relapsed/Refractory Acute Myeloid Leukemia (R/R AML) Treated with Enasidenib Vs Other Lower-Intensity Therapies in the Randomized, Phase 3 IDHentify Trial. Blood 2021, 138: 1243. DOI: 10.1182/blood-2021-147593.Peer-Reviewed Original ResearchClinical Trials CommitteeBest supportive careEvent-free survivalBristol-Myers SquibbIntermediate-dose cytarabineMedian overall survivalR AMLOverall survivalHematologic improvementTransfusion independenceTrials CommitteeCurrent equity holderSpeakers bureauDaiichi SankyoRefractory acute myeloid leukemiaAdvisory CommitteeAdverse-risk AMLConventional care regimensLow-dose cytarabineMedian age overallPrimary refractory AMLStudy drug doseOpen-label trialLow-intensity therapyOverall response rate
2020
Blast MRD AML-1 Trial: Blockade of PD-1 Added to Standard Therapy to Target Measurable Residual Disease in Acute Myeloid Leukemia (AML) 1- an Investigator-Initiated, CTEP-Sponsored, Randomized Phase 2 Study of the Anti-PD-1 Antibody Pembrolizumab in Combination with Conventional Intensive Chemotherapy (IC) As Frontline Therapy in Patients with Acute Myeloid Leukemia (AML)
Zeidan A, Boddu P, Wood B, Zelterman D, Little R, Ivy S, Caldwell A, Sanchez-Espiridion B, Alatrash G, Sharon E, Radich J. Blast MRD AML-1 Trial: Blockade of PD-1 Added to Standard Therapy to Target Measurable Residual Disease in Acute Myeloid Leukemia (AML) 1- an Investigator-Initiated, CTEP-Sponsored, Randomized Phase 2 Study of the Anti-PD-1 Antibody Pembrolizumab in Combination with Conventional Intensive Chemotherapy (IC) As Frontline Therapy in Patients with Acute Myeloid Leukemia (AML). Blood 2020, 136: 15. DOI: 10.1182/blood-2020-139668.Peer-Reviewed Original ResearchMRD-negative complete responsesAcute myeloid leukemiaMinimal residual diseaseEvent-free survivalImmune checkpoint inhibitionPhase 2 studyIntensive chemotherapyDuration of responseComplete responseOverall survivalPD-1AML patientsDay 1Free survivalHematologic improvementInitial treatmentStudy armsResidual diseaseDay IVLeukemia-specific T-cell responsesSingle-arm phase 2 studyPD-1/PD-L1 pathwayTherapy-related acute myeloid leukemiaCancer Therapy Evaluation ProgramImmune cell subsets analysisBlast MRD AML-2: Blockade of PD-1 Added to Standard Therapy to Target Measurable Residual Disease (MDR) in Acute Myeloid Leukemia (AML) 2- a Randomized Phase 2 Study of the Venetoclax, Azacitidine, and Pembrolizumab Versus Venetoclax and Azacitidine As First Line Therapy in Older Patients with AML Who Are Ineligible or Who Refuse Intensive Chemotherapy
Zeidan A, Boddu P, Wood B, Zelterman D, Little R, Ivy S, Caldwell A, Sanchez-Espiridion B, Alatrash G, Sharon E, Radich J. Blast MRD AML-2: Blockade of PD-1 Added to Standard Therapy to Target Measurable Residual Disease (MDR) in Acute Myeloid Leukemia (AML) 2- a Randomized Phase 2 Study of the Venetoclax, Azacitidine, and Pembrolizumab Versus Venetoclax and Azacitidine As First Line Therapy in Older Patients with AML Who Are Ineligible or Who Refuse Intensive Chemotherapy. Blood 2020, 136: 11-12. DOI: 10.1182/blood-2020-139752.Peer-Reviewed Original ResearchEvent-free survivalDuration of responseMRD-negative CRIntensive chemotherapyOverall survivalDay 1Complete remissionFree survivalHematologic improvementOlder patientsSecondary AMLPD-1Study armsAML patientsInitial treatmentHematologic disordersLeukemia-specific T-cell responsesCancer Therapy Evaluation ProgramCR/complete remissionImmune cell subsets analysisRandomized phase 2 studyRandomized phase 2 trialRandomized phase II studyAllogeneic stem cell transplantBetter long-term clinical outcomesThe STIMULUS Program: Clinical Trials Evaluating Sabatolimab (MBG453) Combination Therapy in Patients (Pts) with Higher-Risk Myelodysplastic Syndromes (HR-MDS) or Acute Myeloid Leukemia (AML)
Zeidan A, Esteve J, Giagounidis A, Kim H, Miyazaki Y, Platzbecker U, Schuh A, Sekeres M, Westermann J, Xiao Z, Malek K, Scott J, Niolat J, Peyrard S, Ma F, Kiertsman F, Stegert M, Hertle S, Fenaux P, Santini V. The STIMULUS Program: Clinical Trials Evaluating Sabatolimab (MBG453) Combination Therapy in Patients (Pts) with Higher-Risk Myelodysplastic Syndromes (HR-MDS) or Acute Myeloid Leukemia (AML). Blood 2020, 136: 45-46. DOI: 10.1182/blood-2020-134718.Peer-Reviewed Original ResearchHigh-risk myelodysplastic syndromeHematopoietic stem cell transplantationAcute myeloid leukemiaEvent-free survivalLeukemic stem cellsIntensive chemotherapyOverall survivalPrimary endpointSecondary endpointsCurrent equity holderEligible ptsTim-3Transfusion independenceCombination therapyEastern Cooperative Oncology Group performance statusCR/CRi rateEncouraging overall response rateAdvisory CommitteeDaiichi SankyoDuration of CRTransfusion-free intervalLeukemia-free survivalComplete remission rateHigh-risk MDSProgression-free survivalAML-187: The STIMULUS Clinical Trial Program: Evaluating Combination Therapy with MBG453 in Patients with Higher-Risk Myelodysplastic Syndrome (HR-MDS) or Acute Myeloid Leukemia
Zeidan A, Esteve J, Kim H, Miyazaki Y, Platzbecker U, Schuh A, Westermann J, Malek K, Scott J, Niolat J, Peyrard S, Kiertsman F, Stegert M, Fenaux P. AML-187: The STIMULUS Clinical Trial Program: Evaluating Combination Therapy with MBG453 in Patients with Higher-Risk Myelodysplastic Syndrome (HR-MDS) or Acute Myeloid Leukemia. Clinical Lymphoma Myeloma & Leukemia 2020, 20: s188. DOI: 10.1016/s2152-2650(20)30727-8.Peer-Reviewed Original ResearchHigh-risk myelodysplastic syndromeEvent-free survivalClinical trial programLeukemia-free survivalProgression-free survivalOverall survivalTrial programEligible patientsSecondary endpointsTransfusion independenceCombination therapyAnti-Tim-3 monoclonal antibodyCR/CRi rateMultiple immune cell typesHigher risk MDSMRD negative ratePhase 1b studyOpen-label studyComplete remission rateDouble-blind studyRelapse-free survivalGood safety profileAcute myeloid leukemiaImmune cell typesLeukemic stem cellsEffect of enasidenib (ENA) plus azacitidine (AZA) on complete remission and overall response versus AZA monotherapy in mutant -IDH2 ( mIDH2 ) newly diagnosed acute myeloid leukemia (ND-AML).
Dinardo C, Schuh A, Stein E, Montesinos P, Wei A, De Botton S, Zeidan A, Fathi A, Quek L, Kantarjian H, Frattini M, Lersch F, Gong J, Franovic A, Vyas P, Dohner H. Effect of enasidenib (ENA) plus azacitidine (AZA) on complete remission and overall response versus AZA monotherapy in mutant -IDH2 ( mIDH2 ) newly diagnosed acute myeloid leukemia (ND-AML). Journal Of Clinical Oncology 2020, 38: 7501-7501. DOI: 10.1200/jco.2020.38.15_suppl.7501.Peer-Reviewed Original ResearchOverall response rateDuration of responseGrade 3Randomized phase I/II studyPhase I/II studyResponse rateIDH differentiation syndromePoor-risk cytogeneticsComplete remission rateIntermediate-risk cytogeneticsEvent-free survivalPhase II portionImproved response ratesAcute myeloid leukemiaMost common reasonsMutant IDH2AZA monotherapyDifferentiation syndromeECOG PSFebrile neutropeniaIntensive chemotherapyMedian EFSMedian OSPt ageComplete remission
2019
Cost-Effectiveness Analysis of Gilteritinib Versus Salvage Chemotherapy (SC) for the Treatment of Relapsed or Refractory (R/R) FLT3-Mutated (FLT3 mut+) Acute Myeloid Leukemia (AML)
Zeidan A, Qi C, Pandya B, Garnham A, Yang H, Shah M. Cost-Effectiveness Analysis of Gilteritinib Versus Salvage Chemotherapy (SC) for the Treatment of Relapsed or Refractory (R/R) FLT3-Mutated (FLT3 mut+) Acute Myeloid Leukemia (AML). Blood 2019, 134: 3859. DOI: 10.1182/blood-2019-123819.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationEvent-free survivalAcute myeloid leukemiaIncremental cost-effectiveness ratioUS third-party payer perspectiveThird-party payer perspectiveProbabilistic sensitivity analysesSalvage chemotherapyADMIRAL trialOverall survivalEfficacy inputsDeterministic sensitivity analysesIncremental costPayer perspectiveLifetime horizonAllogeneic hematopoietic stem cell transplantationFavorable cost-effectiveness profileHealth statesPost-progression treatmentTerminal care costsTreatment of RelapsedActive treatment optionsEffective therapeutic optionLong-term survivorsTreatment costs
2018
Effective Immunomodulation with Pomalidomide Beginning at Early Lymphocyte Recovery during Induction Timed Sequential Therapy (TST) for Acute Myeloid Leukemia (AML) and High-Risk Myelodysplasia (HR-MDS)
Zeidner J, Knaus H, Zeidan A, Blackford A, Montiel-Esparza R, Prince G, Gondek L, Ghiaur G, Showel M, DeZern A, Pratz K, Smith B, Levis M, Foster M, Coombs C, Streicher H, Karp J, Luznik L, Gojo I. Effective Immunomodulation with Pomalidomide Beginning at Early Lymphocyte Recovery during Induction Timed Sequential Therapy (TST) for Acute Myeloid Leukemia (AML) and High-Risk Myelodysplasia (HR-MDS). Blood 2018, 132: 335. DOI: 10.1182/blood-2018-99-114961.Peer-Reviewed Original ResearchEarly lymphocyte recoveryTimed sequential therapyAcute myeloid leukemiaMinimal residual diseaseT cellsLymphocyte recoveryCR rateMedian timeDay 1Phase 1 dose-escalation studyALT/AST elevationsAcute respiratory distress syndromeSecondary acute myeloid leukemiaAdverse-risk cytogeneticsHR-MDS patientsNon-hematologic gradePoor-risk subsetStandard induction therapyAcute kidney injuryMedian overall survivalPeripheral blood CD4Disease-free survivalDose-escalation studyEvent-free survivalGrade 3 rashThe effect of iron chelation therapy on overall survival in sickle cell disease and β‐thalassemia: A systematic review
Ballas SK, Zeidan AM, Duong VH, DeVeaux M, Heeney MM. The effect of iron chelation therapy on overall survival in sickle cell disease and β‐thalassemia: A systematic review. American Journal Of Hematology 2018, 93: 943-952. PMID: 29635754, DOI: 10.1002/ajh.25103.Peer-Reviewed Original ResearchConceptsIron chelation therapySickle cell diseaseEvent-free survivalChelation therapyOverall survivalCell diseaseΒ-thalassemiaSystematic reviewRed blood cell transfusionBlood cell transfusionBetter overall survivalSerum ferritin levelsLife-threatening anemiaTransfusion-dependent patientsStandard of careIron chelation agentsLiver iron contentNew oral drugsCell transfusionFrequent transfusionsOral agentsDiabetes mellitusHeart failureLiver cirrhosisFerritin levels
2014
Real-Life Experience of a Brief Arsenic Trioxide-Based Consolidation Chemotherapy in the Management of Acute Promyelocytic Leukemia: Favorable Outcomes With Limited Anthracycline Exposure and Shorter Consolidation Therapy
Leech M, Morris L, Stewart M, Smith BD, Bashey A, Holland K, Solomon S, Zhang X, Carraway HE, Pratz K, Gore SD, Zeidan AM. Real-Life Experience of a Brief Arsenic Trioxide-Based Consolidation Chemotherapy in the Management of Acute Promyelocytic Leukemia: Favorable Outcomes With Limited Anthracycline Exposure and Shorter Consolidation Therapy. Clinical Lymphoma Myeloma & Leukemia 2014, 15: 292-297. PMID: 25499624, PMCID: PMC4409502, DOI: 10.1016/j.clml.2014.11.001.Peer-Reviewed Original ResearchConceptsAcute promyelocytic leukemiaHigh-risk acute promyelocytic leukemiaConsolidation chemotherapyConsolidation therapyPromyelocytic leukemiaEffective consolidation therapyEvent-free survivalLeukemia-free survivalTrans retinoic acidArsenic trioxide combinationsChemotherapy regimenAnthracycline exposureOverall survivalSecondary malignanciesCardiac toxicityFavorable outcomeRetrospective analysisArsenic trioxideTherapyRetinoic acidChemotherapyPatientsSurvivalLeukemiaMonths