2023
Validation of the Molecular International Prognostic Scoring System (IPSS-M) in Patients (Pts) with Myelodysplastic Syndromes/Neoplasms (MDS) Who Were Treated with Hypomethylating Agents (HMA)
Kewan T, Bewersdorf J, Blaha O, Stahl M, Al Ali N, DeZern A, Sekeres M, Carraway H, Desai P, Griffiths E, Stein E, Brunner A, Amaya M, Zeidner J, Savona M, Stempel J, Chandhok N, Logothetis C, Ramaswamy R, Rose A, Roboz G, Rolles B, Wang E, Harris A, Shallis R, Xie Z, Padron E, Maciejewski J, Sallman D, Della Porta M, Komrokji R, Zeidan A. Validation of the Molecular International Prognostic Scoring System (IPSS-M) in Patients (Pts) with Myelodysplastic Syndromes/Neoplasms (MDS) Who Were Treated with Hypomethylating Agents (HMA). Blood 2023, 142: 3240. DOI: 10.1182/blood-2023-186340.Peer-Reviewed Original ResearchComplete remission rateOverall response rateOutcome of ptsMedian overall survivalOverall survivalHypomethylating agentHMA initiationHR-MDSC-indexRisk groupsScoring systemInternational Prognostic Scoring SystemResponse criteriaPrognostic scoring systemHigh-risk diseaseLarge multicenter cohortHigh-risk groupHarrell's C-indexLog-rank testPrediction of outcomeDifferent scoring systemsSubsequent validation studiesHMA cyclesMedian followAllogeneic HSCTValidation of the Composite Complete Response (cCR) Definitions in the International Working Group (IWG) 2023 Criteria in Patients (Pts) with Higher-Risk Myelodysplastic Syndromes/Neoplasms (HR-MDS) Treated with Hypomethylating Agents (HMA) - a Large, Multicenter, Retrospective Analysis from the Validate Database
Bewersdorf J, Kewan T, Blaha O, Stahl M, Al Ali N, DeZern A, Sekeres M, Uy G, Carraway H, Desai P, Griffiths E, Stein E, Brunner A, McMahon C, Zeidner J, Savona M, Stempel J, Chandhok N, Logothetis C, Roboz G, Rolles B, Wang E, Harris A, Amaya M, Hawkins H, Grenet J, Shallis R, Xie Z, Maciejewski J, Sallman D, Della Porta M, Komrokji R, Zeidan A. Validation of the Composite Complete Response (cCR) Definitions in the International Working Group (IWG) 2023 Criteria in Patients (Pts) with Higher-Risk Myelodysplastic Syndromes/Neoplasms (HR-MDS) Treated with Hypomethylating Agents (HMA) - a Large, Multicenter, Retrospective Analysis from the Validate Database. Blood 2023, 142: 324. DOI: 10.1182/blood-2023-180299.Peer-Reviewed Original ResearchImproved OSHypomethylating agentHMA initiationMedian OSResponse assessmentTP53 mutationsResponse definitionsPartial hematologic recoveryPredictors of OSMultivariable Cox modelBone marrow blastsKaplan-Meier analysisLog-rank testOverall response rateEfficacy of therapyMultivariable regression modelsReal-world analysisAllo-HCTBM assessmentBM evaluationHemoglobin thresholdHematologic recoveryMarrow blastsMedian durationMedian ageClinical Implications of TP53 Mutations/Allelic State in Patients (Pts) with Myelodysplastic Syndromes/Neoplasms (MDS) Treated with Hypomethylating Agents (HMA)- a Multicenter, Retrospective Analysis from the Validate Database
Kewan T, Bewersdorf J, Blaha O, Stahl M, Al Ali N, DeZern A, Sekeres M, Carraway H, Desai P, Griffiths E, Stein E, Brunner A, Amaya M, Zeidner J, Savona M, Stempel J, Chandhok N, Cochran H, Ramaswamy R, Singh A, Roboz G, Rolles B, Wang E, Harris A, Shallis R, Xie Z, Padron E, Maciejewski J, Della Porta M, Komrokji R, Sallman D, Zeidan A. Clinical Implications of TP53 Mutations/Allelic State in Patients (Pts) with Myelodysplastic Syndromes/Neoplasms (MDS) Treated with Hypomethylating Agents (HMA)- a Multicenter, Retrospective Analysis from the Validate Database. Blood 2023, 142: 1002. DOI: 10.1182/blood-2023-186875.Peer-Reviewed Original ResearchOverall response rateMedian overall survivalOverall survivalComplete responseHMA initiationHMA therapyMultivariable Cox proportional hazards regression modelsCox proportional hazards regression modelHigh-risk disease featuresComplex karyotypeProportional hazards regression modelsWorse overall survivalLog-rank testHazards regression modelsSignificant differencesLogistic regression modelsAllogenic HSCTBM biopsyHMA cyclesMDS-EBTherapy initiationMedian ageRegression modelsCR ratePoor outcome
2022
Venetoclax-based salvage therapy in patients with relapsed/refractory acute myeloid leukemia previously treated with FLT3 or IDH1/2 inhibitors
Bewersdorf JP, Shallis RM, Derkach A, Goldberg AD, Stein A, Stein EM, Marcucci G, Zeidan AM, Shimony S, DeAngelo DJ, Stone RM, Aldoss I, Ball BJ, Stahl M. Venetoclax-based salvage therapy in patients with relapsed/refractory acute myeloid leukemia previously treated with FLT3 or IDH1/2 inhibitors. Leukemia & Lymphoma 2022, 64: 188-196. PMID: 36287540, PMCID: PMC9905301, DOI: 10.1080/10428194.2022.2136952.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaSalvage therapyIDH1/2 inhibitorsIDH2 inhibitorsMyeloid leukemiaResponse rateRefractory acute myeloid leukemiaEffective salvage therapyLow-dose cytarabineMedian overall survivalRetrospective cohort studyOverall response rateLow response rateCohort studyOverall survivalAML patientsTreatment optionsFLT3 inhibitorsITD mutationPatientsTherapyFLT3Little dataVenetoclaxInhibitorsAML-055 Outcomes Based on Sequential Use of Venetoclax Based Therapy and Targeted Therapy in Acute Myeloid Leukemia (AML)
Stahl M, Shallis R, Derkach A, Goldberg A, Stein A, Stein E, Marcucci G, Zeidan A, Shimony S, DeAngelo D, Stone R, Aldoss I, Ball B, Bewersdorf J. AML-055 Outcomes Based on Sequential Use of Venetoclax Based Therapy and Targeted Therapy in Acute Myeloid Leukemia (AML). Clinical Lymphoma Myeloma & Leukemia 2022, 22: s209-s210. DOI: 10.1016/s2152-2650(22)01214-9.Peer-Reviewed Original ResearchOverall response rateAcute myeloid leukemiaMorphologic leukemia-free stateOverall survivalComplete responseIDH2 inhibitorsMedian OSPartial remissionTargeted therapyEffective salvage therapyIncomplete count recoveryMedian overall survivalShorter median OSRetrospective cohort studyShorter overall survivalAcademic cancer centerFLT3-ITD mutationAlternative therapeutic strategiesSalvage therapyCohort studyCount recoveryInhibitor therapyTargeted agentsClinical efficacyCancer CenterMDS-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 trialsEfficacy of FLT3 and IDH1/2 inhibitors in patients with acute myeloid leukemia previously treated with venetoclax
Bewersdorf JP, Shallis RM, Derkach A, Goldberg AD, Stein A, Stein EM, Marcucci G, Zeidan AM, Shimony S, DeAngelo DJ, Stone RM, Aldoss I, Ball BJ, Stahl M. Efficacy of FLT3 and IDH1/2 inhibitors in patients with acute myeloid leukemia previously treated with venetoclax. Leukemia Research 2022, 122: 106942. PMID: 36108424, DOI: 10.1016/j.leukres.2022.106942.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaIDH2 inhibitorsMyeloid leukemiaResponse rateRetrospective cohort studyOverall response rateRAS pathway mutationsNovel therapeutic strategiesMedian OSR AMLCohort studyShorter OSLandmark trialsTargeted agentsFrontline treatmentMutant FLT3Combination therapyTreatment optionsIDH1/2 inhibitorsDisease progressionTherapeutic strategiesPatientsSmall molecule inhibitorsVenetoclaxTherapyA phase 1b study of glasdegib + azacitidine in patients with untreated acute myeloid leukemia and higher-risk myelodysplastic syndromes
Sekeres MA, Schuster M, Joris M, Krauter J, Maertens J, Breems D, Gyan E, Kovacsovics T, Verma A, Vyas P, Wang ES, Ching K, O’Brien T, Gallo Stampino C, Ma WW, Kudla A, Chan G, Zeidan AM. A phase 1b study of glasdegib + azacitidine in patients with untreated acute myeloid leukemia and higher-risk myelodysplastic syndromes. Annals Of Hematology 2022, 101: 1689-1701. PMID: 35488900, DOI: 10.1007/s00277-022-04853-4.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaHigh-risk myelodysplastic syndromePhase 1b studyChronic myelomonocytic leukemiaMyelodysplastic syndromeExpansion cohortSafety profileMDS cohortMyeloid leukemiaTreatment-emergent adverse eventsUntreated acute myeloid leukemiaNon-hematologic gradeMedian overall survivalAcceptable safety profileOverall response rateDrug-drug interactionsSafety cohortIntensive chemotherapyAdverse eventsOverall survivalClinical benefitQT prolongationAML cohortMyelomonocytic leukemiaGlasdegibA randomized phase 2 trial of azacitidine with or without durvalumab as first-line therapy for older patients with AML
Zeidan AM, Boss I, Beach C, Copeland WB, Thompson E, Fox BA, Hasle VE, Hellmann A, Taussig D, Tormo M, Voso MT, Cavenagh J, O'Connor T, Previtali A, Rose S, Silverman LR. A randomized phase 2 trial of azacitidine with or without durvalumab as first-line therapy for older patients with AML. Blood Advances 2022, 6: 2219-2229. PMID: 34933333, PMCID: PMC9006260, DOI: 10.1182/bloodadvances.2021006138.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaFirst-line therapyOlder patientsDay 1First-line combination therapyTreatment-emergent adverse eventsRandomized phase 2 trialSafety of durvalumabNew safety signalsPD-L1 expressionPhase 2 studyPhase 2 trialDuration of responseOverall response rateDurvalumab 1500Adverse eventsOverall survivalClinical efficacyCombination therapyMyeloid leukemiaSafety signalsTreatment responseCombination treatmentAzacitidineResponse rateA randomized phase 2 trial of azacitidine with or without durvalumab as first-line therapy for higher-risk myelodysplastic syndromes
Zeidan AM, Boss I, Beach C, Copeland WB, Thompson E, Fox BA, Hasle VE, Ogasawara K, Cavenagh J, Silverman LR, Voso MT, Hellmann A, Tormo M, O'Connor T, Previtali A, Rose S, Garcia-Manero G. A randomized phase 2 trial of azacitidine with or without durvalumab as first-line therapy for higher-risk myelodysplastic syndromes. Blood Advances 2022, 6: 2207-2218. PMID: 34972214, PMCID: PMC9006291, DOI: 10.1182/bloodadvances.2021005487.Peer-Reviewed Original ResearchConceptsHigh-risk myelodysplastic syndromeAdverse eventsArm BArm ASubcutaneous azacitidineMyelodysplastic syndromeTreatment cyclesRandomized phase 2 trialInhibitory immune checkpoint moleculesHematologic adverse eventsMedian overall survivalFirst-line therapyFirst-line treatmentPhase 2 studyPhase 2 trialImmune checkpoint moleculesOverall response rateBone marrow granulocytesAzacitidine monotherapyIntravenous durvalumabCheckpoint moleculesOverall survivalClinical outcomesMarrow granulocytesGrade 3
2021
Health-Related Quality of Life (HRQoL) during Treatment with Enasidenib (ENA) Plus Azacitidine (AZA) in Patients with Newly Diagnosed Mutant IDH2 (m IDH2) Acute Myeloid Leukemia (AML) Not Eligible for Intensive Chemotherapy (IC)
DiNardo C, Dohner H, Zeidan A, Schuh A, Vyas P, Stein E, Wei A, de Botton S, Chen C, Lord-Bessen J, Martin-Regueira P, Lersch F, Gong J, Guo S, Shi L, Montesinos P. Health-Related Quality of Life (HRQoL) during Treatment with Enasidenib (ENA) Plus Azacitidine (AZA) in Patients with Newly Diagnosed Mutant IDH2 (m IDH2) Acute Myeloid Leukemia (AML) Not Eligible for Intensive Chemotherapy (IC). Blood 2021, 138: 1244. DOI: 10.1182/blood-2021-147601.Peer-Reviewed Original ResearchClinical Trials CommitteeAcute myeloid leukemiaEQ-5D VASQLQ-C30 domainsCurrent equity holderBristol-Myers SquibbGHS/QoLGlobal health statusOverall response rateTrials CommitteeQLQ-C30 scoresEQ-5DIntensive chemotherapyPhysical functioningRole functioningDaiichi SankyoSpeakers bureauEvaluable ptsBristol-Myers Squibb CompanyHRQoL scoresDyspnea domainEQ-5D visual analog scale scoresJazz PharmaceuticalsMeaningful improvementsVisual analog scale scoreImmune and Epigenetic Landscape of TP53-mutated Acute Myeloid Leukemia (AML) and Higher-Risk Myelodysplastic Syndromes (HR-MDS)
Zeidan A, Bewersdorf J, Hasle V, Thompson E, de Menezes D, Rose S, Boss I, Fox B. Immune and Epigenetic Landscape of TP53-mutated Acute Myeloid Leukemia (AML) and Higher-Risk Myelodysplastic Syndromes (HR-MDS). Blood 2021, 138: 3371. DOI: 10.1182/blood-2021-146329.Peer-Reviewed Original ResearchHigh-risk myelodysplastic syndromeClinical Trials CommitteeAcute myeloid leukemiaBristol-Myers SquibbCurrent equity holderPoor-risk cytogeneticsVariant allele frequencyAML ptsOverall response rateTrials CommitteeMedian OSTP53 mutationsMyeloid neoplasmsFlow cytometryPeripheral bloodT cell genesHigh expressionBone marrowAverage variant allele frequencyRandomized phase 2 studyBone marrow flow cytometryT-cell gene signatureTumor cellsBM blast percentageIPSS-R scoreOutcomes 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 rateEnasidenib plus azacitidine versus azacitidine alone in patients with newly diagnosed, mutant-IDH2 acute myeloid leukaemia (AG221-AML-005): a single-arm, phase 1b and randomised, phase 2 trial
DiNardo CD, Schuh AC, Stein EM, Montesinos P, Wei AH, de Botton S, Zeidan AM, Fathi AT, Kantarjian HM, Bennett JM, Frattini MG, Martin-Regueira P, Lersch F, Gong J, Hasan M, Vyas P, Döhner H. Enasidenib plus azacitidine versus azacitidine alone in patients with newly diagnosed, mutant-IDH2 acute myeloid leukaemia (AG221-AML-005): a single-arm, phase 1b and randomised, phase 2 trial. The Lancet Oncology 2021, 22: 1597-1608. PMID: 34672961, DOI: 10.1016/s1470-2045(21)00494-0.Peer-Reviewed Original ResearchMeSH KeywordsAgedAminopyridinesAntimetabolites, AntineoplasticAntineoplastic Combined Chemotherapy ProtocolsAzacitidineDrug Administration ScheduleDrug-Related Side Effects and Adverse ReactionsFemaleHumansIsocitrate DehydrogenaseLeukemia, Myeloid, AcuteMaleMutationProgression-Free SurvivalRandom AllocationTreatment OutcomeTriazinesConceptsAcute myeloid leukemiaSerious treatment-related adverse eventsTreatment-related adverse eventsDose-finding portionOverall response rateMyeloid leukemiaAdverse eventsFebrile neutropeniaCombination groupInterim analysisEastern Cooperative Oncology Group performance statusCommon treatment-related grade 3Response rateInteractive web response systemTreatment-related grade 3Phase 1b/2 trialPrespecified interim analysisTreatment-related deathsPhase 2 trialWeb response systemPhase 2Acute myeloid leukemia subtypesPhase 2 portionBristol-Myers SquibbAzacitidine monotherapy
2020
Mutant Isocitrate Dehydrogenase 1 Inhibitor Ivosidenib in Combination With Azacitidine for Newly Diagnosed Acute Myeloid Leukemia
DiNardo CD, Stein AS, Stein EM, Fathi AT, Frankfurt O, Schuh AC, Döhner H, Martinelli G, Patel PA, Raffoux E, Tan P, Zeidan AM, de Botton S, Kantarjian HM, Stone RM, Frattini MG, Lersch F, Gong J, Gianolio DA, Zhang V, Franovic A, Fan B, Goldwasser M, Daigle S, Choe S, Wu B, Winkler T, Vyas P. Mutant Isocitrate Dehydrogenase 1 Inhibitor Ivosidenib in Combination With Azacitidine for Newly Diagnosed Acute Myeloid Leukemia. Journal Of Clinical Oncology 2020, 39: 57-65. PMID: 33119479, PMCID: PMC7771719, DOI: 10.1200/jco.20.01632.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaAdverse eventsQT prolongationMyeloid leukemiaMutant acute myeloid leukemiaBone marrow mononuclear cellsExpected safety profileIDH differentiation syndromeIntensive induction chemotherapyTreatment-related gradeMedian treatment durationPhase Ib trialComplete remission rateOverall response rateMarrow mononuclear cellsDifferentiation syndromeIb trialInduction chemotherapyOral ivosidenibSubcutaneous azacitidineComplete remissionComplete respondersRemission rateMedian durationOral inhibitorEffect 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
Enasidenib Plus Azacitidine Significantly Improves Complete Remission and Overall Response Compared with Azacitidine Alone in Patients with Newly Diagnosed Acute Myeloid Leukemia (AML) with Isocitrate Dehydrogenase 2 (IDH2) Mutations: Interim Phase II Results from an Ongoing, Randomized Study
DiNardo C, Schuh A, Stein E, Fernandez P, Wei A, De Botton S, Zeidan A, Fathi A, Quek L, Kantarjian H, Frattini M, Lersch F, Gong J, Franovic A, MacBeth K, Vyas P, Döhner H. Enasidenib Plus Azacitidine Significantly Improves Complete Remission and Overall Response Compared with Azacitidine Alone in Patients with Newly Diagnosed Acute Myeloid Leukemia (AML) with Isocitrate Dehydrogenase 2 (IDH2) Mutations: Interim Phase II Results from an Ongoing, Randomized Study. Blood 2019, 134: 643. DOI: 10.1182/blood-2019-130362.Peer-Reviewed Original ResearchOverall response rateAcute myeloid leukemiaDuration of responseMorphologic leukemia-free stateBone marrow mononuclear cellsAZA armComplete remissionIntensive chemotherapyCelgene CorporationResponse rateDaiichi SankyoVariant allele frequencySpeakers bureauPTC TherapeuticsPartial remissionData cutoffGrade 3Phase I/II studyMedian DORAdvisory CommitteeSeattle GeneticsForty-sevenConventional care regimensIDH differentiation syndromePoor-risk cytogeneticsPS1023 MUTANT IDH1 INHIBITOR IVOSIDENIB (AG‐120) IN COMBINATION WITH AZACITIDINE FOR NEWLY DIAGNOSED ACUTE MYELOID LEUKEMIA
DiNardo C, Stein A, Stein E, Fathi A, Frankfurt O, Schuh A, Döhner H, Martinelli G, Patel P, Raffoux E, Tan P, Zeidan A, de Botton S, Kantarjian H, Stone R, Lam D, Wang X, Gong J, Kapsalis S, Hickman D, Zhang V, Winkler T, Wu B, Vyas P. PS1023 MUTANT IDH1 INHIBITOR IVOSIDENIB (AG‐120) IN COMBINATION WITH AZACITIDINE FOR NEWLY DIAGNOSED ACUTE MYELOID LEUKEMIA. HemaSphere 2019, 3: 460-461. DOI: 10.1097/01.hs9.0000562388.60660.bc.Peer-Reviewed Original ResearchAcute myeloid leukemiaOverall response rateMorphologic leukemia-free stateComplete remissionAdverse eventsFebrile neutropeniaCR rateMyeloid leukemiaMutant IDH1Isocitrate dehydrogenase 1Double-blind placebo-controlled studyGrade 3/4 adverse eventsRefractory acute myeloid leukemiaBone marrow mononuclear cellsCR/CRhGrade adverse eventsIDH differentiation syndromeIDH1 inhibitor ivosidenibMedian response durationPartial hematologic recoveryPhase 1b studyAbsolute neutrophil countPlacebo-controlled studyTreatment of adultsMarrow mononuclear cells
2018
The use of immunosuppressive therapy in MDS: clinical outcomes and their predictors in a large international patient cohort
Stahl M, DeVeaux M, de Witte T, Neukirchen J, Sekeres MA, Brunner AM, Roboz GJ, Steensma DP, Bhatt VR, Platzbecker U, Cluzeau T, Prata PH, Itzykson R, Fenaux P, Fathi AT, Smith A, Germing U, Ritchie EK, Verma V, Nazha A, Maciejewski JP, Podoltsev NA, Prebet T, Santini V, Gore SD, Komrokji RS, Zeidan AM. The use of immunosuppressive therapy in MDS: clinical outcomes and their predictors in a large international patient cohort. Blood Advances 2018, 2: 1765-1772. PMID: 30037803, PMCID: PMC6058241, DOI: 10.1182/bloodadvances.2018019414.Peer-Reviewed Original ResearchConceptsAnti-thymocyte globulinRBC transfusion independenceImmunosuppressive therapyTransfusion independenceOverall response rateHypocellular bone marrowMyelodysplastic syndromeOverall survivalBone marrowRed blood cell transfusion independenceHorse anti-thymocyte globulinRabbit anti-thymocyte globulinInternational Working Group criteriaCox proportional hazards modelSingle-center natureMedian overall survivalKaplan-Meier methodLarge international cohortLarge international patient cohortProportional hazards modelInternational patient cohortPredictors of benefitParoxysmal nocturnal hemoglobinuriaLogistic regression modelsSteroid monotherapy
2017
A Phase 1/2 Study of the Oral Novel JAK1 Inhibitor INCB052793 As Monotherapy and in Combination with Standard Therapies in Patients with Advanced Hematologic Malignancies
Zeidan A, Cook R, Bordoni R, Asatiani E, Zhou G, Faivre T, Byrne M, Savona M. A Phase 1/2 Study of the Oral Novel JAK1 Inhibitor INCB052793 As Monotherapy and in Combination with Standard Therapies in Patients with Advanced Hematologic Malignancies. Blood 2017, 130: 640. DOI: 10.1182/blood.v130.suppl_1.640.640.Peer-Reviewed Original ResearchAdvanced acute myeloid leukemiaMDS/MPNAdvanced hematologic malignanciesOverall response rateHigh-risk myelodysplastic syndromePhase 1/2 studyComplete responseIncyte CorporationMyelodysplastic syndromeCombination therapyMultiple myelomaHematologic malignanciesData cutoffAdverse eventsStandard therapySpeakers bureauJAK/STAT pathwayPreliminary safetyDisease progressionEfficacy dataMorphologic leukemia-free stateOngoing phase 1/2 studyJAK1 inhibitorAdvisory CommitteeAdvanced myeloid malignancies