2024
Cost-effectiveness of Enasidenib versus conventional care for older patients with IDH2-mutant refractory/relapsed AML
Alhajahjeh A, Patel K, Shallis R, Podoltsev N, Kewan T, Stempel J, Mendez L, Huntington S, Stahl M, Goshua G, Bewersdorf J, Zeidan A. Cost-effectiveness of Enasidenib versus conventional care for older patients with IDH2-mutant refractory/relapsed AML. Leukemia & Lymphoma 2024, 66: 488-496. PMID: 39560957, DOI: 10.1080/10428194.2024.2426073.Peer-Reviewed Original ResearchConventional care regimensOlder patientsTreatment of older patientsIDH2 inhibitor enasidenibIncremental cost-effectiveness ratioCost-effectiveness ratioProbabilistic sensitivity analysesR/R AMLRefractory/relapsed AMLEvent-freeCost-effective treatmentEnasidenibCare regimensAMLPatientsCost-effectiveIncremental costLife yearsConventional careR/RIncremental effectSurvivalTreatmentHMA/VEN treatment modifications and associated outcomes in IDH-mutant AML
Chin K, Derkach A, Famulare C, Gupta G, Borge P, Geyer M, Goldberg A, Haque T, Park J, Roeker L, Tallman M, Stahl M, Stein E. HMA/VEN treatment modifications and associated outcomes in IDH-mutant AML. Leukemia & Lymphoma 2024, 66: 270-278. PMID: 39397429, DOI: 10.1080/10428194.2024.2411436.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaTreatment modificationHypomethylating agentsResponse rateAssociated with lower response ratesMedian overall survivalIDH-mutated acute myeloid leukemiaLong-term toxicityCR/CRi rateSignificant neutropeniaFebrile neutropeniaInduction chemotherapyOverall survivalMyeloid leukemiaLow response rateSurvival rateAffect survivalNeutropeniaVenetoclaxSurvivalED visitsPatientsR/RMonthsReal-world settingsRisk of bleeding in patients with essential thrombocythemia and extreme thrombocytosis
Venkat R, Redd R, Harris A, Aryee M, Marneth A, Kamaz B, Kim C, Wazir M, Weeks L, Stahl M, DeAngelo D, Lindsley R, Luskin M, Hobbs G, How J. Risk of bleeding in patients with essential thrombocythemia and extreme thrombocytosis. Blood Advances 2024, 8: 6043-6054. PMID: 39293089, PMCID: PMC11635702, DOI: 10.1182/bloodadvances.2024013777.Peer-Reviewed Original ResearchConceptsRisk of bleedingClinically relevant nonmajor bleedingEssential thrombocythemiaBleeding riskPlatelet countCumulative incidenceDana-Farber Cancer Institute and Massachusetts General HospitalAssociated with acquired von Willebrand syndromeCumulative incidence of thrombosisCumulative incidence of bleedingIncreased bleeding riskIncidence of bleedingReduced bleeding riskVon Willebrand syndromeIncidence of thrombosisNonmajor bleedingDNMT3A mutationsMassachusetts General HospitalThrombotic eventsDana-FarberDiabetes mellitusBleedingPatientsRisk factorsTreatment decisionsPrognostic impact of ‘multi-hit’ versus ‘single-hit’ TP53 alteration in patients with acute myeloid leukemia: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases
Badar T, Nanaa A, Atallah E, Shallis R, Craver E, Li Z, Goldberg A, Saliba A, Patel A, Bewersdorf J, Duvall A, Burkart M, Bradshaw D, Abaza Y, Stahl M, Palmisiano N, Murthy S, Zeidan A, Kota V, Patnaik M, Litzow M. Prognostic impact of ‘multi-hit’ versus ‘single-hit’ TP53 alteration in patients with acute myeloid leukemia: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases. Haematologica 2024, 109: 3533-3542. PMID: 38813716, PMCID: PMC11532685, DOI: 10.3324/haematol.2024.285000.Peer-Reviewed Original ResearchAcute myeloid leukemiaMyelodysplastic syndromeComplex cytogeneticsMyeloid leukemiaAllogeneic hematopoietic stem cell transplantationLower-risk myelodysplastic syndromesHematopoietic stem cell transplantationHigher-risk myelodysplastic syndromesOutcomes of SHStem cell transplantationAllo-HCTTP53 alterationsPrognostic impactMyeloid malignanciesTP53 mutationsCell transplantationFLT3-ITDIDH1 mutationMultivariate analysisSupportive careUS academic institutionsNeoplastic diseasePatientsSuperior EFSPredicting outcomeMolecular ontogeny underlies the benefit of adding venetoclax to hypomethylating agents in newly diagnosed AML patients
Shimony S, Garcia J, Keating J, Chen E, Luskin M, Stahl M, Neuberg D, DeAngelo D, Stone R, Lindsley R. Molecular ontogeny underlies the benefit of adding venetoclax to hypomethylating agents in newly diagnosed AML patients. Leukemia 2024, 38: 1494-1500. PMID: 38538860, PMCID: PMC11216982, DOI: 10.1038/s41375-024-02230-w.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAgedAged, 80 and overAntineoplastic Combined Chemotherapy ProtocolsBridged Bicyclo Compounds, HeterocyclicDNA MethylationFemaleHematopoietic Stem Cell TransplantationHumansLeukemia, Myeloid, AcuteMaleMiddle AgedMutationPrognosisRemission InductionSulfonamidesSurvival RateTumor Suppressor Protein p53Young AdultConceptsAcute myeloid leukemiaDiagnosed AML patientsHypomethylating agentsAML patientsTP53-mutated acute myeloid leukemiaPatients treated with intensive chemotherapyAllogeneic hematopoietic stem cell transplantationHematopoietic stem cell transplantationComposite complete remissionStem cell transplantationGroup of patientsMolecular ontogenyMedian OSOS benefitComplete remissionIntensive chemotherapyCell transplantationClinicopathological variablesMyeloid leukemiaClinical benefitClinical impactSplicing mutationPatientsSecondary groupVenetoclax
2023
When to use which molecular prognostic scoring system in the management of patients with MDS?
Kewan T, Bewersdorf J, Gurnari C, Xie Z, Stahl M, Zeidan A. When to use which molecular prognostic scoring system in the management of patients with MDS? Best Practice & Research Clinical Haematology 2023, 36: 101517. PMID: 38092484, DOI: 10.1016/j.beha.2023.101517.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsInternational Prognostic Scoring SystemPrognostic scoring systemAcute myeloid leukemiaScoring systemRisk stratificationRecurrent molecular alterationsHigh-risk patientsAppropriate risk stratificationManagement of patientsRecurrent genetic mutationsIntensive therapyMyeloid leukemiaTreatment strategiesPrognostic toolDisease pathogenesisMolecular alterationsHematopoietic cancersClinical decisionHeterogeneous groupGenetic mutationsNext-generation sequencingPrognostic systemPatientsVariable propensitySubsequent revisionE7820, an anti-cancer sulfonamide, degrades RBM39 in patients with splicing factor mutant myeloid malignancies: a phase II clinical trial
Bewersdorf J, Stahl M, Taylor J, Mi X, Chandhok N, Watts J, Derkach A, Wysocki M, Lu S, Bourcier J, Hogg S, Rahman J, Chaudhry S, Totiger T, Abdel-Wahab O, Stein E. E7820, an anti-cancer sulfonamide, degrades RBM39 in patients with splicing factor mutant myeloid malignancies: a phase II clinical trial. Leukemia 2023, 37: 2512-2516. PMID: 37814121, PMCID: PMC10681888, DOI: 10.1038/s41375-023-02050-4.Peer-Reviewed Original ResearchSafety and efficacy of FLAG-Ida-based therapy combined with venetoclax for the treatment for newly diagnosed and relapsed/refractory patients with AML – A systematic review
Sherban A, Fredman D, Shimony S, Yeshurun M, Raanani P, Stahl M, Gafter-Gvili A, Wolach O. Safety and efficacy of FLAG-Ida-based therapy combined with venetoclax for the treatment for newly diagnosed and relapsed/refractory patients with AML – A systematic review. Leukemia Research 2023, 133: 107368. PMID: 37598660, DOI: 10.1016/j.leukres.2023.107368.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaIntensive chemotherapyEfficacy outcomesHigh-risk acute myeloid leukemiaResponse to treatmentFLAG-IdaRelapsed/refractory patientsMyeloid leukemiaTreated patientsVenetoclaxSafety outcomesSystematic reviewEfficacyInfection ratePatientsTreatmentOutcomesChemotherapyLeukemiaSafetyTherapyReviewInfectionA systematic reviewCurrent landscape of translational and clinical research in myelodysplastic syndromes/neoplasms (MDS): Proceedings from the 1st International Workshop on MDS (iwMDS) Of the International Consortium for MDS (icMDS)
Bewersdorf J, Xie Z, Bejar R, Borate U, Boultwood J, Brunner A, Buckstein R, Carraway H, Churpek J, Daver N, Porta M, DeZern A, Fenaux P, Figueroa M, Gore S, Griffiths E, Halene S, Hasserjian R, Hourigan C, Kim T, Komrokji R, Kuchroo V, List A, Loghavi S, Majeti R, Odenike O, Patnaik M, Platzbecker U, Roboz G, Sallman D, Santini V, Sanz G, Sekeres M, Stahl M, Starczynowski D, Steensma D, Taylor J, Abdel-Wahab O, Xu M, Savona M, Wei A, Zeidan A. Current landscape of translational and clinical research in myelodysplastic syndromes/neoplasms (MDS): Proceedings from the 1st International Workshop on MDS (iwMDS) Of the International Consortium for MDS (icMDS). Blood Reviews 2023, 60: 101072. PMID: 36934059, DOI: 10.1016/j.blre.2023.101072.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsImmune checkpoint inhibitorsSpecific molecular alterationsNovel animal modelInnate immune systemCheckpoint inhibitorsImmune dysregulationMDS patientsClinical trialsNovel therapiesTherapeutic strategiesAnimal modelsGermline predispositionImmune systemMolecular alterationsClinical researchInternational ConsortiumNeoplasmsClinical workGenetic landscapeInternational WorkshopPatientsCurrent landscapePathogenesisTherapyDisease
2022
Cost-Effectiveness of Azacitidine and Ivosidenib in Newly Diagnosed Older, Intensive Chemotherapy-Ineligible Patients with IDH1-Mutant Acute Myeloid Leukemia
Bewersdorf J, Patel K, Goshua G, Shallis R, Podoltsev N, Stahl M, Stempel J, Stein E, Huntington S, Zeidan A. Cost-Effectiveness of Azacitidine and Ivosidenib in Newly Diagnosed Older, Intensive Chemotherapy-Ineligible Patients with IDH1-Mutant Acute Myeloid Leukemia. Blood 2022, 140: 319-321. DOI: 10.1182/blood-2022-158465.Peer-Reviewed Original ResearchInternational Working Group (IWG) Response Criteria and Association with Overall Survival in Patients with Myelodysplastic Syndromes Treated with Hypomethylating Agents - a Systematic Review and Meta-Analysis
Bewersdorf J, Grimshaw A, Platzbecker U, Fenaux P, Sekeres M, Zeidan A, Stahl M. International Working Group (IWG) Response Criteria and Association with Overall Survival in Patients with Myelodysplastic Syndromes Treated with Hypomethylating Agents - a Systematic Review and Meta-Analysis. Blood 2022, 140: 9811-9812. DOI: 10.1182/blood-2022-158622.Peer-Reviewed Original ResearchIntensive Therapy for NPM1 Mutant AML Patients: Negative Impact of FLT3-ITDhigh but Not FLT3-ITDlow and FLT3-TKD and Role of Transplant in Patients over 60 Years of Age
Winer E, Flamand Y, Shallis R, Stempel J, Goldberg A, Stahl M, Patel A, Duvall A, Abaza Y, Oh T, Ma K, Badar T, Litzow M, Kota V, Bradshaw D, Raman H, Luskin M, Stone R, DeAngelo D. Intensive Therapy for NPM1 Mutant AML Patients: Negative Impact of FLT3-ITDhigh but Not FLT3-ITDlow and FLT3-TKD and Role of Transplant in Patients over 60 Years of Age. Blood 2022, 140: 6285-6287. DOI: 10.1182/blood-2022-170587.Peer-Reviewed Original ResearchMutant Srsf2 Diminishes Jak2V617F -Induced Erythrocytosis in Mice and Is Associated with Lower Hemoglobin in Patients with Chronic Phase JAK2-Mutant MPN
Marneth A, Jutzi J, Kim C, Laurore C, Tishena A, Kramer F, Rocha A, Wazir M, Weeks L, How J, Stahl M, Luskin M, Lindsley R, Pozdnyakova O, Graubert T, Mullally A. Mutant Srsf2 Diminishes Jak2V617F -Induced Erythrocytosis in Mice and Is Associated with Lower Hemoglobin in Patients with Chronic Phase JAK2-Mutant MPN. Blood 2022, 140: 1089-1090. DOI: 10.1182/blood-2022-158823.Peer-Reviewed Original ResearchClinical Outcomes of Patients with TP53-Mutated AML after First Relapse or with Primary Refractory Disease: Results from Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND)
Badar T, Atallah E, Saliba A, Shallis R, Stahl M, Bewersdorf J, De Camargo Correia G, Patel A, Abaza Y, Murthy S, Duvall A, Burkart M, Palmisiano N, Bradshaw D, Kota V, Dinner S, Goldberg A, Litzow M. Clinical Outcomes of Patients with TP53-Mutated AML after First Relapse or with Primary Refractory Disease: Results from Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND). Blood 2022, 140: 6058-6060. DOI: 10.1182/blood-2022-163795.Peer-Reviewed Original ResearchPredictors of Long-Term Outcome in TP53-Mutated Acute Myeloid Leukemia Patients Receiving Allogeneic Stem Cell Transplant after First- or Second-Line Therapy: Results from the Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND)
Badar T, Atallah E, Shallis R, Saliba A, Stahl M, Bewersdorf J, Grenet J, Patel A, Abaza Y, Murthy S, Duvall A, Burkart M, Palmisiano N, Kubiak M, Kota V, Dinner S, Goldberg A, Litzow M. Predictors of Long-Term Outcome in TP53-Mutated Acute Myeloid Leukemia Patients Receiving Allogeneic Stem Cell Transplant after First- or Second-Line Therapy: Results from the Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND). Blood 2022, 140: 1435-1437. DOI: 10.1182/blood-2022-167731.Peer-Reviewed Original ResearchA Phase II Clinical Trial of E7820 for Patients with Relapsed/Refractory Myeloid Malignancies with Mutations in Splicing Factor Genes
Bewersdorf J, Stahl M, Taylor J, Chandhok N, Watts J, Derkach A, Wysocki M, Kostantakis V, Lu S, Bourcier J, Hogg S, Totiger T, Abdel-Wahab O, Stein E. A Phase II Clinical Trial of E7820 for Patients with Relapsed/Refractory Myeloid Malignancies with Mutations in Splicing Factor Genes. Blood 2022, 140: 9065-9067. DOI: 10.1182/blood-2022-156830.Peer-Reviewed Original ResearchMolecular predictors of immunophenotypic measurable residual disease clearance in acute myeloid leukemia
Stahl M, Derkach A, Farnoud N, Bewersdorf J, Robinson T, Famulare C, Cho C, Devlin S, Menghrajani K, Patel M, Cai S, Miles L, Bowman R, Geyer M, Dunbar A, Epstein‐Peterson Z, McGovern E, Schulman J, Glass J, Taylor J, Viny A, Stein E, Getta B, Arcila M, Gao Q, Barker J, Shaffer B, Papadopoulos E, Gyurkocza B, Perales M, Abdel‐Wahab O, Levine R, Giralt S, Zhang Y, Xiao W, Pai N, Papaemmanuil E, Tallman M, Roshal M, Goldberg A. Molecular predictors of immunophenotypic measurable residual disease clearance in acute myeloid leukemia. American Journal Of Hematology 2022, 98: 79-89. PMID: 36251406, PMCID: PMC10080561, DOI: 10.1002/ajh.26757.Peer-Reviewed Original ResearchConceptsMeasurable residual diseaseAcute myeloid leukemiaAllo-SCTInduction chemotherapyPersistent diseaseMyeloid leukemiaAllogeneic stem cell transplantationStem cell transplantationMonosomy 5Residual diseaseDisease clearanceKaryotypic abnormalitiesPrognostic factorsCell transplantationMolecular predictorsMRD clearanceRemissionClinical trialsNext-generation sequencingChemotherapyPatientsMutation patternsTherapyLeukemiaMRDVenetoclax-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 dataVenetoclaxInhibitorsEfficacy 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 inhibitorsVenetoclaxTherapyAre We Moving the Needle for Patients with TP53-Mutated Acute Myeloid Leukemia?
Shallis RM, Bewersdorf JP, Stahl MF, Halene S, Zeidan AM. Are We Moving the Needle for Patients with TP53-Mutated Acute Myeloid Leukemia? Cancers 2022, 14: 2434. PMID: 35626039, PMCID: PMC9140008, DOI: 10.3390/cancers14102434.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsAcute myeloid leukemiaMyeloid leukemiaAllogeneic hematopoietic stem cell transplantationHematopoietic stem cell transplantationCD47/SIRPαIntensive induction therapyAvailable therapeutic optionsStem cell transplantationStandard of careAvailable clinical dataTesting of agentsInduction therapyMedian overallRefractory settingAggressive treatmentTim-3Immune checkpointsPreclinical rationaleTherapeutic optionsCell transplantationEfficacy dataClinical dataPatientsMolecular subgroupsTherapeutic agents
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