2024
Targeted therapies for myelodysplastic syndromes/neoplasms (MDS): current landscape and future directions
Bidikian A, Bewersdorf J, Shallis R, Getz T, Stempel J, Kewan T, Stahl M, Zeidan A. Targeted therapies for myelodysplastic syndromes/neoplasms (MDS): current landscape and future directions. Expert Review Of Anticancer Therapy 2024, 24: 1131-1146. PMID: 39367718, DOI: 10.1080/14737140.2024.2414071.Peer-Reviewed Original ResearchErythropoiesis-stimulating agentsTargeted therapyLR-MDSHR-MDSHypoxia-inducible factorAllogeneic hematopoietic stem cell transplantationLandscape of targeted therapiesHematopoietic stem cell transplantationHeterogeneous group of hematologic malignanciesGroup of hematologic malignanciesMolecular prognostic toolsDuration of responseStem cell transplantationTrial designClinical trial designHypomethylating agentsCell transplantationHematologic malignanciesImprove patient outcomesRNA splicing machineryImmune evasionPrognostic toolTGF-betaTherapyEffective treatmentIntensive induction chemotherapy vs hypomethylating agents in combination with venetoclax in NPM1-mutant AML
Bewersdorf J, Shimony S, Shallis R, Liu Y, Berton G, Schaefer E, Zeidan A, Goldberg A, Stein E, Marcucci G, Bystrom R, Lindsley R, Chen E, Perez J, Stein A, Pullarkat V, Aldoss I, DeAngelo D, Neuberg D, Stone R, Garciaz S, Ball B, Stahl M. Intensive induction chemotherapy vs hypomethylating agents in combination with venetoclax in NPM1-mutant AML. Blood Advances 2024, 8: 4845-4855. PMID: 38941537, PMCID: PMC11416634, DOI: 10.1182/bloodadvances.2024012858.Peer-Reviewed Original ResearchIntensive induction chemotherapyAcute myeloid leukemiaNPM1-Mutant Acute Myeloid LeukemiaInduction chemotherapyHypomethylating agentsMulticenter retrospective cohort study of patientsPatients treated with ICAllogeneic stem cell transplantationRetrospective cohort study of patientsMulticenter retrospective cohort studyCohort study of patientsComposite complete remissionStem cell transplantationYears-oldFLT3-ITD mutationStudy of patientsStandard of careNormal cytogeneticsComplete remissionCell transplantationNPM1 mutationsMyeloid leukemiaFLT3-ITDYounger patientsOlder patientsAcute myeloid leukemia (AML) with chromosome 3 inversion: biology, management, and clinical outcome
Alhajahjeh A, Bewersdorf J, Bystrom R, Zeidan A, Shimony S, Stahl M. Acute myeloid leukemia (AML) with chromosome 3 inversion: biology, management, and clinical outcome. Leukemia & Lymphoma 2024, 65: 1541-1551. PMID: 38962996, DOI: 10.1080/10428194.2024.2367040.Peer-Reviewed Original ResearchAcute myeloid leukemiaIntensive chemotherapyHypomethylating agentsMyeloid leukemiaAllogeneic stem cell transplantationAcute myeloid leukemia casesAcute myeloid leukemia subtypesStem cell transplantationComplex hematological malignancyCurrent treatment modalitiesRare genetic anomalyCell transplantationHematologic malignanciesTreatment modalitiesClinical outcomesTreatment responseInv(3Genetic alterationsLeukemia developmentTreatment strategiesCellular processesGenetic anomaliesLeukemiaFusion geneClinical implicationsPrognostic 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 outcomeCost-effectiveness of adding quizartinib to induction chemotherapy for patients with FLT3-mutant acute myeloid leukemia
Bewersdorf J, Patel K, Shallis R, Podoltsev N, Kewan T, Stempel J, Mendez L, Stahl M, Stein E, Huntington S, Goshua G, Zeidan A. Cost-effectiveness of adding quizartinib to induction chemotherapy for patients with FLT3-mutant acute myeloid leukemia. Leukemia & Lymphoma 2024, 65: 1136-1144. PMID: 38648559, PMCID: PMC11265977, DOI: 10.1080/10428194.2024.2344052.Peer-Reviewed Original ResearchQuality-adjusted life yearsCompletion of consolidation therapyFLT3-mutant acute myeloid leukemiaAllogeneic hematopoietic cell transplantationIncremental cost-effectiveness ratioProbabilistic sensitivity analysesImproved overall survivalHematopoietic cell transplantationPartitioned survival analysis modelAcute myeloid leukemiaCost-effectiveness ratioFLT3 inhibitor quizartinibHealth economic implicationsConsolidation therapyInduction chemotherapyAverage wholesale priceOverall survivalCell transplantationContinuous therapyMyeloid leukemiaITD mutationQuizartinibIncremental costCost-effective optionLife years
2023
Clinical and Genomic-Based Decision Support System to Define the Optimal Timing of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Myelodysplastic Syndromes (MDS)
Tentori C, Gregorio C, Robin M, Gagelmann N, Gurnari C, Ball S, Berrocal J, Lanino L, D'Amico S, Spreafico M, Maggioni G, Travaglino E, Sauta E, Meggendorfer M, Zhao L, Bernardi M, Di Grazia C, Vago L, Rivoli G, Borin L, Chiusolo P, Giaccone L, Voso M, Bewersdorf J, Nibourel O, Díaz-Beyá M, Jerez A, Hernandez F, Kennedy K, Xicoy B, Ubezio M, Campagna A, Russo A, Todisco G, Mannina D, Bramanti S, Zampini M, Riva E, Bicchieri M, Asti G, Viviani F, Buizza A, Tinterri B, Bacigalupo A, Rambaldi A, Passamonti F, Ciceri F, Savevski V, Santoro A, Al Ali N, Sallman D, Sole F, Garcia-Manero G, Germing U, Kordasti S, Santini V, Sanz G, Kern W, Kubasch A, Platzbecker U, Diez-Campelo M, Maciejewski J, Ades L, Fenaux P, Haferlach T, Zeidan A, Castellani G, Komrokji R, Ieva F, Della Porta M. Clinical and Genomic-Based Decision Support System to Define the Optimal Timing of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Myelodysplastic Syndromes (MDS). Blood 2023, 142: 197. DOI: 10.1182/blood-2023-182194.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationStem cell transplantationMyelodysplastic syndromeProlonged life expectancyClinical outcomesOptimal timingCell transplantationLife expectancyValidation cohortImmediate transplantationTransplantation policyRisks of HSCTImmediate hematopoietic stem cell transplantationAllogeneic hematopoietic stem cell transplantationAge groupsDiagnosis of MDSConventional prognostic scoresPost-HSCT outcomesLow-risk diseaseTiming of transplantationDisease-modifying therapiesEarly disease stagesPatient's life expectancyAverage survival timeDifferent time points
2022
Disparities in receiving disease‐directed therapy, allogeneic stem cell transplantation in non‐Hispanic Black patients with TP53‐mutated acute myeloid leukemia
Badar T, Litzow M, Shallis R, Patel A, Saliba A, Burkart M, Bewersdorf J, Stahl M, De Camargo Correia G, Murthy S, Abaza Y, Duvall A, Bradshaw D, Kota V, Dinner S, Goldberg A, Palmisiano N, Al Kali A, Atallah E. Disparities in receiving disease‐directed therapy, allogeneic stem cell transplantation in non‐Hispanic Black patients with TP53‐mutated acute myeloid leukemia. Cancer 2022, 129: 934-945. PMID: 36545710, DOI: 10.1002/cncr.34604.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaAllogeneic stem cell transplantationNHB patientsStem cell transplantationNHW patientsCell transplantationMyeloid leukemiaTherapy-related acute myeloid leukemiaNon-Hispanic black patientsCurative-intent therapyLow-intensity chemotherapyBest supportive careComplete response rateMedian patient ageProportion of patientsSubset of patientsDisease-directed therapyHigher proportionIntensive chemotherapyPatient ageSupportive careBlack patientsClinical outcomesTreatment disparitiesRetrospective studyHitting the brakes on accelerated and blast-phase myeloproliferative neoplasms: current and emerging concepts
Bewersdorf J, Rampal R. Hitting the brakes on accelerated and blast-phase myeloproliferative neoplasms: current and emerging concepts. Hematology 2022, 2022: 218-224. PMID: 36485103, PMCID: PMC9820986, DOI: 10.1182/hematology.2022000341.Peer-Reviewed Original ResearchConceptsMyeloproliferative neoplasmsBlast-phase MPNBlast-phase myeloproliferative neoplasmsAllogeneic hematopoietic cell transplantationBCR-ABL-negative myeloproliferative neoplasmsStages of clinical developmentMedian overall survivalHigh-risk molecular featuresHematopoietic cell transplantationCurative therapeutic modalityPrognosis of patientsAcute myeloid leukemiaMinority of patientsClinical trial enrollmentMPN-BPMyeloid blastsCurative intentHypomethylating agentsOverall survivalCell transplantationPeripheral bloodMyeloid leukemiaPalliative treatmentBone marrowClinical developmentMolecular 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 patternsTherapyLeukemiaMRDAre 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
2021
The Current Understanding of and Treatment Paradigm for Newly-Diagnosed TP53-Mutated Acute Myeloid Leukemia
Shallis R, Stahl M, Bewersdorf J, Zeidan A. The Current Understanding of and Treatment Paradigm for Newly-Diagnosed TP53-Mutated Acute Myeloid Leukemia. Hemato 2021, 2: 748-763. DOI: 10.3390/hemato2040051.Peer-Reviewed Original ResearchAcute myeloid leukemiaMyeloid leukemiaTherapy-related acute myeloid leukemiaMeasurable residual disease statusHematopoietic stem cell transplantationMedian overall survivalResidual disease statusStem cell transplantationCurrent treatment approachesIntensive chemotherapyIntensive regimensRemission rateCytogenetic riskOverall survivalWorse prognosisCell transplantationConditioning intensityTreatment paradigmTreatment approachesTP53 mutationsDisease statusBiological subsetsPatientsPrognosisLeukemiaMulticenter Analysis of Treatment and Outcomes for Patient with TP53 Mutated AML in the Era of Novel Therapies; Significant Impact of Allogeneic Stem Cell Transplantation on Survival
Badar T, Litzow M, Shallis R, Bewersdorf J, Saliba A, De Camargo Correia G, Stahl M, Goldberg A, Atallah E, Foran J. Multicenter Analysis of Treatment and Outcomes for Patient with TP53 Mutated AML in the Era of Novel Therapies; Significant Impact of Allogeneic Stem Cell Transplantation on Survival. Blood 2021, 138: 797. DOI: 10.1182/blood-2021-147639.Peer-Reviewed Original ResearchCR/CRiProportion of patientsStem cell transplantationComplex cytogeneticsVariant allele frequencyCPX-351Overall survivalAML patientsNovel therapiesTP53 mutationsCell transplantationUnivariate analysisAllogeneic hematopoietic stem cell transplantationMedian progression-free survivalAllogeneic stem cell transplantationHematopoietic stem cell transplantationAdvisory CommitteeAML induction therapyExtra-medullary diseaseHigh-risk AMLInduction/consolidationVenetoclax combination therapyVenetoclax-based regimensMedian overall survivalMulticenter observational studyMaintenance therapies in acute myeloid leukemia: the renaissance of an old therapeutic concept
Bewersdorf J, Tallman M, Stahl M. Maintenance therapies in acute myeloid leukemia: the renaissance of an old therapeutic concept. Current Opinion In Oncology 2021, 33: 658-669. PMID: 34341323, DOI: 10.1097/cco.0000000000000778.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaAcute myeloid leukemia patientsMaintenance therapyAllo-HCTOverall survivalIntensive chemotherapyMyeloid leukemiaPersistence of leukemic cellsSafety of maintenance therapyAllogeneic hematopoietic cell transplantationRelapse-free survivalDuration of remissionHematopoietic cell transplantationIndividualized therapeutic approachFLT3 inhibitor sorafenibCC-486Induction chemotherapyOS benefitDisease relapseCell transplantationInhibitor sorafenibImproved survivalPatient selectionTargeted agentsLeukemic cells
2020
Following in the footsteps of acute myeloid leukemia: are we witnessing the start of a therapeutic revolution for higher-risk myelodysplastic syndromes?
Bewersdorf JP, Zeidan AM. Following in the footsteps of acute myeloid leukemia: are we witnessing the start of a therapeutic revolution for higher-risk myelodysplastic syndromes? Leukemia & Lymphoma 2020, 61: 2295-2312. PMID: 32421403, PMCID: PMC7670856, DOI: 10.1080/10428194.2020.1761968.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsHigh-risk myelodysplastic syndromeAcute myeloid leukemiaMyelodysplastic syndromeMyeloid leukemiaAllogeneic hematopoietic stem cell transplantationGenetic testingHematopoietic stem cell transplantationImmune checkpoint inhibitorsCurative treatment optionMainstay of therapyRecent therapeutic advancesStem cell transplantationCurrent treatment approachesAgent azacitidineCheckpoint inhibitorsMost patientsMDS patientsAvailable therapiesCell transplantationPatient subgroupsTreatment optionsTherapeutic advancesIDH inhibitorsIndividualized treatmentNew agents