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, ahead-of-print: 1-9. 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 effectSurvivalTreatmentA review of the isocitrate dehydrogenase inhibitors in management of adult patients with AML and MDS
Norman M, Yamartino K, Gerstein R, Shallis R, Mendez L, Podoltsev N, Stahl M, Eighmy W, Zeidan A. A review of the isocitrate dehydrogenase inhibitors in management of adult patients with AML and MDS. Expert Review Of Hematology 2024, ahead-of-print: 1-13. PMID: 39474840, DOI: 10.1080/17474086.2024.2422554.Peer-Reviewed Original ResearchDiagnosed AMLSurvival benefitManagement of acute myeloid leukemiaDevelopment of oral therapiesIsocitrate dehydrogenase inhibitorsNewly diagnosed AMLManagement of adult patientsPost-transplant maintenanceAcute myeloid leukemiaSingle-arm studyExcellent response ratesIDH inhibitorsRelapsed AMLHypomethylating agentsInhibitor therapyMyelodysplastic syndromeOral therapyCombination therapyPost-transplantMyeloid leukemiaImproved survivalSingle-armAdult patientsAzacitidineRandomized studyGlobal Disparities in the Characteristics and Outcomes of Leukemia Clinical Trials: A Cross-Sectional Study of the ClinicalTrials.gov Database.
Alhajahjeh A, Rotter L, Stempel J, Grimshaw A, Bewersdorf J, Blaha O, Kewan T, Podoltsev N, Shallis R, Mendez L, Stahl M, Zeidan A. Global Disparities in the Characteristics and Outcomes of Leukemia Clinical Trials: A Cross-Sectional Study of the ClinicalTrials.gov Database. JCO Global Oncology 2024, 10: e2400316. PMID: 39621951, DOI: 10.1200/go-24-00316.Peer-Reviewed Original ResearchAnalyzing determinants of premature trial discontinuation in leukemia clinical trials
Rotter L, Alhajahjeh A, Stempel J, Grimshaw A, Bewersdorf J, Blaha O, Kewan T, Podoltsev N, Shallis R, Mendez L, Stahl M, Zeidan A. Analyzing determinants of premature trial discontinuation in leukemia clinical trials. Leukemia & Lymphoma 2024, ahead-of-print: 1-9. PMID: 39440622, DOI: 10.1080/10428194.2024.2416565.Peer-Reviewed Original ResearchClinical trialsSlow accrualLeukemia trialsEarly-phase trialsNon-randomized trialsAcademic-sponsored trialsImprove patient outcomesLeukemia clinical trialsSingle-centerTrial discontinuationSponsored trialsSmall trialsLeukemiaEarly terminationPatient outcomesLate-phaseIndependent reviewersTermination ratesComprehensive searchTrialsTargeted 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 patientsImpact of induction regimens intensity and allogeneic stem cell transplantation on survival of patients with Philadelphia chromosome‐positive acute lymphoblastic leukemia: A multi‐institutional study
Badar T, Narra R, Mims A, Heckman M, Shallis R, Fahad S, Hunter C, Kota V, Othman T, Jonas B, Desai S, de Camargo Correia G, Patel A, DuVall A, Palmisiano N, Curran E, Omer Z, Advani A, Atallah E, Litzow M. Impact of induction regimens intensity and allogeneic stem cell transplantation on survival of patients with Philadelphia chromosome‐positive acute lymphoblastic leukemia: A multi‐institutional study. American Journal Of Hematology 2024, 99: 2388-2391. PMID: 39253997, DOI: 10.1002/ajh.27475.Peer-Reviewed Original ResearchIntensive chemotherapyPhiladelphia chromosome-positive acute lymphoblastic leukemiaAllogeneic stem cell transplantationFirst-generation TKIsStem cell transplantationSurvival of patientsAcute lymphoblastic leukemiaMulti-institutional studyAllo-HCTPropensity score matchingRegimen intensityCell transplantationLymphoblastic leukemiaMultivariate analysisOutcomes of patients with acute myeloid leukemia treated with intensive therapy after failure of venetoclax-inclusive, less-intensive therapy
Achar R, McCormick B, Dworkin E, Geramita E, Im A, Patel A, Badar T, Shallis R. Outcomes of patients with acute myeloid leukemia treated with intensive therapy after failure of venetoclax-inclusive, less-intensive therapy. Leukemia Research 2024, 146: 107577. PMID: 39278123, DOI: 10.1016/j.leukres.2024.107577.Peer-Reviewed Original ResearchBeyond HMAs: Novel targets and therapeutic approaches
Getz T, Bewersdorf J, Kewan T, Stempel J, Bidikian A, Shallis R, Stahl M, Zeidan A. Beyond HMAs: Novel targets and therapeutic approaches. Seminars In Hematology 2024 PMID: 39389839, DOI: 10.1053/j.seminhematol.2024.08.001.Peer-Reviewed Original ResearchAcute myeloid leukemiaDelays progression to acute myeloid leukemiaHeterogeneous group of clonal hematopoietic disordersGroup of clonal hematopoietic disordersMolecular International Prognostic Scoring SystemRandomized phase 3 clinical trialProgression to acute myeloid leukemiaInternational Prognostic Scoring SystemLower-risk MDS patientsRisk stratification of patientsPhase 3 clinical trialsCombination of azacitidineHypomethylating agent combinationsCurrent treatment landscapeFirst line therapyPrognostic Scoring SystemBiomarker-directed therapiesClonal hematopoietic disordersLack of therapeutic agentsStratification of patientsEarly phase trialsErythropoiesis stimulating agentsTreated with therapiesVariable clinical featuresStandard of careReal‐world outcomes of intensive induction approaches in core binding factor acute myeloid leukemia
Rojek A, McCormick B, Cwykiel J, Odetola O, Abaza Y, Nai N, Foucar C, Achar R, Shallis R, Bradshaw D, Standridge M, Kota V, Murthy S, Badar T, Patel A. Real‐world outcomes of intensive induction approaches in core binding factor acute myeloid leukemia. EJHaem 2024, 5: 728-737. PMID: 39157611, PMCID: PMC11327707, DOI: 10.1002/jha2.981.Peer-Reviewed Original ResearchPatients treated with ICEvent-free survivalTreated with ICCore binding factor acute myeloid leukemiaGemtuzumab ozogamicinIntensive chemotherapyCBF-AMLAcute myeloid leukemiaKIT inhibitorsOverall survivalEuropean LeukemiaNetMyeloid leukemiaThree-year overall survivalMedian follow-upInduction treatment regimensAntibody-drug conjugatesStatistically significant differenceImproved OSFavorable riskSurvival benefitTreatment regimensProspective studyFollow-upCompare outcomesAcademic centersTreatment approach and outcomes of patients with accelerated/blast-phase myeloproliferative neoplasms in the current era
Patel A, Yoon J, Johnston H, Davidson M, Shallis R, Chen E, Burkart M, Oh T, Iyer S, Madarang E, Muthiah C, Gross I, Dean R, Kassner J, Viswabandya A, Madero-Marroquin R, Rampal R, Murthy S, Bradley T, Abaza Y, Garcia J, Gupta V, Pettit K, Cursio J, Odenike O. Treatment approach and outcomes of patients with accelerated/blast-phase myeloproliferative neoplasms in the current era. Blood Advances 2024, 8: 3468-3477. PMID: 38739724, PMCID: PMC11260843, DOI: 10.1182/bloodadvances.2024012880.Peer-Reviewed Original ResearchAcute myeloid leukemiaOutcomes of patientsMyeloproliferative neoplasmsMPN-AP/BPAllo-HCTMedian OSIntensive chemotherapyOverall survivalEuropean LeukemiaNetSurvival outcomesAllogeneic hematopoietic stem cell transplantationAssociated with poor survival outcomesHematopoietic stem cell transplantationInvestigate outcomes of patientsProgression of myeloproliferative neoplasmsMedian overall survivalStem cell transplantationFirst-line treatmentPoor survival outcomesMulti-center analysisAssociation of responseBlast-phaseMPN-BPCell transplantationAnalyzed patientsPrognostic 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 outcomeCombination therapy with hypomethylating agents and venetoclax versus intensive induction chemotherapy in IDH1‐ or IDH2‐mutant newly diagnosed acute myeloid leukemia—A multicenter cohort study
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, Ramos J, Stein A, Pullarkat V, Aldoss I, DeAngelo D, Neuberg D, Stone R, Garciaz S, Ball B, Stahl M. Combination therapy with hypomethylating agents and venetoclax versus intensive induction chemotherapy in IDH1‐ or IDH2‐mutant newly diagnosed acute myeloid leukemia—A multicenter cohort study. American Journal Of Hematology 2024, 99: 1640-1643. PMID: 38751104, DOI: 10.1002/ajh.27366.Peer-Reviewed Original ResearchChallenges in management of older patients with chronic myeloid leukemia
Stempel J, Shallis R, Wong R, Podoltsev N. Challenges in management of older patients with chronic myeloid leukemia. Leukemia & Lymphoma 2024, 65: 1219-1232. PMID: 38652861, DOI: 10.1080/10428194.2024.2342559.Peer-Reviewed Original ResearchChronic myeloid leukemiaTyrosine kinase inhibitorsTreatment-free remissionOlder patientsMyeloid leukemiaTyrosine kinase inhibitor dosingGeneration tyrosine kinase inhibitorsManagement of older patientsSecond-line agentsSurvival of patientsSide effect profileFront-line optionPrevalence of comorbiditiesImproved survivalCardiovascular toleranceEffect profileFavorable outcomeKinase inhibitorsPrevent progressionPatientsPersonalized treatmentQuality of lifeTreatment goalsOptimal careDiscontinuation strategiesCost-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 yearsComparing venetoclax in combination with hypomethylating agents to hypomethylating agent-based therapies for treatment naive TP53-mutated acute myeloid leukemia: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND)
Badar T, Nanaa A, Atallah E, Shallis R, Guilherme S, Goldberg A, Saliba A, Patel A, Bewersdorf J, DuVall A, Bradshaw D, Abaza Y, Murthy G, Palmisiano N, Zeidan A, Kota V, Litzow M. Comparing venetoclax in combination with hypomethylating agents to hypomethylating agent-based therapies for treatment naive TP53-mutated acute myeloid leukemia: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND). Blood Cancer Journal 2024, 14: 32. PMID: 38378617, PMCID: PMC10879201, DOI: 10.1038/s41408-024-01000-2.Peer-Reviewed Original ResearchHypomethylating agents plus venetoclax compared with intensive induction chemotherapy regimens in molecularly defined secondary AML
Shimony S, Bewersdorf J, Shallis R, Liu Y, Schaefer E, Zeidan A, Goldberg A, Stein E, Marcucci G, Lindsley R, Chen E, Ramos Perez J, Stein A, DeAngelo D, Neuberg D, Stone R, Ball B, Stahl M. Hypomethylating agents plus venetoclax compared with intensive induction chemotherapy regimens in molecularly defined secondary AML. Leukemia 2024, 38: 762-768. PMID: 38378841, DOI: 10.1038/s41375-024-02175-0.Peer-Reviewed Original ResearchAssociated with improved OSHypomethylating agentsCPX-351Overall survivalSplicing factor mutationsCo-mutationsAllogeneic hematopoietic stem cell transplantationAssociated with better OSAssociated with worse OSSecondary acute myeloid leukemiaHematopoietic stem cell transplantationMedian overall survivalStem cell transplantationPatients aged >Acute myeloid leukemiaTreated with daunorubicinLiposomal daunorubicinMonosomal karyotypeNRAS/KRAS mutationsImproved OSSecondary AMLMyeloid diseasesMyeloid neoplasmsAML patientsAML treatmentClinical outcomes of hypomethylating agents plus Venetoclax as frontline treatment in patients 75 years and older with acute myeloid leukemia: Real‐world data from eight US academic centers
Abaza Y, Winer E, Murthy S, Shallis R, Matthews A, Badar T, Geramita E, Kota V, Swaroop A, Doukas P, Bradshaw D, Helenowski I, Liu Y, Zhang H, Im A, Litzow M, Perl A, Atallah E, Altman J. Clinical outcomes of hypomethylating agents plus Venetoclax as frontline treatment in patients 75 years and older with acute myeloid leukemia: Real‐world data from eight US academic centers. American Journal Of Hematology 2024, 99: 606-614. PMID: 38342997, DOI: 10.1002/ajh.27231.Peer-Reviewed Original ResearchAcute myeloid leukemiaHypomethylating agentsOverall survivalElderly patientsMedian OSMyeloid leukemiaTP53-mutated acute myeloid leukemiaNewly diagnosed acute myeloid leukemiaFailure of hypomethylating agentsMedian duration of responseDiagnosed AMLVIALE-A trialMedian overall survivalIncomplete count recoveryDuration of responseMulticenter retrospective studySingle-center studyTreatment of patientsStandard of careCR/CRi rateIntensive chemotherapyCount recoveryMedian durationFrontline treatmentRetrospective studyIntegrated genetic, epigenetic, and immune landscape of TP53 mutant AML and higher risk MDS treated with azacitidine
Zeidan A, Bewersdorf J, Hasle V, Shallis R, Thompson E, de Menezes D, Rose S, Boss I, Halene S, Haferlach T, Fox B. Integrated genetic, epigenetic, and immune landscape of TP53 mutant AML and higher risk MDS treated with azacitidine. Therapeutic Advances In Hematology 2024, 15: 20406207241257904. PMID: 38883163, PMCID: PMC11180421, DOI: 10.1177/20406207241257904.Peer-Reviewed Original ResearchHigher-risk myelodysplastic syndromesAcute myeloid leukemiaBone marrowMutation statusImmune landscapeImmunological landscapeAnti-PD-L1 antibody durvalumabHR-MDS patientsWild-type acute myeloid leukemiaTP53-mutant acute myeloid leukemiaMutant acute myeloid leukemiaAzacitidine-based therapyWild-type patientsImmune checkpoint proteinsImmune checkpoint expressionT cell populationsWild-typeStatistically significant decreaseAZA therapyImmunosuppressive microenvironmentPD-L1Mutant patientsDNA methylation arraysCheckpoint expressionMyelodysplastic syndrome
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 HSCT