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 treatmentMDS-772 Time Toxicity for Patients Receiving Oral Versus Parenteral Hypomethylating Agents for Myelodysplastic Syndromes/Neoplasms
Zeidan A, Olopoenia A, Costantino H, Modi K, Salimi T, Washington T, Krenitsky J, Epstein R. MDS-772 Time Toxicity for Patients Receiving Oral Versus Parenteral Hypomethylating Agents for Myelodysplastic Syndromes/Neoplasms. Clinical Lymphoma Myeloma & Leukemia 2024, 24: s406. DOI: 10.1016/s2152-2650(24)01382-x.Peer-Reviewed Original ResearchHMA therapyRetrospective analysis of adult patientsAnalysis of adult patientsRoute of administrationMedian life expectancyPropensity score matchingHypomethylating agentsMDS treatmentBurden of treatmentAdult patientsPatient cohortInfusion dayParenteral treatmentMatched cohortResults PatientsRetrospective analysisOlder patientsEmergency room visitsCancer therapyHMA treatmentParenteral administrationTherapyPatientsOutpatient settingScore matchingTime toxicity for patients receiving oral versus parenteral hypomethylating agents for myelodysplastic syndromes/neoplasms (MDS).
Epstein R, Zeidan A, Olopoenia A, Costantino H, Modi K, Salimi T, Washington T, Krenitsky J. Time toxicity for patients receiving oral versus parenteral hypomethylating agents for myelodysplastic syndromes/neoplasms (MDS). Journal Of Clinical Oncology 2024, 42: 6568-6568. DOI: 10.1200/jco.2024.42.16_suppl.6568.Peer-Reviewed Original ResearchHMA therapyHypomethylating agentsRetrospective analysis of adult patientsAnalysis of adult patientsEmergency roomRoute of administrationTime burdenMedian life expectancyPropensity score matchingMDS treatmentBurden of treatmentAdult patientsParenteral treatmentPatient cohortInfusion dayRetrospective analysisCancer therapyPatientsTherapyOutpatient settingScore matchingTreatment groupsCohortHealthcare daysOutpatient visitsToward a more patient‐centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS)
Efficace F, Buckstein R, Abel G, Giesinger J, Fenaux P, Bewersdorf J, Brunner A, Bejar R, Borate U, DeZern A, Greenberg P, Roboz G, Savona M, Sparano F, Boultwood J, Komrokji R, Sallman D, Xie Z, Sanz G, Carraway H, Taylor J, Nimer S, Della Porta M, Santini V, Stahl M, Platzbecker U, Sekeres M, Zeidan A. Toward a more patient‐centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS). HemaSphere 2024, 8: e69. PMID: 38774655, PMCID: PMC11106800, DOI: 10.1002/hem3.69.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsPatient-reported outcomesPatient-reported outcome dataHealth-related qualityPatient-reported outcome endpointsHealth-related quality of lifeClinical trialsImprove patients' health-related qualityPatients' health-related qualityPatient-reported outcome itemsQuality of lifeTime-to-event analysisRandomized controlled trialsTreatment decision-makingPatient carePatient populationTreatment advancesControlled trialsPatientsIncurable diseaseAssessment strategiesHRQoLBenefit/risk assessmentDrug development processTrialsTherapyPatients’ perspectives on oral decitabine/cedazuridine for the treatment of myelodysplastic syndromes/neoplasms
Zeidan A, Perepezko K, Salimi T, Washington T, Epstein R. Patients’ perspectives on oral decitabine/cedazuridine for the treatment of myelodysplastic syndromes/neoplasms. Therapeutic Advances In Hematology 2024, 15: 20406207241257313. PMID: 39091323, PMCID: PMC11292726, DOI: 10.1177/20406207241257313.Peer-Reviewed Original ResearchHypomethylating agentsDEC-CQuality of lifeHypomethylating agent therapyGuideline-recommended treatmentHMA therapyOral therapySurvey of patientsTreatment side effectsAdult patientsImprove quality of lifeUS patientsSide effectsPatientsTreatment administrationTherapyDecitabine/cedazuridineTreatmentDaily activitiesMonthsLittle/no impact
2023
Are We Ready For “Triplet” Therapy in Higher-Risk MDS?
Brunner A, Platzbecker U, DeZern A, Zeidan A. Are We Ready For “Triplet” Therapy in Higher-Risk MDS? Clinical Hematology International 2023, 5: 23-32. PMID: 37933301, PMCID: PMC10625655, DOI: 10.46989/001c.88301.Peer-Reviewed Original ResearchAcute myeloid leukemiaCombination chemotherapyPatient populationOnly disease-modifying therapyNovel combination chemotherapyOngoing therapeutic challengeOlder patient populationUnique patient populationDisease-modifying therapiesCurrent treatment paradigmsTherapeutic challengeTreatment paradigmEffective therapyMyeloid leukemiaNew therapiesAppropriate endpointsTherapyChemotherapyComorbiditiesPatientsTransplantNeoplasmsLeukemiaPopulationDecitabineUpdates on risk stratification and management of lower-risk myelodysplastic syndromes/neoplasms
Badar T, Madanat Y, Zeidan A. Updates on risk stratification and management of lower-risk myelodysplastic syndromes/neoplasms. Future Oncology 2023, 19: 1877-1889. PMID: 37750305, DOI: 10.2217/fon-2023-0454.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsErythropoiesis-stimulating agentsTreatment-naive patientsHigh-risk patientsMeaningful efficacyIndolent courseDismal prognosisRandomized trialsRisk stratificationNeoplasm patientsSerial monitoringClinical trialsNovel therapiesPatientsAppropriate managementModest responseGood responseNeoplasmsTrialsNovel compoundsLuspaterceptLenalidomidePrognosisAnemiaTherapyMore needsCurrent 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 landscapePathogenesisTherapyDiseaseAn overview of novel therapies in advanced clinical testing for acute myeloid leukemia
Venugopal S, Xie Z, Zeidan A. An overview of novel therapies in advanced clinical testing for acute myeloid leukemia. Expert Review Of Hematology 2023, 16: 109-119. PMID: 36718500, DOI: 10.1080/17474086.2023.2174521.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsAML therapyMolecular pathogenesisApproval of midostaurinPathophysiology of AMLMeasurable residual diseaseAdvanced clinical testingClinical trial developmentAcute myeloid leukemiaCell surface antigensDevelopment of agentsDisease relapseImmune environmentAML treatmentResidual diseaseTherapeutic armamentariumMyeloid leukemiaNovel therapiesSurface antigenClinical testingTrial developmentTherapyAMLEscape mechanismsGene mutationsRelapse
2022
Prognostic implications of mono-hit and multi-hit TP53 alterations in patients with acute myeloid leukemia and higher risk myelodysplastic syndromes treated with azacitidine-based therapy
Zeidan A, Bewersdorf J, Hasle V, Shallis R, Thompson E, de Menezes D, Rose S, Boss I, Halene S, Haferlach T, Fox B. Prognostic implications of mono-hit and multi-hit TP53 alterations in patients with acute myeloid leukemia and higher risk myelodysplastic syndromes treated with azacitidine-based therapy. Leukemia 2022, 37: 240-243. PMID: 36437356, DOI: 10.1038/s41375-022-01766-z.Peer-Reviewed Original ResearchThe Characteristics and Prognosis of Patients with Clonal Cytopenias of Undetermined Significance, Including Cancer and Therapy-Related Clonal Cytopenias
Xie Z, Smith A, Komrokji R, Al Ali N, Patel A, Saygin C, Zeidan A, Bewersdorf J, Kishtagari A, Zeidner J, Coombs C, Madanat Y, Foran J, Badar T, Desai P, Tsai C, Griffiths E, Al Malki M, Amanam I, Lai C, Deeg J, Ades L, Yi C, Osman A, Dinner S, Abaza Y, Chandhok N, Soong D, Taylor J, Brunner A, Carraway H, Singh A, Geyer S, Padron E, Patnaik M, Savona M, Al-Kali A. The Characteristics and Prognosis of Patients with Clonal Cytopenias of Undetermined Significance, Including Cancer and Therapy-Related Clonal Cytopenias. Blood 2022, 140: 2887-2890. DOI: 10.1182/blood-2022-162367.Peer-Reviewed Original ResearchEvaluating complete remission with partial hematologic recovery (CRh) as a response criterion in myelodysplastic syndromes (MDS)
Brunner A, Gavralidis A, Ali N, Hunter A, Komrokji R, Zeidan A, Sallman D. Evaluating complete remission with partial hematologic recovery (CRh) as a response criterion in myelodysplastic syndromes (MDS). Blood Cancer Journal 2022, 12: 153. PMID: 36379923, PMCID: PMC9666661, DOI: 10.1038/s41408-022-00748-9.Peer-Reviewed Original ResearchConceptsPartial hematologic recoveryMyelodysplastic syndromeHematologic recoveryResponse criteriaCR/CRhIWG 2006 criteriaDuration of therapyBest overall responseTime of therapyCR responseCRH responseDNMTi therapyOS associationComplete remissionMedian OSOverall survivalAdult patientsAllogeneic transplantsMedian ageMDS patientsMultivariable analysisClinical trialsSimilar survivalPatientsTherapyVenetoclax-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 dataVenetoclaxInhibitorsPoster: AML-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. Poster: 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: s128. DOI: 10.1016/s2152-2650(22)00761-3.Peer-Reviewed Original ResearchTreatment of myelodysplastic syndromes in the era of precision medicine and immunomodulatory drugs: a focus on higher-risk disease
Mohty R, Al Hamed R, Bazarbachi A, Brissot E, Nagler A, Zeidan A, Mohty M. Treatment of myelodysplastic syndromes in the era of precision medicine and immunomodulatory drugs: a focus on higher-risk disease. Journal Of Hematology & Oncology 2022, 15: 124. PMID: 36045390, PMCID: PMC9429775, DOI: 10.1186/s13045-022-01346-9.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsHigh-risk diseaseMyelodysplastic syndromeAcute myeloid leukemiaHeterogeneous clonal diseaseHematopoietic cell transplantationRisk of progressionDifferent hematologic malignanciesImmune therapyImmunomodulatory drugsCell transplantationHematologic malignanciesMyeloid leukemiaNovel therapiesMyeloid neoplasmsTherapeutic approachesClonal diseaseDisease pathophysiologyDiseasePrecision medicineSyndromeTherapyVariable degreesCytopeniasTransplantationMalignancyEfficacy 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 inhibitorsVenetoclaxTherapyLonger-term benefit of luspatercept in transfusion-dependent lower-risk myelodysplastic syndromes with ring sideroblasts
Zeidan AM, Platzbecker U, Garcia-Manero G, Sekeres MA, Fenaux P, DeZern AE, Greenberg PL, Savona MR, Jurcic JG, Verma A, Mufti G, Buckstein R, Santini V, Shetty JK, Ito R, Zhang J, Zhang G, Ha X, Backstrom JT, Komrokji RS. Longer-term benefit of luspatercept in transfusion-dependent lower-risk myelodysplastic syndromes with ring sideroblasts. Blood 2022, 140: 2170-2174. PMID: 35797468, PMCID: PMC10653038, DOI: 10.1182/blood.2022016171.Peer-Reviewed Original ResearchConceptsLower-risk myelodysplastic syndromesLow-risk myelodysplasiaErythropoiesis-stimulating agentsEligible patientsTransfusion independenceMyelodysplastic syndromeLong-term benefitsLuspaterceptMedian durabilityRing sideroblastsPatientsIncremental benefitPlaceboSignificant minorityTherapyTransfusionMyelodysplasiaSideroblastsSyndromeTrialsWeeksMonthsP570: REAL-WORLD EFFICACY OUTCOMES OF VENETOCLAX PLUS AZACITIDINE VS INTENSIVE CHEMOTHERAPY FOR INDUCTION THERAPY IN ADULT PATIENTS WITH ACUTE MYELOID LEUKEMIA
Zeidan A, Pollyea D, Borate U, Vasconcelos A, Potluri R, Rotter D, Kiendrebeogo Z, Gaugler L, Bonifacio G, Prebet T, Chen C. P570: REAL-WORLD EFFICACY OUTCOMES OF VENETOCLAX PLUS AZACITIDINE VS INTENSIVE CHEMOTHERAPY FOR INDUCTION THERAPY IN ADULT PATIENTS WITH ACUTE MYELOID LEUKEMIA. HemaSphere 2022, 6: 469-470. DOI: 10.1097/01.hs9.0000845168.01159.c0.Peer-Reviewed Original Research
2018
Immunosuppressive therapy in myelodysplastic syndromes: a borrowed therapy in search of the right place
Shallis RM, Chokr N, Stahl M, Pine AB, Zeidan AM. Immunosuppressive therapy in myelodysplastic syndromes: a borrowed therapy in search of the right place. Expert Review Of Hematology 2018, 11: 715-726. PMID: 30024293, DOI: 10.1080/17474086.2018.1503049.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsImmunosuppressive therapyMyelodysplastic syndromeImmune pathwaysManagement of MDSTreatment of MDSClonal hematopoietic stem cell disordersMeaningful clinical activityAdaptive immune pathwaysHematopoietic stem cell disordersStem cell disordersImmune dysregulationDisease coursePeripheral cytopeniasClinical benefitImmune activationTherapeutic optionsAplastic anemiaClinical activityHematologic diseasesCell disordersClinical experienceContinued clarificationLeukemic progressionTherapyPatientsCounseling patients with higher-risk MDS regarding survival with azacitidine therapy: are we using realistic estimates?
Zeidan AM, Stahl M, DeVeaux M, Giri S, Huntington S, Podoltsev N, Wang R, Ma X, Davidoff AJ, Gore SD. Counseling patients with higher-risk MDS regarding survival with azacitidine therapy: are we using realistic estimates? Blood Cancer Journal 2018, 8: 55. PMID: 29891916, PMCID: PMC5995881, DOI: 10.1038/s41408-018-0081-8.Peer-Reviewed Original Research