2024
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)
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
Improvement of Patient-Reported Outcomes Among Heavily Pretreated Patients with Lower-Risk Myelodysplastic Syndromes and High Transfusion Burden Treated with Imetelstat on the IMerge Phase 3 Trial
Sekeres M, Diez-Campelo M, Zeidan A, Platzbecker U, Regnault A, Creel K, Sengupta N, Wan Y, Sun L, Xia Q, Berry T, Dougherty S, Shah S, Navada S, Santini V, Valcárcel D. Improvement of Patient-Reported Outcomes Among Heavily Pretreated Patients with Lower-Risk Myelodysplastic Syndromes and High Transfusion Burden Treated with Imetelstat on the IMerge Phase 3 Trial. Blood 2023, 142: 6479. DOI: 10.1182/blood-2023-181103.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesPatient-reported outcomesHigh transfusion burdenTransfusion burdenPlacebo groupMyelodysplastic syndromeTreatment groupsFunctional assessmentCancer Therapy-AnemiaPatient-reported fatigueTransfusion independence rateChronic Illness TherapyPhase 3 trialInferior overall survivalTransfusion-dependent anemiaLeast square meansImprovement of patientsPRO end pointsComposite scorePositive mean changesQuality of lifeFACT-AnTreat populationDyspnea scoreOverall survivalOral therapy for myelodysplastic syndromes/neoplasms and acute myeloid leukemia: a revolution in progress
Venugopal S, Shallis R, Zeidan A. Oral therapy for myelodysplastic syndromes/neoplasms and acute myeloid leukemia: a revolution in progress. Expert Review Of Anticancer Therapy 2023, 23: 903-911. PMID: 37470508, DOI: 10.1080/14737140.2023.2238897.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsAcute myeloid leukemiaOral therapyMyeloid leukemiaAllogeneic hematopoietic stem cell transplantationHematopoietic stem cell transplantationDisease-related complicationsDisease-directed therapyStem cell transplantationQuality of lifeCC-486HR-MDSOral azacitidineClinic visitsMost patientsGood tolerabilityIntensive therapyOptimal regimensCell transplantationTherapy combinationsTreatment optionsMedication administrationPatient outcomesMyeloid neoplasmsClinical developmentMyeloid malignanciesConsiderations for Drug Development in Myelodysplastic Syndromes
Sekeres M, Kim N, DeZern A, Norsworthy K, Garcia J, de Claro R, Theoret M, Jen E, Ehrlich L, Zeidan A, Komrokji R. Considerations for Drug Development in Myelodysplastic Syndromes. Clinical Cancer Research 2023, 29: 2573-2579. PMID: 36688922, PMCID: PMC10349686, DOI: 10.1158/1078-0432.ccr-22-3348.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsMyelodysplastic syndromeTrial designDrug developmentResponse criteriaLow-risk diseaseHigh-risk patientsFuture trial designClinical trial designQuality of lifeValidation of PatientHigh-need populationDurable responsesOverall survivalAnemic patientsTransfusion dependencyClinical benefitPatient populationAdvanced ageClinical trialsDose reductionOutcome instrumentsNew therapiesPreclinical modelingPatientsActive drug
2021
IMerge: A phase 3 study to evaluate imetelstat in transfusion-dependent subjects with IPSS low or intermediate-1 risk myelodysplastic syndromes that are relapsed/refractory to erythropoiesis-stimulating agent treatment.
Platzbecker U, Komrokji R, Fenaux P, Zeidan A, Sekeres M, Savona M, Madanat Y, Sherman L, Dougherty S, Sun L, Huang F, Wan Y, Rizo A, Berry T, Feller F, Santini V. IMerge: A phase 3 study to evaluate imetelstat in transfusion-dependent subjects with IPSS low or intermediate-1 risk myelodysplastic syndromes that are relapsed/refractory to erythropoiesis-stimulating agent treatment. Journal Of Clinical Oncology 2021, 39: tps7056-tps7056. DOI: 10.1200/jco.2021.39.15_suppl.tps7056.Peer-Reviewed Original ResearchInternational Prognostic Scoring SystemErythropoiesis-stimulating agentsPhase 3 partRisk myelodysplastic syndromesMyelodysplastic syndromeRBC-TIRed blood cell (RBC) transfusion-dependent patientsErythropoiesis-stimulating agent treatmentLower-risk myelodysplastic syndromesPhase 2 partProgression of MDSRBC transfusion independenceTransfusion dependent subjectsPlacebo-controlled trialPhase 2/3 studyPhase 3 studyPrognostic scoring systemCurrent treatment optionsTransfusion-dependent patientsQuality of lifeMedian TI durationRate of CRVariant allele frequencyMechanism of actionAdult pts
2020
Feasibility of Peri-Transfusion Quality of Life Assessment for Patients with Myelodysplastic Syndromes
Abel G, Klepin H, Magnavita E, Jaung T, Shallis R, Bahl N, Dellinger-Johnston R, Winer E, Zeidan A. Feasibility of Peri-Transfusion Quality of Life Assessment for Patients with Myelodysplastic Syndromes. Blood 2020, 136: 25-26. DOI: 10.1182/blood-2020-139919.Peer-Reviewed Original ResearchECOG performance statusQuality of lifePerformance statusRBC transfusionMyelodysplastic syndromeExact testRed blood cell transfusionPatients' QOLDana-Farber Cancer InstituteTwo-sided Fisher's exact testMore RBC transfusionsPre-transfusion HbSetting of anemiaBlood cell transfusionHalf of patientsPost-transfusion increaseFisher's exact testCell transfusionPatient characteristicsUnstable anginaMedian HbMean ageTransfusion decisionsHb thresholdQoL assessmentAML-205: Health-Related Quality of Life in Patients with Untreated Higher-Risk Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia Receiving Glasdegib + Azacitidine
Wang E, Bell T, Zeidan A, Bhattcharyya H, Kudla A, Chan G, Sekeres M. AML-205: Health-Related Quality of Life in Patients with Untreated Higher-Risk Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia Receiving Glasdegib + Azacitidine. Clinical Lymphoma Myeloma & Leukemia 2020, 20: s191. DOI: 10.1016/s2152-2650(20)30732-1.Peer-Reviewed Original ResearchHigh-risk myelodysplastic syndromeAcute myeloid leukemiaChronic myelomonocytic leukemiaPatient Global ImpressionMyelodysplastic syndromeIntensive chemotherapyPatient's impressionGlobal ImpressionAML cohortMDS cohortMyeloid leukemiaAML/myelodysplastic syndromeMD Anderson Symptom InventoryFirst-line treatment optionLower symptom burdenHRQoL of patientsOpen-label studyHealth-related qualityPatient-reported outcomesEnd of treatmentMain outcome measuresTotal symptom severityQuality of lifeTrend of improvementStudy medicationHealth-related quality of life (HRQoL) in patients with untreated higher-risk myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and chronic myelomonocytic leukemia (CMML) receiving glasdegib + azacitidine (AZA).
Wang E, Bell T, Zeidan A, Bhattacharyya H, Kudla A, Chan G, Sekeres M. Health-related quality of life (HRQoL) in patients with untreated higher-risk myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and chronic myelomonocytic leukemia (CMML) receiving glasdegib + azacitidine (AZA). Journal Of Clinical Oncology 2020, 38: 7527-7527. DOI: 10.1200/jco.2020.38.15_suppl.7527.Peer-Reviewed Original ResearchChronic myelomonocytic leukemiaAcute myeloid leukemiaMyelodysplastic syndromeIntensive chemotherapyGlobal ImpressionAML cohortAML/myelodysplastic syndromeHigh-risk myelodysplastic syndromeMD Anderson Symptom InventoryFirst-line treatment optionLower symptom burdenPhase Ib studyEnd of treatmentQuality of lifeStudy medicationRemission rateOverall survivalSymptom burdenMDS cohortTreatment optionsMyeloid leukemiaMedian numberMyelomonocytic leukemiaGlasdegibAzacitidine
2017
A call for action: Increasing enrollment of untreated patients with higher‐risk myelodysplastic syndromes in first‐line clinical trials
Zeidan AM, Stahl M, Sekeres MA, Steensma DP, Komrokji RS, Gore SD. A call for action: Increasing enrollment of untreated patients with higher‐risk myelodysplastic syndromes in first‐line clinical trials. Cancer 2017, 123: 3662-3672. PMID: 28759108, DOI: 10.1002/cncr.30903.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsHigh-risk myelodysplastic syndromeAZA-001 trialClinical trialsMinimal survival gainsLarge randomized trialsManagement of patientsPopulation-based studyClinical trial enrollmentQuality of lifeMonotherapy armMedian survivalRandomized trialsSurvival impactTrial enrollmentSurvival gainMyelodysplastic syndromeRegistry analysisAggressive malignancyPatientsNatural historyRoutine useTrialsSurvivalEnrollmentHMAsManagement of lower-risk myelodysplastic syndromes without del5q: current approach and future trends
Stahl M, Zeidan AM. Management of lower-risk myelodysplastic syndromes without del5q: current approach and future trends. Expert Review Of Hematology 2017, 10: 345-364. PMID: 28277851, DOI: 10.1080/17474086.2017.1297704.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsErythropoiesis stimulating agentsMyelodysplastic syndromeLR-MDSImmunosuppressive therapyTreatment modalitiesLower-risk myelodysplastic syndromesTGF-β pathway inhibitorCombination treatment modalitiesLR-MDS patientsPromising investigational agentsSubset of patientsAvailable therapeutic modalitiesGoals of careStandard therapeutic optionPredictors of responseCurrent treatment modalitiesAcute myeloid leukemiaQuality of lifeBone marrow failureRisk assessment toolSymptom controlBlood cytopeniasInvestigational agentsTherapeutic optionsClinical behavior
2014
Clinical utility of lenalidomide in the treatment of myelodysplastic syndromes
Zahr A, Aldin E, Komrokji RS, Zeidan AM. Clinical utility of lenalidomide in the treatment of myelodysplastic syndromes. Journal Of Blood Medicine 2014, Volume 6: 1-16. PMID: 25565910, PMCID: PMC4278786, DOI: 10.2147/jbm.s50482.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMyelodysplastic syndromeClinical trialsClinical utilityRed blood cell transfusion requirementsLower-risk myelodysplastic syndromesHigh-risk myelodysplastic syndromeActivity of lenalidomideDifferent prognostic scoresMajor clinical trialsPeripheral blood cytopeniasEfficacy of lenalidomideClonal hematopoietic disordersQuality of lifeMechanism of actionMDS managementTransfusion requirementsBlood cytopeniasPrognostic scoreClinical outcomesRisk stratificationMechanisms of resistanceIneffective hematopoiesisLenalidomideDrug lenalidomideLeukemic transformation