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 processTrialsTherapy
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 survivalPatient-Reported Outcomes (PRO) of Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent (ESA)-Naïve, Transfusion-Dependent (TD), Lower-Risk Myelodysplastic Syndromes (LR-MDS): Results from the Phase 3 COMMANDS Study
Oliva E, Platzbecker U, Della Porta M, Garcia-Manero G, Santini V, Fenaux P, Shortt J, Komrokji R, Pelligra C, Guo S, Lord-Bessen J, Xiao H, Yucel A, Miteva D, Rose S, Kreitz S, Sekeres M, Zeidan A. Patient-Reported Outcomes (PRO) of Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent (ESA)-Naïve, Transfusion-Dependent (TD), Lower-Risk Myelodysplastic Syndromes (LR-MDS): Results from the Phase 3 COMMANDS Study. Blood 2023, 142: 4596. DOI: 10.1182/blood-2023-174660.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesEORTC QLQ-C30Patient-reported outcomesErythropoietin-stimulating agentsEpoetin alfaQLQ-C30FACT-AnSustained improvementTreatment armsESA-naïveTransfusion-DependentTreatment tolerabilityStratified Cox proportional hazards regression modelDay 1Cox proportional hazards regression modelCancer Therapy-Anemia (FACT-An) questionnaireLife Questionnaire Core 30Proportional hazards regression modelsMajority of patientsTreatment side effectsPost-baseline visitTransfusion-dependent patientsErythropoiesis stimulating agentsHazards regression modelsPhase 3
2020
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
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 medication