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
Updates 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 needs
2022
TP53‐altered acute myeloid leukemia and myelodysplastic syndrome with excess blasts should be approached as a single entity
Shallis R, Daver N, Altman J, Hasserjian R, Kantarjian H, Platzbecker U, Santini V, Wei A, Sallman D, Zeidan A. TP53‐altered acute myeloid leukemia and myelodysplastic syndrome with excess blasts should be approached as a single entity. Cancer 2022, 129: 175-180. PMID: 36397669, DOI: 10.1002/cncr.34535.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsA Multi-Center Phase Ib Trial of the Histone Deactylase Inhibitor (HDACi) Entinostat in Combination with Anti-PD1 Antibody Pembrolizumab in Patients with Refractory/Relapsed Myelodysplastic Syndromes (RR-MDS) or Oligoblastic Acute Myeloid Leukemia (RR-AML) after Hypomethylating Agent (HMA) Failure
Bewersdorf J, Shallis R, Sharon E, Caldwell A, Wei W, Yacoub A, Madanat Y, Zeidner J, Altman J, Odenike O, Yerrabothala S, Kovacsovics T, Podoltsev N, Halene S, Little R, Piekarz R, Gore S, Kim T, Zeidan A. A Multi-Center Phase Ib Trial of the Histone Deactylase Inhibitor (HDACi) Entinostat in Combination with Anti-PD1 Antibody Pembrolizumab in Patients with Refractory/Relapsed Myelodysplastic Syndromes (RR-MDS) or Oligoblastic Acute Myeloid Leukemia (RR-AML) after Hypomethylating Agent (HMA) Failure. Blood 2022, 140: 9084-9086. DOI: 10.1182/blood-2022-158626.Peer-Reviewed Original ResearchOverall Survival and Progression-Free Survival of Patients Following Luspatercept Treatment in the MEDALIST Trial
Santini V, Fenaux P, Zeidan A, Komrokji R, Buckstein R, Oliva E, Ha X, Miteva D, Yucel A, Nadal J, Platzbecker U. Overall Survival and Progression-Free Survival of Patients Following Luspatercept Treatment in the MEDALIST Trial. Blood 2022, 140: 4079-4081. DOI: 10.1182/blood-2022-157489.Peer-Reviewed Original ResearchStimulus MDS-US Trial in Progress: Evaluating Sabatolimab in Combination with Hypomethylating Agents (HMAs) in Patients with Intermediate-, High-, or Very High-Risk Myelodysplastic Syndrome (MDS)
Zeidan A, DeZern A, Borate U, Kobata K, Ide S, Sabo J, Ramos P, Sun H, Lyons R, Garcia-Manero G. Stimulus MDS-US Trial in Progress: Evaluating Sabatolimab in Combination with Hypomethylating Agents (HMAs) in Patients with Intermediate-, High-, or Very High-Risk Myelodysplastic Syndrome (MDS). Blood 2022, 140: 4069-4070. DOI: 10.1182/blood-2022-167259.Peer-Reviewed Original ResearchHow Many Stratification Factors Are Reasonable and Can There be Too Many? Evaluation of Analysis Strategies in the Context of a Randomized Phase 2 Trial in Acute Myeloid Leukemia within the Myelomatch Platform
Moseley A, Freidlin B, Shallis R, Zeidan A, Sallman D, Little R, Erba H, Leblanc M, Othus M. How Many Stratification Factors Are Reasonable and Can There be Too Many? Evaluation of Analysis Strategies in the Context of a Randomized Phase 2 Trial in Acute Myeloid Leukemia within the Myelomatch Platform. Blood 2022, 140: 8932-8933. DOI: 10.1182/blood-2022-170376.Peer-Reviewed Original ResearchUmbrella Trial in Myeloid Malignancies: The Myelomatch National Clinical Trials Network Precision Medicine Initiative
Little R, Othus M, Assouline S, Ansher S, Atallah E, Lindsley R, Freidlin B, Gore S, Harris L, Hourigan C, Ivy S, Jiwani S, Langan E, Luger S, Michaelis L, Odenike O, Patton D, Perales M, Radich J, Ramineni B, Salk J, Stiff P, Stock W, Stone R, Uy G, Williams P, Wood B, Worthington K, Yee L, Zeidan A, Zhang J, Litzow M, Erba H. Umbrella Trial in Myeloid Malignancies: The Myelomatch National Clinical Trials Network Precision Medicine Initiative. Blood 2022, 140: 9057-9060. DOI: 10.1182/blood-2022-169307.Peer-Reviewed Original ResearchCHIPing away the progression potential of CHIP: A new reality in the making
Xie Z, Zeidan AM. CHIPing away the progression potential of CHIP: A new reality in the making. Blood Reviews 2022, 58: 101001. PMID: 35989137, DOI: 10.1016/j.blre.2022.101001.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsClonal hematopoiesisEffective preventive strategiesEvidence-based recommendationsClinical sequelaeInterventional trialsClinical outcomesClinical trialsPreventive strategiesInterventional strategiesProgression potentialClinical implicationsIndeterminate potentialTrialsMolecular mechanismsLatest updatesHematopoiesisUrgent needImmune Checkpoint Inhibition in Acute Myeloid Leukemia and Myelodysplastic Syndromes
Abaza Y, Zeidan AM. Immune Checkpoint Inhibition in Acute Myeloid Leukemia and Myelodysplastic Syndromes. Cells 2022, 11: 2249. PMID: 35883692, PMCID: PMC9318025, DOI: 10.3390/cells11142249.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsAcute myeloid leukemiaImmune checkpoint inhibitorsHigh-risk myelodysplastic syndromeImmune checkpoint inhibitionMyelodysplastic syndromeCheckpoint inhibitionMyeloid leukemiaInnate immune checkpointPD-1 inhibitorsEarly clinical trialsEarly phase trialsLow mutational burdenCheckpoint inhibitorsDurable responsesImmune checkpointsRandomized trialsCTLA-4Clinical trialsImmune escapePhase trialsMutational burdenSolid tumorsMyeloid malignanciesTrialsLack of activityLonger-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 minorityTherapyTransfusionMyelodysplasiaSideroblastsSyndromeTrialsWeeksMonthsA phase I/II multicenter, open-label, dose escalation and randomized trial of BI 836858 in patients with low- or intermediate-1-risk myelodysplastic syndrome.
Komrokji RS, Carraway HE, Germing U, Wermke M, Zeidan AM, Fu E, Rüter B, Burkard U, Osswald A, Foran JM. A phase I/II multicenter, open-label, dose escalation and randomized trial of BI 836858 in patients with low- or intermediate-1-risk myelodysplastic syndrome. Haematologica 2022, 107: 2742-2747. PMID: 35734924, PMCID: PMC9614517, DOI: 10.3324/haematol.2021.280500.Peer-Reviewed Original ResearchP778: LONG-TERM UTILIZATION AND BENEFIT OF LUSPATERCEPT IN PATIENTS (PTS) WITH LOWER-RISK MYELODYSPLASTIC SYNDROMES (LR-MDS) FROM THE MEDALIST TRIAL
Fenaux P, Santini V, Komrokji R, Zeidan A, Garcia‐Manero G, Buckstein R, Miteva D, Keeperman K, Holot N, Zhang J, Nadal J, Rosettani B, Yucel A, Platzbecker U. P778: LONG-TERM UTILIZATION AND BENEFIT OF LUSPATERCEPT IN PATIENTS (PTS) WITH LOWER-RISK MYELODYSPLASTIC SYNDROMES (LR-MDS) FROM THE MEDALIST TRIAL. HemaSphere 2022, 6: 673-674. DOI: 10.1097/01.hs9.0000845996.15086.2d.Peer-Reviewed Original ResearchNeutrophil and platelet increases with luspatercept in lower-risk MDS: secondary endpoints from the MEDALIST trial
Garcia-Manero G, Mufti GJ, Fenaux P, Buckstein R, Santini V, Díez-Campelo M, Finelli C, Ilhan O, Sekeres MA, Zeidan AM, Ito R, Zhang J, Rampersad A, Sinsimer D, Backstrom JT, Platzbecker U, Komrokji RS. Neutrophil and platelet increases with luspatercept in lower-risk MDS: secondary endpoints from the MEDALIST trial. Blood 2022, 139: 624-629. PMID: 34758066, PMCID: PMC8796653, DOI: 10.1182/blood.2021012589.Peer-Reviewed Original Research
2021
Gilteritinib vs salvage chemotherapy in FLT3-mutated acute myeloid leukemia: number needed to treat for clinical outcomes per a secondary analysis of the ADMIRAL trial
Zeidan AM, Qi CZ, Yang H, Garnham A, Shah MV, Pandya BJ. Gilteritinib vs salvage chemotherapy in FLT3-mutated acute myeloid leukemia: number needed to treat for clinical outcomes per a secondary analysis of the ADMIRAL trial. Leukemia & Lymphoma 2021, 63: 762-764. PMID: 34749571, DOI: 10.1080/10428194.2021.2001467.Peer-Reviewed Original ResearchPoster: MDS-364: STIMULUS MDS-US Trial in Progress: Evaluating Sabatolimab in Combination with Hypomethylating Agents (HMAs) in Patients with Intermediate-, High-, or Very High–Risk Myelodysplastic Syndromes (MDS)
Zeidan A, DeZern A, Borate U, Kobata K, Sadek I, Sabo J, Purkayastha D, Ramos P, Sun H, Lyons R, Garcia-Manero G. Poster: MDS-364: STIMULUS MDS-US Trial in Progress: Evaluating Sabatolimab in Combination with Hypomethylating Agents (HMAs) in Patients with Intermediate-, High-, or Very High–Risk Myelodysplastic Syndromes (MDS). Clinical Lymphoma Myeloma & Leukemia 2021, 21: s230. DOI: 10.1016/s2152-2650(21)01452-x.Peer-Reviewed Original Research
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 useTrialsSurvivalEnrollmentHMAs64 A Phase I Trial of Ipilimumab (IPI) in Patients (PTS) with Myelodysplastic Syndromes (MDS) after Hypomethylating Agent (HMAS) Failure
Zeidan A, Knaus H, Robinson T, Zeidner J, Blackford A, Duffield A, Rizzieri D, Frattini M, Levy M, Schroeder M, Ferguson A, Sheldon K, Dezern A, Gojo I, Gore S, Streicher H, Luznik L, Smith B. 64 A Phase I Trial of Ipilimumab (IPI) in Patients (PTS) with Myelodysplastic Syndromes (MDS) after Hypomethylating Agent (HMAS) Failure. Leukemia Research 2017, 55: s43. DOI: 10.1016/s0145-2126(17)30177-7.Peer-Reviewed Original ResearchHypomethylating agents in combination with histone deacetylase inhibitors in higher risk myelodysplastic syndromes: Is there a light at the end of the tunnel?
Stahl M, Zeidan AM. Hypomethylating agents in combination with histone deacetylase inhibitors in higher risk myelodysplastic syndromes: Is there a light at the end of the tunnel? Cancer 2017, 123: 911-914. PMID: 28094843, DOI: 10.1002/cncr.30532.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus Statements