2023
Long-term utilization and benefit of luspatercept in transfusion-dependent, erythropoiesis-stimulating agent-refractory or -intolerant patients with lower-risk myelodysplastic syndromes with ring sideroblasts
Platzbecker U, Santini V, Komrokji R, Zeidan A, Garcia-Manero G, Buckstein R, Miteva D, Keeperman K, Holot N, Nadal J, Lai Y, Vodala S, Rosettani B, Giuseppi A, Yucel A, Fenaux P. Long-term utilization and benefit of luspatercept in transfusion-dependent, erythropoiesis-stimulating agent-refractory or -intolerant patients with lower-risk myelodysplastic syndromes with ring sideroblasts. Leukemia 2023, 37: 2314-2318. PMID: 37752285, PMCID: PMC10624606, DOI: 10.1038/s41375-023-02031-7.Peer-Reviewed Original ResearchMyelodysplastic Syndromes/Neoplasms—Insights Into Pathogenesis Leading to Improved Classification, Risk Stratification, and Treatments
Rose M, Zeidan A. Myelodysplastic Syndromes/Neoplasms—Insights Into Pathogenesis Leading to Improved Classification, Risk Stratification, and Treatments. The Cancer Journal 2023, 29: 109-110. PMID: 37195765, DOI: 10.1097/ppo.0000000000000663.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsSpectrum From Clonal Hematopoiesis to Myelodysplastic Neoplasm/Syndromes and Other Myeloid Neoplasms
Xie Z, Chen E, Mendez L, Komrokji R, Zeidan A. Spectrum From Clonal Hematopoiesis to Myelodysplastic Neoplasm/Syndromes and Other Myeloid Neoplasms. The Cancer Journal 2023, 29: 130-137. PMID: 37195768, DOI: 10.1097/ppo.0000000000000656.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsClonal hematopoiesisHigh-risk patientsRisk of progressionSuch patientsUndetermined significanceAge-related diseasesHematologic malignanciesClonal cytopeniaMyeloid neoplasmsHigh riskUnmet needMyeloid malignanciesNatural historyCH managementPatientsMalignancySignificant knowledge gapsRiskHematopoiesisCytopeniasNeoplasmsSyndrome
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 ResearchTP53‐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 ResearchInternational Working Group (IWG) Response Criteria and Association with Overall Survival in Patients with Myelodysplastic Syndromes Treated with Hypomethylating Agents - a Systematic Review and Meta-Analysis
Bewersdorf J, Grimshaw A, Platzbecker U, Fenaux P, Sekeres M, Zeidan A, Stahl M. International Working Group (IWG) Response Criteria and Association with Overall Survival in Patients with Myelodysplastic Syndromes Treated with Hypomethylating Agents - a Systematic Review and Meta-Analysis. Blood 2022, 140: 9811-9812. DOI: 10.1182/blood-2022-158622.Peer-Reviewed Original ResearchCharacterization of Patients with Lower-Risk Myelodysplastic Syndromes Experiencing Long-Term Responses with Luspatercept in the MEDALIST Study
Platzbecker U, Santini V, Komrokji R, Zeidan A, Garcia-Manero G, Buckstein R, Oliva E, Miteva D, Pozharskaya V, Ha X, Nadal J, Fenaux P. Characterization of Patients with Lower-Risk Myelodysplastic Syndromes Experiencing Long-Term Responses with Luspatercept in the MEDALIST Study. Blood 2022, 140: 9808-9810. DOI: 10.1182/blood-2022-157486.Peer-Reviewed Original ResearchTreatment Patterns and Characteristics Among Patients with Myelodysplastic Syndromes Initiating Oral Decitabine and Cedazuridine or Intravenous/Subcutaneous Hypomethylating Agents in a Real-World Setting
Zeidan A, Divino V, DeKoven M, Wang E, Chen J, Salimi T, Epstein R. Treatment Patterns and Characteristics Among Patients with Myelodysplastic Syndromes Initiating Oral Decitabine and Cedazuridine or Intravenous/Subcutaneous Hypomethylating Agents in a Real-World Setting. Blood 2022, 140: 4047-4049. DOI: 10.1182/blood-2022-157926.Peer-Reviewed Original ResearchPrimary Results of Stimulus-MDS1: A Randomized, Double-Blind, Placebo-Controlled Phase II Study of TIM-3 Inhibition with Sabatolimab Added to Hypomethylating Agents (HMAs) in Adult Patients with Higher-Risk Myelodysplastic Syndromes (MDS)
Zeidan A, Ando K, Rauzy O, Turgut M, Wang M, Cairoli R, Hou H, Kwong Y, Sangerman M, Meers S, Pullarkat V, Santini V, Malek K, Kiertsman F, Lyu J, Ramos P, Fenaux P, Miyazaki Y, Platzbecker U. Primary Results of Stimulus-MDS1: A Randomized, Double-Blind, Placebo-Controlled Phase II Study of TIM-3 Inhibition with Sabatolimab Added to Hypomethylating Agents (HMAs) in Adult Patients with Higher-Risk Myelodysplastic Syndromes (MDS). Blood 2022, 140: 2063-2065. DOI: 10.1182/blood-2022-158612.Peer-Reviewed Original ResearchDisease Characteristics and International Prognostic Scoring Systems (IPSS, IPSS-R, IPSS-M) in Adult Patients with Higher-Risk Myelodysplastic Syndromes (MDS) Participating in Two Randomized, Double-Blind, Placebo-Controlled Studies with Intravenous Sabatolimab Added to Hypomethylating Agents (HMA) (STIMULUS-MDS1 and MDS2)
Santini V, Platzbecker U, Fenaux P, Giagounidis A, Miyazaki Y, Sekeres M, Xiao Z, Sanz G, Van Hoef M, Ma F, Hertle S, Ramos P, Zeidan A. Disease Characteristics and International Prognostic Scoring Systems (IPSS, IPSS-R, IPSS-M) in Adult Patients with Higher-Risk Myelodysplastic Syndromes (MDS) Participating in Two Randomized, Double-Blind, Placebo-Controlled Studies with Intravenous Sabatolimab Added to Hypomethylating Agents (HMA) (STIMULUS-MDS1 and MDS2). Blood 2022, 140: 1340-1342. DOI: 10.1182/blood-2022-160282.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 ResearchImetelstat Achieved Prolonged, Continuous Transfusion Independence (TI) in Patients with Heavily Transfused Non-Del(5q) Lower-Risk Myelodysplastic Syndrome (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis Stimulating Agents (ESAs) within the IMerge Phase 2 Study
Platzbecker U, Komrokji R, Fenaux P, Zeidan A, Sekeres M, Savona M, Madanat Y, Santini V, Van Eygen K, Raza A, Germing U, Berry T, Dougherty S, Shah S, Sun L, Huang F, Feller F, Wan Y, Ikin A, Sherman L. Imetelstat Achieved Prolonged, Continuous Transfusion Independence (TI) in Patients with Heavily Transfused Non-Del(5q) Lower-Risk Myelodysplastic Syndrome (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis Stimulating Agents (ESAs) within the IMerge Phase 2 Study. Blood 2022, 140: 1106-1108. DOI: 10.1182/blood-2022-169050.Peer-Reviewed Original ResearchBurden of Illness in Patients with Higher-Risk Myelodysplastic Syndromes By Baseline Transfusion Status
Zeidan A, Ng C, Yellow-Duke A, Lamarre N, Cheng W, Ma E. Burden of Illness in Patients with Higher-Risk Myelodysplastic Syndromes By Baseline Transfusion Status. Blood 2022, 140: 11032-11034. DOI: 10.1182/blood-2022-157666.Peer-Reviewed Original ResearchPhase 2 Study of Oral Decitabine/Cedazuridine in Combination with Magrolimab for Previously Untreated Subjects with Intermediate to Very High-Risk Myelodysplastic Syndromes (MDS)
Zeidan A, Mosher K, Souza S, Mirakhur B, Keer H, Taylor J. Phase 2 Study of Oral Decitabine/Cedazuridine in Combination with Magrolimab for Previously Untreated Subjects with Intermediate to Very High-Risk Myelodysplastic Syndromes (MDS). Blood 2022, 140: 9779-9780. DOI: 10.1182/blood-2022-158276.Peer-Reviewed Original ResearchSafety of Deferiprone in Patients with Myelodysplastic Syndromes: Results from the Deferiprone US Safety Registry and a Compassionate Use Program
Zeidan A, Fradette C, Rozova A, Temin N, Tricta F. Safety of Deferiprone in Patients with Myelodysplastic Syndromes: Results from the Deferiprone US Safety Registry and a Compassionate Use Program. Blood 2022, 140: 1115-1117. DOI: 10.1182/blood-2022-169074.Peer-Reviewed Original ResearchMultiple Episodes of Transfusion Independence with Luspatercept Treatment and the Impact of Dose Escalation in Patients with Lower-Risk Myelodysplastic Syndromes from the MEDALIST Study
Platzbecker U, Santini V, Komrokji R, Zeidan A, Garcia-Manero G, Buckstein R, Oliva E, Pozharskaya V, Ha X, Nadal J, Miteva D, Fenaux P. Multiple Episodes of Transfusion Independence with Luspatercept Treatment and the Impact of Dose Escalation in Patients with Lower-Risk Myelodysplastic Syndromes from the MEDALIST Study. Blood 2022, 140: 6971-6973. DOI: 10.1182/blood-2022-157487.Peer-Reviewed Original ResearchPoster: MDS-476 Sabatolimab (MBG453) Combination Treatment Regimens for Patients With Higher-Risk Myelodysplastic Syndromes (HR-MDS): The Myelodysplastic Syndromes Studies in the STIMULUS Immuno-Myeloid Clinical Trial Program
Zeidan A, Al-Kali A, Borate U, Cluzeau T, DeZern A, Esteve J, Giagounidis A, Kobata K, Lyons R, Platzbecker U, Sallman D, Santini V, Sanz G, Sekeres M, Wei A, Xiao Z, Van Hoef M, Nourry-Boulot C, Sadek I, Ma F, Iordan A, Sabo J, Garcia-Manero G. Poster: MDS-476 Sabatolimab (MBG453) Combination Treatment Regimens for Patients With Higher-Risk Myelodysplastic Syndromes (HR-MDS): The Myelodysplastic Syndromes Studies in the STIMULUS Immuno-Myeloid Clinical Trial Program. Clinical Lymphoma Myeloma & Leukemia 2022, 22: s155. DOI: 10.1016/s2152-2650(22)00947-8.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 degreesCytopeniasTransplantationMalignancyLonger-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 minorityTherapyTransfusionMyelodysplasiaSideroblastsSyndromeTrialsWeeksMonths