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clinical / clinicalMDS, luspatercept MEDALIST, imetelstat, MDS clinical trials

Uwe Platzbecker

乌韦·普拉茨贝克

MD

🏢Leipzig University Hospital(莱比锡大学医院)🌐Germany

Professor and Director, Medical Clinic and Policlinic I — Hematology and Cell Therapy, Leipzig University Hospital莱比锡大学医院血液学与细胞治疗第一医学诊所主任及教授

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Key Papers
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Awards
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Key Contributions

👥Biography 个人简介

Uwe Platzbecker, MD is Professor and Director of the Medical Clinic and Policlinic I (Hematology and Cell Therapy) at Leipzig University Hospital, Germany. He is one of the foremost MDS clinical researchers in Europe and globally, best known as the principal investigator of the MEDALIST trial — the landmark phase III study (NEJM 2020) that demonstrated luspatercept (a TGF-β pathway activin receptor ligand trap) significantly improved red blood cell transfusion independence in lower-risk MDS with ring sideroblasts (SF3B1 mutation), leading to FDA and EMA approval of luspatercept in this indication. Prof. Platzbecker has been a leading voice in MDS clinical research through the European MDS cooperative group (EMSCO / AGORA) and the German MDS Study Group (GMDS), conducting pivotal trials of hypomethylating agents, lenalidomide, and transplant strategies in MDS. He has contributed extensively to the molecular IPSS (IPSS-M) risk scoring system for MDS, which incorporates 31 somatic gene mutations alongside cytogenetics to provide individualized risk prediction. He has also led pivotal studies examining imetelstat (a telomerase inhibitor) in transfusion-dependent lower-risk MDS (IMerge trial), contributing to the 2024 FDA approval of imetelstat. Prof. Platzbecker serves on the EHA scientific program committee, MDS Foundation board, and has authored over 300 peer-reviewed publications on MDS and related myeloid disorders.

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🧪Research Fields 研究领域

Luspatercept — MEDALIST Trial in Lower-Risk MDS with Ring Sideroblasts卢帕曲塞——MEDALIST试验在低危环形铁粒幼细胞MDS中的应用
Imetelstat — MDS Telomerase Inhibition in Lower-Risk Patients伊美司他——低危MDS患者的端粒酶抑制
MDS Risk Stratification — IPSS-R and Molecular IPSS (IPSS-M)MDS风险分层——IPSS-R与分子IPSS(IPSS-M)
Allogeneic Stem Cell Transplantation in MDS — RICMAC and EBMT TrialsMDS中的异基因造血干细胞移植——RICMAC与EBMT试验
Hypomethylating Agents and Novel Combinations in Higher-Risk MDS高危MDS中的低甲基化药物与新型联合方案

🎓Key Contributions 主要贡献

MEDALIST Trial — Luspatercept in Lower-Risk MDS with Ring Sideroblasts

Served as principal investigator of the phase III MEDALIST trial (NEJM 2020) demonstrating that luspatercept achieved red blood cell transfusion independence (≥8 weeks) in 38% of patients with lower-risk MDS with ring sideroblasts (SF3B1-mutated, predominantly) versus 13% with placebo (OR 5.1; p<0.001), providing the first erythroid maturation agent approval in MDS and establishing luspatercept as the preferred treatment for anemia in this MDS subtype.

IMerge Trial — Imetelstat in Transfusion-Dependent Lower-Risk MDS

Led European participation in the phase III IMerge trial of imetelstat (a telomerase inhibitor) in transfusion-dependent lower-risk MDS patients who had relapsed or were refractory to erythropoiesis-stimulating agents, demonstrating 40% transfusion independence rate versus 15% with placebo (p<0.001) and leading to the 2024 FDA approval of imetelstat (Rytelo) in this setting — the first telomerase inhibitor approved in any cancer.

IPSS-M — Molecular Prognostic Score for MDS

Co-developed and validated the Molecular International Prognostic Scoring System (IPSS-M) for MDS, a comprehensive machine-learning-derived model incorporating 31 gene mutations alongside the 5 clinical parameters of the IPSS-R, published in NEJM Evidence (2022). The IPSS-M provides individualized risk prediction for MDS patients, reclassifies approximately 46% of patients compared with IPSS-R, and has been rapidly adopted in international MDS guidelines and clinical trial stratification.

Transplant Strategies in MDS — European Trial Leadership

Led the EBMT-sponsored RICMAC trial and other European cooperative group studies defining optimal conditioning intensity, donor selection, and post-transplant MRD monitoring for allogeneic stem cell transplantation in MDS, contributing to evidence-based transplant decision algorithms in the ELN/EBMT MDS transplant guidelines that guide when and how to transplant higher-risk MDS patients.

Representative Works 代表性著作

[1]

Luspatercept in patients with lower-risk myelodysplastic syndromes (MEDALIST)

New England Journal of Medicine (2020)

Phase III MEDALIST trial demonstrating luspatercept improved transfusion independence in lower-risk MDS with ring sideroblasts, supporting FDA approval of the first erythroid maturation agent in MDS.

[2]

Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge)

New England Journal of Medicine (2023)

Phase III IMerge trial showing imetelstat achieved 40% transfusion independence in ESA-refractory lower-risk MDS, supporting FDA approval of the first telomerase inhibitor in oncology.

[3]

Molecular International Prognostic Scoring System for Myelodysplastic Syndromes (IPSS-M)

NEJM Evidence (2022)

Development and validation of the IPSS-M incorporating 31 gene mutations for individualized MDS risk prediction, reclassifying 46% of patients versus IPSS-R.

[4]

Azacitidine versus conventional care regimens in elderly patients with newly diagnosed AML with more than 30% bone marrow blasts

Journal of Clinical Oncology (2010)

Pivotal trial establishing azacitidine as an effective option for elderly AML patients with >30% blasts compared with conventional care, supporting MDS/AML treatment decision-making.

🏆Awards & Recognition 奖项与荣誉

🏆MDS Foundation Scientific Advisory Board Member
🏆European Hematology Association (EHA) Scientific Program Committee
🏆EBMT Aplastic Anemia and MDS Working Party Chair
🏆German MDS Study Group (GMDS) Steering Committee
🏆Leipzig University Hospital Department Director

📄Data Sources 数据来源

Last updated: 2026-04-06 | All information from publicly available academic sources

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