Uwe Platzbecker
乌韦·普拉茨贝克
MD
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
👥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.
🧪Research Fields 研究领域
🎓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
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.
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.
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.
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
📄Data Sources 数据来源
Last updated: 2026-04-06 | All information from publicly available academic sources
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