Real‐life experience of luspatercept in transfusion‐dependent lower risk myelodysplastic syndrome patients
Year: 2025
Type: article
Study design: Observational Study
Abstract: Luspatercept has been approved for the treatment of anaemia in transfusion-dependent (TD) patients with lower risk (LR) myelodysplastic syndromes (MDS) after erythroid-stimulated agent (ESA) failure, according to the results of the MEDALIST trial. In this multicentre retrospective study, we report efficacy and safety data of luspatercept administered in 98 TD LR-MDS patients after ESA failure. The percentage of patients that stopped luspatercept due to adverse events was comparable to that reported in the MEDALIST study. Furthermore, we observed that 44.3% patients who had completed 24 weeks of follow-up achieved transfusion independence lasting longer than 8 weeks, compared to 38% in the MEDALIST trial. These positive results may be attributed to the inclusion of patients with lower transfusion needs in our study. All responses were observed within 8 months since luspatercept onset and many were long-lasting, even in the high-transfusion burden patient group. In addition, response to luspatercept and the presence of less than two mutations independently predicted for longer overall survival. Overall, our results confirm luspatercept's safety and efficacy in TD LR-MDS patients who have experienced ESA failure in a real-world setting.
Source: British Journal of Haematology
DOI: 10.1111/bjh.20102
Authors: Anthi Bouchla, Sotirios G. Papageorgiou, Ioannis Kotsianidis, Panagiotis Diamantopoulos, Eleni Gavriilaki, Eleni Bouronikou, Argiris Symeonidis, Panagiotis Zikos, Mustafa Cetiner, Efthymia Vlachaki, Akrivi Kostourou, Athanasios Galanopoulos, Eleftheria Hatzimichael, Elisavet‐Christina Vervesou, Sinem Civriz Bozdağ, Nora‐Athina Viniou, Dimitrios Christoulas, Maria Dellatola, Maria Papaioannou, Menelaos Papoutselis, Dimitra Vlachopoulou, Antonia Syrigou, Maria Mainou, Sofia Chatzileontiadou, Vasiliki Pappa
Institutions: National and Kapodistrian University of Athens, University General Hospital Attikon, Democritus University of Thrace, University Hospital of Alexandroupolis, Laiko General Hospital of Athens, G. Papanikolaou General Hospital, University Hospital of Larissa, General University Hospital of Patras, Agios Andreas Hospital, Acıbadem University, Aristotle University of Thessaloniki, Ippokrateio General Hospital of Thessaloniki, Pammakaristos Hospital, General Hospital of Athens G. Genimatas, University of Ioannina, Henry Dunant Hospital, Koç University, Hellenic Airforce General Hospital, Metropolitan Hospital, AHEPA University Hospital
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $5,190
OpenAlex ID: W4410104428