Real‐life experience of luspatercept in transfusion‐dependent lower risk myelodysplastic syndrome patients
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.
Authors
- Vasiliki Pappa (ORCID: https://orcid.org/0000-0003-0421-9424)
- Maria Dellatola
- Ιoannis Kotsianidis (ORCID: https://orcid.org/0000-0001-7668-0186)
- Eleni Gavriilaki (ORCID: https://orcid.org/0000-0002-8883-8208)
- Athanasios Galanopoulos (ORCID: https://orcid.org/0000-0002-4216-3364)
- Argiris Symeonidis (ORCID: https://orcid.org/0000-0002-3685-3473)
- Nora‐Athina Viniou (ORCID: https://orcid.org/0000-0002-2287-4150)
- Menelaos Papoutselis (ORCID: https://orcid.org/0000-0002-1686-4992)
- Panagiotis Diamantopoulos (ORCID: https://orcid.org/0000-0003-2692-5944)
- Sotirios G. Papageorgiou (ORCID: https://orcid.org/0000-0002-3376-837X)
- Sinem Civriz Bozdağ (ORCID: https://orcid.org/0000-0001-8359-7794)
- Eleftheria Hatzimichael (ORCID: https://orcid.org/0000-0002-4408-5646)
- Antonia Syrigou
- Maria Mainou (ORCID: https://orcid.org/0000-0002-5775-2241)
- Anthi Bouchla (ORCID: https://orcid.org/0000-0002-9234-7073)
- Panagiotis Zikos (ORCID: https://orcid.org/0000-0001-5463-1105)
- Sofia Chatzileontiadou
- Elisavet‐Christina Vervesou
- Eleni Bouronikou
- Dimitra Vlachopoulou (ORCID: https://orcid.org/0000-0001-9537-9591)
- Akrivi Kostourou
- Efthymia Vlachaki (ORCID: https://orcid.org/0000-0002-2043-9630)
- Mustafa Çeti̇ner (ORCID: https://orcid.org/0000-0002-4420-6452)
- Maria Papaioannou (ORCID: https://orcid.org/0000-0002-1999-9622)
- Dimitrios Christoulas
Institutions
- Koç University (TR)
- Democritus University of Thrace (GR)
- University of Ioannina (GR)
- National and Kapodistrian University of Athens (GR)
- Aristotle University of Thessaloniki (GR)
- Agios Andreas Hospital (GR)
- AHEPA University Hospital (GR)
- General University Hospital of Patras (GR)
- Acıbadem University (TR)
- Hellenic Airforce General Hospital (GR)
- University Hospital of Larissa (GR)
- Laiko General Hospital of Athens (GR)
- Athens Medical Center (GR)
- Metropolitan Hospital (GR)
- Pammakaristos Hospital (GR)
- Henry Dunant Hospital (GR)
- G. Papanikolaou General Hospital (GR)
- University Hospital of Alexandroupolis (GR)
Publication Details
- Journal
- British Journal of Haematology
- Published
- 2025-05-05
- DOI
- https://doi.org/10.1111/bjh.20102
- Citations
- 2
- Primary Topic
- Acute Myeloid Leukemia Research
- Type
- article
- Field-Weighted Citation Impact
- 3.54