Bouchla Anthi, Papageorgiou Sotirios G, Kotsianidis Ioannis, Diamantopoulos Panagiotis, Gavriilaki Eleni, Bouronikou Eleni, Symeonidis Argiris, Zikos Panagiotis, Cetiner Mustafa, Vlachaki Efthymia, Kostourou Akrivi, Galanopoulos Athanasios, Hatzimichael Eleftheria, Vervesou Elisavet-Christina, Bozdağ Sinem Civriz, Viniou Nora-Athina, Christoulas Dimitrios, Dellatola Maria, Papaioannou Maria, Papoutselis Menelaos, Vlachopoulou Dimitra, Syrigou Antonia, Mainou Maria, Chatzileontiadou Sofia, Pappa Vasiliki
2nd Department of Internal Medicine and Research Institute, Hematology Unit, Attikon University Hospital, National and Kapodistrian University of Athens, Athens, Greece.
Department of Hematology, University Hospital of Alexandroupolis, Democritus University of Thrace Medical School, Alexandroupolis, Greece.
Br J Haematol. 2025 Jul;207(1):101-109. doi: 10.1111/bjh.20102. Epub 2025 May 5.
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.
根据MEDALIST试验结果，罗特西普已被批准用于治疗红细胞生成刺激剂（ESA）治疗失败后的低危（LR）骨髓增生异常综合征（MDS）输血依赖（TD）患者的贫血。在这项多中心回顾性研究中，我们报告了98例ESA治疗失败后的TD LR-MDS患者使用罗特西普的疗效和安全性数据。因不良事件而停用罗特西普的患者百分比与MEDALIST研究报告的百分比相当。此外，我们观察到，在完成24周随访的患者中，44.3%实现了持续超过8周的输血独立，而在MEDALIST试验中这一比例为38%。这些积极结果可能归因于我们的研究纳入了输血需求较低的患者。所有反应均在罗特西普开始使用后的8个月内观察到，并且许多反应是持久的，即使在高输血负担患者组中也是如此。此外，对罗特西普的反应以及少于两个突变的存在独立预测了更长的总生存期。总体而言，我们的结果证实了罗特西普在现实环境中经历ESA治疗失败的TD LR-MDS患者中的安全性和有效性。
