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伊沙佐米-来那度胺-地塞米松(IRd)用于复发难治性多发性骨髓瘤(RRMM)——来自德国的多中心真实世界分析及文献比较综述

Ixazomib-lenalidomid-dexamethasone (IRd) in relapsed refractory multiple myeloma (RRMM)-multicenter real-world analysis from Germany and comparative review of the literature.

作者信息

Löffler Hanna, Braun Magdalena, Reinhardt Heike, Rösner Amelie, Ihorst Gabriele, Räder Jan, Wenger Sina, Brioli Annamaria, von Lilienfeld-Toal Marie, Klaiber-Hakimi Maika, Fehr Eva-Marie, Munder Markus, Epp Katharina, Trautmann-Grill Karolin, Decker Sarah, Hoff Holger, Wäsch Ralph, Engelhardt Monika

机构信息

Department of Hematology and Oncology, Faculty of Freiburg, University of Freiburg Germany, Freiburg, Germany.

Comprehensive Cancer Center Freiburg, Freiburg, Germany.

出版信息

Ann Hematol. 2025 Jun 5. doi: 10.1007/s00277-025-06441-8.

Abstract

With the introduction of novel agents (proteasome inhibitors (PIs), immunomodulatory drugs (IMiDs), immuntherapeutics), the outcome of multiple myeloma (MM) patients has improved. The oral PI ixazomib was authorized in the EU in 11/2016 and is indicated in combination with lenalidomide and dexamethasone (IRd) for the treatment of patients with relapsed/refractory MM (RRMM). The study aim was to identify the typical real world (RW) RRMM patient population in Germany treated with IRd and to compare the outcome to prior IRd cohorts. Our retrospective study included consecutive RRMM patients receiving IRd in six large German hospitals between 06/2017 and 12/2021. We identified 24 consecutive RRMM patients within these six institutions. Their median age was 68 years at the start of IRd; 21% carrying high-risk cytogenetics [del17p, t(4;14) and/or t(14;16)]. Patients had a median of 2 prior treatment lines (range, 1-5), prior PI/IMiD exposure was 100%/58%, comparable to previous real world evidence (RWE)-studies. With a median follow-up of 37.7 months, the overall response rate was 70.8%, and median PFS and OS were 22.0 and 62.2 months, respectively. With these German data, our study is closing an important gap of IRd real world data (RWD) reporting across Europe, providing median PFS/OS data and comparison to prior IRd studies. IRd is a well-tolerated and effective triplet regimen, often given as 2nd or 3rd-line therapy in RRMM, with results confirming the registration trial and other international RW-analyses.

摘要

随着新型药物(蛋白酶体抑制剂(PIs)、免疫调节药物(IMiDs)、免疫疗法)的引入,多发性骨髓瘤(MM)患者的预后得到了改善。口服蛋白酶体抑制剂伊沙佐米于2016年11月在欧盟获批,与来那度胺和地塞米松联合(IRd)用于治疗复发/难治性MM(RRMM)患者。本研究的目的是确定在德国接受IRd治疗的典型真实世界(RW)RRMM患者群体,并将其结果与既往IRd队列进行比较。我们的回顾性研究纳入了2017年6月至2021年12月期间在德国六家大型医院接受IRd治疗的连续RRMM患者。我们在这六家机构中确定了24例连续的RRMM患者。他们开始接受IRd治疗时的中位年龄为68岁;21%携带高危细胞遗传学特征[del17p、t(4;14)和/或t(14;16)]。患者既往治疗线数的中位数为2(范围1-5),既往PI/IMiD暴露率为100%/58%,与既往真实世界证据(RWE)研究相当。中位随访37.7个月时,总缓解率为70.8%,中位无进展生存期(PFS)和总生存期(OS)分别为22.0个月和62.2个月。通过这些德国数据,我们的研究填补了欧洲IRd真实世界数据(RWD)报告的一个重要空白,提供了中位PFS/OS数据并与既往IRd研究进行了比较。IRd是一种耐受性良好且有效的三联方案,在RRMM中常作为二线或三线治疗,其结果证实了注册试验及其他国际RW分析。

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