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来那度胺治疗复发/难治性多发性骨髓瘤患者的真实世界数据:来自匈牙利的研究。

Pomalidomide Treatment in Relapsed/Refractory Multiple Myeloma Patients-Real-World Data From Hungary.

机构信息

Department of Hematology, Institute for Medicine, Faculty of Medicine, University of Debrecen, Debrecen, Hungary.

Department of Internal Medicine and Hematology, Semmelweis University, Budapest, Hungary.

出版信息

Pathol Oncol Res. 2022 Oct 3;28:1610645. doi: 10.3389/pore.2022.1610645. eCollection 2022.

Abstract

Pomalidomide is a third generation immunomodulatory drug in the treatment of refractory and relapsed multiple myeloma patients. Our aim was to investigate the efficacy and safety of pomalidomide therapy in a real world setting. Eighty-six Hungarian patients were included, 45 of whom received pomalidomide ± an alkylating agent, while in 38 of them pomalidomide was combined with a proteasome inhibitor. 56 patients (65%) showed any response to the treatment with 18 complete or very good partial remissions and 38 partial remissions. At a median duration of follow-up of 18.6 months, the median progression-free survival (PFS) was 9.03 months, while the median overall survival (OS) was 16.53 months in the whole cohort. Patients with early stage disease (R-ISS 1 and 2) had better survival results than those with stage 3 myeloma ( = 0.002). Neither the number of prior treatment lines, nor lenalidomide refractoriness had a significant impact on PFS PFS was found similar between the cohort of patients with impaired renal function and the cohort without kidney involvement. During the study, eight mortal infections and two fatal bleeding complications occurred, however, mild hematologic and gastrointestinal toxicities were identified as the most frequent adverse events. The results of our investigations confirm that pomalidomide is an effective treatment option for relapsed/refractory MM, besides, the safety profile is satisfactory in subjects with both normal and impaired renal function.

摘要

泊马度胺是一种第三代免疫调节药物,可用于治疗难治性和复发性多发性骨髓瘤患者。我们的目的是在真实环境中研究泊马度胺治疗的疗效和安全性。共纳入 86 名匈牙利患者,其中 45 名患者接受泊马度胺±烷化剂治疗,38 名患者接受泊马度胺联合蛋白酶体抑制剂治疗。56 名患者(65%)对治疗有任何反应,其中 18 名完全缓解或非常好的部分缓解,38 名部分缓解。在中位随访时间为 18.6 个月时,全队列的中位无进展生存期(PFS)为 9.03 个月,中位总生存期(OS)为 16.53 个月。疾病早期(R-ISS 1 和 2)的患者比 3 期骨髓瘤患者有更好的生存结果( = 0.002)。先前治疗线的数量和来那度胺耐药性均未对 PFS 产生显著影响。在肾功能受损的患者组和无肾脏受累的患者组之间,PFS 相似。研究期间,发生了 8 例致命感染和 2 例致命出血并发症,但轻度血液学和胃肠道毒性被认为是最常见的不良事件。我们的研究结果证实,泊马度胺是复发性/难治性多发性骨髓瘤的有效治疗选择,此外,在肾功能正常和受损的患者中,安全性特征令人满意。

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