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利用韩国全国性观察性数据对复发/难治性多发性骨髓瘤患者进行卡非佐米安全性的目标试验模拟。

Target trial emulation of carfilzomib safety among patients with relapsed/refractory multiple myeloma using a nationwide observational data in Korea.

机构信息

College of Pharmacy, Seoul National University, Seoul, Republic of Korea.

College of Pharmacy, Gachon University, Incheon, Republic of Korea.

出版信息

J Cancer Res Clin Oncol. 2024 May 20;150(5):266. doi: 10.1007/s00432-024-05800-8.

Abstract

PURPOSE

Carfilzomib, commonly used for relapsed/refractory multiple myeloma (RRMM), has been associated with various adverse events in randomized controlled trials (RCTs). However, real-world safety data for a more diverse population are needed, as carfilzomib received expedited approval. This study aimed to evaluate carfilzomib's safety in Korea by comparing new users of KRd (carfilzomib, lenalidomide, and dexamethasone) to Rd (lenalidomide and dexamethasone) using a nationwide administrative claims database.

METHODS

The retrospective cohort study utilized target trial emulation, focusing on adverse events in various organ systems similar to the ASPIRE trial.

RESULTS

This study included 4,580 RRMM patients between 2007 and 2020, and the KRd group showed significantly higher risks of hematologic adverse events (anemia, neutropenia, thrombocytopenia) and some non-hematologic adverse events (cough, hypokalemia, constipation, hypertension, heart failure) compared to the Rd group. Among non-hematologic adverse events, cardiovascular events (heart failure [HR 2.04; 95% CI 1.24-3.35], hypertension [HR 1.58; 95% CI 1.15-2.17]) had the highest risk in the KRd group.

CONCLUSION

The safety profile of carfilzomib in Korean patients was similar to previous RCTs. Therefore, caution should be exercised when using carfilzomib in Asian individuals with RRMM due to the increased risk of cardiovascular adverse events.

摘要

目的

卡非佐米常用于治疗复发/难治性多发性骨髓瘤(RRMM),在随机对照试验(RCT)中与各种不良事件相关。然而,由于卡非佐米获得了加速批准,因此需要更多样化人群的真实世界安全性数据。本研究旨在通过使用全国性行政索赔数据库,将 KRd(卡非佐米、来那度胺和地塞米松)的新使用者与 Rd(来那度胺和地塞米松)进行比较,来评估卡非佐米在韩国的安全性。

方法

回顾性队列研究采用目标试验模拟,重点关注与 ASPIRE 试验相似的各种器官系统的不良事件。

结果

本研究纳入了 2007 年至 2020 年间的 4580 例 RRMM 患者,与 Rd 组相比,KRd 组的血液学不良事件(贫血、中性粒细胞减少症、血小板减少症)和一些非血液学不良事件(咳嗽、低钾血症、便秘、高血压、心力衰竭)发生风险显著更高。在非血液学不良事件中,心血管事件(心力衰竭[HR 2.04;95%CI 1.24-3.35]、高血压[HR 1.58;95%CI 1.15-2.17])的风险最高。

结论

卡非佐米在韩国患者中的安全性与之前的 RCT 相似。因此,在亚洲 RRMM 患者中使用卡非佐米时应谨慎,因为心血管不良事件的风险增加。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0599/11793395/938de7124408/432_2024_5800_Fig1_HTML.jpg

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