文章：

KarMMa-RW研究：将idecabtagene vicleucel与复发/难治性多发性骨髓瘤真实世界结局进行比较

KarMMa-RW: comparison of idecabtagene vicleucel with real-world outcomes in relapsed and refractory multiple myeloma

原文发布日期：2021-06-18

DOI: 10.1038/s41408-021-00507-2

类型: Article

开放获取: 是

英文摘要：

Patients with relapsed and refractory multiple myeloma (RRMM) who are triple-class exposed (to an immunomodulatory agent, proteasome inhibitor, and anti-CD38 antibody) have limited treatment options and there is no standard of care. Idecabtagene vicleucel (ide-cel, bb2121), a BCMA-directed CAR T-cell therapy, demonstrated efficacy in triple-class exposed RRMM patients in the KarMMa trial (NCT03361748). In this retrospective study (KarMMa-RW), patient-level data from triple-class exposed RRMM patients were merged into a single data model and compared with KarMMa using trimmed stabilized inverse probability of treatment weighting. Endpoints included overall response rate (ORR; primary), rate of very good partial response or better (≥VGPR), progression-free survival (PFS), and overall survival (OS). Of 1949 real-world triple-class exposed RRMM patients, 190 received subsequent (index) line of therapy and met KarMMa eligibility criteria (Eligible RRMM cohort). With a median follow-up of 13.3 months in KarMMa and 10.2 months in Eligible RRMM, ORR, and ≥VGPR were significantly improved in KarMMa versus Eligible RRMM (ORR, 76.4% vs 32.2%; ≥VGPR, 57.9% vs 13.7%; both P < 0.0001) as were PFS (11.6 vs 3.5 months; P = 0.0004) and OS (20.2 vs 14.7 months; P = 0.0006). This study demonstrated that ide-cel significantly improved responses and survival compared with currently available therapies in triple-class exposed RRMM.
 

摘要翻译： 

三重暴露（即使用过免疫调节剂、蛋白酶体抑制剂和抗CD38抗体）的复发难治性多发性骨髓瘤（RRMM）患者治疗选择有限，且目前尚无标准治疗方案。在KarMMa试验（NCT03361748）中，靶向BCMA的CAR T细胞疗法idecabtagene vicleucel（ide-cel, bb2121）在三重暴露的RRMM患者中显示出疗效。本回顾性研究（KarMMa-RW）将来自三重暴露RRMM患者的个体数据合并至统一数据模型中，并采用修剪稳定逆概率治疗加权法与KarMMa试验数据进行对比。研究终点包括总缓解率（ORR；主要终点）、良好部分缓解及以上缓解率（≥VGPR）、无进展生存期（PFS）和总生存期（OS）。在1949例真实世界三重暴露RRMM患者中，190例接受了后续（索引）治疗线且符合KarMMa试验入选标准（符合条件RRMM队列）。KarMMa试验中位随访时间为13.3个月，符合条件RRMM队列为10.2个月。与符合条件RRMM队列相比，KarMMa试验的ORR和≥VGPR均显著改善（ORR：76.4%对比32.2%；≥VGPR：57.9%对比13.7%；均P < 0.0001），PFS（11.6个月对比3.5个月；P = 0.0004）和OS（20.2个月对比14.7个月；P = 0.0006）亦显著延长。本研究表明，与现有疗法相比，ide-cel能显著改善三重暴露RRMM患者的治疗反应和生存结局。

原文链接：

KarMMa-RW: comparison of idecabtagene vicleucel with real-world outcomes in relapsed and refractory multiple myeloma