Idecabtagene vicleucel (Ide-cel) has demonstrated excellent efficacy and durable responses in patients with relapsed/refractory multiple myeloma (RRMM). However, the outcomes with ide-cel in patients with extramedullary disease (EMD) remain incompletely characterized. We included patients with RRMM treated with ide-cel between May 2021 and April 2023 across 11 US academic institutions. Visceral or soft tissue lesions non-contiguous from bone was classified as EMD. Time-to-event analyses were performed from date of ide-cel infusion. Among 351 patients, 84 (24%) had EMD prior to infusion. The median follow-up from ide-cel infusion was 18.2 months (95% CI: 17-19.3). The day 90 overall response rates (ORR) were 52% vs. 82% for the EMD and non-EMD cohorts, respectively (p < 0.001). The median progression-free survival (PFS) was 5.3 months (95% CI: 4.1–6.9) for the EMD cohort vs. 11.1 months (95% CI: 9.2–12.6; p < 0.0001) for the non-EMD cohort. In a multivariable analysis, EMD was an independent predictor of inferior PFS [hazard ratio 1.5 (1.1–2.2), p = 0.02]. The median overall survival was 14.8 months [95% CI: 9-Not reached (NR)] vs. 26.9 months (26.3 vs. NR, p = 0.006) for the EMD and non-EMD cohorts, respectively. Extramedullary disease represents an independent predictor of inferior day 90 ORR and PFS among patients treated with ide-cel.

中文翻译：

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髓外多发性骨髓瘤对 idecabtagene vicleucel 结局的影响


Idecabtagene vicleucel （Ide-cel） 在复发/难治性多发性骨髓瘤 （RRMM） 患者中表现出优异的疗效和持久的疗效。然而，ide-cel 在髓外疾病 （EMD） 患者中的结局仍未完全确定。我们纳入了 2021 年 5 月至 2023 年 4 月期间在美国 11 家学术机构接受 ide-cel 治疗的 RRMM 患者。与骨骼不相邻的内脏或软组织病变被归类为 EMD。从 ide-cel 输注之日起进行事件发生时间分析。在 351 例患者中，84 例 （24%） 在输注前患有 EMD。ide-cel 输注的中位随访时间为 18.2 个月 （95% CI： 17-19.3）。EMD 和非 EMD 队列的第 90 天总体缓解率 （ORR） 分别为 52% 和 82% （p < 0.001）。EMD 队列的中位无进展生存期 （PFS） 为 5.3 个月 （95% CI： 4.1-6.9），而非 EMD 队列为 11.1 个月 （95% CI： 9.2-12.6;p < 0.0001）。在多变量分析中，EMD 是较差 PFS 的独立预测因子 [风险比 1.5 （1.1–2.2），p = 0.02]。EMD 和非 EMD 队列的中位总生存期分别为 14.8 个月 [95% CI： 9-未达到 （NR）] vs. 26.9 个月 （26.3 vs. NR，p = 0.006）。髓外疾病是 ide-cel 治疗患者下第 90 天 ORR 和 PFS 的独立预测因子。