Hematology Hospital, Seoul St. Mary's Hospital, The Catholic University of Korea, 222, Banpo-daero, Seocho-gu, Seoul, 06591, South Korea.
Department of Hematology, Yeouido St. Mary's Hospital, The Catholic University of Korea, Seoul, South Korea.
J Cancer Res Clin Oncol. 2023 Dec;149(18):16279-16291. doi: 10.1007/s00432-023-05385-8. Epub 2023 Sep 12.
Carfilzomib plus dexamethasone (Kd) is widely used in patients with relapsed and/or refractory multiple myeloma (RRMM). However, the treatment outcomes of Kd, especially in trial-unfit patients, have not been extensively studied in the real-world setting.
We analyzed the outcomes of 152 RRMM patients who received Kd at our hospitals from April 2018 to March 2022.
At the commencement of Kd, patients received a median of two (range 1-7) lines of prior anti-myeloma therapy. According to the ENDEAVOR study criteria, 93 (61.2%) and 59 (38.8%) patients were classified as the trial-fit and the trial-unfit group, respectively. The overall response (OR) rate for the entire cohort was 71.1% (95% CI 63.2-78.1%). Progression-free survival (PFS) and overall survival (OS) were 5.6 months (95% CI 3.9-6.9 months) and 24.0 months (95% CI 13.4-38.0 months), respectively. There was no significant difference in the OR rate between the trial-fit and the trial-unfit groups (76.3% vs. 62.7%; P = 0.105). However, the median PFS (3.6 months vs. 7.3 months; P < 0.001) and OS (15.0 vs. 36.8 months; P = 0.009) were significantly shorter in the trial-unfit group. On multivariate analysis, trial-fitness (unfit vs. fit) remained a significant covariate influencing the TRM (HR: 4.84, 95% CI 1.66-14.06; P = 0.004) and PFS (HR: 1.82, 95% CI 1.27-2.62; P = 0.001).
Our data suggest that the treatment outcomes of Kd are acceptable in the real-world setting with significant differences between the trial-fit and the trial-unfit groups, although they are relatively inferior to those of a pivotal trial.
卡非佐米联合地塞米松（Kd）广泛用于治疗复发和/或难治性多发性骨髓瘤（RRMM）患者。然而，Kd 的治疗效果，尤其是在不适合临床试验的患者中的治疗效果，在真实世界环境中尚未得到广泛研究。
我们分析了 2018 年 4 月至 2022 年 3 月在我院接受 Kd 治疗的 152 例 RRMM 患者的治疗结局。
在开始 Kd 治疗时，患者接受了中位数为 2 线（范围 1-7 线）的既往抗骨髓瘤治疗。根据 ENDEAVOR 研究标准，93 例（61.2%）和 59 例（38.8%）患者分别被归类为适合临床试验和不适合临床试验组。整个队列的总缓解（OR）率为 71.1%（95%CI 63.2-78.1%）。无进展生存期（PFS）和总生存期（OS）分别为 5.6 个月（95%CI 3.9-6.9 个月）和 24.0 个月（95%CI 13.4-38.0 个月）。适合临床试验和不适合临床试验组的 OR 率无显著差异（76.3% vs. 62.7%；P=0.105）。然而，不适合临床试验组的中位 PFS（3.6 个月 vs. 7.3 个月；P<0.001）和 OS（15.0 个月 vs. 36.8 个月；P=0.009）明显更短。多变量分析显示，适合临床试验（不适合 vs. 适合）仍然是影响 TRM（HR：4.84，95%CI 1.66-14.06；P=0.004）和 PFS（HR：1.82，95%CI 1.27-2.62；P=0.001）的显著协变量。
我们的数据表明，Kd 的治疗效果在真实世界环境中是可以接受的，适合临床试验和不适合临床试验组之间存在显著差异，尽管它们不如关键试验中的结果好。
