在真实世界中以卡非佐米为基础的方案治疗多发性骨髓瘤的疗效及安全性分析

Efficacy and Safety Analysis of Carfilzomib-Based Regimens in the Treatment of Multiple Myeloma in the Real World

摘要: 目的：探讨在真实世界中以卡非佐米为基础的方案治疗多发性骨髓瘤(MM)的效果及安全性。方法：回顾性病例系列研究。回顾性收集青岛医学院附属医院、潍坊市人民医院、烟台毓璜顶医院、山东大学齐鲁医院青岛院区2023年3月至2025年2月应用以卡非佐米为基础的方案治疗的96例MM患者临床资料。分析患者疗效、生存及不良反应发生情况。根据患者性别、年龄、ECOG评分、ISS分期、R-ISS分期、髓外病变、治疗疗程数、治疗线数、既往移植进行亚组间疗效分析，采用卡方检验法检验组间差别；采用Kaplan-Meier法绘制患者无进展生存期(PFS)和总生存期(OS)生存曲线进行生存分析，采用Log-rank法或Landmark法行生存曲线的比较。结果：进行1个及以上疗程治疗后，96个患者中，MR为11例(11.5%)，PR为17例(17.7%)，VGPR为11例(11.5%)，CR为22例(22.9%)，sCR为3例(3.1%)，ORR为66.7%。≤3个疗程组、4~6个疗程组与≥7个疗程组总体临床疗效比较，差异有统计学意义(P < 0.05)，三组患者PFS生存曲线比较及OS生存曲线比较，差异有统计学意义(P < 0.05)；ECOG不同评分，ISS分期I~III组，R-ISS分期I~III组，有髓外病变组与无髓外病变组，一线用药组、首次复发用药组与三线及以上用药组，既往有移植与无移植组之间的临床疗效、PFS、OS生存曲线比较，差异无统计学意义(P > 0.05)。96例患者的不良反应中，最常见的不良反应依次为血液学不良反应29例(52.09%)、心血管系统症状28例(29.17%)、感染26例(27.09%)，2例发生血栓事件，2例因心衰终止治疗。结论：以卡非佐米为基础的治疗方案治疗MM的ORR较高，且随着治疗疗程数的增加，疗效越明显，OS和PFS获益越明显。

Abstract: Objective: To investigate the efficacy and safety of carfilzomib-based regimens in the treatment of multiple myeloma (MM) in the real world. Methods: This was a retrospective case series study. Clinical data were collected from 96 patients with MM who were treated with carfilzomib-based regimens at the Affiliated Hospital of Qingdao Medical College, Weifang People’s Hospital, Yantai Yuhuangding Hospital, and Qingdao Branch of Qilu Hospital of Shandong University from March 2023 to February 2025. Patient efficacy, survival, and adverse events were analyzed. Subgroup analyses of efficacy were performed based on gender, age, ECOG performance status, ISS stage, R-ISS stage, extramedullary disease, number of treatment cycles, lines of therapy, and prior transplantation status, using the chi-square test for intergroup comparisons. Progression-free survival (PFS) and overall survival (OS) curves were plotted using the Kaplan-Meier method, and survival curves were compared using the Log-rank or Landmark test. Results: After at least one cycle of treatment, among the 96 patients, 11 (11.5%) achieved minimal response (MR), 17 (17.7%) achieved partial response (PR), 11 (11.5%) achieved very good partial response (VGPR), 22 (22.9%) achieved complete response (CR), and 3 (3.1%) achieved stringent complete response (sCR), yielding an overall response rate (ORR) of 66.7%. Statistically significant differences were observed in overall clinical efficacy, PFS, and OS among the ≤3 cycles group, 4~6 cycles group, and ≥7 cycles group (P < 0.05). No statistically significant differences were found in clinical efficacy, PFS, or OS among subgroups based on ECOG performance status, ISS stage (I~III), R-ISS stage (I~III), presence or absence of extramedullary disease, lines of therapy (first-line, first relapse, third-line or beyond), or prior transplantation status (P > 0.05). Among the 96 patients, the most common adverse events were hematologic adverse events in 29 patients (52.09%), followed by cardiovascular system symptoms in 28 patients (29.17%) and infections in 26 patients (27.09%). Thrombotic events occurred in 2 patients, and treatment was discontinued due to heart failure in 2 patients. Conclusion: Carfilzomib-based regimens demonstrate a high ORR in the treatment of MM in the real world, with efficacy becoming more pronounced and OS and PFS benefits increasing with a higher number of treatment cycles.