Venetoclax (Ven), a BCL-2 inhibitor, has demonstrated efficacy in patients with relapsed/refractory multiple myeloma (RRMM) harboring a t(11;14) and/or elevated BCL-2 expression. However, data from clinical trial remain inconclusive. This retrospective study evaluated the efficacy and safety of Ven-based therapies in 232 MM patients without concurrent AL amyloidosis treated at Mayo Clinic sites between Jan 2015 and Dec 2023. The median age was 62 years, with a median of 3 prior lines of therapy. Among the cohort, 82% had t(11;14), and elevated BCL-2 expression was identified in 17 of 18 non-t(11;14) patients tested. Ven combinations included Ven-Dex (VenD; 48.3%), Proteasome Inhibitor-Ven (30.2%), and Daratumumab-Ven (19%) with other combinations making up the rest. The overall response rate was 57%; 64% for t(11;14) patients and 26% for non-t(11;14) patients. Median progression-free survival (PFS) was 9.4 months overall; 11.8 months for t(11;14) patients and 2.9 months for those without (p < 0.001). Among t(11;14) patients, the presence of del(17p) or 1q gain/amplification significantly reduced PFS to 7.7 months. Venetoclax-based regimens remain an important option for t(11;14) patients, but efficacy is limited in patients without a t(11;14). The presence of secondary high-risk cytogenetics imparts an inferior PFS.
维奈托克（Ven）作为一种BCL-2抑制剂，已在对携带t(11;14)和/或BCL-2高表达的复发/难治性多发性骨髓瘤（RRMM）患者中显示出疗效，但临床试验数据尚未形成明确结论。本回顾性研究评估了2015年1月至2023年12月在梅奥诊所医疗系统接受治疗的232例未合并AL淀粉样变性多发性骨髓瘤患者中，以维奈托克为基础的治疗方案的疗效与安全性。患者中位年龄为62岁，既往中位治疗线数为3线。队列中82%的患者存在t(11;14)染色体易位，在18例接受检测的非t(11;14)患者中，有17例存在BCL-2高表达。维奈托克联合方案包括Ven-Dex（VenD；48.3%）、蛋白酶体抑制剂-Ven（30.2%）以及达雷妥尤单抗-Ven（19%），其余为其他联合方案。总体缓解率为57%；其中t(11;14)患者为64%，非t(11;14)患者为26%。中位无进展生存期（PFS）总体为9.4个月；t(11;14)患者为11.8个月，非t(11;14)患者为2.9个月（p < 0.001）。在t(11;14)患者中，若同时存在del(17p)或1q增益/扩增，其PFS显著缩短至7.7个月。基于维奈托克的治疗方案仍是t(11;14)患者的重要选择，但对非t(11:14)患者疗效有限。合并继发性高危细胞遗传学异常会导致较差的PFS。
肿瘤(癌症)患者之家