Department of Haematology, Tel-Aviv Sourasky Medical Center, Tel-Aviv, Israel.
Sackler Faculty of Medicine, Tel-Aviv University, Tel-Aviv, Israel.
Br J Haematol. 2023 Jan;200(1):45-53. doi: 10.1111/bjh.18479. Epub 2022 Oct 7.
Belantamab mafodotin, an immuno-conjugate targeting B-cell maturation antigen, showed single-agent activity in phase 1 and 2 studies, and was recently approved for heavily pretreated relapsed/refractory multiple myeloma (RRMM) patients. Real-world data and long-term follow-up are scarce. We conducted a multisite retrospective study aimed to assess safety and efficacy of belantamab mafodotin monotherapy administered via the GSK expanded access compassionate care programme. One-hundred and six RRMM patients were treated with belantamab mafodotin between July 2019 and March 2021. The median age was 69.4 years. Patients were heavily pretreated with a median of six (range 2-11) prior therapy lines. Major adverse effects included ocular toxicity (keratopathy 68.4%, grade ≥3: 40.5%; blurred vision 36.8%, grade ≥3: 6.3%), thrombocytopenia (27.4%, grade ≥3: 17.9%) and infections (11.3%, grade ≥3: 7.5%). Median follow-up time was 11.9 [95% confidence interval (CI) 10.0-13.8] months. Overall response rate was 45.5%. Median progression-free survival was 4.7 (95% CI 3.5-5.9) months in the entire cohort and 8.8 (95% CI 6.6-10.9) months among responders. Median overall survival was 14.5 (95% CI 9.5-19.6) months, and not reached for responders. To conclude, in a real-world setting, belantamab mafodotin monotherapy showed efficacy comparable with the prospective clinical trials, with a tolerable toxicity profile.
贝兰他单抗mafodotin 是一种针对 B 细胞成熟抗原的免疫偶联药物,在 1 期和 2 期研究中显示出单药活性,最近被批准用于大量预处理的复发/难治性多发性骨髓瘤 (RRMM) 患者。目前缺乏真实世界数据和长期随访结果。我们进行了一项多中心回顾性研究,旨在评估通过 GSK 扩大准入同情护理计划给予贝兰他单抗 mafodotin 单药治疗的安全性和疗效。在 2019 年 7 月至 2021 年 3 月期间,有 106 例 RRMM 患者接受了贝兰他单抗 mafodotin 治疗。中位年龄为 69.4 岁。患者接受了大量预处理,中位数为 6 线(范围 2-11)先前的治疗。主要不良反应包括眼毒性(角膜炎 68.4%,≥3 级:40.5%;视力模糊 36.8%,≥3 级:6.3%)、血小板减少症(27.4%,≥3 级:17.9%)和感染(11.3%,≥3 级:7.5%)。中位随访时间为 11.9 [95%置信区间 (CI) 10.0-13.8] 个月。总缓解率为 45.5%。在整个队列中,无进展生存期的中位数为 4.7(95%CI 3.5-5.9)个月,在缓解者中为 8.8(95%CI 6.6-10.9)个月。中位总生存期为 14.5(95%CI 9.5-19.6)个月,缓解者未达到。总之,在真实环境中,贝兰他单抗 mafodotin 单药治疗显示出与前瞻性临床试验相当的疗效,且毒性谱可耐受。