文章：

单药belantamab mafodotin治疗复发/难治性多发性骨髓瘤：来自关键DREAMM-2研究中冻干剂型队列的分析

Single-agent belantamab mafodotin for relapsed/refractory multiple myeloma: analysis of the lyophilised presentation cohort from the pivotal DREAMM-2 study

原文发布日期：2020-10-23

DOI: 10.1038/s41408-020-00369-0

类型: Article

开放获取: 是

英文摘要：

DREAMM-2 (NCT03525678) is an ongoing global, open-label, phase 2 study of single-agent belantamab mafodotin (belamaf; GSK2857916), a B-cell maturation antigen-targeting antibody-drug conjugate, in a frozen-liquid presentation in patients with relapsed/refractory multiple myeloma (RRMM). Alongside the main study, following identical inclusion/exclusion criteria, a separate patient cohort was enrolled to receive belamaf in a lyophilised presentation (3.4 mg/kg, every 3 weeks) until disease progression/unacceptable toxicity. Primary outcome was independent review committee-assessed overall response rate (ORR). Twenty-five patients were enrolled; 24 received ≥1 dose of belamaf. As of 31 January 2020, ORR was 52% (95% CI: 31.3–72.2); 24% of patients achieved very good partial response. Median duration of response was 9.0 months (2.8–not reached [NR]); median progression-free survival was 5.7 months (2.2–9.7); median overall survival was not reached (8.7 months–NR). Most common grade 3/4 adverse events were keratopathy (microcyst-like corneal epithelial changes, a pathological finding seen on eye examination [75%]), thrombocytopenia (21%), anaemia (17%), hypercalcaemia and hypophosphatemia (both 13%), neutropenia and blurred vision (both 8%). Pharmacokinetics supported comparability of frozen-liquid and lyophilised presentations. Single-agent belamaf in a lyophilised presentation (intended for future use) showed a deep and durable clinical response and acceptable safety profile in patients with heavily pre-treated RRMM.
 

摘要翻译： 

DREAMM-2（临床试验编号NCT03525678）是一项正在进行的全球性、开放标签、二期研究，旨在评估单药belantamab mafodotin（belamaf；GSK2857916）——一种靶向B细胞成熟抗原的抗体药物偶联物——以冷冻液体制剂形式用于复发/难治性多发性骨髓瘤（RRMM）患者的效果。除主要研究外，另设独立患者队列采用相同入排标准，接受冻干制剂belamaf治疗（3.4毫克/千克，每三周一次），直至疾病进展或出现不可耐受毒性。主要终点为独立评审委员会评估的总缓解率（ORR）。共入组25例患者，其中24例接受至少一剂belamaf。截至2020年1月31日，ORR达52%（95%置信区间：31.3–72.2）；24%患者达到非常好的部分缓解。中位缓解持续时间为9.0个月（2.8–未达到）；中位无进展生存期为5.7个月（2.2–9.7）；中位总生存期未达到（8.7个月–未达到）。最常见的3/4级不良事件包括角膜病变（显微镜下囊样角膜上皮改变，眼科检查所见病理表现[75%]）、血小板减少症（21%）、贫血（17%）、高钙血症与低磷血症（均为13%）、中性粒细胞减少症与视力模糊（均为8%）。药代动力学数据支持冷冻液体与冻干制剂的等效性。单药belamaf冻干制剂（拟用于未来临床）在经深度治疗的RRMM患者中展现出深厚持久的临床缓解和可接受的安全性特征。

原文链接：

Single-agent belantamab mafodotin for relapsed/refractory multiple myeloma: analysis of the lyophilised presentation cohort from the pivotal DREAMM-2 study