文章：

多发性骨髓瘤早期临床试验的治疗结果：一项荟萃分析

Therapeutic outcome of early-phase clinical trials in multiple myeloma: a meta-analysis

原文发布日期：2021-03-01

DOI: 10.1038/s41408-021-00441-3

类型: Article

开放获取: 是

英文摘要：

Great progress in the treatment of patients with multiple myeloma (MM) has been made due to the development of novel drugs. Patients with relapsed/refractory MM (RRMM) can be enrolled in early-phase clinical trials, but their performance across the last decade is unknown. We conducted a meta-analysis on the overall response rate (ORR) and toxicity. PubMed, Embase, and Cochrane Library were systematically searched for phase I and phase II trials investigating an experimental compound as a single agent or in combination with dexamethasone, published from January 1, 2010 to July 1, 2020. Eighty-eight articles were included, describing 61 phase I trials involving 1835 patients and 37 phase II trials involving 2644 patients. There was a high degree of heterogeneity. Using a random-effects model, the 95% CIs of the estimated ORR were 8–17% for phase I trials and 18–28% for phase II trials. There were significant subgroup differences in ORR between the years of publication in phase I trials and between drug classes in both phase I and phase II trials. The ORR in early-phase clinical trials in RRMM is substantial, especially in phase II trials, but due to high heterogeneity a general assessment of clinical benefit before participation is difficult to offer to patients.
 

摘要翻译： 

多发性骨髓瘤（MM）的治疗因新药研发已取得重大进展。复发/难治性多发性骨髓瘤（RRMM）患者可纳入早期临床试验，但其在过去十年间的整体疗效尚不明确。我们对总体缓解率（ORR）及毒性反应进行了荟萃分析。系统检索了PubMed、Embase和Cochrane图书馆中自2010年1月1日至2020年7月1日发表的I期和II期临床试验，这些试验研究了实验性药物单药或联合地塞米松的治疗效果。共纳入88篇文献，其中描述了涉及1835名患者的61项I期试验和涉及2644名患者的37项II期试验。研究存在高度异质性。采用随机效应模型估算，I期试验的ORR 95%置信区间为8–17%，II期试验为18–28%。I期试验中不同发表年份之间，以及I期和II期试验中不同药物类别之间的ORR存在显著亚组差异。RRMM早期临床试验的总体缓解率显著，尤其在II期试验中，但由于高度异质性，难以在患者参与前对其临床获益提供普遍性评估。

原文链接：

Therapeutic outcome of early-phase clinical trials in multiple myeloma: a meta-analysis