文章：

多发性骨髓瘤注册试验中患者报告结局的回顾：突显改进领域

Review of patient-reported outcomes in multiple myeloma registrational trials: highlighting areas for improvement

原文发布日期：2021-08-31

DOI: 10.1038/s41408-021-00543-y

类型: Article

开放获取: 是

英文摘要：

Over the past 13 years, there have been advances in characterizing the patient experience in oncology trials, primarily using patient-reported outcomes (PROs). This review aims to provide details on the PRO measures and analyses used in multiple myeloma (MM) registrational trials. We identified registrational trials supporting MM indications from 2007 to 2020 from FDA databases. Trial protocols, statistical analysis plans, and clinical study reports were reviewed for PRO measures used, collection methods, statistical analyses, baseline and instrument completion definitions, and thresholds for clinical meaningfulness. Twenty-five trials supporting 20 MM indications were identified; 17 (68%) contained submitted PRO data. Of the 17 trials, 14 were randomized controlled trials and the remainder were single-arm trials. All but one trial were open label trials. Seven trials collected data electronically and five in paper format. The majority of trials evaluated at least two PRO measures (82%) with two trials (12%) utilizing four measures. Nine unique PRO measures were used, most commonly the EORTC QLQ-30 (87%), EQ-5D (65%), and QLQ-MY20 (47%). All 17 (100%) trials provided descriptive summaries, 10 (59%) carried out longitudinal mixed model analysis, 9 (53%) conducted responder analysis, and 2 (12%) did a basic inferential test. We noted substantial heterogeneity in terms of PRO collection methods, measures, definitions, and analyses, which may hinder the ability to effectively capture and interpret patient experience in future MM clinical trials. Further research is needed to determine the most appropriate approaches for statistical and analytical methodologies for PRO data in MM trials.
 

摘要翻译： 

过去13年间，肿瘤学试验在患者体验特征描述方面取得进展，主要采用患者报告结局（PROs）进行评估。本综述旨在详细阐述多发性骨髓瘤（MM）注册试验中使用的PRO评估工具及其分析方法。我们从FDA数据库中筛选出2007年至2020年间支持MM适应症的注册试验，通过审查试验方案、统计分析计划和临床研究报告，提取PRO评估工具类型、收集方法、统计分析策略、基线定义与工具完成标准以及临床意义阈值等要素。共确定支持20项MM适应症的25项试验，其中17项（68%）提交了PRO数据。这17项试验中14项为随机对照试验，其余为单臂试验；除1项试验外均为开放性试验。7项试验采用电子化数据收集，5项使用纸质收集方式。绝大多数试验（82%）评估至少两种PRO工具，其中两项试验（12%）使用了四种工具。共采用九种不同的PRO工具，最常用的是EORTC QLQ-30（87%）、EQ-5D（65%）和QLQ-MY20（47%）。所有17项试验（100%）均提供描述性统计摘要，10项（59%）进行了纵向混合模型分析，9项（53%）实施应答者分析，仅2项（12%）采用基础推断检验。我们注意到PRO收集方法、评估工具、定义标准和分析策略存在显著异质性，这可能影响未来MM临床试验中患者体验的有效捕获与解读。需要进一步研究以确定MM试验中PRO数据最适宜的统计分析方法学路径。

原文链接：

Review of patient-reported outcomes in multiple myeloma registrational trials: highlighting areas for improvement