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.

在过去的 13 年里，主要使用患者报告的结果 (PRO) 来描述肿瘤学试验中的患者体验方面取得了进展。本综述旨在提供多发性骨髓瘤 (MM) 注册试验中使用的 PRO 测量和分析的详细信息。我们从 FDA 数据库中确定了 2007 年至 2020 年支持 MM 适应症的注册试验。对试验方案、统计分析计划和临床研究报告进行了审查，以了解所使用的 PRO 测量、收集方法、统计分析、基线和仪器完成定义以及临床意义的阈值。确定了支持 20 种 MM 适应症的 25 项试验； 17 (68%) 包含提交的 PRO 数据。在 17 项试验中，14 项为随机对照试验，其余为单组试验。除一项试验外，所有试验均为开放标签试验。七项试验以电子方式收集数据，五项试验以纸质形式收集数据。大多数试验至少评估了两项 PRO 措施 (82%)，其中两项试验 (12%) 使用了四项措施。使用了九种独特的 PRO 测量，最常见的是 EORTC QLQ-30 (87%)、EQ-5D (65%) 和 QLQ-MY20 (47%)。所有 17 项试验（100%）都提供了描述性摘要，10 项试验（59%）进行了纵向混合模型分析，9 项试验（53%）进行了应答者分析，2 项试验（12%）进行了基本推理测试。我们注意到 PRO 收集方法、测量、定义和分析方面存在很大的异质性，这可能会阻碍未来 MM 临床试验中有效捕获和解释患者体验的能力。需要进一步研究来确定 MM 试验中 PRO 数据的最合适的统计和分析方法。