Department of Hematological Malignancies and Cellular Therapeutics, Kansas University Medical Center, Kansas City.
Division of Blood and Marrow Transplantation, University of California, San Diego, La Jolla.
JAMA Netw Open. 2021 Apr 1;4(4):e218084. doi: 10.1001/jamanetworkopen.2021.8084.
A thorough understanding of the optimal role and sequence of agents for treatment of multiple myeloma (MM) requires knowledge of the use and rate of postprotocol therapies in randomized clinical trials (RCTs).
To examine the proportion of MM RCTs that reported postprotocol therapies and, among those, the percentage of patients who received no further therapy and how treatments differed between the control and intervention arms.
The reporting of postprotocol therapies was systematically assessed in published MM RCTs using 3 databases (PubMed, Embase, and Cochrane Registry of Controlled Trials) for MM RCTs from January 1, 2005, to December 30, 2019. All MM RCTs were included, and all other studies, such as editorials, nonrandomized studies, and review articles, were excluded.
A total of 103 RCTs were identified (47 251 patients); of these, 45 (43.7%) reported subsequent treatments in that publication or in any subsequent publication. Trials funded by pharmaceutical companies (26 of 47 [55.3%]) were more likely to report subsequent treatments than cooperative group studies (19 of 56 [33.9%]) (χ21,103 = 4.8; P = .03). Differences were found in the treatments received between the intervention and control arms of RCTs. When data were reported, 5150 of 9351 patients (54.9%) in RCTs of newly diagnosed MM and 2197 of 4501 patients (48.8%) in RCTs of relapsed/refractory MM received any subsequent therapy.
Postprotocol therapies in MM RCTs are often not reported and, when they are, many patients receive no further therapy. Reporting guidelines for postprotocol therapies are needed.
全面了解多发性骨髓瘤（MM）治疗的最佳药物和治疗顺序，需要了解随机临床试验（RCT）中方案后治疗的使用和比例。
检查报告方案后治疗的 MM RCT 的比例，以及在这些 RCT 中，未接受进一步治疗的患者百分比，以及对照臂和干预臂之间的治疗差异。
使用 3 个数据库（PubMed、Embase 和 Cochrane 对照试验登记处）系统地评估了 2005 年 1 月 1 日至 2019 年 12 月 30 日发表的 MM RCT 中报告方案后治疗的 MM RCT，纳入所有 MM RCT，排除其他研究，如社论、非随机研究和综述文章。
共确定了 103 项 RCT（47251 例患者）；其中，45 项（43.7%）在该出版物或任何后续出版物中报告了随后的治疗。由制药公司资助的试验（26/47 [55.3%]）比合作组研究（19/56 [33.9%]）更有可能报告随后的治疗（χ21,103=4.8；P=0.03）。在 RCT 的干预臂和对照臂之间发现了治疗差异。当报告数据时，新诊断 MM 的 RCT 中 9351 例患者中有 5150 例（54.9%）和复发/难治性 MM 的 RCT 中 4501 例患者中有 2197 例（48.8%）接受了任何后续治疗。
MM RCT 中的方案后治疗常常没有报告，而当报告时，许多患者没有接受进一步的治疗。需要制定方案后治疗报告指南。
