Clinical Research and Evidence-Based Medicine Unit, Second Medical Department, Aristotle University of Thessaloniki, Thessaloniki, Greece.
Clinical Research and Evidence-Based Medicine Unit, Second Medical Department, Aristotle University of Thessaloniki, Thessaloniki, Greece.
Crit Rev Oncol Hematol. 2024 Dec;204:104529. doi: 10.1016/j.critrevonc.2024.104529. Epub 2024 Oct 4.
Many clinical trials of therapeutic interventions for multiple myeloma do not use patient important outcomes and rely on the use of surrogate endpoints. The aim of this systematic review was to depict the landscape of randomized controlled trials in myeloma research and compile the endpoints utilized.
We searched Embase, PubMed, and the Cochrane Library for randomized controlled trials in myeloma published in English up to October 2023. We included trials exploring efficacy of therapeutic modalities for myeloma itself or supportive care interventions.
A total of 2181 records, reporting data from 624 trials (448 comparing anti-myeloma treatments and 176 comparing supportive interventions) were deemed eligible. The most common primary outcome reported was disease response, followed by progression free survival (PFS) and overall survival (OS). Across all trials, 119 (19.1 %) used OS as the primary endpoint, while 316 (50.6 %) listed it as a secondary endpoint. Quality of life was less commonly prioritized, featured as primary endpoint only in seven studies (1.1 %) and as secondary endpoint in 115 studies (18.4 %). Studies funded by the pharmaceutical industry were more likely (Odds Ratio [OR] 3.85, 95 % CI 2.41-6.35) to use PFS as primary outcome. Similarly, studies with authors that had conflicts of interest with the funding source were more likely (OR 4.57, 95 % CI 2.72-7.92) to use PFS as the primary outcome.
While randomized controlled trials for multiple myeloma predominantly rely on surrogate endpoints, particularly PFS, the importance of OS as an outcome should not be overlooked.
许多多发性骨髓瘤治疗干预的临床试验未使用患者重要结局，而是依赖替代终点。本系统评价的目的是描绘多发性骨髓瘤研究中随机对照试验的现状，并汇编所使用的结局指标。
我们检索了 Embase、PubMed 和 Cochrane 图书馆中截至 2023 年 10 月发表的英文多发性骨髓瘤随机对照试验。我们纳入了探索骨髓瘤本身治疗方法或支持性护理干预疗效的试验。
共 2181 条记录，来自 624 项试验的数据（448 项比较抗骨髓瘤治疗，176 项比较支持性干预）被认为符合条件。报告最多的主要结局是疾病缓解，其次是无进展生存期（PFS）和总生存期（OS）。在所有试验中，119 项（19.1%）将 OS 作为主要终点，而 316 项（50.6%）将其列为次要终点。生活质量较少被优先考虑，只有 7 项研究（1.1%）将其作为主要终点，115 项研究（18.4%）将其作为次要终点。制药行业资助的研究更有可能（优势比 [OR] 3.85，95%置信区间 2.41-6.35）将 PFS 作为主要结局。同样，作者与资金来源存在利益冲突的研究更有可能（OR 4.57，95%置信区间 2.72-7.92）将 PFS 作为主要结局。
尽管多发性骨髓瘤的随机对照试验主要依赖于替代终点，尤其是 PFS，但不应忽视 OS 作为结局的重要性。
