针对血液癌症的第三期临床试验中患者报告的结果:系统性审查
Kishan Patel1, Alexandra Ivanov2, Tajmah Jocelyn3
1Department of Internal Medicine, Brigham & Women's Hospital, Boston, Massachusetts.
JAMA network open
|June 3, 2024
概括
患者报告结果 (PROs) 在大多数血液癌症试验中收集,但很少作为主要终点. 在出版物中报告PRO数据的数量很低,这引发了对出版物偏见和临床试验中患者的实践经验的担忧.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 临床试验 临床试验
背景情况:
- 患者报告结果 (PROs) 在实体瘤临床试验中未得到充分利用.
- 在血液性恶性瘤中收集和报告PRO的做法尚不清楚.
研究的目的:
- 确定血性恶性瘤随机临床试验 (RCT) 中的PROs作为终点的患病率.
- 评估PRO数据在相关试验出版物中的报告情况.
主要方法:
- 2018-2022年间发表在8个具有影响力的期刊上,针对血液性恶性瘤的3期治疗性RCT的系统综述.
- 从手稿和协议中提取关于试验特征和PRO的数据.
- 单变量分析以确定试验特征与PRO收集/报告之间的关联.
主要成果:
- 在90个符合条件的RCT中,73%的终点是PRO,但只有1%的RCT将PRO作为主要终点.
- 在39%的出版物中报告了PRO数据;结果没有变化 (18) 或改善 (8),没有报道更糟糕的PROs.
- 营利性赞助和特定的血液学疾病 (血细胞疾病,多发性骨髓瘤,多发性骨髓瘤) 与更高的PRO终点纳入和报告有关.
结论:
- 虽然大多数血液癌症RCT收集PRO数据,但其作为主要终点的使用很少.
- 在主要出版物中报告PRO数据方面存在很大的差距,可能是由于出版偏差.
- 迫切需要改善血液癌症临床试验中以患者为中心的数据的传播.
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