从儿童瘤学的随机第三期试验的调查结果的统计脆弱性
Hannah E Olsen1, Pei-Chi Kao2, Caleb Richmond2
1Department of Pediatrics, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts, USA.
Cancer medicine
|December 16, 2024
概括
脆弱性指数 (FI) 和生存推断脆弱性指数 (SIFI) 显示,儿科瘤学试验是强大的. 这些试验的统计结论对小患者组的变化并不过于敏感.
科学领域:
- 在瘤学瘤学.
- 生物统计学 生物统计学
- 临床试验 临床试验
背景情况:
- 脆弱性指数 (FI) 有助于解释临床试验中的p值.
- FI以前没有在儿童瘤学第三阶段试验中进行评估.
研究的目的:
- 评估儿科瘤学第三阶段试验的统计脆弱性.
- 评估患者数量对试验结论的影响.
主要方法:
- 通过PubMed.com确定了113项儿科瘤学第三阶段试验 (1980-2020年).
- 对于二进制结果的计算FI和对于时间到事件结果的生存推断脆弱性指数 (SIFI).
- 根据试验大小来规范脆弱性,确定脆弱性系数 (FQ/SFQ).
主要成果:
- 中位数FI为4.5 (二进制),中位数SIFI为13 (时间到事件).
- 在25%的试验中,FI/SIFI的数量低于随访中丢失的患者数量.
- 随着时间的推移,脆弱率保持稳定,不管样本大小增加.
结论:
- 儿科瘤学试验的结论依赖于小比例的患者.
- 与成人试验相比,这些试验表现出类似或较低的脆弱性.
- 建议在3期儿科瘤学试验中与p值一起报告FI/SIFI.
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