在转移性骨病的多中心随机对照试验中,以患者为中心的分层复合结果的设计
Hadia Farrukh1, Abbey Kunzli1, Olivia Virag1
1Department of Surgery, McMaster University, Hamilton, ON L8S 4K1, Canada.
Current oncology (Toronto, Ont.)
|June 25, 2025
概括
这项研究开发了一种以患者为中心的初级结局,用于近接大腿骨的转移性骨病 (MBD). 结果优先考虑生存,身体功能和家庭时间,指导未来的临床试验评估.
科学领域:
- 整形瘤学 整形瘤学
- 患者为中心的研究结果研究.
- 临床试验方法论 临床试验方法论
背景情况:
- 靠近的股骨是转移性骨病 (MBD) 的常见部位,导致病理性骨折.
- 手术稳定是标准的,但由于改善存活率,重建越来越多地使用.
- 临近大腿骨的MBD以患者为中心的结局尚未得到充分研究.
研究的目的:
- 为近端FEmur重建或内部固定fOR转移 (PERFORM) 试验制定以患者为中心的初级结局.
- 将患者和护理人员的观点纳入MBD治疗结果的选择.
- 建立一个有意义的初级结果,用于评估手术干预在MBD.
主要方法:
- 采用混合方法的方法,从高级癌症患者和护理人员的焦点组开始.
- 与外科医生,患者和护理人员一起进行了离散选择实验 (DCE),以评估结果的重要性.
- 利益相关者的意见指导了关键结果组件的选择.
主要成果:
- 确定的主要结局组件是:在12个月内死亡率,在4个月内身体功能 (PROMIS® 全球身体功能) 和在12个月内在家中的日子.
- DCE证实,生存和身体功能是最优先考虑的结果.
- 利益相关者参与成功定义了一个全面的,以患者为中心的结果指标.
结论:
- 为PERFORM试验开发的初级结局有效地整合了近端大腿骨的MBD患者的优先事项.
- 这种以患者为中心的结果有可能指导外科手术方法的评估,并影响临床实践.
- 优先考虑生存,功能和生活质量 (在家呆一天) 提高了医疗保健临床试验的相关性.
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