混合方法以使用退出面试和临床试验数据来定义有意义的变化,用于患有旋巨细胞瘤 (TGCT) 的患者
Heather L Gelhorn1, Katelyn N Cutts2, Brooke Harrow3
1Patient-Centered Research, Evidera, Wilmington, NC, USA. heather.gelhorn@thermofisher.com.
概括
这项研究为患有紧突巨细胞瘤 (TGCT) 的患者确定了有意义的改善. 关键发现确定了对身体功能,运动范围和度的响应者值,有助于临床试验的解释.
科学领域:
- 骨科和肌肉骨系统疾病
- 临床试验方法论 临床试验方法论
- 患者报告的结果
背景情况:
- 长巨细胞瘤 (TGCT) 是一种局部侵袭性瘤,会引起疼痛,硬和身体功能减弱.
- 在MOTION第3期试验中,对TGCT进行了维姆塞尔提尼布的评估,因此需要确定有意义的临床结果变化.
- 了解患者感知到的有意义的变化对于解释TGCT临床试验结果至关重要.
研究的目的:
- 在TGCT患者中定义积极运动范围 (ROM),身体功能 (PF) 和度的有意义的改善值.
- 将患者的定性观点与MOTION试验的定量数据相结合.
- 在TGCT中建立关键临床结果评估 (COA) 的应答者定义.
主要方法:
- 与来自MOTION试验的患者进行了嵌入式退出访谈.
- 定性数据探讨了患者总体变化印象 (PGIC),PROMIS-PF和最坏的度NRS的有意义的变化.
- 定量MOTION试验数据的基于和分布的分析,通过采访提供信息,定义了应答者值.
主要成果:
- 78%的患者 (96/123) 完成了退场面试,大多数人将"微小改善"的反应定义为有意义的.
- 响应者的估计值各不相同:PROMIS-PF (1.45-4.9),活跃的ROM (6.0-14.8%),以及刚性NRS (-2.3到-0.5).
- 累积分布函数曲线显示,治疗组在拟议的值周围有明显的分离.
结论:
- 确定了响应者定义:PROMIS-PF的≥3点改善,活性ROM的≥10%,最坏度NRS的≥2点改善.
- 定性患者访谈对于选择适当的点和定义临床结果评估的有意义变化至关重要.
- 这些定义的值提高了对TGCT临床试验数据的解释,并纳入了患者的观点.
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