Application of Heterogeneity of Treatment-Effects Methods: Exploratory Analyses of a Trial of Exercise-Based Interventions for Knee Osteoarthritis.
Application of Heterogeneity of Treatment-Effects Methods: Exploratory Analyses of a Trial of Exercise-Based Interventions for Knee Osteoarthritis.
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DOI：
10.1002/acr.24564
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发表时间：
2022-08
影响因子：
4.7
通讯作者：
Allen KD
中科院分区：
文献类型：
--
作者：
Coffman CJ;Arbeeva L;Schwartz TA;Callahan LF;Golightly YM;Goode AP;Huffman KM;Allen KD
在一项以运动为基础的干预膝骨关节炎（OA）的试验中，评估治疗效果（HTE）的异质性。参与者（n=350）被随机分为标准物理治疗组（PT, n=140）、基于互联网的运动训练组（IBET, n=142）和等候名单对照组（WL, n=68）。我们应用定性交互树（QUINT），一种顺序划分方法，和广义无偏交互检测和估计（GUIDE），一种回归树方法，来确定在4个月内西安大略省和麦克马斯特大学骨关节炎指数（WOMAC）评分有较大改善的亚组。预测指标包括24项人口统计学、临床和社会心理特征。我们进行了内部验证，以估计各组平均结果差异范围内的乐观（偏倚）。QUINT和GUIDE均显示，对于身体质量指数（BMI）较低的参与者，IBET优于PT （WOMAC的改善范围为6.3至9.1分），对于BMI较高且膝关节OA持续时间较长的参与者，PT优于IBET （WOMAC的改善为6.3分）。在GUIDE分析中，将PT或IBT与WL进行比较，未就业的参与者使用PT或IBT与WL相比，WOMAC的改善从1.8到6.8分不等。从内部验证来看，各组之间的平均结果差异有很大的修正；然而，在校正后，一些差异仍在临床有意义的范围内。结果表明，可能有亚组在PT或IBET后症状有较大改善，这可以指导转诊和未来的试验。然而，对于特定的治疗效应大小估计以及它们如何应用于本研究样本之外，仍然存在不确定性。NCT02312713
To evaluate heterogeneity of treatment effects (HTE) in a trial of exercise-based interventions for knee osteoarthritis (OA). Participants (n=350) were randomized to standard physical therapy (PT; n=140), Internet-Based Exercise Training (IBET; n=142), or wait list control (WL; n=68). We applied QUalitative INteraction Trees (QUINT), a sequential partitioning method, and Generalized Unbiased Interaction Detection and Estimation (GUIDE), a regression tree approach, to identify subgroups with greater improvements in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) score over 4-months. Predictors included 24 demographic, clinical and psychosocial characteristics. We conducted internal validation to estimate optimism (bias) in the range of mean outcome differences among arms. Both QUINT and GUIDE indicated that for participants with lower body mass index (BMI), IBET was better than PT (improvements of WOMAC ranged from 6.3 to 9.1 points lower) and for those with higher BMI and longer duration of knee OA, PT was better than IBET (WOMAC improvement was 6.3 points). In GUIDE analyses comparing PT or IBET to WL, participants not employed had improvements in WOMAC ranging from 1.8 to 6.8 points lower with PT or IBT vs. WL. From internal validation, there were large corrections to the mean outcome differences among arms; however, after correction some differences remained in the clinically meaningful range. Results suggest there may be subgroups who experience greater improvement in symptoms from PT or IBET, and this could guide referrals and future trials. However, uncertainty persists for specific treatment effect size estimates and how they apply beyond this study sample. NCT02312713
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