Strong Brent, Fritz Michele C, Woodward Amanda, Kozlowski Allan, Reeves Mathew J
Department of Epidemiology and Biostatistics, College of Human Medicine, Michigan State University, East Lansing, MI, USA.
Department of Epidemiology and Biostatistics, College of Human Medicine, Michigan State University, East Lansing, MI, USA; Office of the Dean, College of Veterinary Medicine, Michigan State University, East Lansing, MI, USA.
J Clin Epidemiol. 2023 Apr;156:66-75. doi: 10.1016/j.jclinepi.2023.01.009. Epub 2023 Feb 2.
Interpreting between-group differences in patient-reported outcome measures can be challenging. Responder analyses, which compare the proportions of patients who achieve a meaningful clinical change, represent a more interpretable approach. We conducted a secondary responder analysis of the Michigan Stroke Transitions Trial (MISTT).
The MISTT randomized 265 patients with stroke to three treatment groups: usual care [UC], social work case management [SWCM], or social work case management plus access to a patient-oriented website [SWCM + website]. Two Patient-Reported Outcomes Measurement and Information System (PROMIS) Global-10 subscales (representing physical and mental health) and 5 additional patient-reported outcomes were collected at baseline and 90-days. Responder analyses were conducted using modified Poisson and linear regression using published minimal important differences. Multiple imputation was used to address missing data.
For the PROMIS-10 global physical health subscale, responders were 80% more common in the SWCM + website group compared to the UC group (relative risk = 1.8, 95% confidence interval [CI]: 1.0, 3.1), with a number needed to treat of 7 (95% CI: 3, 112). No significant treatment effects were observed for the PROMIS-10 global mental health subscale.
Results of this responder analysis were largely consistent with the original trial analysis but have the advantage of presenting treatment effects using more clinically interpretable number needed to treat metrics.
解读患者报告的结局指标中的组间差异可能具有挑战性。反应者分析比较了实现有意义临床变化的患者比例，是一种更具可解释性的方法。我们对密歇根中风过渡试验（MISTT）进行了二次反应者分析。
MISTT将265例中风患者随机分为三个治疗组：常规护理[UC]、社会工作病例管理[SWCM]或社会工作病例管理加访问以患者为导向的网站[SWCM + 网站]。在基线和90天时收集了两个患者报告结局测量与信息系统（PROMIS）全球10分量表（代表身心健康）以及另外5个患者报告的结局。使用修正的泊松回归和线性回归，并采用已发表的最小重要差异进行反应者分析。采用多重填补法处理缺失数据。
对于PROMIS - 10全球身体健康分量表，与UC组相比，SWCM + 网站组的反应者更为常见，多出80%（相对风险 = 1.8，95%置信区间[CI]：1.0，3.1），治疗所需人数为7（95% CI：3，112）。对于PROMIS - 10全球心理健康分量表，未观察到显著的治疗效果。
该反应者分析的结果在很大程度上与原始试验分析一致，但具有使用更具临床可解释性的治疗所需人数指标来呈现治疗效果的优势。
