Sayers Adrian, Wylde Vikki, Lenguerrand Erik, Gooberman-Hill Rachael, Dawson Jill, Beard David, Price Andrew, Blom Ashley W
Musculoskeletal Research Unit, School of Clinical Sciences, University of Bristol, Southmead Hospital, Bristol, UK.
School of Social and Community Medicine, University of Bristol, Bristol, UK.
BMJ Open. 2017 Jul 21;7(7):e014041. doi: 10.1136/bmjopen-2016-014041.
This article reviews and compares four commonly used approaches to assess patient responsiveness with a treatment or therapy (return to normal (RTN), minimal important difference (MID), minimal clinically important improvement (MCII), OMERACT-OARSI [Outcome Measures in Rheumatology-Osteoarthris Reseach Society International] (OO)) and demonstrates how each of the methods can be formulated in a multilevel modelling (MLM) framework.
Cohort study.
A cohort of patients undergoing total hip and knee replacement were recruited from a single UK National Health Service hospital.
400 patients from the Arthroplasty Pain Experience cohort study undergoing total hip (n=210) and knee (n=190) replacement who completed the Intermittent and Constant Osteoarthritis Pain questionnaire prior to surgery and then at 3, 6 and 12 months after surgery.
The primary outcome was defined as a response to treatment following total hip or knee replacement. We compared baseline scores, change scores and proportion of individuals defined as 'responders' using traditional and MLM approaches with patient responsiveness.
Using existing approaches, baseline and change scores are underestimated, and the variance of baseline and change scores overestimated in comparison with MLM approaches. MLM increases the proportion of individuals defined as responding in RTN, MID and OO criteria compared with existing approaches. Using MLM with the MCII criteria reduces the number of individuals identified as responders.
MLM improves the estimation of the SD of baseline and change scores by explicitly incorporating measurement error into the model and avoiding regression to the mean when making individual predictions. Using refined definitions of responsiveness may lead to a reduction in misclassification when attempting to predict who does and does not respond to an intervention and clarifies the similarities between existing methods.
本文回顾并比较了四种常用的评估患者对治疗或疗法反应性的方法（恢复正常（RTN）、最小重要差异（MID）、最小临床重要改善（MCII）、OMERACT - OARSI[风湿病学 - 骨关节炎研究协会国际组织（OO）的疗效指标]），并展示了如何在多水平建模（MLM）框架中构建每种方法。
队列研究。
从英国一家国民健康服务医院招募了一组接受全髋关节和膝关节置换的患者。
来自关节置换疼痛体验队列研究的400例患者，其中210例行全髋关节置换，190例行全膝关节置换，这些患者在手术前以及术后3个月、6个月和12个月完成了间歇性和持续性骨关节炎疼痛问卷。
主要结局定义为全髋关节或膝关节置换术后对治疗的反应。我们使用传统方法和MLM方法比较了基线分数、变化分数以及被定义为“反应者”的个体比例与患者反应性。
与MLM方法相比，使用现有方法时，基线和变化分数被低估，基线和变化分数的方差被高估。与现有方法相比，MLM增加了根据RTN、MID和OO标准定义为有反应的个体比例。使用MLM结合MCII标准会减少被确定为反应者的个体数量。
MLM通过在模型中明确纳入测量误差并在进行个体预测时避免均值回归，改进了对基线和变化分数标准差的估计。使用更精确的反应性定义可能会减少在试图预测谁对干预有反应和谁没有反应时的错误分类，并阐明现有方法之间的相似性。
