Gagnier Joel J, Otalike Edith, Firth Andrew D
Department of Epidemiology & Biostatistics, Schulich School of Medicine & Dentistry, Western University, London, ON, Canada.
Department of Surgery, Schulich School of Medicine & Dentistry, Western University, London, ON, Canada.
Qual Life Res. 2025 Jul 21. doi: 10.1007/s11136-025-04022-w.
This study assessed how poor-quality patients reported outcome measures (PROMS) influence treatment effect estimates in randomized patients with anterior cruciate ligament (ACL) injury.
We chose a representative sample of studies by searching top journals of orthopaedic surgery for randomized trials in patients after anterior cruciate ligament injury over the past 5-years. Studies were eligible if they reported at least one of the PROMs assessed in a previous review of their measurement properties. The risk of bias was assessed, and standardized treatment effects were calculated between groups for each PROM and time point. We used mixed-effects regression to predict treatment effect using psychometric summary score, risk of bias score, follow-up duration, sample size, proportion of female patients, and funding source as predictors, accounting for the random effect of the study.
Out of the 4038 studies screened, 38 RCTs that used 9 validated PROMs and reported 139 PROM scores were included. The mean risk of bias score was 7.1 (standard deviation [SD]: 1), and the mean psychometric score was 1.4 (SD = 2.5). While psychometric quality was not significantly associated with treatment effect variance in lower-quality PROMs, the study found that longer follow-up duration was associated with a less likely treatment effect favouring the experimental group.
Poor quality patient-reported outcomes may demonstrate greater variance of treatment effects in randomized trials. More psychometric evidence, particularly of responsiveness, is needed to assess the effect of individual measurement properties on treatment effects. These findings should be considered hypothesis generating only and be followed up in additional related research.
本研究评估了质量较差的患者报告结局测量指标（PROMS）如何影响前交叉韧带（ACL）损伤随机分组患者的治疗效果估计。
我们通过检索过去5年骨科手术顶级期刊中有关前交叉韧带损伤患者的随机试验，选取了具有代表性的研究样本。如果研究报告了先前对其测量特性进行综述时评估的至少一项PROMS，则该研究符合纳入标准。评估偏倚风险，并计算每个PROMS和时间点组间的标准化治疗效果。我们使用混合效应回归，以心理测量汇总评分、偏倚风险评分、随访持续时间、样本量、女性患者比例和资金来源作为预测变量来预测治疗效果，同时考虑研究的随机效应。
在筛选的4038项研究中，纳入了38项随机对照试验，这些试验使用了9项经过验证的PROMS，并报告了139个PROMS评分。平均偏倚风险评分为7.1（标准差[SD]：1），平均心理测量评分为1.4（SD = 2.5）。虽然在质量较低的PROMS中，心理测量质量与治疗效果差异无显著相关性，但研究发现随访持续时间越长，治疗效果越有利于试验组的可能性越小。
在随机试验中，质量较差的患者报告结局可能显示出更大的治疗效果差异。需要更多的心理测量证据，尤其是反应性方面的证据，来评估个体测量特性对治疗效果的影响。这些发现仅应被视为提出假设，需在更多相关研究中进行后续跟进。
