Center for Gait and Motion Analysis, Gillette Children's Specialty Healthcare, St Paul, MN, USA.
Department of Orthopedic Surgery, University of Minnesota, St Paul, MN, USA.
Dev Med Child Neurol. 2021 Feb;63(2):196-203. doi: 10.1111/dmcn.14709. Epub 2020 Oct 21.
To test whether an observational study employing propensity score matching could accurately estimate the causal treatment effects of rectus femoris transfer (RFT) as part of single-event multilevel surgery (SEMLS) in ambulatory children with cerebral palsy.
We used a large clinical database to derive a propensity score for treatment assignment (SEMLS±RFT) and used this score to generate a matched patient cohort. We compared the causal treatment effects estimated from this matched cohort with a previously published randomized controlled trial (RCT).
The treated arms of the observational study and RCT were well matched. There were 129 limbs (81 males) with a mean age of 10 years 7 months (4y 7mo) in the treated arm of the observational study, and 129 limbs (68 males) with a mean age of 10 years 2 months (3y 9mo) in the control arm of the observational study. Differences between the observational study and RCT cohorts were clinically meaningless for knee flexion kinematics (1-4°), timing of knee angle extrema (<3% gait cycle), and speed (<5mm/s). Postoperative changes in the observational study matched those from the RCT. All but one of the observational study confidence intervals were completely contained within the corresponding RCT confidence interval; there were no meaningful differences in magnitude or sign of key outcomes related to stiff knee gait.
Propensity score matching is an accurate method for estimating the causal treatment effects of RFT as part of an SEMLS. It seems reasonable to extend this approach to other common components of SEMLS treatment in this population.
Propensity score matching is an accurate method for estimating the causal treatment effects of rectus femoris transfer (RFT) in ambulatory children with cerebral palsy (CP). The causal treatment effects for RFT surgery in ambulatory children diagnosed with CP were validated.
检验一项观察性研究，该研究采用倾向评分匹配来准确估计作为单事件多层次手术（SEMLS）一部分的股直肌转位（RFT）对脑瘫患儿门诊治疗的因果效应。
我们利用一个大型临床数据库来获得治疗分配（SEMLS±RFT）的倾向评分，并使用该评分生成匹配的患者队列。我们将从匹配队列中估计的因果治疗效果与先前发表的随机对照试验（RCT）进行比较。
观察性研究和 RCT 的治疗组匹配良好。在观察性研究的治疗组中，有 129 条肢体（81 名男性），平均年龄为 10 岁 7 个月（4y 7mo），在观察性研究的对照组中，有 129 条肢体（68 名男性），平均年龄为 10 岁 2 个月（3y 9mo）。在膝关节弯曲运动学（1-4°）、膝关节角度极值的时间（<3%步态周期）和速度（<5mm/s）方面，观察性研究和 RCT 队列之间的差异在临床上无意义。观察性研究的术后变化与 RCT 一致。除了一个之外，观察性研究的置信区间完全包含在相应的 RCT 置信区间内；与僵硬膝关节步态相关的关键结果的幅度或符号没有明显差异。
倾向评分匹配是一种准确估计股直肌转位（RFT）作为 SEMLS 一部分的因果治疗效果的方法。在该人群中，将这种方法扩展到 SEMLS 治疗的其他常见组成部分似乎是合理的。
倾向评分匹配是一种准确估计脑瘫患儿门诊治疗中股直肌转位（RFT）的因果治疗效果的方法。验证了脑瘫患儿门诊接受 RFT 手术的因果治疗效果。
