Epidemiology, Biostatistics and Prevention Institute, University of Zurich, Zürich, Switzerland.
School of Agricultural, Forest and Food Sciences, Bern University of Applied Sciences, Bern, Switzerland.
J Neurotrauma. 2022 Feb;39(3-4):266-276. doi: 10.1089/neu.2020.7407. Epub 2021 Apr 7.
Neurological disorders usually present very heterogeneous recovery patterns. Nonetheless, accurate prediction of future clinical end-points and robust definition of homogeneous cohorts are necessary for scientific investigation and targeted care. For this, unbiased recursive partitioning with conditional inference trees (URP-CTREE) have received increasing attention in medical research, especially, but not limited to traumatic spinal cord injuries (SCIs). URP-CTREE was introduced to SCI as a clinical guidance tool to explore and define homogeneous outcome groups by clinical means, while providing high accuracy in predicting future clinical outcomes. The validity and predictive value of URP-CTREE to provide improvements compared with other more common approaches applied by clinicians has recently come under critical scrutiny. Therefore, a comprehensive simulation study based on traumatic, cervical complete spinal cord injuries provides a framework to investigate and quantify the issues raised. First, we assessed the replicability and robustness of URP-CTREE to identify homogeneous subgroups. Second, we implemented a prediction performance comparison of URP-CTREE with traditional statistical techniques, such as linear or logistic regression, and a novel machine learning method. URP-CTREE's ability to identify homogeneous subgroups proved to be replicable and robust. In terms of prediction, URP-CTREE yielded a high prognostic performance comparable to a machine learning algorithm. The simulation study provides strong evidence for the robustness of URP-CTREE, which is achieved without compromising prediction accuracy. The slightly lower prediction performance is offset by URP-CTREE's straightforward interpretation and application in clinical settings based on simple, data-driven decision rules.
神经系统疾病的恢复模式通常表现出很大的异质性。然而,准确预测未来的临床终点和稳健定义同质队列对于科学研究和有针对性的护理是必要的。为此,无偏递归分区与条件推理树(URP-CTREE)在医学研究中受到越来越多的关注,尤其是但不限于创伤性脊髓损伤(SCI)。URP-CTREE 被引入 SCI 作为一种临床指导工具,通过临床手段探索和定义同质的结果组,同时提供对未来临床结果的高精度预测。URP-CTREE 在提供与临床医生应用的其他更常见方法相比的改进方面的有效性和预测价值最近受到了严格审查。因此,一项基于创伤性、颈段完全性脊髓损伤的综合模拟研究提供了一个框架来调查和量化所提出的问题。首先,我们评估了 URP-CTREE 识别同质亚组的可重复性和稳健性。其次,我们实现了 URP-CTREE 与传统统计技术(如线性或逻辑回归)和一种新的机器学习方法的预测性能比较。URP-CTREE 识别同质亚组的能力被证明是可重复和稳健的。在预测方面,URP-CTREE 产生了与机器学习算法相当的高预后性能。模拟研究为 URP-CTREE 的稳健性提供了强有力的证据,在不影响预测准确性的情况下实现了这一目标。URP-CTREE 的预测性能略低,但可以通过其基于简单数据驱动决策规则的直接解释和在临床环境中的应用来弥补。