David G. Lester, Kevin Talbot, Martin R. Turner, Alexander G. Thompson, the Pooled Resource Open-Access ALS Clinical Trials Consortium
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引用次数: 0
摘要
肌萎缩性侧索硬化症(ALS)的严重体重减轻是常见的,多因素的,并与生存期缩短有关。使用来自三个队列的6000多名ALS患者的纵向体重数据,我们建立了一个加速失效时间模型来预测未来严重(≥10%)体重减轻的风险,使用五个单时间点临床预测指标:症状持续时间、修订的ALS功能评定量表、发病部位、用力肺活量和年龄。采用内外交叉验证和随机效应荟萃分析评估模型的性能和通用性。总体一致性统计量为0.71 (95% CI 0.63 ~ 0.79),校准斜率和截距分别为0.91(0.69 ~ 1.13)和0.05(-0.11 ~ 0.21)。本研究强调了与ALS患者严重体重减轻最相关的临床因素,并为分层工具提供了基础。
A Validated Model to Predict Severe Weight Loss in Amyotrophic Lateral Sclerosis
Severe weight loss in amyotrophic lateral sclerosis (ALS) is common, multifactorial, and associated with shortened survival. Using longitudinal weight data from over 6000 patients with ALS across three cohorts, we built an accelerated failure time model to predict the risk of future severe (≥ 10%) weight loss using five single-timepoint clinical predictors: symptom duration, revised ALS Functional Rating Scale, site of onset, forced vital capacity, and age. Model performance and generalisability were evaluated using internal–external cross-validation and random-effects meta-analysis. The overall concordance statistic was 0.71 (95% CI 0.63–0.79), and the calibration slope and intercept were 0.91 (0.69–1.13) and 0.05 (−0.11–0.21). This study highlights clinical factors most associated with severe weight loss in ALS and provides the basis for a stratification tool.
期刊介绍:
Annals of Clinical and Translational Neurology is a peer-reviewed journal for rapid dissemination of high-quality research related to all areas of neurology. The journal publishes original research and scholarly reviews focused on the mechanisms and treatments of diseases of the nervous system; high-impact topics in neurologic education; and other topics of interest to the clinical neuroscience community.