Czaplinski Adam, Yen Albert A, Simpson Ericka P, Appel Stanley H
Department of Neurology, University of Basel, Basel, Switzerland.
Muscle Nerve. 2006 Dec;34(6):702-8. doi: 10.1002/mus.20658.
The aim of this study was to determine the predictors of disease progression in a group of 832 patients with the diagnosis of definite or probable amyotrophic lateral sclerosis (ALS). Disease progression was defined as the time to 20-point change in Appel ALS (AALS) score. The effects of individual prognostic factors on disease progression were assessed with the Kaplan-Meier life-table method. In addition, the prognostic value of each factor was estimated using both univariate and multivariate Cox proportional hazard analyses. The median time to a 20-point change in AALS score in our patient population was 9 months. Age, site of symptom onset, time between first symptom and first examination, total AALS score at first examination, and AALS preslope (rate of disease progression between first symptom and first examination) were significant and independent covariates of disease progression in our population. Identification of predictors of disease progression will facilitate better design of therapeutic trials, permitting the use of disease progression as a primary endpoint and improving baseline stratification of patient populations.
本研究的目的是确定一组832例确诊或疑似肌萎缩侧索硬化症（ALS）患者疾病进展的预测因素。疾病进展定义为阿佩尔ALS（AALS）评分变化20分所需的时间。采用Kaplan-Meier生存表法评估个体预后因素对疾病进展的影响。此外，使用单变量和多变量Cox比例风险分析估计每个因素的预后价值。在我们的患者群体中，AALS评分变化20分的中位时间为9个月。年龄、症状起始部位、首次症状出现至首次检查的时间、首次检查时的总AALS评分以及AALS斜率前值（首次症状出现至首次检查之间的疾病进展率）是我们群体中疾病进展的显著且独立的协变量。识别疾病进展的预测因素将有助于更好地设计治疗试验，允许将疾病进展用作主要终点，并改善患者群体的基线分层。
