Predictability of disease progression in amyotrophic lateral sclerosis
Predictability of disease progression in amyotrophic lateral sclerosis
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DOI：
10.1002/mus.20658
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发表时间：
2006-12-01
期刊：
影响因子：
3.4
通讯作者：
Appel, Stanley H.
中科院分区：
文献类型：
--
作者：
Czaplinski, Adam;Yen, Albert A.;Appel, Stanley H.
这项研究的目的是在832名确诊或可能患有肌萎缩侧索硬化症(ALS)的患者中确定疾病进展的预测因素。疾病进展定义为AALS(AALS)评分改变20分的时间。用Kaplan-Meier寿命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比例风险分析来估计每个因素的预后价值。在我们的患者群体中，AALS评分改变20分的中位时间为9个月。年龄、起病部位、首发至初诊时间、初诊AALS总分和AALS前期(首发症状至初诊疾病进展率)是影响疾病进展的显著独立协变量。确定疾病进展的预测因素将有助于更好地设计治疗试验，允许使用疾病进展作为主要终点，并改善患者群体的基线分层。
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.