中国散发型肌萎缩侧索硬化的自然病史和临床特征

Objectives To describe the natural history and clinical features of sporadic amyotrophic lateral sclerosis (ALS) in Chinese patients, and to report data on the prognostic factors for survival. Methods All patients referred to our ALS centre between 2003 and 2012 were followed up every 3 months. Survival and tracheotomy were predefined as primary outcome measures. Group differences were analysed using parametric and non-parametric tests as appropriate. Survival was analysed using the Kaplan-Meier method and Cox regression analysis. Results Of the 1624 patients with ALS, 75.1% had limb-onset, 14.0% had bulbar-onset, 7.8% had flail-arm syndrome (FAS), 2.6% had progressive muscular atrophy and 0.5% had primary lateral sclerosis. The male:female ratio was 1.7:1, and the mean age at onset was 49.8 years. The median diagnostic delay was 14 months, and the median survival time after symptom onset was 71 months. Male gender, older age at symptom onset, lower body mass index, shorter diagnostic delay, bulbar-onset ALS phenotype, higher Airlie House category at presentation, rural place of residence, use of traditional Chinese medicine and a history of contact with pesticides were associated with poorer survival, whereas female gender or an FAS phenotype may have a better prognosis. Conclusions The clinical characteristics and outcomes of Chinese patients with sporadic ALS were different compared with patients from other countries. Compared with other studies, the age at onset of Chinese patients was earlier, the percentage of bulbar-onset ALS was lower and the prognosis was better. This study substantially advances the understanding of the clinical features and epidemiology of this rare disease.