Department of Neurosciences, IRCSS, Mario Negri Institute of Pharmacological Research, Milan.
Ann Neurol. 2014 Feb;75(2):287-97. doi: 10.1002/ana.24096. Epub 2014 Feb 24.
To determine the long-term survival in amyotrophic lateral sclerosis (ALS) and identify predictors of prolonged survival in a population-based cohort of newly diagnosed patients.
An incident cohort from a population-based registry during the years 1998 through 2002 in Lombardy, Italy was followed until death or to February 28, 2013. Age, sex, date of onset of symptoms, site of onset, date of diagnosis, and El Escorial diagnostic category were collected. Survival was assessed using Kaplan-Meier curves. Cox proportional hazards function was used to identify independent prognostic predictors. Standardized mortality ratios (SMRs) were used to assess the 5-year and 10-year excess mortality of ALS patients.
Included were 280 men and 203 women aged 18 to 93 years. Spinal onset ALS was present in 312 cases (64.6%). Definite ALS was diagnosed in 213 cases (44.1%), probable ALS in 130 (26.9%), possible ALS in 93 (19.3%), and suspected ALS in 47 (9.7%). The cumulative time-dependent survival at 1, 5, and 10 years from diagnosis was 76.2%, 23.4%, and 11.8%, respectively. Independent predictors included younger age, the diagnosis of possible/suspected ALS, spinal onset, and symptoms having started >12 months previously at diagnosis. SMR was 9.4 at 5 years and 5.4 at 10 years. SMR at 10 years was higher until age 75 year, predominating in women, and became nonsignificant for males thereafter.
The outcome in ALS varies with phenotype. Longer survival is predicted by younger age, spinal onset, male gender, and suspected ALS. After age 75 years, 10-year survival in men with ALS is similar to the general population.
确定肌萎缩侧索硬化症（ALS）的长期生存率，并确定新诊断患者人群中延长生存的预测因素。
意大利伦巴第地区一个基于人群的登记处于 1998 年至 2002 年期间收集了一个发病队列，随访至死亡或 2013 年 2 月 28 日。收集了年龄、性别、症状发作日期、发作部位、诊断日期和 El Escorial 诊断类别。使用 Kaplan-Meier 曲线评估生存情况。使用 Cox 比例风险函数确定独立的预后预测因素。使用标准化死亡率比（SMR）评估 ALS 患者的 5 年和 10 年超额死亡率。
纳入了 280 名男性和 203 名年龄在 18 至 93 岁之间的女性。312 例（64.6%）为脊髓发病 ALS。213 例（44.1%）明确诊断为 ALS，130 例（26.9%）可能诊断为 ALS，93 例（19.3%）可疑诊断为 ALS，47 例（9.7%）疑似诊断为 ALS。从诊断起，1、5 和 10 年的累积时间依赖性生存率分别为 76.2%、23.4%和 11.8%。独立预测因素包括年龄较小、可能/疑似 ALS 的诊断、脊髓发病和症状在诊断前 12 个月以上开始。5 年时的 SMR 为 9.4，10 年时为 5.4。10 年 SMR 在 75 岁之前较高，主要见于女性，此后对于男性则不再显著。
ALS 的结局因表型而异。年轻、脊髓发病、男性和疑似 ALS 预测生存率延长。75 岁后，ALS 男性的 10 年生存率与一般人群相似。
