Outcome measures and prognostic indicators in patients with amyotrophic lateral sclerosis
Outcome measures and prognostic indicators in patients with amyotrophic lateral sclerosis
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DOI：
10.1080/17482960801942695
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发表时间：
2008-01-01
影响因子：
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通讯作者：
Micheli, Anna
中科院分区：
文献类型：
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作者：
Beghi, Ettore;Millul, Andrea;Micheli, Anna
这项研究的目的是评估ALS患者残疾测量的频率和预测因素。在基于人口的登记中，居住在意大利伦巴第八个行政区(人口4947,554人)的114名新诊断患者接受了2570个人月(平均22.5个月)的跟踪调查。根据Kaplan-Meier方法评估轮椅、经皮内窥镜胃造瘘术和辅助呼吸的累积时间相关性风险。用COX比例风险函数评估残疾的预测因素(年龄、性别、确诊时的病程、发病类型、EL-Ecore诊断)。在随访期间，29例(25.4%)患者被轮椅束缚，51例(44.7%)接受胃造口术，47例(41.2%)接受辅助机械通气。中位行走时间为46.7个月(95%可信区间为36.5~56.8)。中位胃造瘘期为31.1个月(95%可信区间26.8~35.4)，中位辅助通气期为34.6个月(95%可信区间29.6~39.6)。脊椎型肌萎缩侧索硬化症是步态障碍的唯一预测因子。胃造瘘术的预测因素是年龄较大、明确的ALS和较短的病程。较短的病程是辅助呼吸机的唯一预测因子。总而言之，肌萎缩侧索硬化症患者在残疾的衡量标准和预测因素方面有所不同。这些因素是随机临床试验中偏差和混淆的来源。
The purpose of the study was to assess frequency and predictors of disability measures in ALS. One hundred and fourteen newly diagnosed patients resident in eight administrative districts of Lombardy, Italy (population 4,947,554), included in a population-based registry, were followed for 2570 person-months (mean 22.5 months). The cumulative time-dependent risk of wheelchair, percutaneous endoscopic gastrostomy, and assisted ventilation was estimated according to the Kaplan-Meier method. Predictors of disability (age, sex, disease duration at diagnosis, type of onset, El-Escorial diagnosis) were assessed with the Cox proportional hazard function. During follow-up, 29 patients (25.4%) became wheelchair bound, 51 (44.7%) received gastrostomy, and 47 (41.2%) received assisted ventilation. The median time to loss of ambulation was 46.7 months (95% CI 36.5-56.8). The median time to gastrostomy and assisted ventilation was 31.1 months (95% CI 26.8-35.4) and 34.6 months (95% CI 29.6-39.6), respectively. Spinal onset ALS was the only predictor of loss of ambulation. Predictors of gastrostomy were older age, definite ALS, and shorter disease duration. Shorter disease duration was the only predictor of assisted ventilation. In conclusion, patients with ALS differ in terms of measures and predictors of disability. These factors are sources of bias and confounding in randomized clinical trials.