Development and evaluation of a clinical staging system for amyotrophic lateral sclerosis
Development and evaluation of a clinical staging system for amyotrophic lateral sclerosis
复制标题
DOI：
10.1136/jnnp-2013-306589
复制
发表时间：
2015-01-01
影响因子：
11
通讯作者：
Filippini, Graziella
中科院分区：
文献类型：
--
作者：
Chio, Adriano;Hammond, Edward R.;Filippini, Graziella
疾病严重程度的分期对于预后、决策和资源规划是有用的。然而，没有常用的，有效的分期系统存在肌萎缩侧索硬化症（ALS）。我们的目的是开发一个ALS分期系统（ALS米兰-都灵分期），捕捉观察到的渐进性丧失的独立性和functions.Methods临床里程碑在ALS进展的定义在ALS功能评定量表（ALSFRS）的四个关键领域的独立性损失：吞咽，行走/自我护理，沟通和呼吸。阶段定义如下：阶段0，功能参与但在任何领域都没有丧失独立性;阶段1-4，丧失独立性的领域数量;阶段5，死亡。对入组ALS护理质量（QOC）研究的患者应用分期标准;终点包括功能（ALSFRS）、生活质量（QOL;简表-36）和医疗服务费用。阶段之间的转换概率进行了评估，在QOC研究和碳酸锂在ALS.Results的第二个临床研究70/118（59.3%）的参与者在QOC研究进展到更高的疾病阶段，在12个月相比，他们的基线阶段。功能（ALSFRS）和QOL指标与疾病分期呈负相关。健康服务费用与疾病分期从0增加到4直接相关（p
Background Staging of disease severity is useful for prognosis, decision-making and resource planning. However, no commonly used, validated staging system exists for amyotrophic lateral sclerosis (ALS). Our purpose was to develop an ALS staging system (ALS Milano-Torino Staging) that captures the observed progressive loss of independence and function.Methods Clinical milestones in ALS progression were defined by loss of independence in four key domains on the ALS Functional Rating Scale (ALSFRS): swallowing, walking/self-care, communicating and breathing. Stages were defined as follows: stage 0, functional involvement but no loss of independence on any domain; stages 1-4, number of domains in which independence was lost; and stage 5, death. Staging criteria were applied to patients enrolled in a Quality of Care in ALS (QOC) study; endpoints included function (ALSFRS), quality of life (QOL; Short Form-36) and health service costs. Between-stage transition probabilities were assessed in the QOC study and in a second clinical study of lithium carbonate in ALS.Results 70/118 (59.3%) participants in the QOC study progressed to higher stages of disease at 12 months compared with their baseline stage. Functional (ALSFRS) and QOL measures were inversely related to disease stage. Health service costs were directly related to increasing disease stages from 0 to 4 (p