A proposed staging system for amyotrophic lateral sclerosis.
A proposed staging system for amyotrophic lateral sclerosis.
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DOI：
10.1093/brain/awr351
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发表时间：
2012-03
期刊：
影响因子：
--
通讯作者：
Al-Chalabi A
中科院分区：
文献类型：
--
作者：
Roche JC;Rojas-Garcia R;Scott KM;Scotton W;Ellis CE;Burman R;Wijesekera L;Turner MR;Leigh PN;Shaw CE;Al-Chalabi A
肌萎缩侧索硬化症是一种神经退行性疾病，其特征在于上、下运动神经元的进行性丧失，中位生存期为2-3年。虽然存在各种表型和研究诊断分类系统，并且已经产生了几个预后模型，但没有分期系统。肌萎缩侧索硬化症的分期标准将有助于提供一个普遍和客观的疾病进展的措施，有利于病人护理，资源分配，研究分类和临床试验设计。因此，我们试图定义容易识别的临床里程碑，这些里程碑可以显示在疾病过程中的特定点发生，反映疾病进展并影响预后和治疗。分析了三级转诊中心的临床数据库，包括1993年至2007年1471例肌萎缩侧索硬化症患者。癫痫定义为症状发作（一个中枢神经系统区域（定义为延髓、上肢、下肢或延髓）的虚弱、消瘦、痉挛、构音障碍或吞咽困难的功能受累）、诊断、第二个区域的功能受累、第三个区域的功能受累、需要胃造口术和无创通气。使用死亡患者的信息，通过将里程碑时间除以疾病持续时间，将里程碑时间标准化为疾病过程中经过的时间比例。在疾病过程中，粟粒细胞以可预测的比例发生。在整个疾病过程中，诊断发生在35%，累及第二区域为38%，第三区域为61%，需要胃造口术为77%，需要无创通气为80%。因此，我们提出一个简单的分期系统肌萎缩侧索硬化症。第一阶段：症状发作（累及第一区域）;阶段2A：诊断;阶段2B：累及第二区域;阶段3：累及第三区域;阶段4A：需要胃造口术;和阶段4 B：需要无创通气。该分期系统的验证需要在其他人群、人群登记和其他临床数据库中进行进一步研究。里程碑的标准化时间在不同的研究和人群之间可能会有所不同，尽管阶段本身及其含义可能保持不变。
Amyotrophic lateral sclerosis is a neurodegenerative disorder characterized by progressive loss of upper and lower motor neurons, with a median survival of 2–3 years. Although various phenotypic and research diagnostic classification systems exist and several prognostic models have been generated, there is no staging system. Staging criteria for amyotrophic lateral sclerosis would help to provide a universal and objective measure of disease progression with benefits for patient care, resource allocation, research classifications and clinical trial design. We therefore sought to define easily identified clinical milestones that could be shown to occur at specific points in the disease course, reflect disease progression and impact prognosis and treatment. A tertiary referral centre clinical database was analysed, consisting of 1471 patients with amyotrophic lateral sclerosis seen between 1993 and 2007. Milestones were defined as symptom onset (functional involvement by weakness, wasting, spasticity, dysarthria or dysphagia of one central nervous system region defined as bulbar, upper limb, lower limb or diaphragmatic), diagnosis, functional involvement of a second region, functional involvement of a third region, needing gastrostomy and non-invasive ventilation. Milestone timings were standardized as proportions of time elapsed through the disease course using information from patients who had died by dividing time to a milestone by disease duration. Milestones occurred at predictable proportions of the disease course. Diagnosis occurred at 35% through the disease course, involvement of a second region at 38%, a third region at 61%, need for gastrostomy at 77% and need for non-invasive ventilation at 80%. We therefore propose a simple staging system for amyotrophic lateral sclerosis. Stage 1: symptom onset (involvement of first region); Stage 2A: diagnosis; Stage 2B: involvement of second region; Stage 3: involvement of third region; Stage 4A: need for gastrostomy; and Stage 4B: need for non-invasive ventilation. Validation of this staging system will require further studies in other populations, in population registers and in other clinic databases. The standardized times to milestones may well vary between different studies and populations, although the stages themselves and their meanings are likely to remain unchanged.
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影响因子：
4.4
作者：
Cedarbaum, JM;Stambler, N;Nakanishi, A
通讯作者：
Nakanishi, A
影响因子：
9.9
作者：
Wijesekera, L. C.;Mathers, S.;Leigh, P. N.
通讯作者：
Leigh, P. N.
DOI：
10.1093/biostatistics/1.4.465
发表时间：
2000-12-01
期刊：
Biostatistics (Oxford, England)
影响因子：
--
作者：
Henderson, R;Diggle, P;Dobson, A
通讯作者：
Dobson, A
影响因子：
11
作者：
Huisman, Mark H. B.;de Jong, Sonja W.;van den Berg, Leonard H.
通讯作者：
van den Berg, Leonard H.
DOI：
10.3109/17482960903420140
发表时间：
2010-08
期刊：
Amyotrophic lateral sclerosis : official publication of the World Federation of Neurology Research Group on Motor Neuron Diseases
影响因子：
--
作者：
Turner MR;Brockington A;Scaber J;Hollinger H;Marsden R;Shaw PJ;Talbot K
通讯作者：
Talbot K