Clinical characteristics of a large cohort of US participants enrolled in the National Amyotrophic Lateral Sclerosis (ALS) Registry, 2010-2015
Clinical characteristics of a large cohort of US participants enrolled in the National Amyotrophic Lateral Sclerosis (ALS) Registry, 2010-2015
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DOI：
10.1080/21678421.2019.1612435
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发表时间：
2019-07-03
影响因子：
2.8
通讯作者：
Horton, Kevin
中科院分区：
文献类型：
--
作者：
Raymond, Jaime;Oskarsson, Bjorn;Horton, Kevin
背景：肌萎缩侧索硬化症（ALS）是一种进行性致死性疾病，具有不同的临床特征。目的：描述国家ALS登记处招募的大型ALS参与者队列的临床特征。方法：对2010-2015年期间完成登记处在线临床调查模块的ALS参与者的数据进行分析，以确定特征，如发病部位、相关症状、症状发作至诊断时间、诊断至临终关怀转诊时间以及药物和非药物干预。结果：在完成调查的1758名参与者中，60.9%为男性，62.1%为50-69岁，95.5%为白色。大约72.0%的患者报告了最初的肢体无力发作，其次是延髓（22.1%）和躯干/全身发作（6.1%）。曾出现的其他症状包括痉挛（56.7%）、肌束震颤（56.3%）和构音障碍（33.0%）。从肌肉痉挛增加到ALS诊断之间的中位时间为12个月;与延髓发作的参与者相比，肢体发作的参与者在诊断前的痉挛时间更长。最常使用的干预措施包括利鲁唑（48.3%目前正在使用）、轮椅/踏板车（32.8%）和无创呼吸设备（30.0%）。躯干/全球发病的参与者被称为临终关怀几乎四倍比others.Conclusions：这些数据显示ALS的临床特征有很大的不同，在一个大的队列参与者之前的诊断，并反映在疾病的发病，进展和预后的变化。更好地表征症状发作可能有助于临床医生更快地诊断ALS，这可能导致更早的治疗干预。
Background:Amyotrophic lateral sclerosis (ALS) is a progressive fatal disease with a varying range of clinical characteristics.Objective:To describe the clinical characteristics in a large cohort of ALS participants enrolled in the National ALS Registry.Methods:Data from ALS participants who completed the Registry's online clinical survey module during 2010-2015 were analyzed to determine characteristics, such as site of onset, associated symptoms, time of symptom onset to diagnosis, time of diagnosis to hospice referral, and pharmacological and non-pharmacological interventions.Results:Of the 1758 participants who completed the survey, 60.9% were male, 62.1% were 50-69 years old, and 95.5% white. Approximately, 72.0% reported initial limb weakness onset of disease, followed by bulbar (22.1%), and trunk/global onset (6.1%). Other symptoms ever experienced included cramps (56.7%), fasciculations (56.3%), and dysarthria (33.0%). The median time between an increase of muscle cramps until an ALS diagnosis was 12 months; limb onset participants had cramps longer preceding diagnosis versus those with bulbar onset. The most frequent interventions used included riluzole (48.3% currently using), wheelchairs/scooters (32.8%), and noninvasive breathing equipment (30.0%). Participants with trunk/global onset were referred to hospice almost four times earlier than others.Conclusions:These data show how ALS clinical characteristics differ widely in a large cohort of participants preceding diagnosis and reflect variations in disease onset, progression, and prognosis. Better characterization of symptom onset may assist clinicians in diagnosing ALS sooner, which could lead to earlier therapeutic interventions.