Requardt Maria Viktoria, Görlich Dennis, Grehl Torsten, Boentert Matthias
Department of Neurology with Institute of Translational Neurology, Münster University Hospital (UKM), 48149 Münster, Germany.
Institute for Biostatistics and Clinical Research, Münster University Hospital, 48149 Muenster, Germany.
J Clin Med. 2021 Apr 12;10(8):1623. doi: 10.3390/jcm10081623.
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that is ultimately fatal but characterized by substantial phenotypic heterogeneity, which is known to impact long-term course and survival. This study investigated clinical determinants of disease progression and outcome in a large cohort of patients with ALS.
Retrospective analysis included comprehensive data from 625 patients who attended a tertiary ALS centre at least twice. Patients were stratified according to five distinct clinical phenotypes: classical ALS; bulbar ALS; ALS with frontotemporal dementia (ALS-FTD); upper motor neuron predominant (UMNP); and lower motor neuron predominant (LMNP).
This study confirmed higher age at symptom onset, shorter latency to diagnosis and more rapid decline in the revised ALS Functional Rating Scale sum score as predictors of poor prognosis. Hazard ratios for shorter survival were higher in patients with ALS-FTD versus classical ALS, and in patients with versus without chronic obstructive pulmonary disease (COPD). Mean survival was longest in the UMNP phenotype group.
This study confirmed established predictors of shorter survival in ALS and showed that concomitant COPD in particular relates to poor outcome.
肌萎缩侧索硬化症（ALS）是一种神经退行性疾病，最终会导致死亡，但其具有显著的表型异质性，已知这种异质性会影响疾病的长期病程和生存情况。本研究调查了一大群ALS患者疾病进展和预后的临床决定因素。
回顾性分析纳入了625名至少两次就诊于三级ALS中心的患者的综合数据。患者根据五种不同的临床表型进行分层：经典型ALS；延髓型ALS；伴额颞叶痴呆的ALS（ALS-FTD）；以上运动神经元为主型（UMNP）；以下运动神经元为主型（LMNP）。
本研究证实，症状出现时年龄较大、诊断延迟时间较短以及修订的ALS功能评定量表总分下降更快是预后不良的预测因素。与经典型ALS相比，ALS-FTD患者以及患有慢性阻塞性肺疾病（COPD）的患者与未患COPD的患者相比，生存时间较短的风险比更高。UMNP表型组的平均生存期最长。
本研究证实了ALS患者生存时间较短的既定预测因素，并表明合并COPD尤其与不良预后相关。
