Guangdong Provincial Key Laboratory of Diagnosis and Treatment of Major Neurological Diseases, National Key Clinical Department and Key Discipline of Neurology, Department of Neurology, The First Affiliated Hospital, Sun Yat-sen University, No. 58 Zhongshan Road 2, Guangzhou 510080, China.
Cells. 2021 May 17;10(5):1220. doi: 10.3390/cells10051220.
(1) Background: The aim of this longitudinal study was to evaluate the association between disease progression according to the Milano-Torino staging (MITOS) system and long-term survival in Chinese patients with amyotrophic lateral sclerosis (ALS). We also examined factors affecting MITOS progression. (2) Methods: Patients were enrolled and underwent follow-up at 6, 12, 18, and 24 months, and their demographic and clinical data, including the Milano-Torino stage, Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score and neuropsychiatric data, were evaluated. The sensitivity and specificity of predicting survival outcomes based on MITOS progression and ALSFRS-R score decline from baseline to 6 months were compared. The associations between MITOS progression from baseline to 6 months and survival outcome at 12, 18 and 24 months were examined, and factors associated with disease progression were evaluated with subgroup analyses. (3) Results: Among the 100 patients included, 74% were in stage 0 at baseline, and approximately 95% progressed to a higher stage of the MITOS system at 24 months. MITOS progression from baseline to 6 months and ALSFRS-R decline showed comparable value for predicting survival at 12, 18, and 24 months. MITOS progression from baseline to 6 months is strongly associated with death outcomes. Older age at onset and increased depression and anxiety scores may be related to disease progression. (4) Conclusions: MITOS progression during the early disease course could serve as a prognostic marker of long-term survival and may have utility in clinical trials. Age at onset and diagnosis and neuropsychiatric factors might be associated with disease progression.
(1) 背景：本纵向研究旨在评估根据米兰-都灵分期（MITOS）系统评估的疾病进展与中国肌萎缩侧索硬化症（ALS）患者长期生存之间的关联。我们还检查了影响 MITOS 进展的因素。(2) 方法：患者在 6、12、18 和 24 个月时接受登记和随访，并评估其人口统计学和临床数据，包括米兰-都灵分期、肌萎缩侧索硬化功能评定量表修订版（ALSFRS-R）评分和神经心理学数据。比较基于 MITOS 进展和从基线到 6 个月的 ALSFRS-R 评分下降预测生存结果的敏感性和特异性。检查从基线到 6 个月的 MITOS 进展与 12、18 和 24 个月的生存结果之间的关联，并通过亚组分析评估与疾病进展相关的因素。(3) 结果：在纳入的 100 例患者中，74%的患者在基线时处于 0 期，大约 95%的患者在 24 个月时进展到更高的 MITOS 系统分期。从基线到 6 个月的 MITOS 进展和 ALSFRS-R 下降对于预测 12、18 和 24 个月的生存具有相似的价值。从基线到 6 个月的 MITOS 进展与死亡结局密切相关。发病年龄较大以及抑郁和焦虑评分增加可能与疾病进展有关。(4) 结论：在疾病早期阶段的 MITOS 进展可以作为长期生存的预后标志物，并且可能在临床试验中有用。发病年龄和诊断以及神经心理学因素可能与疾病进展有关。
