Clinical and Translational Sciences Institute, Tufts Medical Center, Boston, MA, USA.
Department of Communication Sciences and Disorders, MGH Institute of Health Professions, Charlestown, MA, USA.
Amyotroph Lateral Scler Frontotemporal Degener. 2022 May;23(3-4):271-278. doi: 10.1080/21678421.2021.1961805. Epub 2021 Aug 10.
: For patients diagnosed with ALS, dysphagia can result in aspiration, malnutrition, and mortality. The purpose of this study was to develop a clinical prediction model capable of identifying patients with ALS at imminent risk for developing swallowing complications. : A retrospective cohort study using the Pooled Resource Open-Access ALS Clinical Trials Database (PRO-ACT) was conducted. After dividing the PRO-ACT database into development and validation cohorts with dysphagia defined from the ALS Functional Rating Scale (ALSFRS), a multivariable Cox proportional hazards regression model estimated the probability of dysphagia at 3, 6, and 12-months with subsequent evaluation of model discrimination and calibration. : With 2057 participants in the development cohort and 1891 in the validation cohort, the Cox model included 7 clinical variables: spinal-onset; bulbar, fine and gross motor ALSFRS subscale scores; respiratory impairment; functional progression rate; and time from diagnosis. The cumulative incidence of dysphagia was 18% at 3-months, 29% at 6-months, and 45% at 12-months. The mean predicted probability of dysphagia development ranged from 4.5% in the bottommost risk decile to 40% in the topmost decile at 3 months, 10%-72% at 6 months, and 25%-93% at 12 months. In the validation cohort, the model had good discrimination and calibration with an optimism corrected c-statistic of 0.70 and calibration slope of 0.96. : The ALS dysphagia risk score can be used to identify patients with ALS at high risk for self-reported dysphagia development who would benefit from a comprehensive swallowing assessment and proactive dysphagia management strategies.
对于被诊断为 ALS 的患者，吞咽困难可导致吸入性肺炎、营养不良和死亡。本研究旨在开发一种临床预测模型，以识别即将发生吞咽并发症的 ALS 患者。
本研究采用回顾性队列研究，使用 Pooled Resource Open-Access ALS Clinical Trials Database (PRO-ACT) 数据库。在将 PRO-ACT 数据库根据 ALS 功能评定量表 (ALSFRS) 中的吞咽困难定义分为开发和验证队列后，多变量 Cox 比例风险回归模型估计了 3、6 和 12 个月时吞咽困难的概率，并随后评估了模型的区分度和校准度。
在开发队列中有 2057 名参与者，在验证队列中有 1891 名参与者，Cox 模型包括 7 个临床变量：脊髓发病；球部、精细和粗大运动 ALSFRS 子量表评分；呼吸障碍；功能进展率；以及从诊断到发病的时间。吞咽困难的累积发生率在 3 个月时为 18%，在 6 个月时为 29%，在 12 个月时为 45%。预测的吞咽困难发展的平均概率从风险最低的十分位数的 4.5%到最高的十分位数的 40%不等，在 3 个月时为 10%-72%，在 6 个月时为 25%-93%。在验证队列中，该模型具有良好的区分度和校准度，校正后的 C 统计量为 0.70，校准斜率为 0.96。
ALS 吞咽困难风险评分可用于识别吞咽困难风险高的 ALS 患者，这些患者需要进行全面的吞咽评估和积极的吞咽困难管理策略。
