Bingham Indu Navar, Norel Raquel, Roitberg Esteban G, Peller Julián, Trevisan Marcos A, Agurto Carla, Shalom Diego E, Aguirre Felipe, Embon Iair, Taitz Alan, Harris Donna, Wright Amy, Seaver Katie, Sullivan Stacey, Green Jordan R, Ostrow Lyle W, Fraenkel Ernest, Berry James D
Peter Cohen Foundation dba Everything ALS, Seattle, Washington and Los Altos, California, USA.
IBM Research Center, Yorktown Heights, NY, USA.
medRxiv. 2024 Jun 1:2024.05.31.24308140. doi: 10.1101/2024.05.31.24308140.
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative motor neuron disease that causes progressive muscle weakness. Progressive bulbar dysfunction causes dysarthria and thus social isolation, reducing quality of life. The Everything ALS Speech Study obtained longitudinal clinical information and speech recordings from 292 participants. In a subset of 120 participants, we measured speaking rate (SR) and listener effort (LE), a measure of dysarthria severity rated by speech pathologists from recordings. LE intra- and inter-rater reliability was very high (ICC 0.88 to 0.92). LE correlated with other measures of dysarthria at baseline. LE changed over time in participants with ALS (slope 0.77 pts/month; p<0.001) but not controls (slope 0.005 pts/month; p=0.807). The slope of LE progression was similar in all participants with ALS who had bulbar dysfunction at baseline, regardless of ALS site of onset. LE could be a remotely collected clinically meaningful clinical outcome assessment for ALS clinical trials.
肌萎缩侧索硬化症（ALS）是一种神经退行性运动神经元疾病，会导致进行性肌肉无力。进行性延髓功能障碍会导致构音障碍，进而造成社交隔离，降低生活质量。“全面ALS语音研究”从292名参与者那里获取了纵向临床信息和语音记录。在120名参与者的子集中，我们测量了说话速率（SR）和听众努力程度（LE），后者是言语病理学家根据录音对构音障碍严重程度进行评分的一项指标。LE在评分者内和评分者间的可靠性非常高（组内相关系数为0.88至0.92）。LE在基线时与其他构音障碍指标相关。LE在ALS患者中随时间变化（斜率为0.77分/月；p<0.001），但在对照组中无变化（斜率为0.005分/月；p=0.807）。在基线时存在延髓功能障碍的所有ALS参与者中，无论ALS的起病部位如何，LE进展的斜率相似。LE可能是一种可通过远程收集的、对ALS临床试验具有临床意义的临床结局评估指标。
