文章基本信息

标题：Modelling disease course in amyotrophic lateral Sclerosis: pseudo-longitudinal insights from cross-sectional health-related quality of life data
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作者：Tino Prell ; Nayana Gaur ; Robert Steinbach 等
期刊名称：Health and Quality of Life Outcomes
印刷版ISSN：1477-7525
电子版ISSN：1477-7525
出版年度：2020
卷号：18
期号：1
页码：1-5
DOI：10.1186/s12955-020-01372-6
出版社：BioMed Central
摘要：Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive neurodegenerative disorder with limited robust disease-modifying therapies presently available. While several treatments are aimed at improving health-related quality of life (HRQoL), longitudinal data on how QoL changes across the disease course are rare. To explore longitudinal changes in emotional well-being and HRQoL in ALS. Of the 161 subjects initially recruited, 39 received 2 subsequent follow-up assessments at 6 and 12 months after baseline. The ALS Functional Rating Scale-Revised (ALSFRS-R) was used to assess physical impairment. HRQoL was assessed using the ALS Assessment Questionnaire (ALSAQ-40). The D50 disease progression model was applied to explore longitudinal changes in HRQoL. Patients were primarily in the early semi-stable and early progressive model-derived disease phases. Non-linear correlation analyses showed that the ALSAQ-40 summary index and emotional well-being subdomain behaved differently across disease phases, indicating that the response shift occurs early in disease. Both the ALSFRS-R and ALSAQ-40 significantly declined at 6- and 12-monthly follow-ups. ALSAQ-40 summary index and emotional well-being change comparably over both actual time and model-derived phases, indicating that the D50 model enables pseudo-longitudinal interpretations of cross-sectional data in ALS.
关键词：Disease aggressiveness;Health-related quality of life;Longitudinal modelling