Presentation (CTI)

OBJECTIVES: Amyotrophic lateral sclerosis (ALS) is a rare progressive neurodegenerative that rapidly impairs both lower and upper motor neurons. A variety of outcome measures are used to assess the functional ability and quality of life of ALS patients. However, clinical outcome assessments (COAs) are reported inconsistently and vary between randomized controlled trials (RCTs) and observational studies. Therefore, we aimed to evaluate and contrast the COAs utilized across them.
METHODS: We performed a scoping review to identify both RCTs and observational studies that reported COAs in adult ALS patients. We searched PubMed and Embase upto May 2025 and supplemented it with a targeted search.
RESULTS: A total of 3,781 citations were screened to identify 22 RCTs and 31 observational studies reporting different COAs were identified. A greater variety of COAs were evaluated in observational studies (18) compared to RCTs (7). Across both the study designs, Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) was commonly used to assess functional ability. The Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ) was the most commonly reported patient-reported outcome measure (PROM) in RCTs while both ALSAQ and Amyotrophic Lateral Sclerosis Quality of Life (ALSQoL) were frequently assessed in observational studies. The baseline ALSFRS-R scores were similar across the study designs, and it ranged from [Mean, 36-42] in RCTs and [Mean, 32-37] in observational studies. When approved treatments for ALS were assessed with the same tool in both RCTs and observational studies, the direction of treatment effect was inconsistent. A lesser reduction of ALSFRS-R scores was observed in RCTs as compared to the observational studies.
CONCLUSIONS: Variety of COAs limit the ability to explore a standardized tool in observational setting. Patient improvement reported in COAs were pronounced in RCTs while observational studies didn’t show substantial benefit in real-world settings. This necessitates assessment of the confounding factors or selection bias causing these differences.