Reconsidering the revised amyotrophic lateral sclerosis functional rating scale for ALS clinical trials

Introduction

Clinical trials in amyotrophic lateral sclerosis (ALS) aim to identify treatments that can slow functional decline or prolong survival time. In the absence of objective biomarkers, questionnaires, such as the ALS Functional Rating Scale (ALSFRS), are used to evaluate treatment effects in clinical trials.1 Although questionnaires are easy to administer and hold prognostic value, they may miss important treatment clues if questions are not selected appropriately. For example, the ALSFRS was originally designed as a 10-item functional score across bulbar, fine and gross motor and respiratory domains. The ALSFRS was revised in 1999 by adding two respiratory items.1 Whether this revision resulted in an improved endpoint is debatable, as the questionnaire’s construct validity may have worsened, resulting in weaker correlations with respiratory function and survival.1 2 Nevertheless, the ultimate, real-world application of the ALSFRS is to serve as efficacy endpoint. Solely…

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