The ALSFRS as an outcome measure in therapeutic trials and its relationship to symptom onset.

ABSTRACT: The reduction in ALS Functional Rating Score (ALSFRS) from reported symptom onset to diagnosis is used to estimate rate of disease progression. ALSFRS decline may be non-linear or distorted by drop-outs in therapeutic trials, reducing the reliability of change in slope as an outcome measure. The PRO-ACT database uniquely allows such measures to be explored using historical data from negative therapeutic trials. The decline of functional scores was analysed in 18 pooled trials, comparing rates of decline based on symptom onset with rates calculated between interval assessments. Strategies to mitigate the effects of trial drop-out were considered. Results showed that progression rate calculated by symptom onset underestimated the subsequent rate of disability accumulation, although it predic

SUBMITTER: Proudfoot M 

PROVIDER: S-EPMC4950444 | biostudies-literature | 2016 Jul-Aug

REPOSITORIES: biostudies-literature