Comparing the ability of various compositive outcomes to discriminate treatment effects in MS clinical trials

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Abstract
We compared the ability of the Kurtzke Expanded Disability Status Scale (EDSS) and a composite outcome of non-physician-based measures of time to ambulate 25 feet (TA) and manual dexterity (the Box and Block Test [BBT], and 9-Hole Peg Test [9HPT]) to discriminate treatment effects in the Phase III study of interferon beta-1a. A log-rank comparison of Kaplan-Meier curves by treatment group showed the non-physicianbased composite of BBT, 9HPT, and TA was of comparable sensitivity (P=0.013) in discriminating sustained treatment failure as the EDSS alone (P=0.029). The composite of BBT, 9HPT, TA, and EDSS was more sensitive (P=0.009) in discriminating sustained treatment failure than the EDSS alone. Compositive outcomes of the EDSS and non-physician-based measures of manual dexterity and timed ambulation provide an appealing strategy to reduce the number of patients required to discriminate treatment effects in MS clinical trials.