G Piscosquito, M M Reilly, A Schenone, G M Fabrizi, T Cavallaro, L Santoro, F Manganelli, G Vita, A Quattrone, L Padua, F Gemignani, F Visioli, M Laurà, D Calabrese, R A C Hughes, D Radice, A Solari, D Pareyson
BACKGROUND AND PURPOSE: Charcot-Marie-Tooth disease (CMT) is a very slowly progressive neuropathy which makes it difficult to detect disease progression over time and to assess intervention efficacy. Experience from completed clinical trials with ascorbic acid and natural history studies confirm difficulties in detecting such changes. Consequently, sensitive-to-change outcome measures (OMs) are urgently needed. METHODS: The relative responsiveness of clinical scales of the Italian-UK ascorbic acid trial (placebo arm) were assessed by using the standardized response mean (SRM), which is the ratio of the paired scores mean change over time to the standard deviation of the score change (0 is worst responsiveness)...
December 2015: European Journal of Neurology