Are novel outcome measures for Charcot–Marie–Tooth disease sensitive to change? The 6-minute walk test and StepWatch™ Activity Monitor in a 12-month longitudinal study

Abstract Charcot–Marie–Tooth (CMT) is the most common inherited neuropathy, yet has no available pharmacological therapy. Past pharmacotherapy trials failed to provide positive results, possibly due to a poor choice of outcome measures. We previously performed a study in which we validated the 6-minute walk test and StepWatch™ Activity Monitor in CMT. The aim of the current study was to determine if these outcome measures are sensitive to change over a 12-month period. In this longitudinal multicenter study, 149 out of 169 initially enrolled patients were re-evaluated after 12 months using the 6-minute walk test, StepWatch™ Activity Monitor and other outcome measures commonly adopted in CMT disease. Statistical analysis showed a worsening of the CMT-Neuropathy Score ( p p p p  > 0.05), muscular strength as detected by clinical evaluation ( p  > 0.05), 6-minute walk test ( p  > 0.05), pain ( p  > 0.05) and quality of life ( p  > 0.05) showed no change. In the current study, patients showed clinical worsening over 12 months, confirmed by a reduction of activity as detected by StepWatch™ Activity Monitor. The 6-minute walk test failed to detect change.