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Multicenter Study
. 2019 Apr;29(4):310-316.
doi: 10.1016/j.nmd.2019.01.009. Epub 2019 Feb 2.

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

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Multicenter Study

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

Costanza Pazzaglia et al. Neuromuscul Disord. .

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 < 0.05), strength of distal muscles measured by myometry (p < 0.05) and StepWatch™ Activity Monitor outputs (p < 0.05). The 10 meter walking test (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.

Keywords: Charcot–Marie–Tooth; Outcome measures; Rehabilitation; Sensitivity to changes.

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