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Novel outcome measures for Charcot−Marie−Tooth disease: validation and reliability of the 6‐min walk test and StepWatch ™ Activity Monitor and identification of the walking features related to higher quality of life
Author(s) -
Padua L.,
Pazzaglia C.,
Pareyson D.,
Sche A.,
Aiello A.,
Fabrizi G. M.,
Cavallaro T.,
Santoro L.,
Manganelli F.,
Gemignani F.,
Vitetta F.,
Quattrone A.,
Mazzeo A.,
Russo M.,
Vita G.
Publication year - 2016
Publication title -
european journal of neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.881
H-Index - 124
eISSN - 1468-1331
pISSN - 1351-5101
DOI - 10.1111/ene.13033
Subject(s) - medicine , reliability (semiconductor) , quality of life (healthcare) , clinical trial , tooth disease , disease , rehabilitation , physical therapy , test (biology) , validity , physical medicine and rehabilitation , psychometrics , clinical psychology , paleontology , power (physics) , physics , nursing , quantum mechanics , biology
Background and purpose Charcot−Marie−Tooth ( CMT ) disease is the most common inherited neuropathy, but therapeutic options have been limited to symptom management. Past pharmacological trials have failed, possibly due to insensitive outcome measures ( OM s). The aim of the current study was to evaluate the validity and reliability of the 6‐min walk test (6 MWT ) and StepWatch ™ Activity Monitoring ( SAM ) with other previously validated OM s in CMT disease. Methods A prospective multicenter study was performed, consecutively enrolling 168 CMT patients (104 with CMT 1A, 27 with CMT 1B, 37 with X‐linked CMT ) from Italian centers specializing in CMT care. Results Statistical analysis showed that the 6 MWT was highly related with all previously used OM s. Some, but not all, SAM parameters were related to commonly used OM s but may provide more information about quality of life. Conclusions The current study demonstrated the validity and reliability of the 6 MWT and SAM as OM s for CMT . Moreover, SAM provides data that correlate better with quality of life measures, making it useful in future rehabilitation trials.