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

for the CMT-TRIAAL Group

Research output: Contribution to journalArticle

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.

Original languageEnglish
Pages (from-to)310-316
Number of pages7
JournalNeuromuscular Disorders
Volume29
Issue number4
DOIs
Publication statusPublished - Apr 1 2019

Fingerprint

Longitudinal Studies
Outcome Assessment (Health Care)
Multicenter Studies
Quality of Life
Pharmacology
Drug Therapy
Pain
Walk Test
Therapeutics

Keywords

  • Charcot–Marie–Tooth
  • Outcome measures
  • Rehabilitation
  • Sensitivity to changes

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Neurology
  • Clinical Neurology
  • Genetics(clinical)

Cite this

@article{5a262da665b743d682f3df1f0d1b6c55,
title = "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 < 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",
author = "{for the CMT-TRIAAL Group} and Costanza Pazzaglia and Luca Padua and Davide Pareyson and Angelo Schenone and Alessia Aiello and Fabrizi, {Gian Maria} and Tiziana Cavallaro and Lucio Santoro and Fiore Manganelli and Daniele Coraci and Franco Gemignani and Francesca Vitetta and Aldo Quattrone and Anna Mazzeo and Massimo Russo and Giuseppe Vita",
year = "2019",
month = "4",
day = "1",
doi = "10.1016/j.nmd.2019.01.009",
language = "English",
volume = "29",
pages = "310--316",
journal = "Neuromuscular Disorders",
issn = "0960-8966",
publisher = "Elsevier Ltd",
number = "4",

}

TY - JOUR

T1 - 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

AU - for the CMT-TRIAAL Group

AU - Pazzaglia, Costanza

AU - Padua, Luca

AU - Pareyson, Davide

AU - Schenone, Angelo

AU - Aiello, Alessia

AU - Fabrizi, Gian Maria

AU - Cavallaro, Tiziana

AU - Santoro, Lucio

AU - Manganelli, Fiore

AU - Coraci, Daniele

AU - Gemignani, Franco

AU - Vitetta, Francesca

AU - Quattrone, Aldo

AU - Mazzeo, Anna

AU - Russo, Massimo

AU - Vita, Giuseppe

PY - 2019/4/1

Y1 - 2019/4/1

N2 - 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.

AB - 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.

KW - Charcot–Marie–Tooth

KW - Outcome measures

KW - Rehabilitation

KW - Sensitivity to changes

UR - http://www.scopus.com/inward/record.url?scp=85063335346&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85063335346&partnerID=8YFLogxK

U2 - 10.1016/j.nmd.2019.01.009

DO - 10.1016/j.nmd.2019.01.009

M3 - Article

C2 - 30926199

AN - SCOPUS:85063335346

VL - 29

SP - 310

EP - 316

JO - Neuromuscular Disorders

JF - Neuromuscular Disorders

SN - 0960-8966

IS - 4

ER -