Refining clinical trial inclusion criteria to optimize the standardized response mean of the CMTPedS.
Journal
Annals of clinical and translational neurology
ISSN: 2328-9503
Titre abrégé: Ann Clin Transl Neurol
Pays: United States
ID NLM: 101623278
Informations de publication
Date de publication:
09 2020
09 2020
Historique:
received:
27
06
2020
accepted:
13
07
2020
pubmed:
8
8
2020
medline:
21
10
2021
entrez:
8
8
2020
Statut:
ppublish
Résumé
The CMT Pediatric Scale (CMTPedS) is a reliable, valid, and responsive clinical outcome measure of disability in children with CMT. The aim of this study was to identify the most responsive patient subset(s), based on the standardized response mean (SRM), to optimize the CMTPedS as a primary outcome measure for upcoming clinical trials. Analysis was based on a 2-year natural history data from 187 children aged 3-20 years with a range of CMT genetic subtypes. Subsets based on age (3-8 years), disability level (CMTPedS score 0-14), and CMT type (CMT1A) increased the SRM of the CMTPedS considerably. Refining the inclusion criteria in clinical trials to younger, mildly affected cases of CMT1A optimizes the responsiveness of the CMTPedS.
Identifiants
pubmed: 32762141
doi: 10.1002/acn3.51145
pmc: PMC7480902
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1713-1715Informations de copyright
© 2020 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association.
Références
Ann Neurol. 2012 May;71(5):642-52
pubmed: 22522479
Ther Clin Risk Manag. 2019 Oct 02;15:1153-1161
pubmed: 31632042
Neuromuscul Disord. 2019 Nov;29(11):842-856
pubmed: 31704158
Ann Neurol. 2017 Sep;82(3):353-359
pubmed: 28796392
Lancet Neurol. 2016 Jan;15(1):65-77
pubmed: 26549782
Clin Biomech (Bristol, Avon). 2006 Jan;21(1):89-98
pubmed: 16182419
JAMA Neurol. 2016 Jun 1;73(6):645-51
pubmed: 27043305
J Peripher Nerv Syst. 2013 Jun;18(2):177-80
pubmed: 23781965