| Title: |
Validation of MRC Centre MRI calf muscle fat fraction protocol as an outcome measure in CMT1A |
| Authors: |
Morrow, JM; Evans, MRB; Grider, T; Sinclair, CDJ; Thedens, D; Shah, S; Yousry, TA; Hanna, MG; Nopoulos, P; Thornton, JS; Shy, ME; Reilly, MM |
| Source: |
Neurology (2018) |
| Publication Year: |
2018 |
| Collection: |
University College London: UCL Discovery |
| Description: |
OBJECTIVE: To translate the quantitative MRC Centre MRI protocol in Charcot-Marie-Tooth disease type 1A (CMT1A) to a second site; validate its responsiveness in an independent cohort; and test the benefit of participant stratification to increase outcome measure responsiveness. METHODS: Three healthy volunteers were scanned for intersite standardization. For the longitudinal patient study, 11 patients with CMT1A were recruited with 10 patients rescanned at a 12-month interval. Three-point Dixon MRI of leg muscles was performed to generate fat fraction (FF) maps, transferred to a central site for quality control and analysis. Clinical data collected included CMT Neuropathy Score. RESULTS: Test-retest reliability of FF within individual healthy calf muscles at the remote site was excellent: intraclass correlation coefficient 0.79, limits of agreement -0.67 to +0.85 . In patients, mean calf muscle FF was 21.0% and correlated strongly with disease severity and age. Calf muscle FF significantly increased over 12 months (+1.8 ± 1.7 , p = 0.009). Patients with baseline FF >10% showed a 12-month FF increase of 2.9% ± 1.3% (standardized response mean = 2.19). CONCLUSIONS: We have validated calf muscle FF as an outcome measure in an independent cohort of patients with CMT1A. Responsiveness is significantly improved by enrolling a stratified patient cohort with baseline calf FF >10%. |
| Document Type: |
article in journal/newspaper |
| File Description: |
text |
| Language: |
English |
| Relation: |
https://discovery.ucl.ac.uk/id/eprint/10055219/1/WNL.0000000000006214.full.pdf; https://discovery.ucl.ac.uk/id/eprint/10055219/ |
| Availability: |
https://discovery.ucl.ac.uk/id/eprint/10055219/1/WNL.0000000000006214.full.pdf; https://discovery.ucl.ac.uk/id/eprint/10055219/ |
| Rights: |
open |
| Accession Number: |
edsbas.C2A09562 |
| Database: |
BASE |