학술논문
Quantitative Foot Muscle Magnetic Resonance Imaging Reliably Measures Disease Progression in Children and Adolescents with Charcot–Marie–Tooth Disease Type 1A.
Document Type
Article
Author
Doherty, Carolynne M.; Howard, Paige; O'Donnell, Luke F.; Zuccarino, Riccardo; Wastling, Stephen; Milev, Evelin; Banks, Tina; Shah, Sachit; Zafeiropoulos, Nick; Stephens, Katherine J.; Sarkozy, Anna; Grider, Tiffany; Feely, Shawna M. E.; Manzur, Adnan; Shy, Rosemary R.; Skorupinska, Mariola; Pipis, Menelaos; Nicolaisen, Emma; McDowell, Amy; Dilek, Nuran
Source
Subject
*MAGNETIC resonance imaging
*CHARCOT-Marie-Tooth disease
*JUVENILE diseases
*DISEASE progression
*TEENAGERS
*CLUBFOOT
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Language
ISSN
0364-5134
Abstract
Quantitative muscle fat fraction (FF) responsiveness is lower in younger Charcot–Marie–Tooth disease type 1A (CMT1A) patients with lower baseline calf‐level FF. We investigated the practicality, validity, and responsiveness of foot‐level FF in this cohort involving 22 CMT1A patients and 14 controls. The mean baseline foot‐level FF was 25.9 ± 20.3% in CMT1A patients, and the 365‐day FF (n = 15) increased by 2.0 ± 2.4% (p < 0.001 vs controls). Intrinsic foot‐level FF demonstrated large responsiveness (12‐month standardized response mean (SRM) of 0.86) and correlated with the CMT examination score (ρ = 0.58, P = 0.01). Intrinsic foot‐level FF has the potential to be used as a biomarker in future clinical trials involving younger CMT1A patients. ANN NEUROL 2024;96:170–174 [ABSTRACT FROM AUTHOR]