Quantitative Foot Muscle Magnetic Resonance Imaging Reliably Measures Disease Progression in Children and Adolescents with Charcot-Marie-Tooth Disease Type 1A.

Autor: Doherty CM; Center for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK., Howard P; Roy and Lucille Carver College of Medicine, University of Iowa, Iowa City, IA, USA., O'Donnell LF; Center for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK., Zuccarino R; Roy and Lucille Carver College of Medicine, University of Iowa, Iowa City, IA, USA.; Fondazione Serena Onlus, Centro Clinico NeMO Trento, Italy., Wastling S; Lysholm Department of Radiology, National Hospital for Neurology and Neurosurgery, London, UK., Milev E; Dubowitz Neuromuscular Center, Great Ormond Street Hospital, London, UK., Banks T; Department of Radiology, Great Ormond Street Hospital, London, UK., Shah S; Lysholm Department of Radiology, National Hospital for Neurology and Neurosurgery, London, UK., Zafeiropoulos N; Lysholm Department of Radiology, National Hospital for Neurology and Neurosurgery, London, UK., Stephens KJ; Roy and Lucille Carver College of Medicine, University of Iowa, Iowa City, IA, USA., Sarkozy A; Dubowitz Neuromuscular Center, Great Ormond Street Hospital, London, UK., Grider T; Roy and Lucille Carver College of Medicine, University of Iowa, Iowa City, IA, USA., Feely SME; Division of Pediatric Neurology, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, WA, USA., Manzur A; Dubowitz Neuromuscular Center, Great Ormond Street Hospital, London, UK., Shy RR; Roy and Lucille Carver College of Medicine, University of Iowa, Iowa City, IA, USA., Skorupinska M; Center for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK., Pipis M; Center for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK., Nicolaisen E; Roy and Lucille Carver College of Medicine, University of Iowa, Iowa City, IA, USA., McDowell A; Center for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.; Lysholm Department of Radiology, National Hospital for Neurology and Neurosurgery, London, UK., Dilek N; University of Rochester School of Medicine and Dentistry, New York, NY, USA., Rossor AM; Center for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK., Laura M; Center for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK., Clark C; Department of Radiology, Great Ormond Street Hospital, London, UK., Muntoni F; Dubowitz Neuromuscular Center, Great Ormond Street Hospital, London, UK., Thedens D; Roy and Lucille Carver College of Medicine, University of Iowa, Iowa City, IA, USA., Thornton J; Lysholm Department of Radiology, National Hospital for Neurology and Neurosurgery, London, UK., Morrow JM; Center for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK., Shy ME; Roy and Lucille Carver College of Medicine, University of Iowa, Iowa City, IA, USA., Reilly MM; Center for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.
Jazyk: angličtina
Zdroj: Annals of neurology [Ann Neurol] 2024 Jul; Vol. 96 (1), pp. 170-174. Date of Electronic Publication: 2024 Apr 13.
DOI: 10.1002/ana.26934
Abstrakt: 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.
(© 2024 The Authors. Annals of Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association.)
Databáze: MEDLINE
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