Effect of creatine monohydrate on motor function in children with facioscapulohumeral muscular dystrophy: A multicenter, randomized, double-blind placebo-controlled crossover trial
Woodcock IR, de Valle K, Cairns A, Davidson ZE, Kean M, Varma N, Grobler A, Metz D, Carroll K, Dilek N, Heatwole C, Ryan MM, Delatycki MB, Yiu EM
Pharmacotherapy · 4 citas
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Cómo se estudió
- Diseño
- Ensayo controlado aleatorizado (indexado por PubMed)
- Estudiado en
- Personas
- Resultado principal
- Marcadores de salud y funcionamiento
Quién lo pagó
- Financiación
- Financiación independiente
- Organización sin fines de lucro
- FSHD Global Research Foundation
- Autores
- Al menos un autor declara un vínculo económico con la industria
Según 1 financiador declarado y la declaración de financiación del texto completo.
Publicación
- Publicado
- 2025-05-14 · Pharmacotherapy · vol. 45 · número 6 · págs. 341–351
- Editorial
- Wiley
- Citado
- 4 citas · más que el 79% de estudios similares · 1.4× el promedio de su campo
- Referencias
- 62 trabajos
- Acceso
- Acceso abierto (revista híbrida) · CC-BY-NC
- Áreas de investigación
- Muscle Physiology and Disorders · Nutrition and Health in Aging · Muscle metabolism and nutrition
- Palabras clave
- Facioscapulohumeral muscular dystrophy, Crossover study, Medicine, Placebo, Muscular dystrophy, Physical therapy, Confidence interval, Randomized controlled trial, Creatine, Internal medicine, Pathology
- MeSH
- humans, muscular dystrophy, facioscapulohumeral, creatine, magnetic resonance imaging, treatment outcome, cross-over studies, double-blind method, adolescent, child, female, male, muscle strength
14 autores
- Ian R. Woodcock🇦🇺 · autor de correspondenciaRoyal Children's Hospital; The University of Melbourne; Murdoch Children's Research Institute
- Katy de Valle🇦🇺Royal Children's Hospital; The University of Melbourne; Murdoch Children's Research Institute
- Anita G. Cairns🇦🇺Children's Health Queensland Hospital and Health Service
- Zoe E. Davidson🇦🇺Royal Children's Hospital; Murdoch Children's Research Institute; Monash University
- Michael J. Kean🇦🇺Royal Children's Hospital
- Nisha Varma🇦🇺Royal Children's Hospital
- Anneke C Grobler🇦🇺The University of Melbourne; Murdoch Children's Research Institute
- David Metz🇦🇺Royal Children's Hospital; Murdoch Children's Research Institute; Monash University
- Kate Carroll🇦🇺Royal Children's Hospital; Murdoch Children's Research Institute
- Nuran Dilek🇺🇸University of Rochester
- Chad Heatwole🇺🇸University of Rochester
- Monique M. Ryan🇦🇺Royal Children's Hospital; Murdoch Children's Research Institute
- Martin B. Delatycki🇦🇺The University of Melbourne; Victorian Clinical Genetics Services; Murdoch Children's Research Institute
- Eppie Mildred Yiu🇦🇺Royal Children's Hospital; The University of Melbourne; Murdoch Children's Research Institute
Resumen
El texto original del estudio, en el idioma en que se publicó.
Background
Facioscapulohumeral muscular dystrophy (FSHD) is a rare, progressive muscle disease with no available disease-modifying therapy. Creatine monohydrate (CrM) has been shown to improve muscle strength in individuals with muscular dystrophies but has not been tested in young people with FSHD. This study aimed to explore the efficacy of CrM on motor function in children with FSHD.
Methods
In a randomized placebo-controlled double-blind crossover trial, powdered CrM at a dose of 100 mg/kg/day (maximum 10 g daily) was compared with placebo in two 12-week treatment periods with a 6-week washout between crossover arms. The primary outcome measure was the Motor Function Measure for Neuromuscular Disease (MFM-32) with secondary outcomes assessing safety, endurance, strength, patient-reported outcome measures, and muscle morphology measurements as assessed by whole-body magnetic resonance imaging (MRI).
Results
Thirteen children were enrolled (mean (standard deviation, SD) 12.2 (2.67) years of age) and 11 patients completed both trial treatment periods. In an intention-to-treat analysis, no clinically meaningful difference was seen between treatment groups as measured by the mean difference in MFM-32 (0.19, 95% confidence interval (CI) -0.71 to 1.08). However, there was an improvement in 6-minute walk distance of 27.74 m (95% CI -1.41 to 56.88) and trends to improvement in the FSHD-Composite Outcome Measure for Pediatrics (FSHD-COM Peds), 10 meter walk/run, and in MRI measures. There were no serious adverse events. Serum creatinine increased by a mean 12.63 μmol/L (95% CI 1.14 to 24.12) post-CrM treatment, though this was presumed to reflect increased creatinine production. No participants discontinued CrM due to adverse events.
Conclusion
CrM is safe and well tolerated in children with FSHD. Although CrM had no effect on motor function as measured by the MFM-32 compared with placebo, there were trends toward improvement in the 6-minute walk distance and other secondary outcome measures. This study confirms the feasibility of conducting clinical trials in children with FSHD. Further assessment of the efficacy of CrM in pediatric FSHD is warranted in a larger randomized controlled clinical trial. Future studies may benefit from stratifying population cohorts according to functional ability or by MRI fat infiltration measurements.
Resumen vía Europe PMC. Los derechos de autor siguen siendo de los autores o la editorial (CC BY-NC).
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