Safety and effectiveness of resistance training in patients with late onset Pompe disease - a pilot study.

Autor: Bhatnagar C; Department of Pediatrics, Neurology and Pathology, Division of Genetics and Genomic Medicine, University of California, Irvine School of Medicine, 101 The City Drive South, ZC4482, Orange, CA, USA., Shah J; Department of Pediatrics, Neurology and Pathology, Division of Genetics and Genomic Medicine, University of California, Irvine School of Medicine, 101 The City Drive South, ZC4482, Orange, CA, USA., Ramani B; Department of Pediatrics, Neurology and Pathology, Division of Genetics and Genomic Medicine, University of California, Irvine School of Medicine, 101 The City Drive South, ZC4482, Orange, CA, USA., Surampalli A; Department of Pediatrics, Neurology and Pathology, Division of Genetics and Genomic Medicine, University of California, Irvine School of Medicine, 101 The City Drive South, ZC4482, Orange, CA, USA., Avanti M; Department of Pediatrics, Neurology and Pathology, Division of Genetics and Genomic Medicine, University of California, Irvine School of Medicine, 101 The City Drive South, ZC4482, Orange, CA, USA., Radom-Aizik S; Pediatric Exercise & Genomics Research Center, Department of Pediatrics, University of California, Irvine, USA., Knight M; Department of Pediatrics, Neurology and Pathology, Division of Genetics and Genomic Medicine, University of California, Irvine School of Medicine, 101 The City Drive South, ZC4482, Orange, CA, USA., Weiss L; Department of Pediatrics, Neurology and Pathology, Division of Genetics and Genomic Medicine, University of California, Irvine School of Medicine, 101 The City Drive South, ZC4482, Orange, CA, USA., Caiozzo V; Department of Orthopedic Surgery, University of California, Irvine, USA., Kimonis V; Department of Pediatrics, Neurology and Pathology, Division of Genetics and Genomic Medicine, University of California, Irvine School of Medicine, 101 The City Drive South, ZC4482, Orange, CA, USA; Department of Neurology, University of California, Irvine School of Medicine, Orange, CA, USA; Department of Pathology & Laboratory Medicine, University of California, Irvine School of Medicine, Orange, CA, USA. Electronic address: vkimonis@uci.edu.
Jazyk: angličtina
Zdroj: Neuromuscular disorders : NMD [Neuromuscul Disord] 2022 Apr; Vol. 32 (4), pp. 284-294. Date of Electronic Publication: 2022 Feb 17.
DOI: 10.1016/j.nmd.2022.02.002
Abstrakt: Pompe disease is a progressive myopathy resulting from deficiency in lysosomal enzyme acid α-glucosidase (GAA), which leads to glycogen accumulation in lysosomes primarily in skeletal and cardiac muscle. Enzyme replacement therapy (ERT) with recombinant human (rh) GAA works well in alleviating the cardiomyopathy; however, many patients continue to have progressive muscle weakness. The purpose of this study was to evaluate the effectiveness of a respiratory training combined with 24-week supervised resistance training program on muscle strength (measured by Biodex)), and respiratory function including maximum inspiratory pressure (MIP), maximum expiratory pressure (MEP) in subjects with late onset Pompe disease receiving ERT. Ten subjects participated in a 24-week resistance exercise program, three times per week, in addition to respiratory muscle exercise training six days per week. Overall, at the end of the resistance training program, as measured by Biodex dynamometry, the leg extensor strength improved by 10.5 ± 3.2Nm. (2 O (p = 0.03) and the MEP by 6.4 ± 4.4 (p = 0.16). The exercise training significantly improved the trajectories of MIP and 6 MWT outcomes but not FVC when compared with the natural history data available in 6 individuals. These pilot results indicate that resistance training combined with respiratory training and ERT had a positive effect on muscular strength, functional capacity, and respiratory function in patients with late-onset Pompe disease and might be considered as a potential adjunct therapy in this population.
Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.
(Copyright © 2022 The Author(s). Published by Elsevier B.V. All rights reserved.)
Databáze: MEDLINE
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