Effectiveness of Respiratory Muscle Training in Pompe Disease: A Systematic Review and Meta-Analysis.

IF 2 4区 医学 Q2 PEDIATRICS
Mu-Yun Lin, Szu-Han Chen, Jen-Ting Lee, Po-Cheng Hsu
{"title":"Effectiveness of Respiratory Muscle Training in Pompe Disease: A Systematic Review and Meta-Analysis.","authors":"Mu-Yun Lin, Szu-Han Chen, Jen-Ting Lee, Po-Cheng Hsu","doi":"10.3390/children11101209","DOIUrl":null,"url":null,"abstract":"<p><p><b>Background</b>: Pompe disease is a rare metabolic myopathy caused by the lack or deficiency of the lysosomal acid alpha-glucosidase, resulting in skeletal muscle weakness and cardiomyopathy. The disease varies by onset age and genetic mutations and is categorized into infantile-onset and late-onset Pompe disease. Respiratory muscle weakness may persist regardless enzyme replacement therapy. This systemic review and meta-analysis aim to assess the effect of respiratory muscle training (RMT) on respiratory muscle strength, functional endurance, and pulmonary function in patient with Pompe disease. <b>Methods</b>: PubMed, EMBASE, and Cochrane databases were searched up until Aug 2024. Studies examining the therapeutic effects of RMT in patients with Pompe disease were included. Outcome measures included the change in maximal inspiratory pressure (MIP), maximal expiratory pressure (MEP), six-minute walking test (6MWT), pulmonary function before after RMT, quality of life and adverse events. <b>Results</b>: The meta-analysis consisted of 5 single-arm studies, including 31 patients in total. Regarding inspiratory muscle strength, RMT has significantly improving MIP (8.71 cmH<sub>2</sub>O; 95% CI, 6.23-11.19, <i>p</i> < 0.001) and MEP (12.15 cmH<sub>2</sub>O; 95% CI, 10.55-13.74, <i>p</i> < 0.001) in both types of Pompe disease. However, no significant change regarding 6MWT. No serious adverse events were reported. <b>Conclusions</b>: Our meta-analysis revealed that RMT may increase inspiratory muscle and expiratory muscle strength, but may not have an effect on 6MWT in patients with Pompe disease. RMT has potential to be integrated into the cardioplulmonary rehabilitation for patients with Pompe disease. Further large randomized controlled trials are needed to verify the efficacy and safety of RMT in patients with Pompe disease.</p>","PeriodicalId":48588,"journal":{"name":"Children-Basel","volume":null,"pages":null},"PeriodicalIF":2.0000,"publicationDate":"2024-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11505692/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Children-Basel","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.3390/children11101209","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"PEDIATRICS","Score":null,"Total":0}
引用次数: 0

Abstract

Background: Pompe disease is a rare metabolic myopathy caused by the lack or deficiency of the lysosomal acid alpha-glucosidase, resulting in skeletal muscle weakness and cardiomyopathy. The disease varies by onset age and genetic mutations and is categorized into infantile-onset and late-onset Pompe disease. Respiratory muscle weakness may persist regardless enzyme replacement therapy. This systemic review and meta-analysis aim to assess the effect of respiratory muscle training (RMT) on respiratory muscle strength, functional endurance, and pulmonary function in patient with Pompe disease. Methods: PubMed, EMBASE, and Cochrane databases were searched up until Aug 2024. Studies examining the therapeutic effects of RMT in patients with Pompe disease were included. Outcome measures included the change in maximal inspiratory pressure (MIP), maximal expiratory pressure (MEP), six-minute walking test (6MWT), pulmonary function before after RMT, quality of life and adverse events. Results: The meta-analysis consisted of 5 single-arm studies, including 31 patients in total. Regarding inspiratory muscle strength, RMT has significantly improving MIP (8.71 cmH2O; 95% CI, 6.23-11.19, p < 0.001) and MEP (12.15 cmH2O; 95% CI, 10.55-13.74, p < 0.001) in both types of Pompe disease. However, no significant change regarding 6MWT. No serious adverse events were reported. Conclusions: Our meta-analysis revealed that RMT may increase inspiratory muscle and expiratory muscle strength, but may not have an effect on 6MWT in patients with Pompe disease. RMT has potential to be integrated into the cardioplulmonary rehabilitation for patients with Pompe disease. Further large randomized controlled trials are needed to verify the efficacy and safety of RMT in patients with Pompe disease.

庞贝氏症患者呼吸肌训练的有效性:系统回顾与元分析》。
背景:庞贝病是一种罕见的代谢性肌病,由溶酶体酸α-葡萄糖苷酶缺乏或不足引起,导致骨骼肌无力和心肌病。该病因发病年龄和基因突变而异,分为婴儿发病型和晚期发病型庞贝氏症。无论采用何种酶替代疗法,呼吸肌无力都可能持续存在。本系统综述和荟萃分析旨在评估呼吸肌训练(RMT)对庞贝病患者呼吸肌力量、功能耐力和肺功能的影响。研究方法检索了截至 2024 年 8 月的 PubMed、EMBASE 和 Cochrane 数据库。纳入了研究 RMT 对庞贝病患者治疗效果的研究。结果指标包括最大吸气压(MIP)、最大呼气压(MEP)、六分钟步行测试(6MWT)、RMT 治疗前的肺功能变化、生活质量和不良事件。分析结果荟萃分析包括 5 项单臂研究,共涉及 31 名患者。在吸气肌力方面,RMT能显著改善两种类型庞贝氏症患者的MIP(8.71 cmH2O; 95% CI, 6.23-11.19, p < 0.001)和MEP(12.15 cmH2O; 95% CI, 10.55-13.74, p < 0.001)。但 6MWT 无明显变化。无严重不良事件报告。结论我们的荟萃分析表明,RMT 可增强庞贝病患者的吸气肌和呼气肌力量,但对 6MWT 可能没有影响。RMT 有可能被纳入庞贝病患者的心肺康复治疗中。要验证 RMT 对庞贝病患者的疗效和安全性,还需要进一步的大型随机对照试验。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
Children-Basel
Children-Basel PEDIATRICS-
CiteScore
2.70
自引率
16.70%
发文量
1735
审稿时长
6 weeks
期刊介绍: Children is an international, open access journal dedicated to a streamlined, yet scientifically rigorous, dissemination of peer-reviewed science related to childhood health and disease in developed and developing countries. The publication focuses on sharing clinical, epidemiological and translational science relevant to children’s health. Moreover, the primary goals of the publication are to highlight under‑represented pediatric disciplines, to emphasize interdisciplinary research and to disseminate advances in knowledge in global child health. In addition to original research, the journal publishes expert editorials and commentaries, clinical case reports, and insightful communications reflecting the latest developments in pediatric medicine. By publishing meritorious articles as soon as the editorial review process is completed, rather than at predefined intervals, Children also permits rapid open access sharing of new information, allowing us to reach the broadest audience in the most expedient fashion.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信