Respiratory muscle training in late-onset Pompe disease: Results of a sham-controlled clinical trial.
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2020-11
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To address progressive respiratory muscle weakness in late-onset Pompe disease (LOPD), we developed a 12-week respiratory muscle training (RMT) program. In this exploratory, double-blind, randomized control trial, 22 adults with LOPD were randomized to RMT or sham-RMT. The primary outcome was maximum inspiratory pressure (MIP). Secondary and exploratory outcomes included maximum expiratory pressure (MEP), peak cough flow, diaphragm ultrasound, polysomnography, patient-reported outcomes, and measures of gross motor function. MIP increased 7.6 cmH2O (15.9) in the treatment group and 2.7 cmH2O (7.6) in the control group (P = 0.4670). MEP increased 14.0 cmH2O (25.9) in the treatment group and 0.0 cmH2O (12.0) in the control group (P = 0.1854). The only statistically significant differences in secondary/exploratory outcomes were improvements in time to climb 4 steps (P = 0.0346) and daytime sleepiness (P = 0.0160). The magnitude of changes in MIP and MEP in the treatment group were consistent with our pilot findings but did not achieve statistical significance in comparison to controls. Explanations for this include inadequate power and baseline differences in subject characteristics between groups. Additionally, control group subjects appeared to exhibit an active response to sham-RMT and therefore sham-RMT may not be an optimal control condition for RMT in LOPD.
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Jones, Harrison N, Maragatha Kuchibhatla, Kelly D Crisp, Lisa D Hobson-Webb, Laura Case, Milisa T Batten, Jill A Marcus, Richard M Kravitz, et al. (2020). Respiratory muscle training in late-onset Pompe disease: Results of a sham-controlled clinical trial. Neuromuscular disorders : NMD, 30(11). pp. 904–914. 10.1016/j.nmd.2020.09.023 Retrieved from https://hdl.handle.net/10161/27298.
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Scholars@Duke

Harrison N. Jones

Maragatha Kuchibhatla
Statistical research methodology, analysis of repeated measurements, latent growth curve models, latent class growth models, classification and regression trees,
designing clinical trials, designing clinical trials in psychiatry -- both treatment and non-treatment
trials in various comorbid populations.
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