Respiratory muscle training in late-onset Pompe disease: Results of a sham-controlled clinical trial.

Journal Article (Journal Article)

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.

Full Text

Duke Authors

Cited Authors

  • Jones, HN; Kuchibhatla, M; Crisp, KD; Hobson-Webb, LD; Case, L; Batten, MT; Marcus, JA; Kravitz, RM; Kishnani, PS

Published Date

  • November 2020

Published In

Volume / Issue

  • 30 / 11

Start / End Page

  • 904 - 914

PubMed ID

  • 33127291

Pubmed Central ID

  • PMC7680435

Electronic International Standard Serial Number (EISSN)

  • 1873-2364

Digital Object Identifier (DOI)

  • 10.1016/j.nmd.2020.09.023


  • eng

Conference Location

  • England