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

Publication ,  Journal Article
Jones, HN; Kuchibhatla, M; Crisp, KD; Hobson-Webb, LD; Case, L; Batten, MT; Marcus, JA; Kravitz, RM; Kishnani, PS
Published in: Neuromuscul Disord
November 2020

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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Published In

Neuromuscul Disord

DOI

EISSN

1873-2364

Publication Date

November 2020

Volume

30

Issue

11

Start / End Page

904 / 914

Location

England

Related Subject Headings

  • Ultrasonography
  • Respiratory Muscles
  • Respiratory Function Tests
  • Neurology & Neurosurgery
  • Muscle Weakness
  • Middle Aged
  • Male
  • Inhalation
  • Humans
  • Glycogen Storage Disease Type II
 

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Jones, H. N., Kuchibhatla, M., Crisp, K. D., Hobson-Webb, L. D., Case, L., Batten, M. T., … Kishnani, P. S. (2020). Respiratory muscle training in late-onset Pompe disease: Results of a sham-controlled clinical trial. Neuromuscul Disord, 30(11), 904–914. https://doi.org/10.1016/j.nmd.2020.09.023
Jones, Harrison N., Maragatha Kuchibhatla, Kelly D. Crisp, Lisa D. Hobson-Webb, Laura Case, Milisa T. Batten, Jill A. Marcus, Richard M. Kravitz, and Priya S. Kishnani. “Respiratory muscle training in late-onset Pompe disease: Results of a sham-controlled clinical trial.Neuromuscul Disord 30, no. 11 (November 2020): 904–14. https://doi.org/10.1016/j.nmd.2020.09.023.
Jones HN, Kuchibhatla M, Crisp KD, Hobson-Webb LD, Case L, Batten MT, et al. Respiratory muscle training in late-onset Pompe disease: Results of a sham-controlled clinical trial. Neuromuscul Disord. 2020 Nov;30(11):904–14.
Jones, Harrison N., et al. “Respiratory muscle training in late-onset Pompe disease: Results of a sham-controlled clinical trial.Neuromuscul Disord, vol. 30, no. 11, Nov. 2020, pp. 904–14. Pubmed, doi:10.1016/j.nmd.2020.09.023.
Jones HN, Kuchibhatla M, Crisp KD, Hobson-Webb LD, Case L, Batten MT, Marcus JA, Kravitz RM, Kishnani PS. Respiratory muscle training in late-onset Pompe disease: Results of a sham-controlled clinical trial. Neuromuscul Disord. 2020 Nov;30(11):904–914.
Journal cover image

Published In

Neuromuscul Disord

DOI

EISSN

1873-2364

Publication Date

November 2020

Volume

30

Issue

11

Start / End Page

904 / 914

Location

England

Related Subject Headings

  • Ultrasonography
  • Respiratory Muscles
  • Respiratory Function Tests
  • Neurology & Neurosurgery
  • Muscle Weakness
  • Middle Aged
  • Male
  • Inhalation
  • Humans
  • Glycogen Storage Disease Type II