Effects of respiratory muscle training (RMT) in children with infantile-onset Pompe disease and respiratory muscle weakness.

Journal Article (Journal Article)

PURPOSE: Respiratory muscle weakness is a primary therapeutic challenge for patients with infantile Pompe disease. We previously described the clinical implementation of a respiratory muscle training (RMT) regimen in two adults with late-onset Pompe disease; both demonstrated marked increases in inspiratory and expiratory muscle strength in response to RMT. However, the use of RMT in pediatric survivors of infantile Pompe disease has not been previously reported. METHOD: We report the effects of an intensive RMT program on maximum inspiratory pressure (MIP) and maximum expiratory pressure (MEP) using A-B-A (baseline-treatment-posttest) single subject experimental design in two pediatric survivors of infantile Pompe disease. Both subjects had persistent respiratory muscle weakness despite long-term treatment with alglucosidase alfa. RESULTS: Subject 1 demonstrated negligible to modest increases in MIP/MEP (6% increase in MIP, d=0.25; 19% increase in MEP, d=0.87), while Subject 2 demonstrated very large increases in MIP/MEP (45% increase in MIP, d=2.38; 81% increase in MEP, d=4.31). Following three-month RMT withdrawal, both subjects maintained these strength increases and demonstrated maximal MIP and MEP values at follow-up. CONCLUSION: Intensive RMT may be a beneficial treatment for respiratory muscle weakness in pediatric survivors of infantile Pompe disease.

Full Text

Duke Authors

Cited Authors

  • Jones, HN; Crisp, KD; Moss, T; Strollo, K; Robey, R; Sank, J; Canfield, M; Case, LE; Mahler, L; Kravitz, RM; Kishnani, PS

Published Date

  • 2014

Published In

Volume / Issue

  • 7 / 3

Start / End Page

  • 255 - 265

PubMed ID

  • 25260508

Electronic International Standard Serial Number (EISSN)

  • 1875-8894

Digital Object Identifier (DOI)

  • 10.3233/PRM-140294


  • eng

Conference Location

  • Netherlands