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Effects of respiratory muscle training (RMT) in children with infantile-onset Pompe disease and respiratory muscle weakness.
J Pediatr Rehabil Med 2014; 7(3):255-65JP

Abstract

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.

Authors+Show Affiliations

Department of Surgery, Division of Speech Pathology and Audiology, Duke University, Durham, NC, USA.Division of Speech Pathology and Audiology, Duke University, Durham, NC, USA.Division of Speech Pathology and Audiology, Duke University, Durham, NC, USA.Division of Speech Pathology and Audiology, Duke University, Durham, NC, USA.Communication Disorders, University of Virginia, Charlottesville, VA, USA.Division of Pediatric Pulmonary and Sleep Medicine, Duke University, Durham, NC, USA.Division of Medical Genetics, Department of Pediatrics, Duke University, Durham, NC, USA.Physical Therapy Division, Duke University, Durham, NC, USA.Department of Communicative Disorders, University of Rhode Island, Kingston, RI, USA.Division of Pediatric Pulmonary and Sleep Medicine, Duke University, Durham, NC, USA.Division of Medical Genetics, Department of Pediatrics, Duke University, Durham, NC, USA.

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

25260508

Citation

Jones, Harrison N., et al. "Effects of Respiratory Muscle Training (RMT) in Children With Infantile-onset Pompe Disease and Respiratory Muscle Weakness." Journal of Pediatric Rehabilitation Medicine, vol. 7, no. 3, 2014, pp. 255-65.
Jones HN, Crisp KD, Moss T, et al. Effects of respiratory muscle training (RMT) in children with infantile-onset Pompe disease and respiratory muscle weakness. J Pediatr Rehabil Med. 2014;7(3):255-65.
Jones, H. N., Crisp, K. D., Moss, T., Strollo, K., Robey, R., Sank, J., ... Kishnani, P. S. (2014). Effects of respiratory muscle training (RMT) in children with infantile-onset Pompe disease and respiratory muscle weakness. Journal of Pediatric Rehabilitation Medicine, 7(3), pp. 255-65. doi:10.3233/PRM-140294.
Jones HN, et al. Effects of Respiratory Muscle Training (RMT) in Children With Infantile-onset Pompe Disease and Respiratory Muscle Weakness. J Pediatr Rehabil Med. 2014;7(3):255-65. PubMed PMID: 25260508.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Effects of respiratory muscle training (RMT) in children with infantile-onset Pompe disease and respiratory muscle weakness. AU - Jones,Harrison N, AU - Crisp,Kelly D, AU - Moss,Tronda, AU - Strollo,Katherine, AU - Robey,Randy, AU - Sank,Jeffrey, AU - Canfield,Michelle, AU - Case,Laura E, AU - Mahler,Leslie, AU - Kravitz,Richard M, AU - Kishnani,Priya S, PY - 2014/9/28/entrez PY - 2014/9/28/pubmed PY - 2015/5/29/medline KW - Glycogen storage disease type II KW - breathing exercises KW - muscle KW - neuromuscular diseases KW - rehabilitation KW - skeletal SP - 255 EP - 65 JF - Journal of pediatric rehabilitation medicine JO - J Pediatr Rehabil Med VL - 7 IS - 3 N2 - 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. SN - 1875-8894 UR - https://www.unboundmedicine.com/medline/citation/25260508/Effects_of_respiratory_muscle_training__RMT__in_children_with_infantile_onset_Pompe_disease_and_respiratory_muscle_weakness_ L2 - https://content.iospress.com/openurl?genre=article&id=doi:10.3233/PRM-140294 DB - PRIME DP - Unbound Medicine ER -