Literature DB >> 32658385

Randomized trial of lung hyperinflation therapy in children with congenital muscular dystrophy.

Hemant Sawnani1,2, Oscar H Mayer3, Avani C Modi2,4, John E Pascoe1,2, Keith McConnell1, Joseph M McDonough3, Anne M Rutkowski5, Md Monir Hossain2,6, Rhonda Szczesniak2,6, Dawit G Tadesse6, Christine L Schuler2,7, Raouf Amin1,2.   

Abstract

OBJECTIVE: Respiratory compromise in congenital muscular dystrophy (CMD) occurs, in part, from chest wall contractures. Passive stretch with hyperinsufflation therapy could reduce related costo-vertebral joint contractures. We sought to examine the impact of hyperinsufflation use on lung function and quality of life in children with CMD. STUDY
DESIGN: We conducted a randomized controlled trial on hyperinsufflation therapy in children with CMD at two centers. An individualized hyperinsufflation regimen of 15 minutes twice daily using a cough assist device over a 12 months period was prescribed. We measured lung function, quality of life, and adherence. To demonstrate reproducibility, pulmonary function was measured twice on the same day. A mixed-effects regression model adjusting for confounders was used to assess the effects of hyperinsufflation.
RESULTS: We enrolled 34 participants in the study; 31 completed the trial (n = 17 treatment group and n = 14 controls). Participants in the treatment group demonstrated a relative gain in lung volume measured at 4 and 8 months, but not at 12 months. The control group required increases in the maximum insufflation pressures to achieve maximum lung volumes while the treatment group did not. Adherence was best early in the study, peaking at the first visit and decreasing at subsequent visits. Caregiver-reported quality of life was higher in the treatment group.
CONCLUSION: Hyperinsufflation therapy is effective in increasing and sustaining lung volume over time. Adherence, however, was inconsistent and difficult to maintain. Further research should determine if improved adherence leads to sustained benefits of hyperinsufflation.
© 2020 Wiley Periodicals LLC.

Entities:  

Keywords:  adherence; neuromuscular disease; respiratory insufficiency

Mesh:

Year:  2020        PMID: 32658385      PMCID: PMC9047443          DOI: 10.1002/ppul.24954

Source DB:  PubMed          Journal:  Pediatr Pulmonol        ISSN: 1099-0496


  27 in total

1.  Active lung volume recruitment to preserve vital capacity in Duchenne muscular dystrophy.

Authors:  Michael Chiou; John R Bach; Lavina Jethani; Michael F Gallagher
Journal:  J Rehabil Med       Date:  2017-01-19       Impact factor: 2.912

2.  Natural history of Ullrich congenital muscular dystrophy.

Authors:  A Nadeau; M Kinali; M Main; C Jimenez-Mallebrera; A Aloysius; E Clement; B North; A Y Manzur; S A Robb; E Mercuri; F Muntoni
Journal:  Neurology       Date:  2009-07-07       Impact factor: 9.910

3.  Childhood progressive muscular dystrophy and the role of physical therapy.

Authors:  S E Harris; D B Cherry
Journal:  Phys Ther       Date:  1974-01

4.  Mechanism of CO(2) retention in patients with neuromuscular disease.

Authors:  G Misuri; B Lanini; F Gigliotti; I Iandelli; A Pizzi; M G Bertolini; G Scano
Journal:  Chest       Date:  2000-02       Impact factor: 9.410

5.  Mechanical insufflation/exsufflation improves vital capacity in neuromuscular disorders.

Authors:  Florian Stehling; Anastasios Bouikidis; Ulrike Schara; Uwe Mellies
Journal:  Chron Respir Dis       Date:  2014-12-03       Impact factor: 2.444

6.  IPPB-assisted coughing in neuromuscular disorders.

Authors:  Christian Dohna-Schwake; Regine Ragette; Helmut Teschler; Thomas Voit; Uwe Mellies
Journal:  Pediatr Pulmonol       Date:  2006-06

Review 7.  Health-related quality of life and symptom reporting: similarities and differences between children and their parents.

Authors:  Christine Eiser; James W Varni
Journal:  Eur J Pediatr       Date:  2013-05-29       Impact factor: 3.183

8.  Distortion of chest wall and work of diaphragm in preterm infants.

Authors:  G P Heldt; M B McIlroy
Journal:  J Appl Physiol (1985)       Date:  1987-01

9.  Longitudinal changes in clinical outcome measures in COL6-related dystrophies and LAMA2-related dystrophies.

Authors:  Minal S Jain; Katherine Meilleur; Eunhee Kim; Gina Norato; Melissa Waite; Leslie Nelson; Michelle McGuire; Tina Duong; Katherine Keller; Donovan J Lott; Allan Glanzman; Kristy Rose; Marion Main; Courtney Fiorini; Irene Chrismer; Melody Linton; Monal Punjabi; Jeffrey Elliott; Fatoumata Tounkara; Ruhi Vasavada; Ranjani Logaraj; Jocelyn Winkert; Sandra Donkervoort; Meganne Leach; Jahannaz Dastgir; Linda Hynan; Carmel Nichols; Elizabeth Hartnett; Gilberto M Averion; James C Collins; Eunice S Kim; Angela Kokkinis; Alice Schindler; Kristen Zukosky; Robert Fee; Veronica Hinton; Payam Mohassel; Diana Bharucha-Goebel; Carole Vuillerot; Peter McGraw; Mark Barton; Joseph Fontana; Anne Rutkowski; A Reghan Foley; Carsten G Bönnemann
Journal:  Neurology       Date:  2019-10-25       Impact factor: 9.910

10.  Characterization of pulmonary function in Duchenne Muscular Dystrophy.

Authors:  O H Mayer; R S Finkel; C Rummey; M J Benton; A M Glanzman; J Flickinger; B-M Lindström; T Meier
Journal:  Pediatr Pulmonol       Date:  2015-03-09
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  2 in total

1.  The Use of Autologous Blood Patch in Ullrich Muscular Dystrophy and Recurrent Pneumothorax.

Authors:  Aledie Navas Nazario; Felicia I Cooper; Fabiola Weber-Guzman; Richard S Finkel
Journal:  Cureus       Date:  2022-06-15

2.  Merosin deficient congenital muscular dystrophy type 1A: An international workshop on the road to therapy 15-17 November 2019, Maastricht, the Netherlands.

Authors:  Hubert J M Smeets; Bram Verbrugge; Pierre Springuel; Nicol C Voermans
Journal:  Neuromuscul Disord       Date:  2021-05-01       Impact factor: 4.296

  2 in total

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