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

Hemant Sawnani, Oscar H Mayer, Avani C Modi, John E Pascoe, Keith McConnell, Joseph M McDonough, Anne M Rutkowski, Md Monir Hossain, Rhonda Szczesniak, Dawit G Tadesse, Christine L Schuler, Raouf Amin, Hemant Sawnani, Oscar H Mayer, Avani C Modi, John E Pascoe, Keith McConnell, Joseph M McDonough, Anne M Rutkowski, Md Monir Hossain, Rhonda Szczesniak, Dawit G Tadesse, Christine L Schuler, Raouf Amin

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.

Keywords: adherence; neuromuscular disease; respiratory insufficiency.

Conflict of interest statement

SUMMARY CONFLICT OF INTEREST STATEMENTS

No conflicts: HS, ACM, JEP, KMC, JMM, AMR, MMH, RS, DGT, CLS, RA

OHM reports grants from Cure CMD Foundation, during the conduct of the study.

© 2020 Wiley Periodicals LLC.

Figures

Figure 1:
Figure 1:
Titration plot of a pressure-volume curve (PVC) of a subject with inspiratory capacity (IC) noted. The black arrow indicates the inflection point of the curve, and represents the identified hyperinsufflating pressure subsequently prescribed.
Figure 2:
Figure 2:
Mean (± SD) absolute FVC values at individual visits for the control and treatment groups. The asterix (*) indicates significant difference between the treatment and control groups after adjusting for baseline FVC and other covariates (age, gender, BMI, and adherence rate between visits). The magnitude of the change in absolute FVC from visit 1 was significant at visit 2 and 3 for the treatment group.
Figure 3:
Figure 3:
Mean (± SD) FVC% predicted values at individual visits for the control and treatment groups. The asterix (*) indicates significant difference between the treatment and control groups after adjusting for baseline FVC and other covariates (age, gender, BMI, and adherence rate between visits). The magnitude of the change in FVC% predicted values from visit 1 was significant at visit 2 and 3 for the treatment group.
Figure 4:
Figure 4:
Mean (± SD) maximal insufflation pressures at individual visits for the control and treatment groups. The asterix (*) indicates significant difference between the treatment and control groups after adjusting for baseline FVC and other covariates (age, gender, BMI, and adherence rate between visits) and results are reported expressed as LS means (± SE).
Figure 5:
Figure 5:
Least square mean (LSM) and standard error (SE) estimates of mother PedsQL total score by visit and treatment type. Models are adjusted for the baseline score and the FVC values (* indicates statistically significant difference at 5% level between treatment and control groups)

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Source: PubMed

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