Lung Volume Recruitment and Quality of Life in Duchenne Muscular Dystrophy: Secondary Analysis of the STEADFAST Randomized Controlled Trial.

Abstract

Background: Lung volume recruitment (LVR) is prescribed for children with Duchenne muscular dystrophy (DMD), to maintain chest wall compliance and assist airway clearance. We aimed to determine if twice-daily LVR compared to standard treatment in children with DMD improved health related quality of life (HRQOL) over 2 years.

Methods: A multicentre, assessor-blinded randomized controlled trial was conducted, including boys 6-16 years with DMD with forced vital capacity >30% predicted to undergo conventional treatment or conventional treatment and manual LVR twice-daily for two years. Outcome was HRQOL measured by two validated instruments (PedsQL, DMD PedsQL) every 6 months.

Results: Sixty-two boys (33 LVR group and 29 controls), median (IQR) age 11.4 (9.4,13.4) years completed the study. Median (IQR) baseline HRQOL assessed by parent/caregiver PedsQL was 58.0 (41.9, 67.0) and 55.2 (47.8, 63.5) for the LVR and control groups. HRQOL trajectories were stable over the 2-year study period except parent communication, which improved. No statistically significant interactions were detected between visit and treatment group for any PedsQL outcomes. DMD PedsQL communication scores in the LVR group showed more positive change compared to controls. Treatment scores in the LVR group showed less positive change over time compared to controls; however, after correcting for multiple testing, were no longer significant. Respiratory symptom rate did not differ between groups and was not associated with any HRQOL outcome.

Conclusion: There was no difference in HRQOL between the LVR and control groups after two years. The burden of LVR did not appear to adversely affect HRQOL.