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

Rationale: Lung volume recruitment (LVR) is prescribed for children with Duchenne muscular dystrophy (DMD) to maintain chest wall compliance and assist airway clearance.

Objective

We aimed to determine if twice-daily LVR compared with standard treatment in children with DMD improved health-related quality of life (HRQOL) over 2 years.

Methods

A multicenter, assessor-blinded randomized controlled trial was conducted among boys aged 6-16 years with DMD with forced vital capacity >30% predicted to undergo conventional treatment or conventional treatment and manual LVR twice daily for 2 years. The outcome was HRQOL measured by two validated instruments (Pediatric Quality of Life Inventory [PedsQL] generic version 4.0 and PedsQL DMD version 3.0) every 6 months.

Results

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

Conclusions

There was no difference in HRQOL between the LVR and control groups after 2 years. The burden of LVR did not appear to adversely affect HRQOL.