Early NIV is associated with accelerated lung function decline in Duchenne muscular dystrophy treated with glucocorticosteroids

Abstract

Use of non-invasive ventilation (NIV) in adolescents with Duchenne muscular dystrophy (DMD) has increased with concomitant extended survival. Aim To describe lung function (LF) changes with NIV in adolescents with DMD and to assess differences between Steroid Users and Steroid Naïve subjects. Method A retrospective cohort of adolescents with DMD initiating NIV over 10 years was conducted. Serial LF before and after NIV initiation was collated. Use of systemic glucocorticosteroids, adherence to NIV and presence of cardiac disease were assessed. Results Twenty-nine men started NIV, median age 14.66 years (IQR 2.35, 10.47-17.96). Nine were Steroid Users and eight were Steroid Naïve. Indications for NIV were apnoea-hypopnoea index >5 and/or nocturnal hypoventilation. LF is better (forced vital capacity (FVC) z-score -3.26 vs -5.41, p < 0.02) and decline slower (FVC z-score -0.58 per annum (pa) vs -0.68 pa, p<0.001) in Steroid Users compared with Steroid Naïve subjects. Following NIV initiation, FVC z-score decline slowed for the whole (-0.72 pa (95% CI -0.79 to 0.64) to -0.46 pa (95% CI -0.54 to 0.38) p < 0.001) and Steroid Naïve groups (-0.74 (95% CI -0.85 to 0.63) to -0.44 pa (95% CI -0.56 to 0.32) p < 0.001) but accelerated in the Steroid User group (-0.56 (95% CI -0.70 to 0.42) to -0.75 pa (95% CI -0.89 to 0.61) p < 0.001). Adherence to NIV and cardiac disease did not impact decline. Conclusion Overall, LF decline is reduced on NIV. Steroid Naïve patients have lower LF and faster decline, which slows following NIV initiation. An accelerated LF decline was seen on NIV in Steroid Users which requires further prospective research.