Elsevier

Neuromuscular Disorders

Volume 28, Issue 11, November 2018, Pages 897-909
Neuromuscular Disorders

Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids

https://doi.org/10.1016/j.nmd.2018.07.004Get rights and content
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Highlights

  • Natural history changes in pulmonary function tests across time in Duchenne muscular dystrophy.

  • Treatment with glucocorticoids (steroids) > 1 year was compared to steroid naïve treatment.

  • Steroid treatment slowed the rate of pulmonary decline as measured by FVC%p in 7–9.9 year olds;

  • Steroid treatment resulted in higher peak absolute FVC and PEFr values with later onset of decline.

  • Time to FVC < 1 liter was delayed by steroid treatment; FVC < 1 liter increased risk of death 4-fold.

Abstract

We describe changes in pulmonary function measures across time in Duchenne muscular dystrophy patients treated with glucocorticoids (GCs) > 1 year compared to GC naïve patients in the Cooperative International Research Group Duchenne Natural History Study, a multicenter prospective cohort study. 397 participants underwent 2799 pulmonary function assessments over a period up to 10 years. Fifty-three GC naïve participants (< 1 month exposure) were compared to 322 subjects with > 1 year cumulative GC treatment. Forced vital capacity (FVC), peak expiratory flow rate (PEFr), maximal inspiratory and expiratory pressures were performed and calculated as a percent predicted (%p). GC treatment slowed the rate of pulmonary decline as measured by FVC%p, in patients aged 7–9.9 years. GC treatment slowed 12 and 24-month progression of percent predicted spirometry to a greater degree in those with baseline FVC%p from < 80–34%. GC treatment resulted in higher peak absolute FVC and PEFr values with later onset of decline. Progression to an absolute FVC < 1 liter was delayed by GC treatment. Patients who reached a FVC below 1 L were 4.1 times more likely to die (p = 0.017). Long-term glucocorticoid treatment slows pulmonary disease progression in Duchenne dystrophy throughout the lifespan.

Keywords

Duchenne
Muscular dystrophy
Pulmonary
Steroids
Mortality

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