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Scientific Articles

Longitudinal Pulmonary Function Testing Outcome Measures in Duchenne Muscular Dystrophy: Long-term Natural History with and without Glucocorticoids

key information

source: Neuromuscular Disorders

year: 2018

authors: McDonald CM, Gordish-Dressman H, Henricson EK, Duong T, Joyce, NC, Jhawar S, Leinonen M, Hsu F, Connolly AM, Cnaan A, Abresch RT


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 to 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% to 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 liter 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.

organization: University of California Davis Health, USA; Center for Genetic Medicine, Children's National Health System and the George Washington University School of Medicine and Health Sciences, USA; Stanford University, USA; Clinical Data Science, Basel, Switzerland; Washington University, USA; Cooperative International Neuromuscular Research Group

DOI: 10.1016/j.nmd.2018.07.004

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