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

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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

summary/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 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.

organisation: 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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