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Neuromuscul Disord. 2018 Nov;28(11):897-909. doi: 10.1016/j.nmd.2018.07.004. Epub 2018 Aug 29.

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

Author information

1
University of California Davis Health, Sacramento, CA, USA. Electronic address: cmmcdonald@ucdavis.edu.
2
Center for Genetic Medicine, Children's National Health System, United States; The George Washington University School of Medicine and Health Sciences, Washington DC, USA.
3
University of California Davis Health, Sacramento, CA, USA.
4
Stanford University, Stanford, CA, United States.
5
Clinical Data Science, Basel, Switzerland.
6
Washington University, St. Louis, MO, USA.
7
Buenos Aires, Argentina.
8
Melbourne, VIC, Australia.
9
Sydney, NSW.
10
Toronto, ON, Canada.
11
Calgary, AB.
12
Edmonton, AB.
13
Chennai, India.
14
Jerusalem, Israel.
15
Milan, Italy.
16
San Juan, Puerto Rico.
17
Göteborg, Sweden.
18
Houston, TX, USA.
19
Memphis, TN.
20
Minneapolis, MN.
21
Pittsburgh, PA.
22
Richmond, VA.
23
Rochester, MN.
24
St Louis, MO.
25
Sacramento, CA.
26
Washington, DC.

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; Mortality; Muscular dystrophy; Pulmonary; Steroids

PMID:
30336970
DOI:
10.1016/j.nmd.2018.07.004
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