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J Pediatr. 2019 Jul;210:194-200.e2. doi: 10.1016/j.jpeds.2019.02.037. Epub 2019 Apr 5.

Comparison of Pulmonary Function Decline in Steroid-Treated and Steroid-Naïve Patients with Duchenne Muscular Dystrophy.

Author information

1
Division of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH. Electronic address: Hemant.Sawnani@cchmc.org.
2
Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
3
Wayne State University School of Medicine, Detroit, MI.
4
Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
5
Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH; Division of Endocrinology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
6
Division of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH.
7
Division of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
8
Pediatric Non-invasive Ventilation and Sleep Unit, Necker University Hospital, Paris, France; ASV Santé, Gennevilliers, France.
9
Pediatric Non-invasive Ventilation and Sleep Unit, Necker University Hospital, Paris, France.

Abstract

OBJECTIVE:

To describe and compare the lung function decline in patients with Duchenne muscular dystrophy on glucocorticoid therapy in contrast with glucocorticoid-naïve patients, and to define the deciles of pulmonary decline in glucocorticoid-treated patients.

STUDY DESIGN:

This retrospective study examined lung function of patients with Duchenne muscular dystrophy over 6 years of age followed between 2001 and 2015 at 2 centers-glucocorticoid-treated patients in Cincinnati, Ohio, and glucocorticoid-naïve patients in Paris, France. Forced vital capacity (FVC, FVC%), forced expiratory volume in 1 second, maximal inspiratory pressure, maximal expiratory pressure, and peak expiratory flow data were analyzed. Only FVC data were available for the French cohort.

RESULTS:

There were 170 glucocorticoid-treated patients (92%), 5 patients (2.7%) with past glucocorticoid use, and 50 French glucocorticoid-naïve patients. The peak absolute FVC was higher and was achieved at earlier ages in glucocorticoid-treated compared with glucocorticoid-naïve patients (peak FVC, 2.4 ± 0.6 L vs 1.9 ± 0.7 L; P < .0001; ages 13.5 ± 3.0 years vs 14.3 ± 2.8 years; P = .03). The peak FVC% was also higher and was achieved at earlier ages in glucocorticoid-treated patients (peak FVC%, 105.1 ± 25.1% vs 56 ± 20.9%; P < .0001; ages 11.9 ± 2.9 years vs 13.6 ± 3.2 years; P = .002). Rates of decline for both groups varied with age. Maximal rates of decline were 5.0 ± 0.26% per year (12-20 years) for glucocorticoid-treated and 5.1 ± 0.39% per year for glucocorticoid-naïve patients (11-20 years; P = .2). Deciles of FVC% decline in glucocorticoid-treated patients show that patients experience accelerated decline at variable ages.

CONCLUSIONS:

These data describe nonlinear rates of decline of pulmonary function in patients with Duchenne muscular dystrophy, with improved function in glucocorticoid-treated patients. FVC% deciles may be a useful tool for clinical and research use.

KEYWORDS:

DMD progression; FVC%; muscular dystrophy; pulmonary decline

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