Associations between timing of corticosteroid treatment initiation and clinical outcomes in Duchenne muscular dystrophy

Sunkyung Kim; Yong Zhu; Paul A Romitti; Deborah J Fox; Daniel W Sheehan; Rodolfo Valdez; Dennis Matthews; Brent J Barber; the MD STAR

doi:10.1016/j.nmd.2017.05.019

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Associations between timing of corticosteroid treatment initiation and clinical outcomes in Duchenne muscular dystrophy

Journal article

Peer reviewed

Associations between timing of corticosteroid treatment initiation and clinical outcomes in Duchenne muscular dystrophy

Sunkyung Kim, Yong Zhu, Paul A Romitti, Deborah J Fox, Daniel W Sheehan, Rodolfo Valdez, Dennis Matthews, Brent J Barber and the MD STAR

Neuromuscular disorders : NMD, Vol.27(8), pp.730-737

08/2017

DOI: 10.1016/j.nmd.2017.05.019

PMCID: PMC5824693

PMID: 28645460

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https://www.osti.gov/biblio/1550150View

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Abstract

•This study examined corticosteroid treatment and initiation of treatment on clinical outcomes.•Cases initiating corticosteroids at age ≤5 years had earlier onset of cardiomyopathy.•These cases also had higher risk of fracture and decreased respiratory function.•Timing of corticosteroid initiation deserves careful consideration. The long-term efficacy of corticosteroid treatment and timing of treatment initiation among Duchenne muscular dystrophy (DMD) patients is not well-understood. We used data from a longitudinal, population-based DMD surveillance program to examine associations between timing of treatment initiation (early childhood [before or at age 5 years], late childhood [after age 5 years], and naïve [not treated]) and five clinical outcomes (age at loss of ambulation; ages at onset of cardiomyopathy, scoliosis, and first fracture; and pulmonary function). Hazard ratios (HRs) and 95% confidence intervals (CIs) were estimated using survival analysis. DMD patients who initiated corticosteroid treatment in early childhood had a higher risk of earlier onset cardiomyopathy compared to cases who initiated treatment in late childhood (HR = 2.0, 95% CI = [1.2, 3.4]) or treatment naïve patients (HR = 1.9, 95% CI = [1.1, 3.2]), and higher risk of suffering a fracture (HR = 2.3, 95% CI = [1.4, 3.7] and HR = 2.6, 95% CI = [1.6, 4.2], respectively). Patients with early childhood treatment had slightly decreased respiratory function compared with those with late childhood treatment. Ages at loss of ambulation or scoliosis diagnosis did not differ statistically among treatment groups. We caution that the results from our study are subject to several limitations, as they were based on data abstracted from medical records. Further investigations using improved reporting of disease onset and outcomes are warranted to obtain a more definitive assessment of the association between the timing of corticosteroid treatment and disease severity.

Cardiomyopathy

Corticosteroid

Fractures

Pulmonary function

Ambulation

Scoliosis

Duchenne muscular dystrophy

Details

Title: Subtitle: Associations between timing of corticosteroid treatment initiation and clinical outcomes in Duchenne muscular dystrophy
Creators: Sunkyung Kim - Division of Human Development and Disability, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, 1600 Clifton Rd, Atlanta, GA 30333, USA
Yong Zhu - Department of Epidemiology, College of Public Health, The University of Iowa, 145 N Riverside Dr, S416 CPHB, Iowa City, IA 52242, USA
Paul A Romitti - Department of Epidemiology, College of Public Health, The University of Iowa, 145 N Riverside Dr, S416 CPHB, Iowa City, IA 52242, USA
Deborah J Fox - New York State Department of Health, Empire State Plaza, Corning Tower, Albany, NY 12237, USA
Daniel W Sheehan - University at Buffalo, State University of New York
Rodolfo Valdez - Division of Human Development and Disability, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, 1600 Clifton Rd, Atlanta, GA 30333, USA
Dennis Matthews - Children's Hospital Colorado, 13123 East 16th Avenue, Aurora, CO 80045, USA
Brent J Barber - College of Medicine, University of Arizona, Tucson, AZ 85724, USA
the MD STAR
Resource Type: Journal article
Publication Details: Neuromuscular disorders : NMD, Vol.27(8), pp.730-737
DOI: 10.1016/j.nmd.2017.05.019
PMID: 28645460
PMCID: PMC5824693
NLM abbreviation: Neuromuscul Disord
ISSN: 0960-8966
eISSN: 1873-2364
Publisher: Elsevier B.V
Grant note: DOI: 10.13039/100000030, name: Centers for Disease Control and Prevention, award: U01 DD000187, U01DD0001915, U01 DD000189, U01 DD000190; DOI: 10.13039/100006229, name: Oak Ridge Institute for Science and Education; DOI: 10.13039/100000015, name: U.S. Department of Energy; DOI: 10.13039/100000030, name: Centers for Disease Control and Prevention
Language: English
Date published: 08/2017
Academic Unit: Epidemiology; Biostatistics
Record Identifier: 9983995177702771

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