Corticosteroid Treatments in Males With Duchenne Muscular Dystrophy: Treatment Duration and Time to Loss of Ambulation

Sunkyung Kim; Kimberly A Campbell; Deborah J Fox; Dennis J Matthews; Rodolfo Valdez; Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet)

doi:10.1177/0883073814558120

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Corticosteroid Treatments in Males With Duchenne Muscular Dystrophy: Treatment Duration and Time to Loss of Ambulation

Journal article

Peer reviewed

Corticosteroid Treatments in Males With Duchenne Muscular Dystrophy: Treatment Duration and Time to Loss of Ambulation

Sunkyung Kim, Kimberly A Campbell, Deborah J Fox, Dennis J Matthews, Rodolfo Valdez and Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet)

Journal of child neurology, Vol.30(10), pp.1275-1280

09/2015

DOI: 10.1177/0883073814558120

PMCID: PMC4439376

PMID: 25414237

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https://www.ncbi.nlm.nih.gov/pmc/articles/4439376View

Open Access

Abstract

This population-based study examines the association between corticosteroid treatment and time to loss of ambulation, stratifying by treatment duration (short: 0.25-3 years, long: >3 years), among 477 Duchenne muscular dystrophy cases identified by the Muscular Dystrophy Surveillance Tracking and Research Network (MDSTARnet). Those cases who received short-term corticosteroid treatment had a time to loss of ambulation that was 0.8 years shorter (t test) and an annual risk of losing ambulation 77% higher than the untreated (Cox regression). Conversely, cases who received long-term corticosteroid treatment had a time to loss of ambulation that was 2 years longer and an annual risk of losing ambulation 82% lower than the untreated, up to age 11 years; after which the risks were not statistically different. The relationship of corticosteroids and time to loss of ambulation is more complex than depicted by previous studies limited to treatment responders or subjects who lost ambulation during study follow-up.

United States

Adrenal Cortex Hormones - therapeutic use

Age of Onset

Child

Disease Progression

Follow-Up Studies

Humans

Longitudinal Studies

Male

Muscular Dystrophy, Duchenne - drug therapy

Muscular Dystrophy, Duchenne - physiopathology

Neuromuscular Agents - therapeutic use

Prednisone - therapeutic use

Pregnenediones - therapeutic use

Proportional Hazards Models

Time Factors

Walking

Details

Title: Subtitle: Corticosteroid Treatments in Males With Duchenne Muscular Dystrophy: Treatment Duration and Time to Loss of Ambulation
Creators: Sunkyung Kim - Division of Human Development and Disability, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA wox0@cdc.gov
Kimberly A Campbell - New York State Department of Health, Albany, NY, USA
Deborah J Fox - New York State Department of Health, Albany, NY, USA
Dennis J Matthews - Children's Hospital Colorado, Aurora, CO, USA
Rodolfo Valdez - Division of Human Development and Disability, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA
Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet)
Contributors: P A Romitti (Contributor) - University of Iowa, Epidemiology
Resource Type: Journal article
Publication Details: Journal of child neurology, Vol.30(10), pp.1275-1280
DOI: 10.1177/0883073814558120
PMID: 25414237
PMCID: PMC4439376
NLM abbreviation: J Child Neurol
ISSN: 0883-0738
eISSN: 1708-8283
Grant note: U01 DD000189 / NCBDD CDC HHS U01 DD001119 / NCBDD CDC HHS CC999999 / Intramural CDC HHS U01 DD000191 / NCBDD CDC HHS 5 U01 DD000191 / NCBDD CDC HHS U01 DD001118 / NCBDD CDC HHS
Language: English
Date published: 09/2015
Academic Unit: Epidemiology; Biostatistics
Record Identifier: 9984214725802771

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