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Observational Study
. 2015 Sep;30(10):1275-80.
doi: 10.1177/0883073814558120. Epub 2014 Nov 20.

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

Sunkyung Kim  1 , Kimberly A Campbell  2 , Deborah J Fox  2 , Dennis J Matthews  3 , Rodolfo Valdez  4 ; MD STARnet
Affiliations
Observational Study

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

Sunkyung Kim et al. J Child Neurol. 2015 Sep.

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.

Keywords: Duchenne muscular dystrophy; ambulation; corticosteroid; treatment duration.

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Conflict of interest statement

Declaration of Conflicting Interests

The authors declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article.

Figures

Figure 1
Figure 1
Boxplots of the distribution of time to ambulation loss (years) by medication and treatment duration.
Figure 2
Figure 2
Distribution of age at onset (years) by treatment duration.
See this image and copyright information in PMC

References

    1. TREAT-NMD. [Accessed May 10, 2014]; http://www.treat-nmd.eu/.
    1. Hoffman EP, Brown RH, Jr, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987;51:919–928. - PubMed
    1. Bushby K, Muntoni F, Urtizberea A, Hughes R, Griggs R. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids. Neuromuscul Disord; Report on the 124th ENMC International Workshop; 2–4 April 2004; Naarden, The Netherlands. 2004. pp. 526–534. - PubMed
    1. Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2008;23 CD003725. - PubMed
    1. Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol. 2010;9:177–189. - PubMed

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