title: Corticosteroid Treatments in Males With Duchenne Muscular Dystrophy: Treatment Duration and Time to Loss of Ambulation.
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.
authors.affiliation: 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.
authors.forename: Sunkyung
authors.initials: S
authors.lastname: Kim
authors.affiliation: New York State Department of Health, Albany, NY, USA.
authors.forename: Kimberly A
authors.initials: KA
authors.lastname: Campbell
authors.affiliation: New York State Department of Health, Albany, NY, USA.
authors.forename: Deborah J
authors.initials: DJ
authors.lastname: Fox
authors.affiliation: Children's Hospital Colorado, Aurora, CO, USA.
authors.forename: Dennis J
authors.initials: DJ
authors.lastname: Matthews
authors.affiliation: Division of Human Development and Disability, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA.
authors.forename: Rodolfo
authors.initials: R
authors.lastname: Valdez
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D017668:Age of Onset; D002648:Child; D018450:Disease Progression; D005500:Follow-Up Studies; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000503:physiopathology*; D009465:Neuromuscular Agents / Q000627:therapeutic use; D011241:Prednisone / Q000627:therapeutic use*; D011282:Pregnenediones / Q000627:therapeutic use*; D016016:Proportional Hazards Models; D013997:Time Factors; D014481:United States; D016138:Walking
chemical_list: D000305:Adrenal Cortex Hormones; D009465:Neuromuscular Agents; D011282:Pregnenediones; C021988:deflazacort; D011241:Prednisone
keywords: Duchenne muscular dystrophy; ambulation; corticosteroid; treatment duration