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Variability and trends in corticosteroid use by male United States participants with Duchenne muscular dystrophy in the Duchenne Registry

BMC Neurology(2019)

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摘要
Background Treatment options for Duchenne muscular dystrophy remain limited, although consensus treatment guidelines recommend corticosteroid use. Methods This retrospective analysis assessed corticosteroid use in ambulatory and nonambulatory US males with Duchenne, age 35 and under, or Becker muscular dystrophy, who enrolled in The Duchenne Registry from 2007 to 2016 (formerly DuchenneConnect). Results The mean (SD) age of corticosteroid use initiation was 5.9 (2.5) years, and deflazacort use (54%) was slightly more common than prednisone/prednisolone (46%). Among all responses from those with Duchenne, 63% were currently on corticosteroids, 12% were no longer on corticosteroids, and 25% had never been on corticosteroids. Among those who were nonambulatory, 49% were currently on corticosteroids, 28% had discontinued corticosteroids, and 23% had never used corticosteroids. Primary reasons for never initiating therapy were that corticosteroids were not prescribed or recommended and concerns about side effects. Corticosteroid use was maximal at age 8 (84% on corticosteroids) and gradually declined from age 10 to 19. The primary reasons for corticosteroid discontinuation were problems with side effects (65%) or not enough benefit (28%). Average doses of corticosteroids were below recommended doses. In the 159 responses with Becker muscular dystrophy, 20% were currently using corticosteroids. Conclusions Recognizing the self-selected nature of participation, it appears that a considerable proportion of US participants registered with The Duchenne Registry were either not on corticosteroids or not on recommended doses despite consensus recommendations. Side effects were a consideration in initiating and discontinuing treatment. These data reinforce the need for additional treatment options for those affected by Duchenne.
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关键词
Duchenne muscular dystrophy, DuchenneConnect, The Duchenne Registry, Rare disease registries, Corticosteroids
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