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

Abstract

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.

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial

“…In this age group at the early ambulatory stage of the disease, a substantial number of boys who have been diagnosed with DMD are not being treated with corticosteroids. In both the US and Europe, over 30%of boys in this age range were reported not to be on steroids, in some cases because parents declined or deferred treatment [ 32, 33 ]. The protocol guided investigators to consider the clinical trajectory of patients at enrollment and as they continued in the trial and to recommend transition to steroids if they felt that clinical progression warranted withdrawal and steroid initiation.…”

Health Profile of Preterm Males With Duchenne Muscular Dystrophy

“…The results are consistent with other studies that have reported the average age of corticosteroid initiation as 6.5 years 19 and 5.9 years. 31 For our analytic sample, the hazard for corticosteroid use was similar between preterm and full-term males with Duchenne muscular dystrophy (hazard ratio = 0.93, 95% confidence interval: 0.61, 1.42).…”

Characterizing demographics, comorbidities, and costs of care among populations with Duchenne muscular dystrophy with Medicaid and commercial coverage

“…First, the generally short follow-up (2-4 years) and differences in median follow-up duration between the cohorts makes directly comparing some outcomes between the commercial and Medicaid DMD cohorts challenging. For example, while corticosteroids are known to delay progression of DMD, 21,[41][42][43][44] expected levels of corticosteroid use were not observed in this study, presumably because not all cohort members appeared in the data during the critical window in which these would have been prescribed. Thus, a comprehensive understanding of each individual's lifetime corticosteroid use was not possible and may impact the interpretation of comparisons between the DMD cohorts.…”

“…It should be noted that the low rate of observed corticosteroid use in this study is consistent with other reports from studies using real-world data and highlights the need for additional treatment options for DMD. 43 Identifying patients based on DMD-genotype specific treatments (such as eteplirsen) or ICD-10-CM diagnositic codes was also explored but did not identify any additional patients beyond those already captured by the study inclusion criteria. The number of patients identified with eteplirsen use was low, since this treatment had only become available for the subset of eligible DMD patients towards the end of the study period.…”