We have exciting news to share from The Duchenne Registry. A study using the Registry data was published this week in a well-known international journal, BMC Neurology. This study was led by researchers at Catabasis Pharmaceuticals and focused on the Registry’s corticosteroid data. The title of the manuscript is “Variability and trends in corticosteroid use by male United States participants with Duchenne muscular dystrophy in The Duchenne Registry.”

The Catabasis team, led by Joanne Donovan, MD, PhD, wanted to gain a better understanding of corticosteroid use in the United States since the establishment of the Duchenne Care Guidelines. The researchers performed a retrospective analysis of corticosteroid use in ambulatory and nonambulatory males with Duchenne or Becker muscular dystrophy who enrolled in The Duchenne Registry from 2007 to 2016. Only data from registrants living in the US were used in this study.

FINDINGS

The Catabasis team had survey responses from a total of 1538 Duchenne registrants and 154 Becker registrants. The primary surveys used for this study were the Corticosteroid Survey and the Muscle Function Survey. If you completed both of these surveys between 2007-2016 and you live in the US, it is very likely that your data was used in this study!

The following is a summary of the descriptive statistics resulting from this study.

• The researchers found that the mean age for initiating corticosteroid use was 5.9 years (with a standard deviation of 2.5 years). Deflazacort use was slightly more common than prednisone/prednisolone use (54% versus 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 highest 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 the recommended doses set forth in the Duchenne Care Guidelines (67% of the recommended doses). These findings reaffirm the uncertainties that still exist regarding corticosteroid dosing and regimens.
• For the 154 registrants with Becker muscular dystrophy who responded to the Corticosteroid Survey, only 20% were currently using corticosteroids.

THE VALUE OF THIS WORK

Using data from The Duchenne Registry, it appears that a considerable proportion of US participants 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.

THE DUCHENNE REGISTRY

Thank you to all the families who have participated in the Registry and completed their Medical Surveys. This work would not be possible without YOU! As stated by the authors, “Patient and family participation in The Duchenne Registry is extremely valuable in order to further clinical development of new treatments for Duchenne.”

Thank you for being citizen scientists and for contributing data that advances our fight to End Duchenne!

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