Earlier this month, the publication, A Mixed-Method Study Exploring Patient-Experienced and Caregiver-Reported Benefits and Side Effects of Corticosteroid Use in Duchenne Muscular Dystrophy, was accepted into the Journal of Neuromuscular Diseases.

Background 

PPMD has led the way in patient-focused drug development—quantifying how people with Duchenne and their caregivers think and feel about emerging therapies and clinical trials. While corticosteroids are considered the standard of care in Duchenne, caregiver and patient views are not well characterized. To fill this gap, in early 2020, PPMD partnered with Catabasis Pharmaceuticals, PTC Therapeutics, Mallinckrodt Pharmaceuticals, and Santhera Pharmaceuticals and collaborated with RTI in order to better understand these perspectives. 

About the study design 

All participants in the study were either currently taking or had previously taken corticosteroids, or were parents of people with Duchenne or Becker with corticosteroid experience.  

• Twenty-eight individuals participated in the qualitative one-on-one interviews, half parents and half teens or adults with Duchenne or Becker.  
• 236 people completed an online survey, 90% of whom were parents.
• People with Duchenne and their caregivers were asked to rate whether they experienced a particular benefit or side effect and also how important that side effect or benefit was, as well as their corticosteroid use history, including when they started and if they took any breaks. 

What we learned

Benefits and Side Effects 

• The benefits of corticosteroids to breathing, heart function, arm strength, slowing progression of weakness, and getting around were rated as particularly important by people with Duchenne and their caregivers.  

• All participants expressed concern about increased fracture risk, unwanted weight gain, and diabetes.  
• Parents reported considering behavior issues highly important, whereas adults with Duchenne indicated they considered delayed puberty as highly important.  

Treatment Satisfaction 

• Overall, participants in this study were satisfied with corticosteroids and felt that the benefits of corticosteroids outweighed the risks.
• The type of corticosteroids did not significantly affect responses about side effects, benefits, or satisfaction.  

Download the Study

Conclusion  

Corticosteroids and the benefits they provide are important to people with Duchenne and their caregivers. PPMD is hopeful that this data can be used to help families make decisions about initiating or restarting corticosteroids and may help in understanding patient and caregiver perspectives in future drug development. 

Thank you to all the families who participated in this important study and to our sponsors for their support of this key research.