Although prednisolone remains the most common steroid medication prescribed in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), many patients opt to switch to deflazacort for a number of reasons. A study published in The Journal of Comparative Effectiveness Research demonstrates that outcomes reported by treating physicians were consistent with addressing patients’ primary reasons for switching to that medication. 

Read more about DMD etiology

DMD and BMD are both X-linked muscular disorders that are degenerative and chronic in nature. Although patients with BMD may survive to their 40s and beyond, most patients with DMD do not survive past the age of 30. There is currently no cure for both of these dystrophinopathies. Physicians use corticosteroid treatment as the primary way to delay disease progression (particularly the loss of ambulation) and to possibly lengthen survival. 

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Although prednisolone remains the first-line corticosteroid of choice, deflazacort (another corticosteroid) became available in 2017 when it gained FDA approval for use in DMD patients. There has been evidence that deflazacort improves clinical outcomes and reduces adverse effects associated with prednisolone. Research has found that deflazacort is associated with less weight gain compared to prednisolone and that DMD patients receiving deflazacort experienced significantly slower rates of functional decline over 48 weeks compared to patients who were on prednisolone. 

Read more about DMD prognosis 

This study was a retrospective chart review of patients with DMD or BMD who were on prednisolone before switching to deflazacort. Physicians treating eligible patients were recruited from a panel of neurologists from across the US. A total of 50 neurologists contributed data from 104 males with DMD or BMD, who switched from prednisolone to deflazacort. 

The most common reasons for the switch were in order from the most common to the least were:  

  • The desire to slow the disease progression (79.31% in DMD, 83.05% in BMD). 
  • Tolerability issues (46.67% in DMD, 67.21% in BMD). 
  • Parent/caregiver preference or request (37.93% in DMD, 33.33% in BMD). 
  • Patient perspective or request (37.93% in DMD, 20.00% in BMD)

Having recorded the reasons for the patient’s switch from prednisolone to deflazacort, the treating physicians were asked to give their perspective on the effectiveness of the switch. Below were the results: 

  • Somewhat effective (63.33% in DMD, 58.06% in BMD) 
  • Very effective (26.67% in DMD, 38.71% in BMD) 
  • Not effective (10.00% in DMD, 3.23% in BMD) 

This study provides important insights into the most common reasons for DMD and BMD patients switching from prednisolone to deflazacort and their associated outcomes. The results show that switching was “very” or “somewhat” effective in addressing primary concerns in 90%-95% of patients. 

Reference

Jessica RM, Claudio S, Brian P, et al. Steroid switching in dystrophinopathy treatment: a US chart review of patient characteristics and clinical outcomes. J Comp Eff Res. Published online July 19, 2021. doi:10.2217/cer-2021-0110

Becker muscular dystrophy deflazacort Emflaza Neurologists prednisolone