A recent systematic review and meta-analysis published in the Annals of Medicine and Surgery offers new insights into the treatment of Duchenne muscular dystrophy (DMD) by comparing the efficacy and safety of daily versus intermittent use of corticosteroids.
While targeted drugs to treat DMD are now being studied, there’s no cure for the disorder, and corticosteroids, along with rehabilitation and psychosocial support, are the current standard of care to manage symptoms. However, there remains a lack of clarity on optimal dosing regimens, or whether it’s best to take corticosteroids daily or intermittently to give the body a break from the drugs.
To help shed some light on this, researchers analyzed data from six studies with a total of 708 participants with DMD comparing daily vs intermittent prednisone, the primary corticosteroid used in the management of DMD.
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The results showed there were no statistically significant differences between the two dosing regimens in terms of efficacy. To determine this, the researchers looked at measures like respiratory function, changes in weight, bone density, bone fracture risk, the ability to walk independently and behavioral changes.
However, the two dosing regimens did score differently in terms of safety profile. Compared to the daily dose regimen, intermittent prednisone was associated with a higher risk of cushingoid appearance (characterized by a round, puffy “moon-shaped” face), excessive hair growth and high blood pressure.
The researchers said these results could help inform physicians when prescribing prednisone to individuals with DMD. “These findings provide valuable insights for clinicians when choosing treatment strategies and highlight the need for personalized approaches to minimize side effects while maintaining efficacy,” the authors said.
However, the authors also noted important limitations in the studies they analyzed, including inconsistencies in reporting key confounding factors, such as disease severity and concurrent therapies. The authors said the results of their meta-analysis should be interpreted with caution, and called for more research comparing intermittent versus daily prednisone in DMD to validate their findings
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