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Comparing Deflazacort and Prednisone in Duchenne Muscular Dystrophy.

W Douglas Biggar1, Andrew Skalsky2, Craig M McDonald3

  • 1University of Toronto, 15583 22nd Side Road, Georgetown, Ontario, Canada.

Journal of Neuromuscular Diseases
|June 20, 2022
PubMed
Summary

Deflazacort and prednisone offer benefits for Duchenne muscular dystrophy (DMD) patients. Deflazacort shows similar efficacy to prednisone with potentially fewer side effects like weight gain and behavioral issues, though bone health and cataracts may be concerns.

Keywords:
Deflazacortduchenne muscular dystrophyglucocorticoidsprednisoloneprednisone

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Area of Science:

  • Pharmacology and Clinical Therapeutics
  • Neuromuscular Disorders

Background:

  • Deflazacort and prednisone/prednisolone are standard treatments for Duchenne muscular dystrophy (DMD).
  • Both corticosteroids have demonstrated efficacy in improving muscle strength, motor function, pulmonary function, and survival in DMD patients.
  • Deflazacort received FDA approval for DMD in 2017, following years of off-label use.

Purpose of the Study:

  • To compare the pharmacological features, efficacy, and safety profiles of deflazacort and prednisone/prednisolone in DMD patients.
  • To elucidate differentiating characteristics including lipid solubility, pharmacokinetics, gene expression, mineralocorticoid receptor affinity, and glucose metabolism impact.

Main Methods:

  • Review of evidence from randomized clinical trials, prospective studies, meta-analyses, and post-hoc analyses.
  • Comparative analysis of pharmacological properties and clinical outcomes.
  • Assessment of safety profiles, including side effects and impact on growth and bone health.

Main Results:

  • Deflazacort demonstrates comparable or slower rates of functional decline versus prednisone/prednisolone in DMD patients.
  • Prednisone/prednisolone may be associated with greater weight gain and behavioral side effects.
  • Deflazacort use is linked to potentially worse outcomes in bone health, growth parameters, and cataract development.

Conclusions:

  • Deflazacort and prednisone/prednisolone are effective treatments for Duchenne muscular dystrophy.
  • Key pharmacological differences influence their respective safety and efficacy profiles.
  • Treatment choice may depend on balancing benefits against specific side effect concerns for individual DMD patients.