Mechanisms and Clinical Applications of Glucocorticoid Steroids in Muscular Dystrophy

Mattia Quattrocelli*, Aaron S. Zelikovich, Isabella M. Salamone, Julie A. Fischer, Elizabeth M. McNally

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

43 Scopus citations

Abstract

Glucocorticoid steroids are widely used as immunomodulatory agents in acute and chronic conditions. Glucocorticoid steroids such as prednisone and deflazacort are recommended for treating Duchenne Muscular Dystrophy where their use prolongs ambulation and life expectancy. Despite this benefit, glucocorticoid use in Duchenne Muscular Dystrophy is also associated with significant adverse consequences including adrenal suppression, growth impairment, poor bone health and metabolic syndrome. For other forms of muscular dystrophy like the limb girdle dystrophies, glucocorticoids are not typically used. Here we review the experimental evidence supporting multiple mechanisms of glucocorticoid action in dystrophic muscle including their role in dampening inflammation and myofiber injury. We also discuss alternative dosing strategies as well as novel steroid agents that are in development and testing, with the goal to reduce adverse consequences of prolonged glucocorticoid exposure while maximizing beneficial outcomes.

Original languageEnglish (US)
Pages (from-to)39-52
Number of pages14
JournalJournal of neuromuscular diseases
Volume8
Issue number1
DOIs
StatePublished - 2021

Funding

Supported by NIH U54 AR052646, NIH RO1 NS047726, NIH F31 AR073655 (IMS), American Heart Association 20PRE35210837 (JAF), the Parent Project for Muscular Dystrophy. MQ is supported by NIH K01 DK121875 (NIDDK).

Keywords

  • Glucocorticoid steroids
  • immunomodulation
  • metabolism
  • molecular signaling
  • muscle physiology
  • muscular dystrophy
  • neuromuscular diseases

ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology

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