Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy

Muscle Nerve. 2019 Jun;59(6):650-657. doi: 10.1002/mus.26441. Epub 2019 Feb 23.

Abstract

Introduction: Glucocorticosteroids (GC) are effective in slowing weakness in boys with Duchenne muscular dystrophy (DMD).

Methods: This is a multisite, 1-year, open-label trial of twice-weekly prednisolone (5 mg/kg/dose) in infants/young boys (0.4-2.4 years) with DMD. We compared changes in Bayley III Scales of Infant Development (Bayley-III) with untreated boys followed for 1 year (historical control cohort [HCC]). Twenty-three of 25 participants completed the study.

Results: Treated boys gained an average of 0.5 points on the Bayley-III gross motor scaled score (GMSS) compared with the HCC who, on average, declined 1.3 points (P = 0.03). All boys maintained linear growth, and none developed Cushingoid features. Excessive weight gain occurred in 13 of 23 (56%) boys.

Discussion: This study provides evidence that twice-weekly GC is well tolerated in infants and young boys with DMD and improves GMSS. Excessive weight gain is a potential risk. Longer follow-up is required to determine whether early GC initiation is feasible in most infants/boys with DMD. Muscle Nerve 59:650-657, 2019.

Trial registration: ClinicalTrials.gov NCT02167217.

Keywords: Bayley-III Scales of Infant and Toddler Development; DMD; Duchenne muscular dystrophy; GC; infants; twice-weekly glucocorticosteroid.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Case-Control Studies
  • Child, Preschool
  • Cohort Studies
  • Disease Progression
  • Glucocorticoids / administration & dosage*
  • Humans
  • Infant
  • Male
  • Muscle Weakness / physiopathology
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Muscular Dystrophy, Duchenne / physiopathology
  • Prednisolone / administration & dosage*
  • Weight Gain

Substances

  • Glucocorticoids
  • Prednisolone

Associated data

  • ClinicalTrials.gov/NCT02167217