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.
Original language | English (US) |
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Pages (from-to) | 650-657 |
Number of pages | 8 |
Journal | Muscle and Nerve |
Volume | 59 |
Issue number | 6 |
DOIs | |
State | Published - Jun 2019 |
Funding
Abbreviations: AE, adverse event; Bayley-III, Bayley-III Scales of Infant Development; CDC, US Centers for Disease Control and Prevention; CE, clinical evaluator; DMD, Duchenne muscular dystrophy; FMSS, fine motor scaled score; GC, glucocorticosteroids; GMSS, gross motor scaled score; HCC, historical control cohort; TC, treated cohort Key words: Bayley-III Scales of Infant and Toddler Development; DMD; Duchenne muscular dystrophy; GC; infants; twice-weekly glucocorticosteroid Funding: All phases of this study were supported by a grant from the Muscular Dystrophy Association USA (Grant 266053). Conflicts of Interest: No author has relevant disclosures for this work. Clinical Trial Registration: ClinicalTrials.gov ID NCT02167217 “Historically controlled Trial of Corticosteroids in Young boys with Duchenne Muscular Dystrophy” ClinicalTrials.gov PRS Protocol ID 201308062
Keywords
- Bayley-III Scales of Infant and Toddler Development
- DMD
- Duchenne muscular dystrophy
- GC
- infants
- twice-weekly glucocorticosteroid
ASJC Scopus subject areas
- Clinical Neurology
- Physiology (medical)
- Cellular and Molecular Neuroscience
- Physiology