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

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

Author information

1
Department of Neurology, Washington University School of Medicine in Saint Louis, St Louis, Missouri, 63110, USA.
2
Department of Pediatrics, Washington University School of Medicine in Saint Louis, St Louis, Missouri, USA.
3
Department of Pediatrics, Nationwide Children's Hospital, Ohio State University, Columbus, Ohio, USA.
4
Department of Neurology, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.
5
Department of Pediatrics, Nemours Children's Hospital, Orlando, Florida, USA.
6
Department of Physical Medicine and Rehabilitation, University of California, Davis Medical Center, Sacramento, California, USA.
7
Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, USA.

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

PMID:
30706490
DOI:
10.1002/mus.26441
[Indexed for MEDLINE]

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