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

• Published in: Muscle & nerve 2019-06, Vol.59 (6), p.650-657
• Main Authors: Connolly, Anne M., Zaidman, Craig M., Golumbek, Paul T., Cradock, Mary M., Flanigan, Kevin M., Kuntz, Nancy L., Finkel, Richard S., McDonald, Craig M., Iannaccone, Susan T., Anand, Pallavi, Siener, Catherine A., Florence, Julaine M., Lowes, Linda P., Alfano, Lindsay N., Johnson, Linda B., Nicorici, Alina, Nelson, Leslie L., Mendell, Jerry R.
• Format: Article
• Language: English
• Subjects: Bayley‐III Scales of Infant and Toddler Development; DMD; Duchenne muscular dystrophy; Duchenne's muscular dystrophy; Dystrophy; Infants; Muscles; Muscular dystrophy; Prednisolone; twice‐weekly glucocorticosteroid; Weight
• ISSN: 0148-639X; 1097-4598
• DOI: 10.1002/mus.26441

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 See editorial on pages 638–639 in this issue.