학술논문
Twice‐weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy.
Document Type
Article
Author
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.
Source
Subject
Language
ISSN
0148-639X
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. [ABSTRACT FROM AUTHOR]