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.
journal_name
Muscle Nervejournal_title
Muscle & nerveauthors
Connolly AM,Zaidman CM,Golumbek PT,Cradock MM,Flanigan KM,Kuntz NL,Finkel RS,McDonald CM,Iannaccone ST,Anand P,Siener CA,Florence JM,Lowes LP,Alfano LN,Johnson LB,Nicorici A,Nelson LL,Mendell JR,MDA DMD Clinical Reseadoi
10.1002/mus.26441subject
Has Abstractpub_date
2019-06-01 00:00:00pages
650-657issue
6eissn
0148-639Xissn
1097-4598journal_volume
59pub_type
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