Twice weekly Glucocorticosteroids in Infants and Young boys with Duchenne Muscular Dystrophy

Connolly AM, Zaidman CM, Golumbek PT, et al. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle & nerve 2019;59:650-657

Connolly_2019_uscle Nerve. Jun;59(6);650-657

Editorial

McMillan HJ. Intermittent glucocorticoid regimes for younger boys with duchenne muscular dystrophy: Balancing efficacy with side effects. Muscle & nerve 2019;59:638-639

McMillan_2019_Muscle Nerve. Jun;59(6);638-639

Abstract

INTRODUCTION: Glucocorticosteroids (GC) are effective in slowing weakness in boys with Duchenne muscular dystrophy (DMD).

METHODS: This is a multisite, one-year, open-label trial of twice-weekly prednisolone (5mg/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) to untreated boys followed for one year (historical control cohort-HCC). Twenty-three of 25 completed the study.

RESULTS: Treated boys gained an average of 0.5 points on the Bayley-III gross motor scaled score (GMSS) compared to 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 13/23 (56%).

DISCUSSION: This study provides evidence that twice weekly GC are well tolerated in infants and young boys with DMD and improves GMSS. Excessive weight gain is a potential risk. Longer follow-up is needed to determine if early GC initiation is feasible in most infants/boys with DMD.

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