Connolly AM, Zaidman CM, Golumbek PT, et al. Twice weekly Glucocorticosteroids in Infants and Young boys with Duchenne Muscular Dystrophy. Muscle & nerve 2019.
At the moment corticosteroid treatment is usually started when patients are a bit older (>5 years of age). In this study the benefits of early start were investigated. The patients seemed to show improved motor functions compared to non-users and side effects were mild. The study, however, only used historical controls, whereas in the latest years e.g. functionality is already improving just by improved care, making it harder to distinguish real improvement by the corticosteroids. Furthermore only a few patients were included in the trial.
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.