Study of Different Modalities of Steroids Use in Children with Duchenne Muscular Dystrophy: An Exploratory Prospective Study

Our aim was to compare the effects of Prednisolone (Pred) and Deflazacort (DFZ) on anthropometric parameters, quality of life (QoL), emotional wellbeing, functional status, muscles ultrasonography (US) and pulmonary function tests (PFT) in ambulatory patients with Duchenne muscular dystrophy (DMD). Thirty-one steroid naive ambulant boys with DMD were included. Twelve were started on prdnisone (Pred) and 19 on deflazacort (DFZ). Initial and 9 months later assessments included anthropometric measurements, QoL and behavioral evaluation, GSGC (Gait, Stairs, Gower, Chair) scores to assess motor functions, PFT and US was used to assess rectus femoris muscles and diaphragmatic muscle thickness. By the end of the study caregivers of the 2 groups were asked whether their children developed some of the known steroids side effects. Patients’ weight increased significantly within each group upon comparing initial and follow up data 23.24+/-4.45 and 24.77+/-5.14, p = 0.028 for Pred group and 21.43+/-4.02 and 22.33+/-4.29, p = 0.002 for DFZ group. HRQOL score was higher both initially and in the 9 months follow up assessment in DFZ patients 92.37+/-12.84 and 88.88+/-12.26 compared to Pred patients 75.67+/-14.06 and 76.67+/-12.00. DFZ group showed better gait and chair rising scores (2 (1–3)), (1 (1–2)) compared to Pred group (2.5 (2-4.5)) (2 (2–4.5) p < 0.05. No differences were found upon comparing ultrasonography parameters or PFT of the 2 participating groups. Patients in Pred group developed more side effects than those in DFZ group. Patients on DFZ had better motor functions and less side effects compared to those on Pred.