Retrospective cohort study comparing the efficacy of prednisolone and deflazacort in children with muscular dystrophy: A 6 years' experience in a South Indian teaching hospital.

BACKGROUND: Muscular dystrophies are inherited myogenic disorders characterized by progressive muscle wasting and weakness of variable distribution and severity. They are a heterogeneous group characterized by variable degree of skeletal and cardiac muscle involvement. The most common and the most severe form of muscular dystrophy is DMD. Currently, there is no curative treatment for muscular dystrophies. Several drugs have been studied to retard the progression of the muscle weakness. There is much controversy about steroid usage in muscular dystrophy with respect to regimen, adverse effects, and whether long term benefits outweigh side effects. This study is to assess steroid efficacy in children with muscular dystrophy.

MATERIALS AND METHODS: All children with diagnosed muscular dystrophy by muscle biopsy, immunohistochemistry and/or genetic test were enrolled in the study. They were started on either prednisolone (0.75 mg/kg/day) or deflazacort 0.9 mg/kg/day based on affordability. All were followed up every 6 months with clinical assessment, quality of life questionnaire and clinical and laboratory assessment of side effects. Outcome measures of children on deflazacort and prednisolone at 1 year followup were summarized as numbers and percentages and were compared using Fisher's exact test.

RESULTS: Twenty two children with muscular dystrophy were included (prednisolone group: 10 and deflazacort group: 12). The mean age was 7.7 years at an average followup of 26.4 months. Twenty children were diagnosed to have Duchenne's; one had Becker's muscular dystrophy while one had sarcoglycanopathy by Type 2C. All children from prednisolone group maintained their ambulatory status at 2 and 4 years followups while three on deflazacort lost their ability to walk at an average age of 11.3 years. All activities of daily living were found to be better in prednisolone group. Muscle function and time taken to walk improved in prednisolone group. Weight gain in children on prednisolone was three times more.

CONCLUSIONS: Prednisolone is more beneficial than deflazacort at doses of 0.75 mg/kg/day and 0.9 mg/kg/day, respectively, however it is associated with adverse effects.