Conclusions Examining individual studies it appears that DFZ improves strength and functional outcomes compared to placebo , but it remains unclear if it has a benefit over prednisone on similar outcomes .
Two trials found that DFZ causes less weight gain than prednisone
Background To complete a systematic review and meta- analysis based on the clinical question : Is Deflazacort ( DFZ ) , a prednisolone derivative , an effective therapy for improving strength , with acceptable side effects , in children with Duchenne Dystrophy ( DD ) ?
Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group.
A r and omized , controlled trial of daily prednisone was conducted in 99 boys ( aged 5 to 15 years ) with Duchenne dystrophy to define the time course of improvement and the dose response to treatment . Prednisone at 0.3 mg/kg ( n = 33 ) , prednisone at 0.75 mg/kg ( n = 34 ) , and placebo ( n = 32 ) were administered for 6 months . Patients were examined using manual muscle and myometry testing , timed functional testing , pulmonary function testing , and laboratory measurements at 10 days , 1 month , 2 months , 3 months , and 6 months of treatment . Boys treated with prednisone had stronger average muscle strength scores , than did boys treated with placebo as early as 10 days after starting therapy . At the 3-month visit , the boys in the group given 0.75 mg/kg of prednisone were significantly stronger than those in the group given 0.3 mg/kg of prednisone , indicating a dose response . At 6 months , significant side effects occurred in the group treated with 0.75 mg/kg of prednisone , including weight gain , cushingoid appearance , and excessive hari growth . Only weight gain was observed in the group taking prednisone at a dose of 0.3 mg/kg . Importantly , no side effects were evident at 10 days or 1 month of treatment , despite improvement in muscle strength and function . We conclude that prednisone produces a rapid increase in muscle strength in patients with Duchenne dystrophy and that this improvement is maximal at a prednisone dosage of 0.75 mg/kg or less
Conclusions Examining individual studies it appears that DFZ improves strength and functional outcomes compared to placebo , but it remains unclear if it has a benefit over prednisone on similar outcomes .
Two trials found that DFZ causes less weight gain than prednisone
Background To complete a systematic review and meta- analysis based on the clinical question : Is Deflazacort ( DFZ ) , a prednisolone derivative , an effective therapy for improving strength , with acceptable side effects , in children with Duchenne Dystrophy ( DD ) ?