title: Steroids in duchenne muscular dystrophy — Deflazacort trial A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy. Deflazacort vs. prednisone in Duchenne muscular dystrophy: trends of an ongoing study Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group. A comparison of daily and alternate-day prednisone therapy in the treatment of Duchenne muscular dystrophy. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. Deflazacort in Duchenne dystrophy: study of long-term effect. Duchenne dystrophy
abstract: We conducted a double blind controlled trial in 28 Duchenne muscular dystrophy ( DMD ) patients with Deflazacort ( DF ) , an oxazoline derivative of prednisolone which reduces its side-effects . Myometric muscle strength measurements , Scott Score and timed tests showed statistically significant improvement for the treated group ( P less than 0.05 ) . Side-effects after 9 months of treatment included mild cushingoid appearance in four patients ( 28 % ) and moderate in only one ( 7 % ) , increased appetite in seven ( 50 % ) , increased body hair in four ( 28 % ) , irritability and hyperactivity in three ( 21 % ) . Increased body weight was not prominent and was controlled with dietary measures . No patient had to be withdrawn from medication . More research and long-term follow-up are needed in order to establish the mechanism of improvement and the consequences of long-term steroid administration in DMD . In this regard DF appears as an alternative to prednisone preserving its benefits but with fewer side-effects We r and omized 18 Duchenne muscular dystrophy ( DMD ) boys whose age ranged from 5.2 to 14.6 years ( mean , 7.3 years ) for treatment with either deflazacort ( 0.9 mg/kg/day ) or prednisone ( 0.75 mg/kg/day ) on the basis of age and functional score at the onset of treatment . We followed the patients every 3 months for 1 year , evaluating four limb muscles with the Medical Research Council scale and performance of four functions ( walking , climbing stairs , Gowers ' maneuver , and rising from a chair ) . Side effects were monitored by a question naire and by routine blood examination , and weight and height were recorded at each visit . At 12 months , the effect of both steroids was examined by comparing the status of the treated patients with another group of untreated DMD patients that served as natural history control . The two steroids were equally effective in improving motor function and functional performances . At 9 months , the average weight increase with respect to baseline value was 5 % ( 2 kg ) in the deflazacort group but 18 % in the prednisone group ( P < 0 . 005 ) , and the change remained significant after 12 months ( P < 0.05 ) . Other minor but nonsignificant side effects were observed . Steroid treatment with deflazacort appears to cause fewer side effects than with prednisone , particularly weight gain , which could be important to maximize motor performances Several studies have demonstrated the slowing effect of corticosteroids on the decline of muscle strength in Duchenne muscular dystrophy ( DMD ) .  Deflazacort ( DFC ) is supposed to have fewer side effects than prednisone ( PRED ) . An ongoing double blind multicenter study is comparing the effects and side effects of deflazacort ( 0.9 mg/kg/day ) and prednisone ( 0.75 mg/kg/day ) in DMD . This interim report includes data for 67 boys between age 5 years and loss of ambulation . Besides the common clinical and laboratory data for chronic corticoid treatment , motor performance has been tested . Interim results , 3 - 15 months after starting the medication , show some scattering but no grouping of data for all the functions tested : timed motor functions , sum of the strength of 20 muscles according to a 10-point scale on manual testing , weight gain , osteocalcin and alkaline phosphatase . Only the changes in CK activity after 3 months medication might reflect two equal groups without any correlation with the initial activity or with other parameters . On average , there was no clear-cut loss of muscle strength or performance . Except for in 4 patients , who were excluded due to unacceptable weight gain and /or loss of ambulation  , there were no side effects considered to be serious . The results suggest that ( i ) DFC and PRED in equal anti-inflammatory dosage are similarly or equally efficient in slowing down the decline of muscle strength in DMD ; ( ii ) benefits outweigh the side effects . This allows the study to continue as design ed 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 We previously reported the results of a r and omized , double-blind 6-month trial of prednisone therapy in which 102 boys aged 5 to 15 years with Duchenne muscular dystrophy received daily doses of 1.5 and 0.75 mg/kg per day and were compared with those receiving placebo . The strength and function in both prednisone-treated groups improved equally and were significantly better than in the placebo group . To compare alternate-day and daily dosing of prednisone with respect to benefits and adverse side effects , the placebo group was started on alternate-day prednisone therapy , and the treatment group regimens were changed to equivalent doses of alternate-day prednisone without breaking the double-blind nature . At the end of 6 months , the group that was changed from daily to alternate-day therapy had declined in strength back to levels observed 12 months previously , at the start of daily therapy . The group in which alternate-day therapy was started showed a significant improvement in strength at 3 months , similar in magnitude to the response of boys treated with daily therapy . However , their strength declined significantly in the subsequent 3 months compared with boys who received daily therapy . The frequency of side effects was not significantly different for alternate-day therapy compared with daily therapy . We conclude that alternate-day prednisone therapy effectively increases strength but does not sustain the improvement to the same extent as daily therapy or mitigate side effects We performed a r and omized , double-blind , controlled six-month trial of prednisone in 103 boys with Duchenne 's muscular dystrophy ( age , 5 to 15 years ) . The patients were assigned to one of three regimens : prednisone , 0.75 mg per kilogram of body weight per day ( n = 33 ) ; prednisone , 1.5 mg per kilogram per day ( n = 34 ) ; or placebo ( n = 36 ) . The groups were initially comparable in all measures of muscle function . Both prednisone groups had significant improvement of similar degree in the summary scores of muscle strength and function . Improvement began as early as one month and peaked by three months . At six months the high-dose prednisone group , as compared with the placebo group , had improvement in the time needed to rise from a supine to a st and ing position ( 3.4 vs. 6.2 seconds ) , to walk 9 m ( 7.0 vs. 9.7 seconds ) , and to climb four stairs ( 4.0 vs. 7.1 seconds ) , in lifting a weight ( 2.1 vs. 1.2 kg ) , and in forced vital capacity ( 1.7 vs. 1.5 liters ) ( P less than 0.001 for all comparisons ) . There was an increase in urinary creatinine excretion ( 261 vs. 190 mg per 24 hours ) , which suggested an increase in total muscle mass . However , the prednisone-treated patients who had required long-leg braces ( n = 5 ) or wheelchairs ( n = 11 ) continued to require them . The most frequent side effects were weight gain , cushingoid appearance , and excessive hair growth . We conclude from this six-month study that prednisone improves the strength and function of patients with Duchenne 's muscular dystrophy . However , further research is required to identify the mechanisms responsible for these improvements and to determine whether prolonged treatment with corticosteroids may be warranted despite their side effects A r and omized double-blind controlled trial of deflazacort was conducted in 28 Duchenne muscular dystrophy patients either treated with deflazacort 2.0 mg/kg alternate-day therapy or placebo .  The deflazacort group showed significant improvement in climbing stairs ( P < 0.01 ) , in rising from a chair , Gower 's maneuver , and walking ( P < 0.0025 ) after 6 months of treatment . After 1 year , all the above changes remained significantly improved and the MRC index was significantly better ( P < 0.05 ) in the treated group . After 2 years , a significant change was found in the MRC index : higher scores in walking , chair rising ( P < 0.02 ) , and grade and time of Gower 's maneuver ( P < 0.05 ) were found . The mean time for loss of ambulation for the treated group after we started the trial was 33.2 + /- 9 months ; for the placebo group it was 20.5 + /- 11 months ( deflazacort vs. placebo group , P < 0.05 ) [ corrected ] . Our treated patients lost their ambulation at a median age of 11.8 years vs. 10.5 years in the placebo group . Side effects were mild , consisting of moderate weight gain and slight behavioral changes Prednisone has been shown to improve strength in Duchenne dystrophy .  Azathioprine often benefits corticosteroid-responsive diseases and can reduce the dose of prednisone needed . The present study reports a r and omized , controlled trial of prednisone and azathioprine design ed to assess the longer-term effects of prednisone and to determine whether azathioprine alone , or in combination with prednisone , improves strength .  Ninety-nine boys ( aged five to 15 years ) with Duchenne dystrophy were r and omized to one of three groups : ( I ) placebo ; ( II ) prednisone 0.3 mg/kg/d ; or ( III ) prednisone 0.75 mg/kg/d . After 6 months , azathioprine 2 to 2.5 mg/kg/d was added in groups I and II and placebo added in group III . The study showed that the beneficial effect of prednisone ( 0.75 mg/kg/d ) is maintained for at least 18 months and is associated with a 36 % increase in muscle mass . There was weight gain , growth retardation , and other side effects .  Azathioprine did not have a beneficial effect . This study suggests that prednisone 's beneficial effect is not due to immunosuppression
target: 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: 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 ) ?