Steroids in duchenne muscular dystrophy — Deflazacort trial Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study Deflazacort vs. prednisone in Duchenne muscular dystrophy: trends of an ongoing study A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy. Deflazacort in Duchenne dystrophy: study of long-term effect. A comparison of daily and alternate-day prednisone therapy in the treatment of Duchenne muscular dystrophy. Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group. Duchenne dystrophy Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. Prednisone versus deflazacort in the treatment of autoimmune thrombocytopenic purpura: evaluation of clinical response and immunological modifications. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study Factorial randomised controlled trial of glucocorticoids and combination disease modifying drugs in early rheumatoid arthritis Effects of deflazacort versus prednisone on bone mass, body composition, and lipid profile: a randomized, double blind study in kidney transplant patients. Comparative Safety Profile of Levofloxacin in 2523 Children With a Focus on Four Specific Musculoskeletal Disorders Additive effects of prednisolone and beclomethasone dipropionate in patients with stable chronic obstructive pulmonary disease. The reflex dystrophy syndrome response to treatment with systemic corticosteroids. The effect of methylphenidate on prefrontal cognitive functioning, inattention, and hyperactivity in velocardiofacial syndrome.
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 Objective : We aim ed to perform an observational study of age at loss of independent ambulation ( LoA  ) and side-effect profiles associated with different glucocorticoid corticosteroid ( GC ) regimens in Duchenne muscular dystrophy ( DMD ) . Methods : We studied 340 participants in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study ( CINRG-DNHS ) . LoA was defined as continuous wheelchair use . Effects of prednisone or prednisolone (PRED)/deflazacort ( DFZ ) , administration frequency , and dose were analyzed by time-varying Cox regression . Side-effect frequencies were compared using χ2 test . Results : Participants treated ≥1 year while ambulatory ( n = 252/340 ) showed a 3-year median delay in LoA ( p < 0.001 ) . Fourteen different regimens were observed . Nondaily treatment was common for PRED ( 37 % ) and rare for DFZ ( 3 % ) . DFZ was associated with later LoA than PRED ( hazard ratio 0.294 ± 0.053 vs 0.490 ± 0.08 , p = 0.003 ; 2-year difference in median LoA with daily administration , p < 0.001 ) . Average dose was lower for daily PRED ( 0.56 mg/kg/d , 75 % of recommended ) than daily DFZ ( 0.75 mg/kg/d , 83 % of recommended , p < 0.001 ) . DFZ showed higher frequencies of growth delay ( p < 0.001 ) , cushingoid appearance ( p = 0.002 ) , and cataracts ( p < 0.001 ) , but not weight gain . Conclusions : Use of DFZ was associated with later LoA and increased frequency of side effects . Differences in st and ards of care and dosing complicate interpretation of this finding , but stratification by PRED/DFZ might be considered in clinical trials . This study emphasizes the necessity of a r and omized , blinded trial of GC regimens in DMD . Classification of evidence : This study provides Class IV evidence that GCs are effective in delaying LoA in patients with DMD 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 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 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 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 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 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 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 We report the results of a r and omized clinical trial of two different coricosteroids ( prednisone versus deflazacort ) in patients affected by autoimmune thrombocytopenic purpura ( ATP ) . We have evaluated the efficacy of the two steroids on platelet count , antiplatelet antibodies , lymphocyte subsets and the occurrence of side effects .  Twenty-seven patients were evaluable : 13 were treated with PDN and 14 with DFC .  After 24 weeks of treatment , 4/12 ( 33 % ) , subjects treated with PDN were refractory while complete responses were obtained in 2/12 ( 17 % ) and partial responses in 6/12 ( 50 % ) . Among patients treated with DFC , 4/11 ( 36 % ) were considered as refractory , 2/11 ( 18 % ) had a complete response and 5/11 ( 46 % ) a partial response . A statistically significant decrease of antiplatelet antibodies was recorded in both groups after 4 weeks of therapy , but only in subjects receiving PDN did the reduction last until the 24th week . We observed an increase of T lymphocyte subsets ( CD3 , CD2 , CD4 , CD8 ) in absolute number , due to an increase in circulating lymphocytes , after 4 weeks . No substantial modifications were observed in these population s regarding the percentage or the CD4/CD8 ratio . After 24 weeks , 91 % ( 10/11 ) of patients treated with PDN presented an increase of body weight and 1 had a stable increase in blood pressure . Among the subjects treated with DFC , 64 % ( 7/11 ) showed an increase of body weight after the same follow-up . In conclusion , no difference was observed the two steroids studied BACKGROUND Glucocorticoid treatment is recommended as a st and ard of care in Duchenne muscular dystrophy ; however , few studies have assessed the long-term benefits of this treatment . We examined the long-term effects of glucocorticoids on milestone-related disease progression across the lifespan and survival in patients with Duchenne muscular dystrophy . METHODS For this prospect i ve cohort study , we enrolled male patients aged 2 - 28 years with Duchenne muscular dystrophy at 20 centres in nine countries . Patients were followed up for 10 years . We compared no glucocorticoid treatment or cumulative treatment duration of less than 1 month versus treatment of 1 year or longer with regard to progression of nine disease-related and clinical ly meaningful mobility and upper limb milestones . We used Kaplan-Meier analyses to compare glucocorticoid treatment groups for time to st and from supine of 5 s or longer and 10 s or longer , and loss of st and from supine , four-stair climb , ambulation , full overhead reach , h and -to-mouth function , and h and function . Risk of death was also assessed . This study is registered with Clinical Trials.gov , number NCT00468832 . FINDINGS 440 patients were enrolled during two recruitment periods ( 2006 - 09 and 2012 - 16 ) . Time to all disease progression milestone events was significantly longer in patients treated with glucocorticoids for 1 year or longer than in patients treated for less than 1 month or never treated ( log-rank p<0·0001 ) . Glucocorticoid treatment for 1 year or longer was associated with increased median age at loss of mobility milestones by 2·1 - 4·4 years and upper limb milestones by 2·8 - 8·0 years compared with treatment for less than 1 month . Deflazacort was associated with increased median age at loss of three milestones by 2·1 - 2·7 years in comparison with prednisone or prednisolone ( log-rank p<0·012 ) . 45 patients died during the 10-year follow-up . 39 ( 87 % ) of these deaths were attributable to Duchenne-related causes in patients with known duration of glucocorticoids usage . 28 ( 9 % ) deaths occurred in 311 patients treated with glucocorticoids for 1 year or longer compared with 11 ( 19 % ) deaths in 58 patients with no history of glucocorticoid use ( odds ratio 0·47 , 95 % CI 0·22 - 1·00 ; p=0·0501 ) . INTERPRETATION In patients with Duchenne muscular dystrophy , glucocorticoid treatment is associated with reduced risk of losing clinical ly meaningful mobility and upper limb disease progression milestones across the lifespan as well as reduced risk of death . FUNDING US Department of Education/National Institute on Disability and Rehabilitation Research ; US Department of Defense ; National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases ; and Parent Project Muscular Dystrophy Objective : Treating early active rheumatoid arthritis ( RA ) with disease modifying antirheumatic drug ( DMARD ) monotherapy achieves incomplete outcomes and intensive treatment seems preferable . As the relative benefits of combining two DMARDs , one DMARD with glucocorticoids and two DMARDs with glucocorticoids are uncertain we defined them in a factorial trial . Methods : A 2-year r and omised double-blind factorial trial in patients with RA within 2 years of diagnosis treated with methotrexate  studied the benefits of added ciclosporin , 9 months intensive prednisolone or both ( triple therapy ) . The primary outcome was the number of patients with new erosions . Secondary outcomes included Larsen ’s x-ray scores , disability , quality of life and adverse events . Findings : 1391 patients were screened and 467 r and omised . Over 2 years 132 ( 28 % ) changed therapy and 88 ( 19 % ) were lost to follow-up . The number of patients with new erosions was reduced by nearly half by adding ciclosporin or prednisolone ( p = 0.01 and 0.03 ) ; both treatments reduced increases in Larsen ’s x-ray scores by over 2 units ( p = 0.008 and 0.003 ) . A further reduction in erosive damage was seen with combined use of both treatments . Their effects on erosive damage appeared independent . Triple therapy reduced disability and improved quality of life compared with methotrexate ; ciclosporin and prednisolone acted synergistically . More patients withdrew because of adverse events with triple therapy , without an increase in serious adverse effects . Conclusions : This study confirms the existence of a “ window of opportunity ” in early RA , when intensive combination therapy produces sustained benefits on damage and disability . Although methotrexate – prednisolone combinations reduce erosive damage , the synergistic effect of two DMARDs is needed to improve quality of life To compare the effects of deflazacort ( DEFLA ) vs. prednisone ( PRED ) on bone mineral density ( BMD ) , body composition , and lipids , 24 patients with end-stage renal disease were r and omized in a double blind design and followed 78 weeks after kidney transplantation .  BMD and body composition were assessed using dual energy x-ray absorptiometry . Seventeen patients completed the study .  Glucocorticosteroid doses , cyclosporine levels , rejection episodes , and drop-out rates were similar in both groups . Lumbar BMD decreased more in PRED than in DEFLA ( P < 0.05 ) , the difference being particularly marked after 24 weeks ( 9.1 + /- 1.8 % vs. 3.0 + /- 2.4 % , respectively ) . Hip BMD decreased from baseline in both groups ( P < 0.01 ) , without intergroup differences . Whole body BMD decreased from baseline in PRED ( P < 0.001 ) , but not in DEFLA . Lean body mass decreased by approximately 2.5 kg in both groups after 6 - 12 weeks ( P < 0.001 ) , then remained stable . Fat mass increased more ( P < 0.01 ) in PRED than in DEFLA ( 7.1 + /- 1.8 vs. 3.5 + /- 1.4 kg ) . Larger increases in total cholesterol ( P < 0.03 ) , low density lipoprotein cholesterol ( P < 0.01 ) , lipoprotein B2 ( P < 0.03 ) , and triglycerides ( P = 0.054 ) were observed in PRED than in DEFLA . In conclusion , using DEFLA instead of PRED in kidney transplant patients is associated with decreased loss of total skeleton and lumbar spine BMD , but does not alter bone loss at the upper femur . DEFLA also helps to prevent fat accumulation and worsening of the lipid profile Background : Fluoroquinolones , including levofloxacin , have not been recommended for use in children largely because studies in juvenile laboratory animals suggest there may be an increased risk of fluoroquinolone-associated cartilage lesions . A large prospect i ve trial is needed to assess the risks associated with using levofloxacin in children . Objective : Assess the safety and tolerability of levofloxacin therapy in children based on observations for 1 year after therapy . Methods : Safety data were collected in children who participated in 1 of 3 efficacy trials ( N = 2523 ) and a subset of these children who also subsequently participated in a long-term 1-year surveillance trial ( N = 2233 ) . Incidence of adverse events in children r and omized to receive levofloxacin versus nonfluoroquinolone antibiotics  was compared . Based on assessment s by treating physicians and an independent data safety monitoring committee , events related to the musculoskeletal system were further categorized as 1 of 4 predefined musculoskeletal disorders ( arthralgia , arthritis , tendinopathy , gait abnormality ) considered most likely clinical correlates of fluoroquinolone-associated cartilage lesions observed in laboratory animals . Results : Levofloxacin was well tolerated during and for 1 month after therapy as evidence d by similar incidence and character of adverse events compared with nonfluoroquinolone antibiotics . However , incidence of at least 1 of the 4 predefined musculoskeletal disorders ( largely due to reports of arthralgia ) was greater in levofloxacin-treated compared with nonfluoroquinolone-treated children at 2 months ( 2.1 % vs. 0.9 % ; P = 0.04 ) and 12 months ( 3.4 % vs. 1.8 % ; P = 0.03 ) after starting therapy . Conclusions : The incidence of 1 or more of the 4 predefined musculoskeletal disorders identified in nonblinded , prospect i ve evaluations , was statistically greater in levofloxacin-treated compared with comparator-treated children It remains unclear whether inhaled corticosteroids can produce the maximum benefits of corticosteroids  in patients with chronic obstructive pulmonary disease ( COPD ) . To assess the additive effects of 30 mg/day prednisolone to high-dose , inhaled beclomethasone dipropionate ( BDP ) , we conducted a r and omised double-blind , placebo-controlled cross-over trial . The study population consisted of 21 men with stable COPD . The mean age of the patients was 69.1 + /- 6.8 years , and FEV(1)was 0.86 + /- 0.28 l. Seventeen out of the 21 patients ( 81 % ) were considered susceptible to steroids in a previous trial ( FEV(1)increased at least 15 % from baseline after receiving 14 days of 30 mg/day prednisolone ) . All of the patients had been on 1600 microg/day BDP for more than 3 months .  Spirometry was performed before the entry , and at the end of 3-week placebo and prednisolone periods . The peak expiratory flow ( PEF ) , symptoms , and Guyatt 's Chronic Respiratory Disease Question naire ( CRQ ) as a disease specific health-related quality of life over the last seven days of each period were also evaluated . Although a marginal increase in PEF was found during the prednisolone period , no significant differences in FEV(1 ) , FVC , symptoms or CRQ scores were observed between the two treatment periods . We conclude that the therapeutic effects of steroid therapy may be achieved by the long-term use of high-dose , inhaled corticosteroid in some patients with stable COPD Twenty-three patients with reflex dystrophy syndrome were r and omly allocated to medication with oral prednisone , 10 mg thrice daily , or placebo , previous reports having indicated effect of systemic corticosteroids . The medication was continued until clinical remission , maximally 12 weeks . The diagnosis was based on fulfillment of at least four of seven criteria which included clinical , radiological and circulatory changes . All 13 patients in the prednisone-treated group showed more than 75 % clinical improvement within the twelve-week period . Of the ten patients who received placebo , only two reported improvement .  Prednisone appears to be superior to other treatment in reflex dystrophy syndrome , although the mode of action is not known OBJECTIVE Methylpheni date ( MPH ) is commonly used to treat attention-deficit/hyperactivity disorder ( ADHD ) in all children , including those with velocardiofacial syndrome ( VCFS ) . Yet concerns have been raised regarding its safety and efficacy in VCFS . The goal of this study was to examine the safety and efficacy of MPH in children with VCFS . METHODS Thirty-four children and adolescents with VCFS and ADHD participated in a r and omized , controlled trial with a 2:1 ratio of MPH versus placebo . All subjects underwent a cardiological evaluation before and after MPH administration . The primary outcome measure was prefrontal cognitive performance following a single dose of MPH or placebo . A follow-up assessment was conducted after a 6-month treatment with MPH . RESULTS Compared with placebo , single MPH administration was associated with a more robust improvement in prefrontal cognitive performance , including achievements in the Hearts and Flowers executive function task and the visual continuous performance task . After 6 months of treatment , a 40 % reduction in severity of ADHD symptoms was reported by parents on the Revised Conners Rating Scale . All subjects treated with MPH reported at least one side effect , but it did not necessitate discontinuation of treatment . MPH induced an increase in heart rate and blood pressure that was usually minor , but was clinical ly significant in two cases . No differences in response to MPH were observed between catechol-O-methyltransferase Met versus Val carriers . CONCLUSION The use of MPH in children with VCFS appears to be effective and relatively safe . A comprehensive cardiovascular evaluation for children with VCFS before and during stimulant treatment is recommended
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 ) ?