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90 results

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Filters applied: Meta-Analysis, Randomized Controlled Trial. Clear all
Page 1
Deflazacort for the treatment of Duchenne Dystrophy: a systematic review.
Campbell C, Jacob P. Campbell C, et al. BMC Neurol. 2003 Sep 8;3:7. doi: 10.1186/1471-2377-3-7. Epub 2003 Sep 8. BMC Neurol. 2003. PMID: 12962544 Free PMC article.
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)? ...
BACKGROUND: To complete a systematic review and meta-analysis based on the clinical question: Is Deflazacort (DFZ), a prednisolone de …
Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Speed C, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Griggs RC; FOR-DMD Investigators of the Muscle Study Group; Straub V, van Ruiten H, Childs AM, Ciafaloni E, Shieh PB, Spinty S, Maggi L, Baranello G, Butterfield RJ, Horrocks IA, Roper H, Alhaswani Z, Flanigan KM, Kuntz NL, Manzur A, Darras BT, Kang PB, Morrison L, Krzesniak-Swinarska M, Mah JK, Mongini TE, Ricci F, von der Hagen M, Finkel RS, O'Reardon K, Wicklund M, Kumar A, McDonald CM, Han JJ, Joyce N, Henricson EK, Schara-Schmidt U, Gangfuss A, Wilichowski E, Barohn RJ, Statland JM, Campbell C, Vita G, Vita GL, Howard JF Jr, Hughes I, McMillan HJ, Pegoraro E, Bello L, Burnette WB, Thangarajh M, Chang T. Guglieri M, et al. JAMA. 2022 Apr 19;327(15):1456-1468. doi: 10.1001/jama.2022.4315. JAMA. 2022. PMID: 35381069 Free PMC article. Clinical Trial.

RESULTS: Among the 196 boys randomized (mean age, 5.8 years [SD, 1.0 years]), 164 (84%) completed the trial. Both daily prednisone and daily deflazacort were more effective than intermittent prednisone for the primary outcome (P < .001 for daily prednisone vs intermitte

RESULTS: Among the 196 boys randomized (mean age, 5.8 years [SD, 1.0 years]), 164 (84%) completed the trial. Both daily prednisone and daily …
Deflazacort dose optimization and safety evaluation in Duchenne muscular dystrophy (DOSE): A randomized, double-blind non-inferiority trial.
Reddy C, Patil AN, Suthar R, Sankhyan N, Sirari T, Kumar A, Bhattacharjee S, Saxena S, Saini AG, Sahu JK. Reddy C, et al. Eur J Paediatr Neurol. 2022 May;38:77-84. doi: 10.1016/j.ejpn.2022.04.004. Epub 2022 Apr 20. Eur J Paediatr Neurol. 2022. PMID: 35500465 Clinical Trial.
RESULT: A total of 97 patients were enrolled, 40 receiving 0.45 mg/kg/d and 45 receiving 0.9 mg/kg/d deflazacort comprised of mITT population. For primary endpoint analysis the mean (SD) change in 6MWD from baseline to week-24 was 9.7 m (41.5) in deflazacort 0.45 mg …
RESULT: A total of 97 patients were enrolled, 40 receiving 0.45 mg/kg/d and 45 receiving 0.9 mg/kg/d deflazacort comprised of mITT po …
Corticosteroids for the treatment of Duchenne muscular dystrophy.
Matthews E, Brassington R, Kuntzer T, Jichi F, Manzur AY. Matthews E, et al. Cochrane Database Syst Rev. 2016 May 5;2016(5):CD003725. doi: 10.1002/14651858.CD003725.pub4. Cochrane Database Syst Rev. 2016. PMID: 27149418 Free PMC article.
PRIMARY OUTCOME MEASURE: one two-year deflazacort RCT (n = 28) used prolongation of ambulation as an outcome measure but data were not adequate for drawing conclusions. ...Very low quality evidence suggested less weight gain with deflazacort than with prednisone at …
PRIMARY OUTCOME MEASURE: one two-year deflazacort RCT (n = 28) used prolongation of ambulation as an outcome measure but data were no …
Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis.
Landfeldt E, Alemán A, Abner S, Zhang R, Werner C, Tomazos I, Ferizovic N, Lochmüller H, Kirschner J. Landfeldt E, et al. J Neuromuscul Dis. 2024;11(3):579-612. doi: 10.3233/JND-230220. J Neuromuscul Dis. 2024. PMID: 38669554 Free PMC article.
Glucocorticoid therapy was associated with delayed loss of ambulation (overall meta-analysis HR deflazacort/prednisone/prednisolone: 0.44 [95% CI: 0.40-0.48]) (n = 25 studies). Earlier onset of first signs or symptoms, earlier loss of developmental milestones, lower baseli …
Glucocorticoid therapy was associated with delayed loss of ambulation (overall meta-analysis HR deflazacort/prednisone/prednisolone: …
Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials.
McDonald CM, Sajeev G, Yao Z, McDonnell E, Elfring G, Souza M, Peltz SW, Darras BT, Shieh PB, Cox DA, Landry J, Signorovitch J; ACT DMD Study Group and the Tadalafil DMD Study Group. McDonald CM, et al. Muscle Nerve. 2020 Jan;61(1):26-35. doi: 10.1002/mus.26736. Epub 2019 Nov 7. Muscle Nerve. 2020. PMID: 31599456 Free PMC article.
INTRODUCTION: In this study we characterized disease progression over 48 weeks among boys receiving deflazacort vs prednisone/prednisolone placebo arm treatment in two recent Duchenne muscular dystrophy (DMD) clinical trials. ...DISCUSSION: Deflazacort-treated patie …
INTRODUCTION: In this study we characterized disease progression over 48 weeks among boys receiving deflazacort vs prednisone/prednis …
Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy.
Shieh PB, Elfring G, Trifillis P, Santos C, Peltz SW, Parsons JA, Apkon S, Darras BT, Campbell C, McDonald CM; Members of the Ataluren Phase IIb Study Group; Members of the Ataluren Phase IIb Study Clinical Evaluator Training Group; Members of the ACT DMD Study Group; Members of the ACT DMD Clinical Evaluator Training Group. Shieh PB, et al. J Comp Eff Res. 2021 Dec;10(18):1337-1347. doi: 10.2217/cer-2021-0018. Epub 2021 Oct 25. J Comp Eff Res. 2021. PMID: 34693725 Free article.
Aim: Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. ...Significant and clinically meaningful improvements in 4-stair clim …
Aim: Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical mile …
Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.
Shieh PB, Mcintosh J, Jin F, Souza M, Elfring G, Narayanan S, Trifillis P, Peltz SW, Mcdonald CM, Darras BT; THE ACT DMD STUDY GROUP. Shieh PB, et al. Muscle Nerve. 2018 Nov;58(5):639-645. doi: 10.1002/mus.26191. Epub 2018 Sep 27. Muscle Nerve. 2018. PMID: 30028519 Free PMC article. Clinical Trial.
This post hoc analysis compares efficacy and safety for deflazacort and prednisone/prednisolone in the placebo arm. METHODS: Patients received deflazacort (n = 53) or prednisone/prednisolone (n = 61). ...Mean changes in 4-stair climb were 3.79 s (deflazacort; …
This post hoc analysis compares efficacy and safety for deflazacort and prednisone/prednisolone in the placebo arm. METHODS: Patients …
Effects of deflazacort vs. methylprednisone: a randomized study in kidney transplant patients.
Ferraris JR, Pasqualini T, Alonso G, Legal S, Sorroche P, Galich AM, Jasper H; Deflazacort Study Group. Ferraris JR, et al. Pediatr Nephrol. 2007 May;22(5):734-41. doi: 10.1007/s00467-006-0403-0. Epub 2007 Feb 9. Pediatr Nephrol. 2007. PMID: 17294225 Clinical Trial.
Metabolic effects of deflazacort vs. methylprednisone were studied in prepubertal patients after kidney transplantation. Thirty-one patients participated: 15 received deflazacort and 16 remained on methylprednisone. The study started at a mean of 2.1 years after tra …
Metabolic effects of deflazacort vs. methylprednisone were studied in prepubertal patients after kidney transplantation. Thirty-one p …
Deflazacort in Duchenne dystrophy: study of long-term effect.
Angelini C, Pegoraro E, Turella E, Intino MT, Pini A, Costa C. Angelini C, et al. Muscle Nerve. 1994 Apr;17(4):386-91. doi: 10.1002/mus.880170405. Muscle Nerve. 1994. PMID: 8170484 Clinical Trial.
A randomized 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 sta …
A randomized double-blind controlled trial of deflazacort was conducted in 28 Duchenne muscular dystrophy patients either treated wit …
90 results