Trial reportNeurology2016
Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy.
Open access · hybridAbstract readClinical Trial, Phase IIIComparative StudyMulticenter Study
In one paragraph
Trial report in Neurology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 92 papers, 5 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
92citing papers in PubMed, 5 pooled it
8.9field-weighted citation impact, top 2% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
92 citing papers in PubMed, 5 syntheses or guidelines pooled it, 171 citations in OpenAlex.
- Meta-analysis of clinical trials assessing the safety of pharmacological treatments for muscular degeneration in Duchenne muscular dystrophy.BMC pharmacology & toxicology · 2026Pooled it
- Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A Systematic Review.Paediatric drugs · 2024Pooled it
- Efficacy and safety of different doses of vamorolone in boys with Duchenne muscular dystrophy: a systematic review and network meta-analysis.Frontiers in neurology · 2024Pooled it
- The Effectiveness and Value of Deflazacort and Exon-Skipping Therapies for the Management of Duchenne Muscular Dystrophy.Journal of managed care & specialty pharmacy · 2020Pooled it
- Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials.Muscle & nerve · 2020Pooled it
- Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy.Journal of neurology · 2026Trial
- Ductal lavage followed by observation versus oral corticosteroids in idiopathic granulomatous mastitis: A randomized trial.Nature communications · 2024Trial
- Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.Neurology · 2024Trial
- Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status.Journal of comparative effectiveness research · 2023Trial
- Chidamide plus prednisone, etoposide, and thalidomide for untreated angioimmunoblastic T-cell lymphoma in a Chinese population: A multicenter phase II trial.American journal of hematology · 2022Trial
- Trial
- Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.PLoS medicine · 2020Trial
- Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.Muscle & nerve · 2018Trial
- Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.Pharmacological research · 2018Trial
- Status of Clinical Care of Duchenne Muscular Dystrophy: Global Perspective and Situation in India.Indian journal of pediatrics · 2026Review
- Age-Dependent Effect of Myostatin Blockade in theMuscles (Basel, Switzerland) · 2026Article
- Influencing factors and early warning indicators for nonvertebral fractures in patients with Duchenne muscular dystrophy.JBMR plus · 2026Article
- Duchenne muscular dystrophy: from gene to gene-ius therapies.Skeletal muscle · 2026Review
- A Validated Prognostic Score for Time to Loss of Ambulation in Patients With Duchenne Muscular Dystrophy.Neurology · 2026Article
- Gene Editing Strategies for Duchenne Muscular Dystrophy: From Molecular Mechanisms to Clinical Translation.Cells · 2026Review
32 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors at 5 institutions in 2 countries.
Robert C GriggsFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL. Robert_Griggs@URMC.Rochester.edu.
J Phillip MillerFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
Cheryl R GreenbergFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
Darcy L FehlingsFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
Alan PestronkFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
Jerry R MendellFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
Richard T MoxleyFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
Wendy KingFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
John T KisselFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
Valerie CwikFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
Michel VanasseFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
Julaine M FlorenceFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
Shree PandyaFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
Jordan S DubowFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
James M MeyerFrom the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
Muscular Dystrophy Association · USThe Ohio State University Wexner Medical Center · USCentre Hospitalier Universitaire Sainte-Justine · CANationwide Children's Hospital · USUniversity of Rochester Medical Center · US
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveTo assess safety and efficacy of deflazacort (DFZ) and prednisone (PRED) vs placebo in Duchenne muscular dystrophy (DMD).
methodsThis phase III, double-blind, randomized, placebo-controlled, multicenter study evaluated muscle strength among 196 boys aged 5-15 years with DMD during a 52-week period. In phase 1, participants were randomly assigned to receive treatment with DFZ 0.9 mg/kg/d, DFZ 1.2 mg/kg/d, PRED 0.75 mg/kg/d, or placebo for 12 weeks. In phase 2, placebo participants were randomly assigned to 1 of the 3 active treatment groups. Participants originally assigned to an active treatment continued that treatment for an additional 40 weeks. The primary efficacy endpoint was average change in muscle strength from baseline to week 12 compared with placebo. The study was completed in 1995.
resultsAll treatment groups (DFZ 0.9 mg/kg/d, DFZ 1.2 mg/kg/d, and PRED 0.75 mg/kg/d) demonstrated significant improvement in muscle strength compared with placebo at 12 weeks. Participants taking PRED had significantly more weight gain than placebo or both doses of DFZ at 12 weeks; at 52 weeks, participants taking PRED had significantly more weight gain than both DFZ doses. The most frequent adverse events in all 3 active treatment arms were Cushingoid appearance, erythema, hirsutism, increased weight, headache, and nasopharyngitis.
conclusionsAfter 12 weeks of treatment, PRED and both doses of DFZ improved muscle strength compared with placebo. Deflazacort was associated with less weight gain than PRED. CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that for boys with DMD, daily use of either DFZ and PRED is effective in preserving muscle strength over a 12-week period.
Indexed as
AdolescentAnti-Inflammatory AgentsBody WeightChildChild, PreschoolDouble-Blind MethodHumansLeast-Squares AnalysisMaleMotor ActivityMuscle StrengthMuscular Dystrophy, DuchennePrednisonePregnenedionesTreatment OutcomeAnti-Inflammatory AgentsdeflazacortPrednisonePregnenediones
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