Evidence map›Paper›PMID 21753160›Full record

Trial reportNeurology2011

Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy.

D M Escolar, L P Hache, P R Clemens, A Cnaan, C M McDonald, V Viswanathan, A J Kornberg, T E Bertorini, Y Nevo, T Lotze and 11 more

3 registry-linked trialsOpen access · bronzeAbstract readRandomized Controlled Trial
In one paragraph

Trial report in Neurology, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 87 papers, 6 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
87citing papers in PubMed, 6 pooled it
5.1field-weighted citation impact, top 4% of its field
1 · What the graph read from it

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.

2 · The registry

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.

NCT02167217 phase2completednot on this mapstarted 2014, after this paper: background citation

Phase 2 Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

TypeinterventionalSponsorWashington University School of MedicineRan2014 to 2017Enrolled25ConditionsDuchenne Muscular DystrophyArmsPrednisolone
NCT03439670 phase2completednot on this mapstarted 2018, after this paper: background citation

A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)

TypeinterventionalSponsorReveraGen BioPharma, Inc.Ran2018 to 2021Enrolled121ConditionsDuchenne Muscular DystrophyArmsVamorolone, Prednisone, Placebo
NCT04054375 phase2completednot on this mapstarted 2019, after this paper: background citation

Open Label Safety and Efficacy of Once Weekly Steroid in Patients With LGMD and Becker Muscular Dystrophy

TypeinterventionalSponsorNorthwestern UniversityRan2019 to 2022Enrolled20ConditionsLimb-girdle Muscular Dystrophy, Becker Muscular DystrophyArmsPrednisone
3 · Its place in the literature

Who cites it

87 citing papers in PubMed, 6 syntheses or guidelines pooled it, 161 citations in OpenAlex.

  1. Exploring the natural history of bone mineral density in Duchenne muscular dystrophy: a systematic literature review.Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA · 2025
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  5. Corticosteroids for the treatment of Duchenne muscular dystrophy.The Cochrane database of systematic reviews · 2016
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  20. Automated bone age calculation is an independent predictor of vertebral fracture burden in Duchenne muscular dystrophy.Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA · 2025
    Article

27 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

21 authors at 1 institution in 1 country.

D M EscolarChildren's National Medical Center, Washington, DC, USA. diana.escolar@gmail.com
L P Hache
P R Clemens
A Cnaan
C M McDonald
V Viswanathan
A J Kornberg
T E Bertorini
Y Nevo
T Lotze
A Pestronk
M M Ryan
E Monasterio
J W Day
A Zimmerman
A Arrieta
E Henricson
J Mayhew
J Florence
F Hu
A M Connolly
Baylor College of Medicine · US

Funding

University of Pittsburgh Clinical and Translational Science InstituteUL1TR000005 · NCATS · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI REIS, STEVEN E · 2012 to 2015
$50.8M
ZAP-70 EXPRESSION, CD38 EXPRESSION AND PROGNOSIS IN CHRONIC LYMPHOCYTIC LEUKEMIAM01RR020359 · NCRR · CHILDREN'S RESEARCH INSTITUTE · PI RAKHMANINA, NATELLA Y · 2005 to 2010
$13.8M
Mentored Patient Oriented Research Career DevelopmentK23RR016281 · NCRR · CHILDREN'S RESEARCH INSTITUTE · PI ESCOLAR, DIANA M · 2001 to 2005
$622k
NCATS NIH HHS UL1 TR000005NCRR NIH HHS 5M01 RR020359NCRR NIH HHS K23 RR16281-01
6 · The paper itself

Abstract

objectiveTo perform a double-blind, randomized study comparing efficacy and safety of daily and weekend prednisone in boys with Duchenne muscular dystrophy (DMD).

methodsA total of 64 boys with DMD who were between 4 and 10 years of age were randomized at 1 of 12 centers of the Cooperative International Neuromuscular Research Group. Efficacy and safety of 2 prednisone schedules (daily 0.75 mg/kg/day and weekend 10 mg/kg/wk) were evaluated over 12 months.

resultsEquivalence was met for weekend and daily dosing of prednisone for the primary outcomes of quantitative muscle testing (QMT) arm score and QMT leg score. Secondary strength scores for QMT elbow flexors also showed equivalence between the 2 treatment groups. Overall side effect profiles of height and weight, bone density, cataract formation, blood pressure, and behavior, analyzed at 12 months, did not differ between weekend and daily dosing of prednisone.

conclusionsWeekend dosing of prednisone is equally beneficial to the standard daily dosing of prednisone. Analysis of side effect profiles demonstrated overall tolerability of both dosing regimens. CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that weekend prednisone dosing is as safe and effective as daily prednisone in preserving muscle strength and preventing body mass index increases in boys with DMD over a 12-month period.

Indexed as

Age FactorsBody Mass IndexChildChild, PreschoolDose-Response Relationship, DrugDouble-Blind MethodDrug Administration ScheduleFollow-Up StudiesGlucocorticoidsHumansMaleMuscle StrengthMuscular Dystrophy, DuchennePrednisoneTreatment OutcomeGlucocorticoidsPrednisone

Identifiers

PMID21753160
PMCPMC3146308
OpenAlexW2060963350

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.