Evidence map›Paper›PMID 26833937›Full record

ReviewNeurology2016

Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy [RETIRED]: Report of the Guideline Development Subcommittee of the American Academy of Neurology.

David Gloss, Richard T Moxley, Stephen Ashwal, Maryam Oskoui

Retracted Registry-linked trialOpen access · bronzeAbstract readReviewRetracted Publication
PubMed Full text at PMC Publisher
In one paragraph

Review in Neurology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It has been retracted, and should not be counted. It is linked to trial NCT04054375 (Open Label Safety and Efficacy of Once Weekly Steroid in Patients With LGMD and Becker Muscular Dystrophy), which is not on this map. Cited by 105 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
105citing papers in PubMed, 3 pooled it
14.3field-weighted citation impact, top 1% 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.

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

105 citing papers in PubMed, 3 syntheses or guidelines pooled it, 261 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
    Pooled it
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  3. Corticosteroids for the treatment of Duchenne muscular dystrophy.The Cochrane database of systematic reviews · 2016
    Pooled it
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45 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

4 authors at 3 institutions in 1 country.

David GlossFrom the Department of Neurology (D.G.), Geisinger Health System, Danville, PA; Department of Neurology (R.T.M.), University of Rochester School of Medicine and Dentistry, NY; Department of Neurology (S.A.), Loma Linda University Medical Center, CA; and Departments of Pediatric and Neurology/Neurosurgery (M.O.), McGill University, Montréal, Canada.
Richard T MoxleyFrom the Department of Neurology (D.G.), Geisinger Health System, Danville, PA; Department of Neurology (R.T.M.), University of Rochester School of Medicine and Dentistry, NY; Department of Neurology (S.A.), Loma Linda University Medical Center, CA; and Departments of Pediatric and Neurology/Neurosurgery (M.O.), McGill University, Montréal, Canada.
Stephen AshwalFrom the Department of Neurology (D.G.), Geisinger Health System, Danville, PA; Department of Neurology (R.T.M.), University of Rochester School of Medicine and Dentistry, NY; Department of Neurology (S.A.), Loma Linda University Medical Center, CA; and Departments of Pediatric and Neurology/Neurosurgery (M.O.), McGill University, Montréal, Canada.
Maryam OskouiFrom the Department of Neurology (D.G.), Geisinger Health System, Danville, PA; Department of Neurology (R.T.M.), University of Rochester School of Medicine and Dentistry, NY; Department of Neurology (S.A.), Loma Linda University Medical Center, CA; and Departments of Pediatric and Neurology/Neurosurgery (M.O.), McGill University, Montréal, Canada.
Loma Linda University Medical Center · USGeisinger Health System · USLoma Linda University · US

Funding

Training and Educational CoreU54NS048843 · NINDS · UNIVERSITY OF ROCHESTER · PI THORNTON, CHARLES A · 2003 to 2017
$21.9M
Phase 2 Study of Mexiletine for the Treatment of Myotonic Dystrophy R01FD003716 · FDA · UNIVERSITY OF ROCHESTER · PI MOXLEY, RICHARD T · 2011 to 2015
$1.5M
CIHRFDA HHS 1R01FD003716NINDS NIH HHS 2U54NS048843PHS HHS HHSN2612012003188P
6 · The paper itself

Abstract

objectiveTo update the 2005 American Academy of Neurology (AAN) guideline on corticosteroid treatment of Duchenne muscular dystrophy (DMD).

methodsWe systematically reviewed the literature from January 2004 to July 2014 using the AAN classification scheme for therapeutic articles and predicated recommendations on the strength of the evidence.

resultsThirty-four studies met inclusion criteria. RECOMMENDATIONS: In children with DMD, prednisone should be offered for improving strength (Level B) and pulmonary function (Level B). Prednisone may be offered for improving timed motor function (Level C), reducing the need for scoliosis surgery (Level C), and delaying cardiomyopathy onset by 18 years of age (Level C). Deflazacort may be offered for improving strength and timed motor function and delaying age at loss of ambulation by 1.4-2.5 years (Level C). Deflazacort may be offered for improving pulmonary function, reducing the need for scoliosis surgery, delaying cardiomyopathy onset, and increasing survival at 5-15 years of follow-up (Level C for each). Deflazacort and prednisone may be equivalent in improving motor function (Level C). Prednisone may be associated with greater weight gain in the first years of treatment than deflazacort (Level C). Deflazacort may be associated with a greater risk of cataracts than prednisone (Level C). The preferred dosing regimen of prednisone is 0.75 mg/kg/d (Level B). Over 12 months, prednisone 10 mg/kg/weekend is equally effective (Level B), with no long-term data available. Prednisone 0.75 mg/kg/d is associated with significant risk of weight gain, hirsutism, and cushingoid appearance (Level B).

Indexed as

Academies and InstitutesAdrenal Cortex HormonesHumansMuscular Dystrophy, DuchenneNeurologyPractice Guidelines as TopicPregnenedionesResearch ReportTreatment OutcomeUnited StatesAdrenal Cortex HormonesdeflazacortPregnenediones

Identifiers

PMID26833937
PMCPMC4773944
OpenAlexW2306713582

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.