Evidence map›Paper›PMID 26311750›Full record

Observational studyNeurology2015

Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study.

Luca Bello, Heather Gordish-Dressman, Lauren P Morgenroth, Erik K Henricson, Tina Duong, Eric P Hoffman, Avital Cnaan, Craig M McDonald, CINRG Investigators

Open access · bronzeAbstract readObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in Neurology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 125 papers, 9 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
125citing papers in PubMed, 9 pooled it
7.2field-weighted citation impact, top 2% 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

125 citing papers in PubMed, 9 syntheses or guidelines pooled it, 213 citations in OpenAlex.

  1. Meta-analysis of the efficacy and safety of vamorolone in Duchenne muscular dystrophy.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025
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  9. Corticosteroids for the treatment of Duchenne muscular dystrophy.The Cochrane database of systematic reviews · 2016
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65 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors at 2 institutions in 1 country.

Luca BelloFrom the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
Heather Gordish-DressmanFrom the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
Lauren P MorgenrothFrom the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
Erik K HenricsonFrom the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
Tina DuongFrom the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
Eric P HoffmanFrom the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
Avital CnaanFrom the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
Craig M McDonaldFrom the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC. cmmcdonald@ucdavis.edu.
CINRG Investigators
University of California Davis Medical Center · USChildren's National · US

Funding

Washington University Institute of Clinical and Translational Sciences (UL1)UL1RR024992 · NCRR · WASHINGTON UNIVERSITY · PI EVANOFF, BRADLEY A · 2007 to 2011
$45.4M
Washington University Institute of Clinical and Translational SciencesUL1TR000448 · NCATS · WASHINGTON UNIVERSITY · PI EVANOFF, BRADLEY A · 2012 to 2016
$41.4M
SUPPLEMENT FOR RENOVATION OF ANIMAL FACILITIES AT INSTITUTE OF NEUROBIOLOGYG12RR003051 · NCRR · UNIVERSITY OF PUERTO RICO MED SCIENCES · PI RODRIGUEZ-MEDINA, JOSE R · 1986 to 2011
$28.1M
NCMRR-DC Core Molecular & Functional Outcome Measures in Rehabilitation MedicineR24HD050846 · NICHD · CHILDREN'S RESEARCH INSTITUTE · PI KNOBLACH, SUSAN M · 2005 to 2014
$8.6M
TRCLU54RR026139 · NCRR · UNIVERSITY OF PUERTO RICO MED SCIENCES · PI FRONTERA, WALTER R. · 2010 to 2011
$8.4M
CTSA INFRASTRUCTURE FOR PEDIATRIC RESEARCHUL1RR031988 · NCRR · CHILDREN'S RESEARCH INSTITUTE · PI GUAY-WOODFORD, LISA MARIE · 2010 to 2011
$8.2M
Wellstone Muscular Dystrophy Center: Children's Nat'l Med CtrU54HD053177 · NICHD · CHILDREN'S RESEARCH INSTITUTE · PI HOFFMAN, ERIC P. · 2005 to 2009
$7.3M
Biomarker discovery and validation in a Duchenne dystrophy natural history studyR01AR062380 · NIAMS · UNIVERSITY OF CALIFORNIA AT DAVIS · PI HATHOUT, YETRIB, MCDONALD, CRAIG M. · 2011 to 2014
$1.5M
Clinically Meaningful Outcomes for Duchenne Muscular Dystrophy Therapeutic TrialsR01AR061875 · NIAMS · UNIVERSITY OF CALIFORNIA AT DAVIS · PI MCDONALD, CRAIG M. · 2011 to 2014
$1.3M
NCATS NIH HHS UL1 TR000448NCRR NIH HHS G12RR003051NCRR NIH HHS U54RR026139NCRR NIH HHS UL1RR024992NCRR NIH HHS UL1RR031988NIAMS NIH HHS 1R01AR061875NIAMS NIH HHS R01 AR061875NIAMS NIH HHS R01AR062380NICHD NIH HHS R24HD050846NICHD NIH HHS U54HD053177
6 · The paper itself

Abstract

objectiveWe aimed 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).

methodsWe 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 χ(2) test.

resultsParticipants 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.

conclusionsUse of DFZ was associated with later LoA and increased frequency of side effects. Differences in standards 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 randomized, 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.

Indexed as

InternationalityMuscular Dystrophy, DuchennePrednisolonePrednisonePregnenedionesAdolescentAdultAnti-Inflammatory AgentsChildChild, PreschoolFollow-Up StudiesGlucocorticoidsHumansMaleYoung AdultAnti-Inflammatory AgentsdeflazacortGlucocorticoidsPrednisolonePrednisonePregnenediones

Identifiers

PMID26311750
PMCPMC4603595
OpenAlexW2159586986

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

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.