Evidence map›Paper›PMID 31594858›Full record

Trial reportNeurology2019

Relationships between

Mathula Thangarajh, Jos Hendriksen, Michael P McDermott, William Martens, Kimberly A Hart, Robert C Griggs, Muscle Study Group and TREAT-NMD

Open access · greenAbstract readMulticenter StudyRandomized Controlled Trial
In one paragraph

Trial report in Neurology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
34citing papers in PubMed, 3 pooled it
2.7field-weighted citation impact, top 9% 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

34 citing papers in PubMed, 3 syntheses or guidelines pooled it, 75 citations in OpenAlex.

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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

7 authors at 2 institutions in 2 countries.

Mathula ThangarajhFrom the Department of Neurology (M.T.), Children's National Health System, Washington, DC; Kempenhaeghe Center for Neurological Learning Disabilities (J.H.), Heeze, the Netherlands; and School of Medicine and Dentistry (M.P.M., W.M., K.A.H., R.C.G.), University of Rochester Medical Center, NY. M.T. is currently affiliated with the Department of Neurology, Virginia Commonwealth University, Richmond. mathula.thangarajh@vcuhealth.org.
Jos HendriksenFrom the Department of Neurology (M.T.), Children's National Health System, Washington, DC; Kempenhaeghe Center for Neurological Learning Disabilities (J.H.), Heeze, the Netherlands; and School of Medicine and Dentistry (M.P.M., W.M., K.A.H., R.C.G.), University of Rochester Medical Center, NY. M.T. is currently affiliated with the Department of Neurology, Virginia Commonwealth University, Richmond.
Michael P McDermottFrom the Department of Neurology (M.T.), Children's National Health System, Washington, DC; Kempenhaeghe Center for Neurological Learning Disabilities (J.H.), Heeze, the Netherlands; and School of Medicine and Dentistry (M.P.M., W.M., K.A.H., R.C.G.), University of Rochester Medical Center, NY. M.T. is currently affiliated with the Department of Neurology, Virginia Commonwealth University, Richmond.
William MartensFrom the Department of Neurology (M.T.), Children's National Health System, Washington, DC; Kempenhaeghe Center for Neurological Learning Disabilities (J.H.), Heeze, the Netherlands; and School of Medicine and Dentistry (M.P.M., W.M., K.A.H., R.C.G.), University of Rochester Medical Center, NY. M.T. is currently affiliated with the Department of Neurology, Virginia Commonwealth University, Richmond.
Kimberly A HartFrom the Department of Neurology (M.T.), Children's National Health System, Washington, DC; Kempenhaeghe Center for Neurological Learning Disabilities (J.H.), Heeze, the Netherlands; and School of Medicine and Dentistry (M.P.M., W.M., K.A.H., R.C.G.), University of Rochester Medical Center, NY. M.T. is currently affiliated with the Department of Neurology, Virginia Commonwealth University, Richmond.
Robert C GriggsFrom the Department of Neurology (M.T.), Children's National Health System, Washington, DC; Kempenhaeghe Center for Neurological Learning Disabilities (J.H.), Heeze, the Netherlands; and School of Medicine and Dentistry (M.P.M., W.M., K.A.H., R.C.G.), University of Rochester Medical Center, NY. M.T. is currently affiliated with the Department of Neurology, Virginia Commonwealth University, Richmond.
Muscle Study Group and TREAT-NMD
Children's National · USKempenhaeghe · NL

Funding

FOR-DMD:Double-blind randomized trial to optimize steroid regimen in Duchenne MDU01NS061799 · NINDS · UNIVERSITY OF ROCHESTER · PI GRIGGS, ROBERT C, GUGLIERI, MICHELA · 2010 to 2019
$13.7M
Biostatistics and Data Management for a Trial of Corticosteroid Regimens in DMDU01NS061795 · NINDS · UNIVERSITY OF ROCHESTER · PI MCDERMOTT, MICHAEL P, TAWIL, RABI · 2010 to 2019
$3.5M
NINDS NIH HHS U01 NS061795NINDS NIH HHS U01 NS061799
6 · The paper itself

Abstract

objectiveWe performed a prospective, cross-sectional analysis of neurodevelopmental concerns and psychosocial adjustment in relation to

methodsWe evaluated 196 steroid-naive boys with dystrophinopathy who were enrolled in the Finding the Optimal Regimen for Duchenne Muscular Dystrophy trial. The neurodevelopmental concerns and psychosocial adjustment challenges were analyzed in relation to

resultsWe found that 39% of boys were reported to have speech delay with a mean age of speaking at 28 months (range 7-66 months). Learning difficulties were reported in 28% of participants. Inattentive-overactive and oppositional-defiant behavior was reported in 8% and 5% of participants, respectively. Psychosocial adjustment challenges were reported in 4% of participants. An ASD diagnosis was reported in 3 participants. Speech delay and learning difficulties were more common in boys with mutations downstream of

conclusionOur data support evidence for a relationship between neurodevelopmental concerns and

Indexed as

MutationChildChild, PreschoolCross-Sectional StudiesDouble-Blind MethodDystrophinHumansMaleMuscular Dystrophy, DuchenneNeurodevelopmental DisordersNeuromuscular AgentsSteroidsDMD protein, humanDystrophinNeuromuscular AgentsSteroids

Identifiers

PMID31594858
PMCPMC6946466
OpenAlexW2979555170

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.