Evidence map›Paper›PMID 42443978›Full record

Trial reportSkeletal muscle2026

Evaluation of a serum protein signature as monitoring biomarker for Duchenne muscular dystrophy in a long-term clinical trial with corticosteroids.

Chiara Degan, Rebecca A Tobin, Sharon I de Vries, Albert Jiménez-Requena, Amela Peco, Michela Guglieri, Jordi Diaz-Manera, Yuri E M van der Burgt, Bart J M van Vlijmen, FOR-DMD investigators of the Muscle Study Group and 5 more

Registry-linked trialAbstract readRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Skeletal muscle, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01603407 (Duchenne Muscular Dystrophy), which is not on this map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
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.

NCT01603407 phase3completednot on this map

Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen

TypeinterventionalSponsorUniversity of RochesterRan2013 to 2019Enrolled196ConditionsDuchenne Muscular DystrophyArmsPrednisone, Deflazacort
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

15 authors.

Chiara DeganDepartment of Biomedical Data Sciences, Leiden University Medical Center, Leiden, The Netherlands. c.degan@lumc.nl.ORCID https://orcid.org/0009-0001-9993-9346
Rebecca A TobinCarleton University, Ottawa, ON, Canada.
Sharon I de VriesDepartment of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
Albert Jiménez-RequenaDepartment of Protein Science, School of Chemistry, Biology and Health, KTH Royal Institute of Technology, Stockholm, Sweden.
Amela PecoDepartment of Protein Science, School of Chemistry, Biology and Health, KTH Royal Institute of Technology, Stockholm, Sweden.
Michela GuglieriThe John Walton Muscular Dystrophy Research Center, Newcastle University, Newcastle Upon Tyne, UK.
Jordi Diaz-ManeraThe John Walton Muscular Dystrophy Research Center, Newcastle University, Newcastle Upon Tyne, UK.
Yuri E M van der BurgtDepartment of Clinical Chemistry and Laboratory Medicine, Leiden University Medical Center, Leiden, The Netherlands.
Bart J M van VlijmenDepartment of Medicine, Division of Thrombosis and Hemostasis, Leiden University Medical Center, Leiden, The Netherlands.
FOR-DMD investigators of the Muscle Study Group
Yetrib HathoutSchool of Pharmacy and Pharmaceutical Sciences, Binghamton University, Binghamton, NY, USA.
Cristina Al-Khalili SzigyartoDepartment of Protein Science, School of Chemistry, Biology and Health, KTH Royal Institute of Technology, Stockholm, Sweden.
Utkarsh J DangCarleton University, Ottawa, ON, Canada.
Roula TsonakaDepartment of Biomedical Data Sciences, Leiden University Medical Center, Leiden, The Netherlands.
Pietro SpitaliDepartment of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands. p.spitali@lumc.nl.

Funding

Biomarker Signatures for Duchenne Muscular DystrophyR61NS119639 · NINDS · STATE UNIVERSITY OF NY,BINGHAMTON · PI DANG, UTKARSH J, HATHOUT, YETRIB · 2022 to 2024
$1.6M
NIH HHS R61NS119639NINDS NIH HHS R61 NS119639Parent Project Muscular Dystrophy 22.010
6 · The paper itself

Abstract

backgroundDuchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder for which monitoring biomarkers are urgently needed. We aimed to evaluate whether proteins in serum can accurately monitor patients' function within the duration of a clinical trial.

methodsIn this study, we evaluated longitudinal serum proteins of DMD patients participating in the FOR-DMD clinical trial, comparing daily and intermittent corticosteroid regimens in boys aged 4-8 years at baseline. Using the aptamer-based protein platform SomaScan, we profiled 1500 proteins. Associations between protein levels and motor function outcomes, such as Rise from the Floor Velocity (RFV), 10-Meter Run/Walk Velocity (10MRWV), and North Star Ambulatory Assessment (NSAA), were assessed using linear mixed models. In particular, we explored whether patients with higher protein levels also tended to have better functional scores (across-patients analysis), and whether changes in protein levels within the same patient over time were linked to changes in their functional performance (within-patient analysis). Finally, penalized (lasso) mixed models were applied to evaluate the predictive function of the proteins. The prediction accuracy of the models (evaluated by optimism-corrected Root Mean Squared Error) was compared to that of a simpler model with only age and treatment as predictors.

resultsAcross-patients and within-patient analyses revealed consistent associations with three functional tests for a subset of proteins, notably RGMA, ART3, ANTXR2, and CFB. Multivariate models incorporating the proteins significantly associated with at least two tests, improved prediction accuracy by 12% for NSAA, and by 33-35% for RFV and 10MRWV. These models also revealed a subset of proteins that were consistently selected. Quantification of CFB, RGMA, ANTXR2, SERPINF1 and ATP5PF using SomaScan showed strong agreement with measurements obtained using orthogonal methods such as ELISA, MRM-MS and an in-house developed bead-based sandwich immunoassay.

conclusionsThese findings support the utility of serum protein signatures as objective, quantitative tools for monitoring disease progression and treatment response in DMD during clinical visits and clinical trials.

trial registrationThe FOR-DMD clinical trial was registered at ClinicalTrials.gov (registration no. NCT01603407). First submission: 03/04/2012.

Indexed as

Adrenal Cortex HormonesBlood ProteinsMuscular Dystrophy, DuchenneBiomarkersChildChild, PreschoolHumansLongitudinal StudiesMaleAdrenal Cortex HormonesBiomarkersBlood ProteinsBiomarkersDuchenne muscular dystrophyMuscle degeneration

Identifiers

PMID42443978
PMCPMC13647910

What Socratic holds

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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 Socratic graph.