Evidence map›Paper›PMID 42304472›Full record

Trial reportJournal of translational medicine2026

Proteomic and machine learning analysis predicts treatment response signatures in Myasthenia Gravis.

Karli Gilbert, Amrita K Cheema, Henry J Kaminski, Linda L Kusner

Abstract readClinical Trial, Phase IIIRandomized Controlled Trial
In one paragraph

Trial report in Journal of translational medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

What it found

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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

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

4 authors.

Karli GilbertLaboratory for Myasthenia Gravis Research, George Washington University, Washington, DC, USA.
Amrita K CheemaDepartments of Oncology, Biochemistry, Molecular and Cellular Biology, Lombardi Comprehensive Cancer Center, Georgetown University Medical Center, Washington, DC, USA.
Henry J KaminskiLaboratory for Myasthenia Gravis Research, George Washington University, Washington, DC, USA. hkaminski@mfa.gwu.edu.ORCID 0000-0002-8195-0141
Linda L KusnerLaboratory for Myasthenia Gravis Research, George Washington University, Washington, DC, USA. lkusner@gwu.edu.ORCID 0000-0002-5530-2140

Funding

Tissue Culture Shared ResourceP30CA051008 · NCI · GEORGETOWN UNIVERSITY · PI MARCUS S NOEL · 1990 to 2026
$71.5M
Rare Disease Network for Myasthenia GravisU54NS115054 · NINDS · GEORGE WASHINGTON UNIVERSITY · PI HENRY J KAMINSKI · 2019 to 2026
$11.9M
NCI NIH HHS P30 CA051008NINDS NIH HHS U54 NS115054
6 · The paper itself

Abstract

backgroundMyasthenia gravis (MG) is a prototypical antibody-mediated autoimmune disease with variable treatment responses with a need for biomarkers to guide therapeutic decision making. Proteomic profiling, coupled with machine learning, offers a hypothesis-free approach to identify multi-protein signatures associated with treatment response.

methodsWe analyzed sera collected at entry (baseline) from participants in a phase 3 trial randomized trial comparing thymectomy plus prednisone versus prednisone alone, along with matched controls using liquid chromatography-mass spectrometry. We derived disease-specific proteomic signatures and evaluated associations between baseline proteins and 6-month clinical outcomes using multiple machine-learning approaches with internal validation.

resultsBaseline serum proteomes distinguished MG from controls, with pathway enrichment implicating complement activation, immunoglobulin production, and T-cell receptor signaling. Distinct protein panels predicted 6-month clinical improvement within each treatment arm. In the thymectomy-plus-prednisone group, models captured non-linear relationships of predictive proteins in contrast with the predominant additive patterns observed in the prednisone-alone group. Predictive proteins were enriched for T-cell signaling and leukocyte trafficking functions, providing insight into treatment-specific biology.

conclusionsBaseline serum proteomics captures core disease characteristics of MG and predicts short-term clinical response in a treatment-specific manner. While our results require validation in independent cohorts, these findings could enable biomarker-guided selection of thymectomy, refine risk stratification, and furnish mechanistic readouts for future MG trials and clinical care. We aim to conduct future studies using -omic approaches to validate these baseline predictive biomarkers and pathways of treatment response in patients with MG.

Indexed as

Machine LearningMyasthenia GravisProteomicsAdultFemaleHumansMaleMiddle AgedPredictive Learning ModelsPrednisoneProteomeThymectomyTreatment OutcomePrednisoneProteomeMachine learningMass spectrometryMyasthenia gravisPrednisoneProteomicsThymectomyTreatment response

Identifiers

PMID42304472
PMCPMC13508445

What OpenQuestion holds

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Registered trials

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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.