Evidence map›Paper›PMID 42064048›Full record

ArticleFrontiers in immunology2026

Beyond the interferon score: neurofilament light chain and glial fibrillary acidic protein capture immune-mediated neuroinjury and response to JAK inhibition in Aicardi-Goutières syndrome.

Lisa Wege, Christian Klemann, Sandy Siegert, Annette Bley, Sarah Koss, Anne Koy, Thorsten Langer, Franziska Dunst, Barbara Plecko, Martin Fleger and 14 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Frontiers in immunology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the 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.

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

1 citing paper in PubMed.

  1. Review
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

24 authors.

Lisa WegeDepartment of Pediatrics, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
Christian KlemannDepartment of Pediatric Immunology, Rheumatology and Infectiology, Hospital for Childrens and Adolescents, University of Leipzig, Leipzig, Germany.
Sandy SiegertDepartment of Pediatrics and Adolescent Medicine, Division of Pediatric Pulmonology, Allergology and Endocrinology, Medical University of Vienna, Vienna, Austria.
Annette BleyUniversity Children's Hospital, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Sarah KossDepartment of Pediatrics, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
Anne KoyDepartment of Pediatrics and Centre for Rare Diseases, Faculty of Medicine and University Hospital of Cologne, University of Cologne, Cologne, Germany.
Thorsten LangerDepartment of Neuropediatrics and Muscle Disorders, Children's Hospital, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
Franziska DunstDepartment of Pediatric Immunology, Rheumatology and Infectiology, Hospital for Childrens and Adolescents, University of Leipzig, Leipzig, Germany.
Barbara PleckoDepartment of Pediatrics, Division of General Pediatrics, Medical University of Graz, Graz, Austria.
Martin FlegerDepartment of Pediatrics, State Hospital of Bregenz, Bregenz, Austria.
Fabian SpethDepartment of Pediatric Rheumatology, Pediatric Bone Marrow Transplantation and Immunology Unit, Center for Obstetrics and Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Tim NiehuesDepartment of Pediatrics, Helios Klinik Krefeld, Krefeld, Germany.
Peter KaufmannDepartment of Pediatrics, Gesundheitsbezirk Bozen, Südtiroler Sanitätsbetrieb, Bozen, Italy.
Jurek SchultzDepartment of Pediatric Surgery, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
Mohammed AttiaVinzentius Krankenhaus Landau, Landau, Germany.
Johannes StoffelsDivision of Neuropediatrics and Integrated Health Care, Department of Pediatrics and Adolescent Medicine, KJF Klinikum Josefinum, Augsburg, Germany.
Beata Wolska-KuśnierzDepartment of Immunology, Children's Memorial Health Institute, Warsaw, Poland.
Roman RolkeDepartment of Palliative Medicine, Medical Faculty Rheinisch-Westfälische Technische Hochschule (RWTH) Aachen University, Aachen, Germany.
Gabriele ReicheltDepartment of Pediatrics, University Medical Center Mainz, Mainz, Germany.
Friederike BlankenburgPediatric Rheumatology, Olgahospital, Stuttgart, Germany.
Jürgen BrunnerPediatric Rheumatology, Department of Pediatrics, Medizinische Universität Innsbruck, Innsbruck, Austria.
Katja AkgünMultiple Sklerose Zentrum Dresden, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
Christine WolfDepartment of Pediatrics, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
Min Ae Lee-KirschDepartment of Pediatrics, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background and objective: Aicardi-Goutières syndrome (AGS) is a prototypical type I interferon-driven neuroimmunological disorder in which immune-mediated inflammation causes progressive brain injury. Targeted immunosuppression with Janus kinase (JAK) inhibitors has shown clinical benefit, yet neurological outcomes remain difficult to quantify, and the interferon (IFN) score poorly reflects neuroaxonal damage. We investigated whether plasma neurofilament light chain (pNfL) and glial fibrillary acidic protein (pGFAP) serve as sensitive biomarkers of neurological involvement and response to immunosuppressive therapy. Methods: Plasma samples from 55 patients with genetically confirmed AGS and 55 age- and sex-matched healthy controls were analyzed using single molecule array assays. pNfL and pGFAP levels were assessed in relation to age, clinical disease severity, IFN score, and treatment with JAK inhibitors. Longitudinal samples before and during therapy were available from 11 patients. Results: Treatment-naïve AGS patients exhibited significantly elevated pNfL and pGFAP levels compared with controls, and biomarker concentrations correlated with clinical disease severity. pNfL and pGFAP were strongly correlated with each other but showed only weak to moderate associations with the IFN score. Longitudinal within-patient analyses demonstrated significant declines in pNfL and pGFAP following initiation of JAK inhibitor therapy, whereas the IFN score remained unchanged. Discussion: pNfL and pGFAP are sensitive indicators of neuroaxonal and astroglial injury in AGS, capturing neurological disease burden and treatment-associated changes more accurately than the IFN score. These findings support their use as objective biomarkers for monitoring immune-mediated brain injury and therapeutic response, and warrant validation in larger longitudinal studies.

Indexed as

Autoimmune Diseases of the Nervous SystemGlial Fibrillary Acidic ProteinJanus Kinase InhibitorsNervous System MalformationsNeurofilament ProteinsAdolescentAdultBiomarkersChildChild, PreschoolFemaleHumansMaleBiomarkersGFAP protein, humanGlial Fibrillary Acidic ProteinJanus Kinase Inhibitorsneurofilament protein LNeurofilament ProteinsAicardi-Goutières syndromebiomarkerglial fibrillary acidic proteinimmune-mediated neurodegenerationinflammationinterferonJAK inhibitionneurofilament light chain

Identifiers

PMID42064048
PMCPMC13124696

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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.