Evidence map›Paper›PMID 39107839›Full record

ArticleActa neuropathologica communications2024

Efficacy of BRAF/MEK-inhibitor therapy for epithelioid glioblastoma with a novel BRAFV600 mutation.

J Steininger, C Buszello, R Oertel, M Meinhardt, S Schmid, K Engellandt, S Herold, S Stasik, A Ebrahimi, B Renner and 8 more

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PubMed Full text at PMC Publisher
In one paragraph

Article in Acta neuropathologica communications, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
  4. Article
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

18 authors.

J Steininger *Department of Dermatology, University Hospital Carl Gustav Carus, Technische Universität Dresden, Fetscherstraβe 74, 01307, Dresden, Germany. Julian.steininger@ukdd.de.ORCID 0000-0001-9094-4512
C Buszello *Department of Neurosurgery, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
R OertelInstitute of Clinical Pharmacology, Medical Faculty, Technische Universität Dresden, Dresden, Germany.
M MeinhardtInstitute of Pathology, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
S SchmidDepartment of Neuropathology, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Charitéplatz 1, Berlin, Germany.
K EngellandtInstitute of Diagnostic and Interventional Neuroradiology, Faculty of Medicine and University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
S HeroldInstitute of Pathology, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
S StasikMedical Department I, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
A EbrahimiDepartment of Neuropathology, DGNN Brain Tumor Reference Center, University of Bonn, Bonn, Germany.
B RennerInstitute of Clinical Pharmacology, Medical Faculty, Technische Universität Dresden, Dresden, Germany.
C ThiedeMedical Department I, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
I Y EyüpogluDepartment of Neurosurgery, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
G SchackertDepartment of Neurosurgery, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
S BeissertDepartment of Dermatology, University Hospital Carl Gustav Carus, Technische Universität Dresden, Fetscherstraβe 74, 01307, Dresden, Germany.
F MeierDepartment of Dermatology, University Hospital Carl Gustav Carus, Technische Universität Dresden, Fetscherstraβe 74, 01307, Dresden, Germany.
J RadkeDepartment of Pathology, University Medicine Greifswald, Greifswald, Germany.
D Westphal *Department of Dermatology, University Hospital Carl Gustav Carus, Technische Universität Dresden, Fetscherstraβe 74, 01307, Dresden, Germany.
T A Juratli *Department of Neurosurgery, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Epithelioid glioblastoma (eGB), a very aggressive and rare brain tumour, is associated with a dismal median overall survival. Effective therapies for patients with eGB, particularly with leptomeningeal dissemination, are still lacking. Here, we describe a case of a 25-year-old male diagnosed with an intramedullary cervical tumour with subsequent leptomeningeal disease. Histopathology identified a highly necrotising, epithelioid-type tumour with high cell density, most compatible with the diagnosis of an eGB. DNA analysis revealed an unprecedented B-Raf protooncogene, serine/threonine kinase (BRAF) gene variant in exon 15 (ENST00000288602.6, c.1799_1810delinsATG, p.(V600_W604delinsDG)), triggering activation of the mitogen-activated protein kinase (MAPK) pathway. Consequently, we initiated MAPK inhibitor (MAPKi) therapy, utilizing a combination of BRAF and mitogen-activated protein kinase kinase (MEK) inhibitors. Liquid chromatography-tandem mass spectrometry analysis confirmed the drugs' presence in the patient's cerebrospinal fluid, indicating their capacity to cross the blood-brain barrier. Remarkably, the patient responded very well to therapy and transitioned from a near-comatose state to significantly improved health, sustained for over three months. This study highlights that MAPKi, particularly targeted towards novel BRAFV600 mutations, might offer promising advancements in eGB treatment strategies.

Indexed as

Brain NeoplasmsGlioblastomaMutationProtein Kinase InhibitorsProto-Oncogene Proteins B-rafAdultHumansMaleMitogen-Activated Protein Kinase KinasesBRAF protein, humanMitogen-Activated Protein Kinase KinasesProtein Kinase InhibitorsProto-Oncogene Proteins B-rafBRAF mutationEpithelioid glioblastomaLeptomeningeal diseaseMAPK inhibitors

Identifiers

PMID39107839
PMCPMC11302837

What OpenQuestion holds

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LicenceCC BY
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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.