ArticleNeuro-oncology advances
Dual BRAF/MEK blockade restores CNS responses in BRAF-mutant Erdheim-Chester disease patients following BRAF inhibitor monotherapy.
Article in Neuro-oncology advances. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
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Who cites it
10 citing papers in PubMed, 21 citations in OpenAlex.
- Delayed Neurologic Response to Dabrafenib and Trametinib in the Case of Mixed Histiocytosis (LCH/ECD): Case Report and Literature Review.Reports (MDPI) · 2026Article
- Refractory Tumorous and Neurodegenerative Histiocytosis Treated With Intra-Arterial Chemotherapy.Neurology(R) neuroimmunology & neuroinflammation · 2025Article
- Article
- Molecular Mutations in Histiocytosis: A Comprehensive Survey of Genetic Alterations.Molecular biotechnology · 2025Review
- Drug-related macular edema: a real-world FDA Adverse Event Reporting System database study.BMC pharmacology & toxicology · 2025Article
- Real-world experience with targeted therapy in patients with histiocytic neoplasms in the Netherlands and in Belgium.Blood neoplasia · 2024Article
- Langerhans cell histiocytosis: promises and caveats of targeted therapies in high-risk and CNS disease.Hematology. American Society of Hematology. Education Program · 2023Review
- MicroRNA-15a-5p acts as a tumor suppressor in histiocytosis by mediating CXCL10-ERK-LIN28a-let-7 axis.Leukemia · 2022Article
- Article
- The Contribution of MicroRNAs to the Inflammatory and Neoplastic Characteristics of Erdheim-Chester Disease.Cancers · 2020Article
Corrections and comments
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Authors and funding
10 authors at 3 institutions in 2 countries.
Funding
Abstract
backgroundErdheim-Chester disease (ECD), a rare inflammatory myeloid neoplasm, is known to be fundamentally reliant on the constitutive activation of the MAPK signaling pathway in the majority of patients. Consequently, inhibition of the V600E-mutant BRAF kinase has proven to be a safe and efficacious long-term therapeutic strategy for
methodsWe retrospectively describe 3
resultsThree patients with a mean age of 52.6 years were treated with Vemurafenib for a mean duration of 26.6 months (range: 6-52). Monotherapies were upgraded to Vemurafenib/Cobimetinib dual therapy. The combination therapy was administered for a mean duration of 21 months (range: 19-23). All patients exhibited clinical and neurological improvement. Regression of lesions on MRI was noted in 2 patients. Both patients characterized by a PET-avid disease responded to the biological treatment regimen with complete metabolic remissions.
conclusionDual inhibition of BRAF and downstream MEK may be a safe and effective therapeutic strategy for
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