ArticleBlood advances2022
Vemurafenib acts as a molecular on-off switch governing systemic inflammation in Langerhans cell histiocytosis.
Article in Blood advances, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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Who cites it
22 citing papers in PubMed, 29 citations in OpenAlex.
- Histiocytosis development and clinical variation through the lens of genomics.The Journal of pathology · 2026Review
- Long-term MAPK inhibition of childhood refractory-Langerhans cell histiocytosis: an observational study of 288 patients.Blood advances · 2026Observational
- Cell-free BRAFHaematologica · 2026Article
- Histiocyte Society blueprint for Langerhans cell histiocytosis research: from cell-of-origin to a more comprehensive cure.Haematologica · 2025Review
- Driver mutations in myeloid and lymphoid cells point to multipotent progenitor origin of diverse histiocytic neoplasms.Blood neoplasia · 2025Article
- BRAFV600E induces key features of LCH in iPSCs with cell type-specific phenotypes and drug responses.Blood · 2025Article
- Effective vemurafenib monotherapy for refractory Langerhans cell histiocytosis with sustained results post-withdrawal for over two years: a case report.Frontiers in oncology · 2025Article
- Review
- Real-world experience with targeted therapy in patients with histiocytic neoplasms in the Netherlands and in Belgium.Blood neoplasia · 2024Article
- Dabrafenib and trametinib in Langerhans cell histiocytosis and other histiocytic disorders.Haematologica · 2024Article
- Vemurafenib combined with chemotherapy achieved sustained remission in pediatric LCH: a multi-center observational study.Journal of cancer research and clinical oncology · 2024Observational
- Treatment of children with refractory/relapse high risk langerhans cell histiocytosis with the combination of cytarabine, vindesine and prednisone.BMC pediatrics · 2024Article
- Identification of Neutrophil Extracellular Trap-Related Gene Expression Signatures in Ischemia Reperfusion Injury During Lung Transplantation: A Transcriptome Analysis and Clinical Validation.Journal of inflammation research · 2024Article
- Comprehensive considerations for dermatologists: the application of FDG-PET in evaluating cutaneous lesions in pediatric Langerhans cell histiocytosis.Frontiers in medicine · 2024Review
- Langerhans cell histiocytosis: promises and caveats of targeted therapies in high-risk and CNS disease.Hematology. American Society of Hematology. Education Program · 2023Review
- Vemurafenib combined with cladribine and cytarabine results in durable remission of pediatric BRAF V600E-positive LCH.Blood advances · 2023Article
- Cladribine and cytarabine in children refractory high risk multisystem Langerhans cell histiocytosis.Heliyon · 2023Article
- Dabrafenib, alone or in combination with trametinib, in BRAF V600-mutated pediatric Langerhans cell histiocytosis.Blood advances · 2023Article
- Lineage switching of the cellular distribution of BRAFV600E in multisystem Langerhans cell histiocytosis.Blood advances · 2023Article
- Clinicogenomic associations in childhood Langerhans cell histiocytosis: an international cohort study.Blood advances · 2023Article
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Authors and funding
8 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Langerhans cell histiocytosis (LCH) is a neoplasm marked by the accumulation of CD1A+CD207+ cells. It is most commonly driven by a somatic, activating mutation in the BRAF serine-threonine kinase (BRAFV600E). Multisystem disease with risk-organ involvement requires myelotoxic chemotherapy, making BRAF-inhibitors an attractive treatment option. Here, we present a comprehensive analysis of the course of an LCH patient treated with the combination of vemurafenib and salvage chemotherapy who achieved sustained clinical and molecular remission. We show that there is no relationship between peripheral blood BRAFV600E levels and clinical presentation during treatment with vemurafenib, but that vemurafenib leads to a fast, efficient, but reversible inhibition of clinical manifestations of systemic inflammation. In line, serum levels of inflammatory cytokines exactly mirror vemurafenib administration. Genotyping analysis identified the BRAFV600E mutation in multiple hematopoietic cell types, including NK cells and granulocytes. Single-cell transcriptome analyses of peripheral blood and bone marrow cells at time of diagnosis and during treatment indicate that RAF-inhibition abrogates the expression of inflammatory cytokines previously implicated in LCH such as IL1B and CXCL8. Together, our data suggest that while the CD1A+CD207+ histiocytes are the hallmark of LCH, other BRAF-mutated cell populations may contribute significantly to morbidity in patients with multisystem LCH.
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