ArticleInternational journal of hematology2021
Vemurafenib provides a rapid and robust clinical response in pediatric Langerhans cell histiocytosis with the BRAF V600E mutation but does not eliminate low-level minimal residual disease per ddPCR using cell-free circulating DNA.
Article in International journal of hematology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 27 citations in OpenAlex.
- Langerhans cell histiocytosis: molecular mechanisms underlying pathogenesis and emerging targeted therapeutics.Cancer metastasis reviews · 2026Review
- Long-term MAPK inhibition of childhood refractory-Langerhans cell histiocytosis: an observational study of 288 patients.Blood advances · 2026Observational
- Article
- Disease response criteria in Langerhans cell histiocytosis: a global view.International journal of hematology · 2025Review
- Current State of Targeted Therapy in Adult Langerhans Cell Histiocytosis and Erdheim-Chester Disease.Targeted oncology · 2024Review
- Targeted Therapy With Vemurafenib in Brazilian Children With Refractory Langerhans Cell Histiocytosis: Two Case Reports and Review of Literature.Cancer reports (Hoboken, N.J.) · 2024Review
- 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
- 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
- Dabrafenib, alone or in combination with trametinib, in BRAF V600-mutated pediatric Langerhans cell histiocytosis.Blood advances · 2023Article
- Vemurafenib improves muscle histopathology in a mouse model of LAMA2-related congenital muscular dystrophy.Disease models & mechanisms · 2023Article
- Lineage switching of the cellular distribution of BRAFV600E in multisystem Langerhans cell histiocytosis.Blood advances · 2023Article
- Signaling pathways, microenvironment, and targeted treatments in Langerhans cell histiocytosis.Cell communication and signaling : CCS · 2022Review
- Dramatic Response After Switching MEK Inhibitors in a Patient With Refractory Mixed Histiocytosis.Journal of hematology · 2022Article
- Gastrointestinal langerhans cell histiocytosis in an adult presenting with anal protrusion and multiple colorectal ulcers: a case report.Therapeutic advances in rare diseaseArticle
Corrections and comments
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Authors and funding
10 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundLangerhans cell histiocytosis (LCH) is a disease that arises from myeloid cells that phenotypically resemble Langerhans cells (LC), which is typically driven by the BRAF V600E mutation. High-risk LCH has a poor prognosis. PROCEDURE: Fifteen children with BRAF V600E + LCH received vemurafenib between March 2016 and February 2020. The median age at LCH onset was 2 months and the median age at the start of vemurafenib treatment was 22 months. The median disease activity score (DAS) at the start of vemurafenib treatment was 12 points.
resultsThe median duration of vemurafenib treatment was 29 months. All patients responded to treatment, with median DAS of 4 points at week 4 and 1 point at 6 months. Two patients died: 1 of hepatic failure after NSAID overdose and 1 of neutropenic sepsis. Cessation of vemurafenib resulted in relapse in 5 patients and was only possible for 1 patient. Serial measurements of BRAF V600E using cell-free circulating DNA revealed that 7 patients had persistently high mutant allele levels.
conclusionVemurafenib is effective in children with BRAF V600E + LCH. However, treatment with vemurafenib does not eradicate the disease and its long-term toxicity has not been established.
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