ArticleCancer research and treatment2021
Effectiveness and Safety of Dabrafenib in the Treatment of 20 Chinese Children with BRAFV600E-Mutated Langerhans Cell Histiocytosis.
Article in Cancer research and treatment, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers.
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Who cites it
30 citing papers in PubMed, 44 citations in OpenAlex.
- Langerhans cell histiocytosis: molecular mechanisms underlying pathogenesis and emerging targeted therapeutics.Cancer metastasis reviews · 2026Review
- [A rare case of Rosai-Dorfman disease involving the pituitary stalk and presenting initially with diabetes insipidus].Zhong nan da xue xue bao. Yi xue ban = Journal of Central South University. Medical sciences · 2026Article
- Origin, diagnosis and treatment of periocular Histiocytic disorders- state-of-the-art review.Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie · 2026Review
- Long-term MAPK inhibition of childhood refractory-Langerhans cell histiocytosis: an observational study of 288 patients.Blood advances · 2026Observational
- Efficacy, safety, and relapse outcomes of MAPK inhibitors in pediatric Langerhans cell histiocytosis: A real-world study.International journal of cancer · 2026Article
- Dabrafenib or trametinib in pediatric Langerhans cell histiocytosis with bone lesions: a retrospective efficacy and safety study.BMC cancer · 2026Article
- Targeted therapy for Langerhans cell histiocytosis with maxillofacial involvement in 20 children.Orphanet journal of rare diseases · 2026Article
- Advances in the diagnosis and management of pediatric Langerhans cell histiocytosis and Rosai-Dorfman disease: therapies, biomarkers, and response assessment.Hematology. American Society of Hematology. Education Program · 2025Review
- Histiocyte Society blueprint for Langerhans cell histiocytosis research: from cell-of-origin to a more comprehensive cure.Haematologica · 2025Review
- Efficacy and safety of dabrafenib-targeted therapy for pediatric langerhans cell histiocytosis: a systematic review and meta-analysis.Translational pediatrics · 2025Article
- [Immune microenvironment of Langerhans cell histiocytosis: from immune suppression to targeted therapy].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Review
- Real-world experience with targeted therapy in patients with histiocytic neoplasms in the Netherlands and in Belgium.Blood neoplasia · 2024Article
- Advancements in the understanding and management of histiocytic neoplasms.Blood research · 2024Review
- Clinical features and treatment outcomes of liver involvement in paediatric Langerhans cell histiocytosis.BMC pediatrics · 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
- Liver transplantation in a child with sclerosing cholangitis due to Langerhans cell histiocytosis: a case report.Frontiers in pediatrics · 2024Article
- Article
- Mutant PIK3CA is a targetable driver alteration in histiocytic neoplasms.Blood advances · 2023Article
Corrections and comments
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Authors and funding
14 authors at 2 institutions in 1 country.
Funding
Abstract
purposeWe sought to investigate the effectiveness and safety of dabrafenib in children with BRAFV600E-mutated Langerhans cell histiocytosis (LCH). MATERIALS AND
methodsA retrospective analysis was performed on 20 children with BRAFV600E-mutated LCH who were treated with dabrafenib.
resultsThe median age at which the patients started taking dabrafenib was 2.3 years old (range, 0.6 to 6.5 years). The ratio of boys to girls was 2.3:1. The median follow-up time was 30.8 months (range, 18.9 to 43.6 months). There were 14 patients (70%) in the risk organ (RO)+ group and six patients (30%) in the RO- group. All patients were initially treated with traditional chemotherapy and then shifted to targeted therapy due to poor control of LCH or intolerance to chemotherapy. The overall objective response rate and the overall disease control rate were 65% and 75%, respectively. During treatment, circulating levels of cell-free BRAFV600E (cfBRAFV600E) became negative in 60% of the patients within a median period of 3.0 months (range, 1.0 to 9.0 months). Grade 2 or 3 adverse effects occurred in five patients.
conclusionSome children with BRAFV600E-mutated LCH may benefit from monotherapy with dabrafenib, especially high-risk patients with concomitant hemophagocytic lymphohistiocytosis and intolerance to chemotherapy. The safety of dabrafenib is notable. A prospective study with a larger sample size is required to determine the optimal dosage and treatment duration.
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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.