ArticleBlood advances2023
Successful treatment of non-Langerhans cell histiocytosis with the MEK inhibitor trametinib: a multicenter analysis.
Article in Blood advances, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.
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Who cites it
33 citing papers in PubMed, 39 citations in OpenAlex.
- BRAF V600E Positive Non Langerhans Cell Histiocytosis Presenting as Hemophagocytic Lymphocytosis in a Young Child.Indian journal of pediatrics · 2026Article
- Article
- Histiocytosis development and clinical variation through the lens of genomics.The Journal of pathology · 2026Review
- Outcome of treatment with trametinib in adults with histiocytic neoplasms in the United Kingdom.British journal of haematology · 2026Article
- Combinatorial Targeting of Avapritinib-Driven MAP Kinase Activation in High-Grade Glioma.Research square · 2026Article
- Targetable alterations and personalized treatment in ameloblastoma: results from a prospective observational precision oncology study.NPJ precision oncology · 2026Article
- Rosai-Dorfman disease withOxford medical case reports · 2026Article
- Red-yellow Papules and Nodules All over the Body: A Quiz.Acta dermato-venereologica · 2026Article
- Targeted Therapy in Erdheim-Chester Disease: A Case Report and Review of the Literature.Cureus · 2026Article
- Extranodal Rosai-Dorfman disease in a male involving the breast and subcutaneous soft tissue: An uncommon distribution.Radiology case reports · 2026Article
- When Erdheim-Chester Disease Reaches the Pectoral Muscle.European journal of case reports in internal medicine · 2026Article
- Clinicopathological insights into ophthalmic Rosai-Dorfman disease: A 15-year retrospective analysis.Indian journal of ophthalmology · 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
- The Clinicopathologic Spectrum of Rosai-Dorfman-Destombes Disease in Adults: An Analysis of 16 Cases.European journal of haematology · 2025Article
- Histiocyte Society blueprint for non-Langerhans cell histiocytosis research: unraveling complex diseases through collaboration.Haematologica · 2025Review
- Symptoms, unmet needs, and quality of life in Erdheim-Chester disease: a longitudinal registry-based analysis.Blood advances · 2025Article
- Integrated In Silico and Experimental Validation of Antrocin as a Plant-Derived Multi-Target Therapeutic for BRAF/MEK/PI3K-Driven Colorectal Cancer.International journal of molecular sciences · 2025Article
- Kidney Involvement in Erdheim-Chester Disease.Kidney international reports · 2025Article
- Tumour-agnostic kinase inhibitors.Nature reviews. Drug discovery · 2025Review
- Langerhans Cell Histiocytosis WithEJHaem · 2025Article
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Authors and funding
13 authors at 6 institutions in 1 country.
Funding
Abstract
Erdheim-Chester disease (ECD) and Rosai-Dorfman disease (RDD) are rare non-Langerhans cell histiocytoses (non-LCHs), for which therapeutic options are limited. MAPK pathway activation through BRAFV600E mutation or other genomic alterations is a histiocytosis hallmark and correlates with a favorable response to BRAF inhibitors and the MEK inhibitor cobimetinib. However, there has been no systematic evaluation of alternative MEK inhibitors. To assess the efficacy and safety of the MEK inhibitor trametinib, we retrospectively analyzed the outcomes of 26 adult patients (17 with ECD, 5 with ECD/RDD, 3 with RDD, and 1 with ECD/LCH) treated with orally administered trametinib at 4 major US care centers. The most common treatment-related toxicity was rash (27% of patients). In most patients, the disease was effectively managed at low doses (0.5-1.0 mg trametinib daily). The response rate of the 17 evaluable patients was 71% (73% [8/11] without a detectable BRAFV600E achieving response). At a median follow-up of 23 months, treatment effects were durable, with a median time-to-treatment failure of 37 months, whereas the median progression-free and overall survival were not reached (at 3 years, 90.1% of patients were alive). Most patients harbored mutations in BRAF (either classic BRAFV600E or other BRAF alterations) or alterations in other genes involved in the MAPK pathway, eg, MAP2K, NF1, GNAS, or RAS. Most patients required lower than standard doses of trametinib but were responsive to lower doses. Our data suggest that the MEK inhibitor trametinib is an effective treatment for ECD and RDD, including those without the BRAFV600E mutation.
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