ArticleBritish journal of cancer2017
MEK inhibition appears to improve symptom control in primary NRAS-driven CNS melanoma in children.
Article in British journal of cancer, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
23 citing papers in PubMed, 64 citations in OpenAlex.
- Living with a rare disease: Why lived experience must shape research.PLoS medicine · 2026Article
- Efficacy of azacitidine and trametinib against leptomeningeal melanosis associated with congenital melanocytic nevus syndrome.Molecular and cellular pediatrics · 2026Review
- Neurocutaneous Melanosis in Children and Adults With Clinical, Radiological, and Pathogenic Evaluation: Case Report and Review of the Literature.Clinical case reports · 2025Article
- Congenital melanocytic neoplasms: clinical, histopathological and recent molecular developments.Virchows Archiv : an international journal of pathology · 2025Review
- Preclinical evidence for employing MEK inhibition in NRAS mutated pediatric gastroenteropancreatic neuroendocrine-like tumors.Translational oncology · 2024Article
- Review
- Clinical management of a rare melanoma case arising from congenital melanocytic nevus.Genes & diseases · 2024Article
- Pearls & Oy-sters: Primary Diffuse Leptomeningeal Melanocytosis: A Diagnostic Conundrum.Neurology · 2023Article
- Review
- Article
- Advances in the diagnosis, evaluation, and management of leptomeningeal disease.Neuro-oncology advances · 2021Article
- Medical Therapy for Craniopharyngiomas.TouchREVIEWS in endocrinology · 2021Review
- Article
- Trametinib therapy for children with neurofibromatosis type 1 and life-threatening plexiform neurofibroma or treatment-refractory low-grade glioma.Cancer medicine · 2021Article
- Novel Insights into Diagnosis, Biology and Treatment of Primary Diffuse Leptomeningeal Melanomatosis.Journal of personalized medicine · 2021Article
- Novel insights into the pathogenesis and treatment of NRAS mutant melanoma.Expert review of precision medicine and drug development · 2021Article
- Does the gene matter? Genotype-phenotype and genotype-outcome associations in congenital melanocytic naevi.The British journal of dermatology · 2020Article
- Can Combination MEK and Akt Inhibition Slay the Giant Congenital Nevus?The Journal of investigative dermatology · 2019Article
- Melanoma arising in a Giant congenital melanocytic nevus: two case reports.Diagnostic pathology · 2019Article
- Tumour compartment transcriptomics demonstrates the activation of inflammatory and odontogenic programmes in human adamantinomatous craniopharyngioma and identifies the MAPK/ERK pathway as a novel therapeutic target.Acta neuropathologica · 2018Article
Corrections and comments
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Authors and funding
5 authors at 2 institutions in 1 country.
Funding
Abstract
backgroundPrimary melanoma of the CNS in children is extremely rare, and usually linked to congenital melanocytic naevus syndrome, caused by mosaicism for oncogenic NRAS mutations. Outcome is fatal in all cases. Data from murine and in vitro studies suggest that MEK inhibition is a possible therapeutic option.
methodsFour children with NRAS-mutated CNS melanoma were treated with Trametinib on a compassionate basis.
resultsAll four had an improvement in symptoms and objectively in signs. These varied from mild improvement for 1 month, to a sustained symptom-free period of 9 months in one case. In all cases there was eventual disease progression through treatment, followed by rapid death after discontinuation. There were no clinically-significant side effects.
conclusionsTrametinib is the first therapy to show any objective or measurable effect in NRAS-mutated primary CNS melanoma, with few side effects in this small series. The role of this therapy should be explored further in this rare paediatric tumour.
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Registered trials
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