ArticleCancer medicine2021
Trametinib therapy for children with neurofibromatosis type 1 and life-threatening plexiform neurofibroma or treatment-refractory low-grade glioma.
Article in Cancer medicine, 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, 31 citations in OpenAlex.
- Selumetinib in the treatment of plexiform neurofibromas in neurofibromatosis type 1: a 6-year prospective case series with review of the literature.BMC pediatrics · 2026Review
- Clinical benefit of additional whole-exome sequencing over panel sequencing in an all-comer real-world molecular tumor board.ESMO open · 2025Article
- [Molecular targeted therapy for progressive low-grade gliomas in children].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2025Article
- Article
- How modern treatments have modified the role of surgery in pediatric low-grade glioma.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2024Review
- Visual Deficits and Diagnostic and Therapeutic Strategies for Neurofibromatosis Type 1: Bridging Science and Patient-Centered Care.Vision (Basel, Switzerland) · 2024Review
- Article
- Optic Pathway Gliomas in Pediatric Population-Current Approach in Diagnosis and Management: Literature Review.Journal of clinical medicine · 2023Review
- Unlocking the power of precision medicine for pediatric low-grade gliomas: molecular characterization for targeted therapies with enhanced safety and efficacy.Frontiers in oncology · 2023Review
- Benign Peripheral Non-cranial Nerve Sheath Tumors of the Neck.Advances in therapy · 2022Review
- Efficacy and Safety of Trametinib in Neurofibromatosis Type 1-Associated Plexiform Neurofibroma and Low-Grade Glioma: A Systematic Review and Meta-Analysis.Pharmaceuticals (Basel, Switzerland) · 2022Review
- Current State of Pediatric Cardio-Oncology: A Review.Children (Basel, Switzerland) · 2022Review
- Rethinking the Management of Optic Pathway Gliomas: A Single Center Experience.Frontiers in surgery · 2022Article
- Neurosurgery for Optic Pathway Glioma: Optimizing Multidisciplinary Management.Frontiers in surgery · 2022Review
- Safety and Efficacy of Mek Inhibitors in the Treatment of Plexiform Neurofibromas: A Retrospective Study.Cancer control : journal of the Moffitt Cancer CenterArticle
- Sporadic Metastatic Malignant Peripheral Nerve Sheath Tumour with an NF1 Mutation Responding to Trametinib: A Case Report.Case reports in oncologyArticle
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Authors and funding
13 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeTo describe a series of children with extensive PNF or treatment refractory PLGG treated on a compassionate basis with trametinib.
methodsWe report on six patients with NF-1 treated with trametinib on a compassionate basis at British Columbia Children's Hospital since 2017. Data were collected retrospectively from the patient record. RAPNO and volumetric criteria were used to evaluate the response of intracranial and extracranial lesions, respectively.
resultsSubjects were 21 months to 14 years old at the time of initiation of trametinib therapy and 3/6 subjects are male. Duration of therapy was 4-28 months at the time of this report. All patients had partial response or were stable on analysis. Two patients with life-threatening PNF had a partial radiographic response in tandem with significant clinical improvement and developmental catch up. One subject discontinued therapy after 6 months due to paronychia and inadequate response. The most common adverse effect (AE) was grade 1-2 paronychia or dermatitis in 5/6 patients. There were no grade 3 or 4 AEs. At the time of this report, five patients remain on therapy.
conclusionTrametinib is an effective therapy for advanced PNF and refractory PLGG in patients with NF-1 and is well tolerated in children. Further data and clinical trials are required to assess tolerance, efficacy and durability of response, and length of treatment required in such patients.
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