Synapse
⌘+K
Synapse
PulseExploreClubsResearchersJournals
Instagram
HomeClubsExplore
June 13, 2026Journal of Paediatrics and Child HealthOpen Access

Selumetinib as a Target Therapy in Progressive Paediatric Low‐Grade Gliomas—Case Series ( pLGG )

View Full Paper
Ask AI
Bookmark
Share

Authors

LTLaura TrapaniGGGiulia GortaniAMAndrea Magnolato

Discussion

Loading...

Member takes

Overview

Case series evaluates selumetinib's efficacy in pediatric NF1-related optic pathway gliomas, suggesting promising outcomes.

Key Points

  • This case series aims to evaluate the efficacy and safety of selumetinib in treating children with NF1-related progressive optic pathway gliomas.
  • Reviewed three pediatric patients with NF1 and progressive OPGs treated with selumetinib 25 mg/m2/day.
  • Selumetinib was given as second-line therapy after chemotherapy in one case and first-line in two cases under compassionate use.
  • Tumour response assessed by MRI; visual function evaluated through serial ophthalmological exams.
  • All patients exhibited radiological tumour shrinkage or stabilization with significant improvements in visual acuity.
  • One child achieved near-complete recovery of vision.
  • Treatment was well tolerated with mild, predominantly dermatological adverse events and no severe systemic toxicities.

Cite This Study

Trapani et al. (2026) studied this question.

synapsesocial.com/papers/6a2cf604faef96ed7f057d23https://doi.org/10.1111/jpc.70455
View Full Paper
Ask AI
Bookmark
Share

Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1LGG-49. EVALUATING THE EFFECTS OF MIRDAMETINIB IN NF1-MUTANT LOW-GRADE GLIOMAS USING BOTH CULTURED CELLS AND A NOVEL ZEBRAFISH XENOGRAFT MODEL2024 · 1 citations
  2. 2Visual outcomes in children with neurofibromatosis type 1-associated optic pathway glioma following chemotherapy: a multicenter retrospective analysis2012 · 321 citations
  3. 3A new era for optic pathway glioma: A developmental brain tumor with life-long health consequences2023 · 19 citations
  4. 4A European randomised controlled trial of the addition of etoposide to standard vincristine and carboplatin induction as part of an 18-month treatment programme for childhood (≤16 years) low grade glioma – A final report2017 · 156 citations
  5. 5Neurofibroma Development in Neurofibromatosis Type 1: Insights from Cellular Origin and Schwann Cell Lineage Development2022 · 23 citations