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June 10, 2026BMC PediatricsOpen Access

Selumetinib in the treatment of plexiform neurofibromas in neurofibromatosis type 1: a 6-year prospective case series with review of the literature

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Authors

FBFrancesco BaldoUniversity of TriesteLMLuca MessanaUniversity of TriesteAAAlberto AndresaniUniversity of Trieste

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Implication

Prospective case series shows selumetinib reduces plexiform neurofibromas in NF1 patients, suggesting a new treatment option.

Key Points

  • This study aims to evaluate the effect of selumetinib on plexiform neurofibromas in patients with neurofibromatosis type 1.
  • Prospective case series of 23 patients with NF1 and plexiform neurofibromas treated with selumetinib from January 2017 to June 2023.
  • MRI volume measurements compared before and after treatment to assess tumor response.
  • Follow-up duration averaged 1085 days.
  • 13 patients (57%) showed a tumor reduction greater than 20%.
  • 7 subjects (30%) experienced tumor stabilization while 3 (13%) had tumor growth.
  • The median volumetric reduction of the tumors was 23%, and no severe adverse events were reported.

Cite This Study

Baldo et al. (2026) studied this question.

synapsesocial.com/papers/6a2901536f82f25be989daf9https://doi.org/10.1186/s12887-026-07081-1
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Also Consider

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

  1. 1NFS-27. SELUMETINIB FOR SYMPTOMATIC, INOPERABLE PLEXIFORM NEUROFIBROMAS IN NEUROFIBROMATOSIS TYPE 1: A SINGLE-INSTITUTION EXPERIENCE2024 · 1 citations
  2. 2Real-world experience with selumetinib in children with neurofibromatosis type 1: a multicentric retrospective study2025 · 5 citations
  3. 3Management of selumetinib-associated toxicity in children with neurofibromatosis type 1 and plexiform neurofibromas2025
  4. 4Respiratory function in children and young adults affected by neurofibromatosis type 1 treated with selumetinib2026
  5. 5Pharmacotherapy of plexiform neurofibromas in patients with neurofibromatosis type 1. Possible adverse events and their management2024 · 2 citations