[Correspondence] Clinical benefits of selumetinib in adults with neurofibromatosis type 1

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Source: The Lancet

Original: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(25)02111-7/fullt...

Published: 2025-12-13

The KOMET1 trial is the first randomized, placebo-controlled trial to demonstrate the efficacy of selumetinib in adult patients with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PN). At cycle 16, the selumetinib group achieved an objective response rate (ORR) of 20%, compared to 5% in the placebo group (p=0.011). Selumetinib caused a reduction in tumor volume and an improvement in symptoms such as pain, with no disease progression during the follow-up period. The study also confirmed a manageable side effect profile, with acneiform rash being the most common side effect. Selumetinib is thus a significant therapeutic benefit for adult patients with NF1 and inoperable PN, while the results support its use outside the pediatric population as well. However, the clinical benefits need to be evaluated with regard to the response rate and other clinical parameters.