Safety, pharmacokinetics and efficacy of selumetinib in Chinese adult and paediatric patients with neurofibromatosis type 1 and inoperable plexiform neurofibromas: The primary analysis of a phase 1 open‐label study.

This article presents data from a phase 1 study on the safety, pharmacokinetics, and efficacy of selumetinib in Chinese pediatric and adult patients with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PN). The study found that selumetinib was well-tolerated, with most adverse events being mild. No unexpected trends or serious adverse events were observed, and no patients stopped treatment due to adverse events. The document also provides information on the adverse events experienced by patients receiving selumetinib, as well as the pharmacokinetics of the medication. Additionally, the document summarizes a study on the efficacy and safety of selumetinib in adults with NF1-PN in China. The study found promising efficacy and improvements in quality of life, but caution should be exercised when interpreting the efficacy data as it was a secondary endpoint. The study concluded that selumetinib has an acceptable benefit-risk profile and may address the medical needs of patients with NF1-PN in China. The study was funded by AstraZeneca and Merck Sharp & Dohme LLC. [Extracted from the article]