J Dermatol. 2025 Oct 23. doi: 10.1111/1346-8138.70030. Online ahead of print.
ABSTRACT
Neurofibromatosis 1 (NF1) is a multisystem genetic disorder characterized by cutaneous, neurological, and skeletal manifestations. Plexiform neurofibroma (PN) is a benign peripheral nerve sheath tumor, often leading to considerable morbidity due to its progressive nature. Selumetinib, a MEK inhibitor, represents a novel therapeutic agent for the treatment of symptomatic, inoperable PNs in pediatric NF1 patients. This report presents NF1 cases managed at Tottori University Hospital, in which the clinical application of selumetinib was evaluated under the guidance of institutional advisory boards and coordinated by a multidisciplinary team (MDT). Treatment with selumetinib resulted in clinical benefits, accompanied by manageable adverse events such as acneiform rash and transient elevations in serum creatine kinase levels. The coordinated efforts of the MDT facilitated appropriate patient selection, individualized dose adjustments, effective management of adverse events, and long-term follow-up. These cases highlight the clinical utility of selumetinib and underscore the importance of MDT involvement in the management of pediatric NF1 patients with PNs in Japan.
PMID:41127924 | DOI:10.1111/1346-8138.70030
