Case report: Treatment response of NF-1-associated bladder ganglioneuroma to trametinib

Autor: Marcus C. Y. Chan, Kevin K. F. Fung, Wai-Fu Ng, Ho-Ming Luk, Dennis T. L. Ku, Anthony P. Y. Liu
Jazyk: angličtina
Rok vydání: 2024
Předmět:
Zdroj: Frontiers in Oncology, Vol 14 (2024)
Druh dokumentu: article
ISSN: 2234-943X
DOI: 10.3389/fonc.2024.1433073
Popis: We present the clinical course of a 4-year-old girl with neurofibromatosis type 1-associated, unresectable, symptomatic urinary bladder ganglioneuroma. She was initially trialed on sirolimus without response and subsequently responded to MEK inhibitor trametinib, with improvement clinically and radiographically over 10 months. This report broadens the repertoire of therapeutic strategies for MEK inhibition in diseases related to the MAPK pathway.
Databáze: Directory of Open Access Journals