AUTHOR=Chan Marcus C. Y. , Fung Kevin K. F. , Ng Wai-Fu , Luk Ho-Ming , Ku Dennis T. L. , Liu Anthony P. Y. 

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

JOURNAL=Frontiers in Oncology

VOLUME=14

YEAR=2024

URL=https://www.frontiersin.org/journals/oncology/articles/10.3389/fonc.2024.1433073

DOI=10.3389/fonc.2024.1433073

ISSN=2234-943X

ABSTRACT=<p>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.</p>