Phase II study of axitinib in patients with <i>NF2</i>-related schwannomatosis and progressive vestibular schwannomas.

ABSTRACT:

Background

Axitinib is an oral multi-receptor tyrosine kinase inhibitor targeting vascular endothelial growth factor receptor (VEGFR), platelet-derived growth factor receptor (PDGFR), and c-KIT. These represent a clinically and/or preclinically validated molecular targets in vestibular schwannoma (VS).

Methods

Eligible patients were age > 5 years with a clinical diagnosis of NF2-related schwannomatosis (NF2-SWN) and at least one volumetrically measurable, progressive VS. Axitinib was given continuously in 28-day cycles for up to of 12 cycles. Primary endpoint was objective volumetric response rate to axitinib, hearing response was a secondary endpoint, along with validated quality of life assessments (NFTI-QOL).

Results

Twelve patients were enrolled and

SUBMITTER: Garcia MR 

PROVIDER: S-EPMC12199335 | biostudies-literature | 2025 Jan-Dec

REPOSITORIES: biostudies-literature