Phase II study of axitinib in patients with

Publication/Presentation Date

1-1-2025

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

RESULTS: Twelve patients were enrolled and 8 completed 12 cycles, including 2 pediatric patients. Ten patients were evaluated for the primary endpoint, defined as ≥ 20% decrease in VS volume, with 2 volumetric responses observed; both were reached after 3 cycles and sustained during treatment. The best volumetric response was -53.9% after 9 cycles. Three hearing responses were observed, one of which was sustained during treatment. All patients experienced drug-related toxicities, the most common were diarrhea, hematuria, and skin toxicity, not exceeding grade 2, as well as hypertension, not exceeding grade 3. NFTI-QOL scores remained stable or improved during treatment.

CONCLUSIONS: Axitinib therapy targeting VEGFR, PDGFR and c-KIT is feasible in this population and associated with volumetric and hearing responses in a subset of patients. However, convenience of oral administration should be balanced with respect to efficacy and safety of axitinib in comparison with other molecular-targeted therapies, including intravenous bevacizumab.

Volume

7

Issue

1

First Page

083

Last Page

083

ISSN

2632-2498

Disciplines

Medicine and Health Sciences | Pediatrics

PubMedID

40575410

Department(s)

Department of Pediatrics

Document Type

Article

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