Overview
- In a single-center retrospective series, 11 children aged 5 to 17 received pazopanib alongside standard first-line therapy between 2016 and 2024 at the Warsaw Mother and Child Institute.
- Two-year overall survival was 85.7% and event-free survival was 68.2%, results the authors say exceed historical benchmarks for this high-risk population.
- Imaging showed responses in all but one patient; at reporting, 10 children were alive and six were still taking pazopanib.
- The oral anti-angiogenic drug, originally developed for renal cell carcinoma, was generally well tolerated with minimal, manageable side-effects and could be taken at home after IV treatment.
- Authors emphasize the study’s limits—small, non-randomized, single-center design with heterogeneous concomitant therapies—and urge prospective multicenter controlled trials to validate efficacy and safety.