Multicenter real-world experience of the clinical efficacy and tolerance of pazopanib in high-risk pediatric solid tumors (PazoPed)

Pazopanib, a receptor tyrosine kinase inhibitor, exhibits anti-tumor activity in adult bone and soft-tissue sarcomas (STS), but has not yet been approved for pediatric tumors. The primary objective was to evaluate pazopanib efficacy when used alone or in combination with topotecan. This real-world multicenter retrospective study included patients with solid tumors, aged 25 years or less at the time of initial diagnosis, treated with pazopanib outside of a clinical trial. Nineteen patients were eligible for efficacy analysis: 14 bone tumors and 5 STS. At pazopanib initiation, the median age was 16.9 years, 18 patients had metastatic disease with a median of 2 prior therapeutic lines. With 6.2 months of median follow-up, no objective response was observed, but 10 patients (52.6%) had stable disease at 8 weeks and the 6-month disease control rate was 26.3%. The median progression free (PFS) and overall survival (OS) were 3.0 months and 6.2 months, respectively. Multivariate analysis showed an inverse relationship between the number of prior treatment lines and PFS and OS (hazard ratio = 1.73 (p = 0.04) and 1.76 (p = 0.03), respectively). Our study showed a potential tumor control activity of pazopanib in pediatric bone and soft tissue sarcomas. Further studies are warranted to determine the optimal timing and condition for pazopanib introduction to maximize the effect.