Single-center phase 1a-1b trial of anlotinib in children with high-risk, recurrent or refractory sarcomas

Anlotinib is a novel highly potent multitargeted TKI. The safety, recommended dosage, and efficacy in pediatric patients have not been fully studied. A phase 1a-1b trial at Sun Yat-sen University Cancer Center evaluated anlotinib once daily on a 2-week-on/1-week-off schedule for children aged 5 to 18 year with high-risk, recurrent or refractory sarcomas. The MTD was 8 mg for those under 35 kg and 12 mg for those 35 kg or more. All children had adverse events, with most being grade 1 or 2 in severity. Among patients receiving anlotinib as maintenance therapy during one year, two-year progression-free survival and overall survival rates were 84.6% and 92.3%, respectively. The authors conclude that anlotinib was well tolerated, with preliminary evidence of efficacy.