Efficacy of oral multikinase inhibitors in refractory Ewing sarcoma

Abstract

We report the case of a nine-year-old boy with localized Ewing sarcoma (EWS) of the right thorax. He was treated with standard chemotherapy but was inoperable because of his poor response to treatment. Subsequent high-dose chemotherapy with autologous peripheral blood stem cell transplantation, irradiation, and multiple additional chemotherapy regimens failed to show sufficient efficacy. He was then started on pazopanib, and the tumor shrank and remained stable for six months. However, the tumor regrew, and his respiratory status worsened. Therefore, he was started on cabozantinib, and his respiratory status improved. The use of these two oral multikinase inhibitors suppressed tumors in pediatric refractory EWS, eliminated the need for hospitalization and inpatient management, and allowed him to be cared for at home for nine months.