Relapsed high-risk neuroblastoma has few effective therapies currently available or in development. Cabozantinib is an Food and Drug Administration approved multitargeted tyrosine kinase inhibitor for select adult malignancies with preclinical data suggesting efficacy against neuroblastoma. A safe and tolerable dose has been identified for children, but its efficacy remains unknown. We describe four children with relapsed metastatic neuroblastoma treated with cabozantinib. All four patients had extended disease control (two complete responsesfor >12 months, 2 stable disease >6 months) with manageable predictable toxicities requiring dose reduction in two patients. We discuss the potential for the use of cabozantinib in neuroblastoma.
Keywords: CNS relapse; CNS tumors; cancer biology; chemotherapy; molecular biology of neuroblastoma; neuroblastoma; neuroblastoma biology; new agents; oncology (general); pediatric oncology; phase 2 clinical trial agents.
© 2020 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC.