Conflict of interest: Nothing to declare.
Lessons learned from adult clinical experience to inform evaluations of VEGF pathway inhibitors in children with cancer
Article first published online: 24 APR 2014
Published 2014. This article is a U.S. Government work and is in the public domain in the USA.
Pediatric Blood & Cancer
Volume 61, Issue 8, pages 1497–1505, August 2014
How to Cite
Smith, M. A. (2014), Lessons learned from adult clinical experience to inform evaluations of VEGF pathway inhibitors in children with cancer. Pediatr. Blood Cancer, 61: 1497–1505. doi: 10.1002/pbc.25036
- Issue published online: 10 JUN 2014
- Article first published online: 24 APR 2014
- Manuscript Accepted: 27 FEB 2014
- Manuscript Received: 8 DEC 2013
- kinase inhibitors;
- vascular endothelial growth factor
Agents targeting the vascular endothelial growth factor (VEGF) pathway have been studied in adults with cancer for nearly two decades. It is important to assess the lessons learned from this adult experience and to see how these lessons can help inform pediatric development of agents in this class. The benefit achieved from the use of VEGF pathway targeted agents for adult cancers has primarily been to delay for several months disease progression and less commonly time to death for conditions in which cure is not a reasonable expectation. VEGF pathway targeted agents have shown no efficacy when applied in the adjuvant setting. For adults with advanced cancer, prolongation of survival by 2–3 months is considered an important achievement in some settings. However, the primary goal of pediatric oncology clinical research is to identify treatments that allow children to be cured of their cancer and to grow to adulthood without treatment-induced limitations that lower their quality of survival. An important question for the pediatric oncology research community, pharmaceutical companies, and regulatory agencies to address in planning for future clinical trials is whether existing data support a role for VEGF pathway targeted agents in contributing to a therapeutic pathway to cure for children with cancer. Pediatr Blood Cancer 2014; 61:1497–1505. Published 2014. This article is a U.S. Government work and is in the public domain in the USA.