Bevacizumab as Single Agent in Children and Teenagers with Optic Pathway Glioma

SIMPLE SUMMARY: Nowadays, there is no univocal therapeutical care for children with optic pathway gliomas (OPG): different chemotherapy regimens are proposed, but no one has clearly proved its superiority over the others on the PFS (Progression free survival). The efficacy of bevacizumab, an anti-VEGF monoclonal antibody, used in combination with Irinotecan, has been raised by several recent publications. However, Irinotecan has demonstrated side effects, especially digestive. Our main goal is to understand if bevacizumab could be efficacious used as a single agent against OPG. ABSTRACT: This is a retrospective study conducted on patients with OPG, aged less than 19 years, treated with bevacizumab as a single agent, since 2010 at IHOPe (Institute of Pediatric Hematology and Oncology). Efficacy of the treatment was evaluated on the tumor response rate on MRI with a centralized review basing upon RAPNO criteria and with visual assessment basing upon a 0.2 log change in the logMAR scale. Thirty-one patients with OPG have been included. From a radiological point of view, best anytime responses were: 1 major response, 6 partial responses, 7 minor responses and 14 stable diseases; achieving disease control in 28 (96%) out of 29 patients. Ophthalmological response was evaluated in 25 patients and disease control was achieved in 22 (88%) out of 25, with 14 steady states and 8 significant improvements. Among patients treated with chemotherapy after the bevacizumab course, nine relapsed and have been retreated with objective responses. Bevacizumab used as single agent seems effective in children and adolescents with OPG. Our work paves the way for a phase II study in which bevacizumab alone could be used as frontline therapy.