Marked recovery of vision in children with optic pathway gliomas treated with bevacizumab.
OBJECTIVE
Children with optic pathway gliomas (OPGs) frequently experience vision loss from their tumors. Standard front-line treatment using carboplatin-based chemotherapy typically produces only a modest benefit (eg, stabilization or 0.2 logMAR improvement) in visual acuity (VA). Bevacizumab is a monoclonal antibody that targets vascular endothelial growth factor and acts primarily as an anti-angiogenic agent. Recent reports suggest a qualitative improvement in vision after bevacizumab-based treatment in children with OPGs.


METHODS
We report 4 cases of pediatric OPGs (2 neurofibromatosis type 1-related and 2 sporadic cases) that received treatment with bevacizumab due to progressive VA or visual field (VF) loss despite prior treatment with chemotherapy or proton-beam radiation. All 4 subjects demonstrated a marked improvement in their VA, VF, or both while receiving bevacizumab-based therapy. Three patients had complete resolution of their VA or VF loss in at least 1 eye-2 of whom had previously received bevacizumab therapy.


CONCLUSIONS
Given that most patients with OPG-related visual impairment will show modest or no visual improvement with standard treatment, the incorporation of bevacizumab in these cases may greatly improve visual outcomes and should be considered in appropriate clinical situations.
Center for Neuroscience and Behavioral Medicine, Children's National Medical Center, Washington, DC2The Gilbert Family Neurofibromatosis Institute, Children's National Medical Center, Washington, DC3Division of Neurology, Children's National Medical Cente.
Robert A
RA
Avery
The Brain Tumor Institute, Children's National Medical Center, Washington, DC7Division of Hematology-Oncology, Children's National Medical Center, Washington, DC.
Eugene I
EI
Hwang
Division of Hematology-Oncology, Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania.
Regina I
RI
Jakacki
Center for Neuroscience and Behavioral Medicine, Children's National Medical Center, Washington, DC2The Gilbert Family Neurofibromatosis Institute, Children's National Medical Center, Washington, DC3Division of Neurology, Children's National Medical Cente.
Roger J
RJ
Packer
D000293:Adolescent; D020533:Angiogenesis Inhibitors / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D000068258:Bevacizumab; D002648:Child; D005260:Female; D006801:Humans; D007275:Injections, Intravenous; D008279:Magnetic Resonance Imaging; D009897:Optic Chiasm / Q000187:drug effects* / Q000503:physiopathology; D020339:Optic Nerve Glioma / Q000188:drug therapy* / Q000503:physiopathology*; D019574:Optic Nerve Neoplasms / Q000188:drug therapy* / Q000503:physiopathology; D020127:Recovery of Function / Q000502:physiology; D042461:Vascular Endothelial Growth Factor A / Q000037:antagonists & inhibitors; D014785:Vision, Ocular / Q000502:physiology*; D014792:Visual Acuity / Q000187:drug effects; D058609:Visual Field Tests; D014794:Visual Fields / Q000187:drug effects
D020533:Angiogenesis Inhibitors; D061067:Antibodies, Monoclonal, Humanized; C467484:VEGFA protein, human; D042461:Vascular Endothelial Growth Factor A; D000068258:Bevacizumab