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Case Report/Case Series
January 2014

Marked Recovery of Vision in Children With Optic Pathway Gliomas Treated With Bevacizumab

Author Affiliations
  • 1Center for Neuroscience and Behavioral Medicine, Children’s National Medical Center, Washington, DC
  • 2The Gilbert Family Neurofibromatosis Institute, Children’s National Medical Center, Washington, DC
  • 3Division of Neurology, Children’s National Medical Center, Washington, DC
  • 4Division of Ophthalmology, Children’s National Medical Center, Washington, DC
  • 5The Brain Tumor Institute, Children’s National Medical Center, Washington, DC
  • 7Division of Hematology-Oncology, Children’s National Medical Center, Washington, DC
  • 6Division of Hematology-Oncology, Children’s Hospital of Pittsburgh, Pittsburgh, Pennsylvania

Copyright 2014 American Medical Association. All Rights Reserved. Applicable FARS/DFARS Restrictions Apply to Government Use.

JAMA Ophthalmol. 2014;132(1):111-114. doi:10.1001/jamaophthalmol.2013.5819

Importance  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.

Observations  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 and Relevance  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.