Visual outcomes following everolimus targeted therapy for neurofibromatosis type 1-associated optic pathway gliomas in children

Pediatr Blood Cancer. 2021 Apr;68(4):e28833. doi: 10.1002/pbc.28833. Epub 2020 Dec 18.

Abstract

Data for visual acuity (VA) after treatment of neurofibromatosis type 1-associated optic pathway gliomas (NF1-OPGs) are limited. We retrospectively collected VA, converted to logMAR, before and after targeted therapy with everolimus for NF1-OPG, and compared to radiologic outcomes (14/18 with NF1-OPG, 25 eyes [three without quantifiable vision]). Upon completion of treatment, VA was stable in 19 eyes, improved in four eyes, and worsened in two eyes; visual and radiologic outcomes were discordant. In summary, the majority of children with NF1-OPG exhibited stabilization of their VA after everolimus treatment. A larger, prospective study will help delineate visual outcomes after targeted therapy.

Keywords: everolimus; neurofibromatosis; optic glioma; visual acuity.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Adolescent
  • Adult
  • Antineoplastic Agents / therapeutic use*
  • Child
  • Child, Preschool
  • Everolimus / therapeutic use*
  • Female
  • Humans
  • Infant
  • Male
  • Neurofibromatosis 1 / drug therapy*
  • Neurofibromatosis 1 / physiopathology
  • Optic Nerve Glioma / drug therapy*
  • Optic Nerve Glioma / physiopathology
  • Retrospective Studies
  • Treatment Outcome
  • Visual Acuity / drug effects*
  • Young Adult

Substances

  • Antineoplastic Agents
  • Everolimus