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

NF Clinical Trials Consortium

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11 Scopus citations

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.

Original languageEnglish (US)
Article numbere28833
JournalPediatric Blood and Cancer
Volume68
Issue number4
DOIs
StatePublished - Apr 2021

Funding

The primary clinical trial was supported by the United States Army Medical Research and Material Command, Office of the Congressionally Directed Medical Research Program, Department of Defense Neurofibromatosis Research Program, Grant number: W81XWH‐05‐01‐0615. We thank the children and parents who participated in this study.

Keywords

  • everolimus
  • neurofibromatosis
  • optic glioma
  • visual acuity

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Pediatrics, Perinatology, and Child Health

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