Visual outcomes following everolimus targeted therapy for
neurofibromatosis type 1-associated optic pathway gliomas in children
Abstract
Data for visual acuity (VA) after treatment of neurofibromatosis type
1-associated optic pathway gliomas (NF1-OPGs) is 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 [3 without quantifiable
vision]). Upon completion of treatment, VA was stable in 19 eyes,
improved in 4 eyes, and worse in 2 eyes; visual and radiologic outcome
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.