Visual outcomes following everolimus targeted therapy for neurofibromatosis type 1‐associated optic pathway gliomas in children
Year: 2020
Type: article
Study design: Observational Study
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.
Source: Pediatric Blood & Cancer
DOI: 10.1002/pbc.28833
Institutions: Boston Children's Hospital, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Children's National, Lurie Children's Hospital, Children's Hospital of Los Angeles, NYU Langone Health, New York University, University of Utah, Washington University in St. Louis, Cincinnati Children's Hospital Medical Center, University of Alabama at Birmingham, UAB Medicine, Children's Hospital of Philadelphia
Language: English
Subfield: Neurology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $3,550
OpenAlex ID: W3116077434