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Visual outcomes following everolimus targeted therapy for neurofibromatosis type 1-associated optic pathway gliomas in children
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  • Nicole Ullrich,
  • Sanjay Prabhu,
  • Roger Packer,
  • Stewart Goldman,
  • Nathan Robison,
  • Jeffrey Allen,
  • David Viskochil,
  • David Gutmann,
  • John Perentesis,
  • Bruce Korf,
  • Michael Fisher,
  • Mark Kieran
Nicole Ullrich
Children's Hospital Boston
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Sanjay Prabhu
Boston Children's Hospital
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Roger Packer
Children's National Health System
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Stewart Goldman
Ann & Robert H Lurie Children's Hospital of Chicago
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Nathan Robison
Children's Hospital Los Angeles, University of Southern California Keck School of Medicine
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Jeffrey Allen
NYU Langone Health
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David Viskochil
University of Utah
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David Gutmann
Washington University in Saint Louis School of Medicine
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John Perentesis
Childrens Hospital Medical Center
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Bruce Korf
University of Alabama at Birmingham
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Michael Fisher
Children's Hospital of Philadelphia
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Mark Kieran
Dana-Farber Cancer Institute
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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.

Peer review status:Published

09 Oct 2020Submitted to Pediatric Blood & Cancer
09 Oct 2020Submission Checks Completed
09 Oct 2020Assigned to Editor
13 Oct 2020Reviewer(s) Assigned
09 Nov 2020Review(s) Completed, Editorial Evaluation Pending
10 Nov 2020Editorial Decision: Revise Minor
11 Nov 20201st Revision Received
11 Nov 2020Submission Checks Completed
11 Nov 2020Assigned to Editor
12 Nov 2020Review(s) Completed, Editorial Evaluation Pending
12 Nov 2020Editorial Decision: Accept
18 Dec 2020Published in Pediatric Blood & Cancer. 10.1002/pbc.28833