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

NF Clinical Trials Consortium

doi:10.1002/pbc.28833

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

NF Clinical Trials Consortium

Pediatrics

Research output: Contribution to journal › Article › peer-review

7 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 language	English (US)
Article number	e28833
Journal	Pediatric Blood and Cancer
Volume	68
Issue number	4
DOIs	https://doi.org/10.1002/pbc.28833
State	Published - Apr 2021

Keywords

everolimus
neurofibromatosis
optic glioma
visual acuity

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health
Hematology
Oncology

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10.1002/pbc.28833

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title = "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) 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",

author = "{NF Clinical Trials Consortium} and Ullrich, {Nicole J.} and Prabhu, {Sanjay P.} and Packer, {Roger J.} and Stewart Goldman and Robison, {Nathan J.} and Allen, {Jeffrey C.} and Viskochil, {David H.} and Gutmann, {David H.} and Perentesis, {John P.} and Korf, {Bruce R.} and Fisher, {Michael J.} and Kieran, {Mark W.}",

note = "Funding Information: 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. Publisher Copyright: {\textcopyright} 2020 Wiley Periodicals LLC",

year = "2021",

month = apr,

doi = "10.1002/pbc.28833",

language = "English (US)",

volume = "68",

journal = "Pediatric Blood and Cancer",

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publisher = "Wiley-Liss Inc.",

number = "4",

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T1 - Visual outcomes following everolimus targeted therapy for neurofibromatosis type 1-associated optic pathway gliomas in children

AU - NF Clinical Trials Consortium

AU - Ullrich, Nicole J.

AU - Prabhu, Sanjay P.

AU - Packer, Roger J.

AU - Goldman, Stewart

AU - Robison, Nathan J.

AU - Allen, Jeffrey C.

AU - Viskochil, David H.

AU - Gutmann, David H.

AU - Perentesis, John P.

AU - Korf, Bruce R.

AU - Fisher, Michael J.

AU - Kieran, Mark W.

N1 - Funding Information: 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. Publisher Copyright: © 2020 Wiley Periodicals LLC

PY - 2021/4

Y1 - 2021/4

N2 - 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.

AB - 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.

KW - everolimus

KW - neurofibromatosis

KW - optic glioma

KW - visual acuity

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

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