Risk factors for treatment-refractory and relapsed optic pathway glioma in children with neurofibromatosis type 1.

Abstract

BACKGROUND:

Nearly one-third of patients with neurofibromatosis type 1-associated optic pathway glioma (NF1-OPG) fail frontline chemotherapy; however, little is known about risk factors for treatment failure.

METHODS:

We performed a retrospective multi-institutional cohort study to identify baseline risk factors for treatment-refractory/relapsed disease and poor visual outcome in children with NF1-OPG. Refractory/relapsed NF1-OPG was defined as a requirement of two or more treatment regimens due to progression or relapse.

RESULTS:

Of 111 subjects eligible for inclusion, adequate clinical and visual data were available for 103 subjects from 7 institutions. Median follow-up from the initiation of first chemotherapy regimen was 95 months (range 13-185). Eighty-four (82%) subjects received carboplatin-based frontline chemotherapy. Forty-five subjects (44%) experienced refractory/relapsed disease, with a median time of 21.5 months (range 2-149) from the initiation of first treatment to the start of second treatment. The proportion of patients without refractory/relapsed disease at 2 and 5 years was 78% and 60%. In multivariable analyses, age less than 24 months at initial treatment, posterior tumor location, and familial inheritance were associated with refractory/relapsed NF1-OPG by 2 years. Both age less than 24 months and posterior tumor location were associated with refractory/relapsed NF1-OPG by 5 years. Subjects with moderate to severe vision loss at last follow-up were more likely to have posterior tumor location, optic disc abnormalities, or abnormal visual acuity at initial treatment.

CONCLUSION:

Young age, posterior tumor location, and optic disc abnormalities may identify patients with the greatest likelihood of refractory/relapsed NF1-OPG and poor visual outcomes, and who may benefit from newer treatment strategies.

• Authors: Chelsea Kotch, Robert Avery, Kelly D Getz, Eric Bouffet, Peter de Blank, Robert Listernick, David H Gutmann, Miriam Bornhorst, Cynthia Campen, Grant T Liu, Richard Aplenc, Yimei Li, Michael J Fisher
• Journal: Neuro-oncology (Neuro Oncol) Vol. 24 Issue 8 Pg. 1377-1386 (08 01 2022) ISSN: 1523-5866 [Electronic] England
• PMID: 35018469 (Publication Type: Journal Article, Research Support, N.I.H., Extramural)
• Copyright: © The Author(s) 2022. Published by Oxford University Press on behalf of the Society for Neuro-Oncology. All rights reserved. For permissions, please e-mail: [email protected].

Topics

• Child
• Cohort Studies
• Humans
• Infant
• Neurofibromatosis 1 (complications, therapy)
• Optic Nerve Glioma (complications)
• Retrospective Studies
• Risk Factors