Risk factors for treatment-refractory and relapsed optic pathway glioma in children with neurofibromatosis type 1. Issue 8 (9th January 2022)

• Record Type: Journal Article
• Authors: Kotch, Chelsea; Avery, Robert; Getz, Kelly D; Bouffet, Eric; de Blank, Peter; Listernick, Robert; Gutmann, David H; Bornhorst, Miriam; Campen, Cynthia; Liu, Grant T; Aplenc, Richard; Li, Yimei; Fisher, Michael J
• Is Part Of: Neuro-oncology. Volume 24:Issue 8(2022)
• Page Start: 1377
• Page End: 1386
• Publication Date: 2022-01-09
• Subjects: cancer epidemiology -- neurofibromatosis type 1 -- optic pathway glioma -- refractory -- relapsed; Brain Neoplasms -- Periodicals; Brain -- Tumors -- Periodicals; Brain -- Cancer -- Periodicals; Nervous system -- Cancer -- Periodicals; 616.99481
• DOI: 10.1093/neuonc/noac013
• Languages: English
• ISSNs: 1522-8517

Abstract

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.