{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,25]],"date-time":"2026-09-25T05:16:47Z","timestamp":1790313407855,"version":"4.1.0"},"reference-count":19,"publisher":"Oxford University Press (OUP)","issue":"8","license":[{"start":{"date-parts":[[2022,1,9]],"date-time":"2022-01-09T00:00:00Z","timestamp":1641686400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/journals\/pages\/open_access\/funder_policies\/chorus\/standard_publication_model"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["T32-CA-09679"],"award-info":[{"award-number":["T32-CA-09679"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2022,8,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:sec>\n                  <jats:title>Background<\/jats:title>\n                  <jats:p>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.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Methods<\/jats:title>\n                  <jats:p>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.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>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.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusion<\/jats:title>\n                  <jats:p>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.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1093\/neuonc\/noac013","type":"journal-article","created":{"date-parts":[[2022,1,5]],"date-time":"2022-01-05T20:15:47Z","timestamp":1641413747000},"page":"1377-1386","source":"Crossref","is-referenced-by-count":31,"title":["Risk factors for treatment-refractory and relapsed optic pathway glioma in children with neurofibromatosis type 1"],"prefix":"10.1093","volume":"24","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6561-575X","authenticated-orcid":false,"given":"Chelsea","family":"Kotch","sequence":"first","affiliation":[{"name":"Division of Oncology, Department of Pediatrics, Children\u2019s Hospital of Philadelphia , Philadelphia, Pennsylvania , USA"},{"name":"University of Pennsylvania Perelman School of Medicine , Philadelphia, Pennsylvania , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"Avery","sequence":"additional","affiliation":[{"name":"Division of Oncology, Department of Pediatrics, Children\u2019s Hospital of Philadelphia , Philadelphia, Pennsylvania , USA"},{"name":"Division of Ophthalmology, Department of Surgery, Children\u2019s Hospital of Philadelphia , Philadelphia, Pennsylvania , USA"},{"name":"University of Pennsylvania Perelman School of Medicine , Philadelphia, Pennsylvania , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kelly D","family":"Getz","sequence":"additional","affiliation":[{"name":"University of Pennsylvania Perelman School of Medicine , Philadelphia, Pennsylvania , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6832-6539","authenticated-orcid":false,"given":"Eric","family":"Bouffet","sequence":"additional","affiliation":[{"name":"Division of Haematology\/Oncology, The Hospital for Sick Children, University of Toronto , Toronto, Ontario , Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"de Blank","sequence":"additional","affiliation":[{"name":"Division of Oncology, Cincinnati Children\u2019s Hospital Medical Center , Cincinnati, Ohio , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"Listernick","sequence":"additional","affiliation":[{"name":"Division of Advanced General Pediatrics, Department of Pediatrics, Ann & Robert H. Lurie Children\u2019s Hospital of Chicago , Chicago, Illinois , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David H","family":"Gutmann","sequence":"additional","affiliation":[{"name":"Department of Neurology, Washington University School of Medicine , St Louis, Missouri , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miriam","family":"Bornhorst","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology-Oncology, Children\u2019s National Hospital , Washington DC , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cynthia","family":"Campen","sequence":"additional","affiliation":[{"name":"Department of Neurology , Stanford University, Palo Alto, California , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Grant T","family":"Liu","sequence":"additional","affiliation":[{"name":"Division of Ophthalmology, Department of Surgery, Children\u2019s Hospital of Philadelphia , Philadelphia, Pennsylvania , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Aplenc","sequence":"additional","affiliation":[{"name":"University of Pennsylvania Perelman School of Medicine , Philadelphia, Pennsylvania , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yimei","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Biostatistics, University of Pennsylvania , Philadelphia, Pennsylvania , USA"},{"name":"University of Pennsylvania Perelman School of Medicine , Philadelphia, Pennsylvania , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael J","family":"Fisher","sequence":"additional","affiliation":[{"name":"Division of Oncology, Department of Pediatrics, Children\u2019s Hospital of Philadelphia , Philadelphia, Pennsylvania , USA"},{"name":"University of Pennsylvania Perelman School of Medicine , Philadelphia, Pennsylvania , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2022,1,9]]},"reference":[{"key":"2022080109010592300_CIT0001","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1097\/WNO.0000000000000550","article-title":"Optic pathway gliomas in neurofibromatosis type 1: an update: surveillance, treatment indications, and biomarkers of vision","volume":"37","author":"de Blank","year":"2017","journal-title":"J Neuroophthalmol."},{"issue":"1","key":"2022080109010592300_CIT0002","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1177\/0883073817739509","article-title":"Optic pathway gliomas in neurofibromatosis type 1","volume":"33","author":"Campen","year":"2018","journal-title":"J Child Neurol."},{"issue":"6","key":"2022080109010592300_CIT0003","doi-asserted-by":"crossref","first-page":"790","DOI":"10.1093\/neuonc\/nos076","article-title":"Visual outcomes in children with neurofibromatosis type 1-associated optic pathway glioma 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