{"doi":"10.1093/neuonc/noac013","title":"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.","journal":"Neuro-Oncology","year":2022,"id":248310,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":30,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9555,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2022-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":887671,"name":"Robert Avery","orcid":null,"position":1,"is_corresponding":false},{"id":887672,"name":"Kelly D Getz","orcid":null,"position":2,"is_corresponding":false},{"id":351812,"name":"Éric Bouffet","orcid":"0000-0002-6832-6539","position":3,"is_corresponding":false},{"id":887673,"name":"Peter de Blank","orcid":null,"position":4,"is_corresponding":false},{"id":841412,"name":"Robert Listernick","orcid":"0000-0001-5564-4661","position":5,"is_corresponding":false},{"id":887674,"name":"David H Gutmann","orcid":null,"position":6,"is_corresponding":false},{"id":109187,"name":"Miriam Bornhorst","orcid":"0000-0002-7595-7726","position":7,"is_corresponding":false},{"id":721733,"name":"Cynthia Campen","orcid":"0000-0002-3519-1375","position":8,"is_corresponding":false},{"id":887675,"name":"Grant T Liu","orcid":null,"position":9,"is_corresponding":false},{"id":85409,"name":"Richard Aplenc","orcid":"0000-0001-7482-5644","position":10,"is_corresponding":false},{"id":302800,"name":"Yimei Li","orcid":"0000-0002-7046-4316","position":11,"is_corresponding":false},{"id":887676,"name":"Michael J Fisher","orcid":null,"position":12,"is_corresponding":false},{"id":794118,"name":"Chelsea Kotch","orcid":"0000-0001-6561-575X","position":0,"is_corresponding":true}],"reference_count":19,"raw_metadata":null,"created_at":"2026-07-19T00:24:09.893688Z","pmid":"35018469","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}