{"doi":"10.1093/noajnl/vdad011","title":"Phase II study of everolimus for recurrent or progressive pediatric ependymoma","abstract":"Abstract Background Preclinical studies have suggested that mTOR pathway signaling may be a potential therapeutic target for childhood ependymoma. Methods A phase II clinical trial (ClinicalTrials.gov identifier: NCT02155920) of single-agent everolimus was performed to test the hypothesis that mTOR pathway inhibition would result in tumor responses for children with recurrent and/or progressive ependymomas. Results Eleven subjects [sex: 4 females (36.4%); median age: 8 years (range: 2-15 years); race: 9 white; prior therapies: median 6 (range: 3-9)] were enrolled on the study. Ten primary tumors were located in the posterior fossa and one primary tumor was located in the spinal cord. Eight of 9 tumors were PF-A subtype epenydmomas. All subjects were treated with oral everolimus 4.5 mg/m2/day (each cycle = 28 days) that was titrated to achieve serum trough levels of 5-15 ng/ml. Overall, everolimus was well tolerated; except for a single event of grade 3 pneumonia, all adverse events were grade 1-2. No objective tumor responses were observed. Participating subjects experienced tumor progression and discontinued therapy after a median of 2 cycles of therapy (1 cycle = 2; 2 cycles = 6; 3, 4, and 8 cycles = 1 each). Conclusions Everolimus does not appear to have activity for children with recurrent or progressive PF-A ependymoma.","journal":"Neuro-Oncology Advances","year":2023,"id":372184,"datarank":0.3437950615515668,"base_score":1.791759469228055,"endowment":1.791759469228055,"self_citation_contribution":0.26876392038420827,"citation_network_contribution":0.07503114116735854,"self_endowment_contribution":0.26876392038420827,"citer_contribution":0.07503114116735854,"corpus_percentile":null,"corpus_rank":null,"citation_count":5,"citer_count":5,"citers_with_citation_signal":3,"citers_with_endowment":3,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9313,"is_data_producer":true,"deposit_databanks":{"ClinicalTrials.gov":["NCT02155920"]},"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2023-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":764657,"name":"Veena Rajaram","orcid":"0000-0002-2128-2006","position":1,"is_corresponding":false},{"id":236638,"name":"Matthias A. Karajannis","orcid":"0000-0002-7151-6528","position":2,"is_corresponding":false},{"id":282350,"name":"Sharon L. Gardner","orcid":"0000-0002-8857-5487","position":3,"is_corresponding":false},{"id":674156,"name":"Jack M. Su","orcid":"0000-0003-2125-3659","position":4,"is_corresponding":false},{"id":558758,"name":"Patricia Baxter","orcid":"0000-0003-3703-8742","position":5,"is_corresponding":false},{"id":558759,"name":"Sonia Partap","orcid":"0000-0001-5615-7689","position":6,"is_corresponding":false},{"id":729783,"name":"Laura J. Klesse","orcid":"0000-0003-1323-7720","position":7,"is_corresponding":false},{"id":647522,"name":"Daniel C. Bowers","orcid":"0000-0002-3947-2481","position":0,"is_corresponding":true}],"reference_count":43,"raw_metadata":null,"created_at":"2026-07-19T01:15:54.000926Z","pmid":"36950217","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":[]}