{"doi":"10.1093/neuonc/noac151","title":"Phase II study of alisertib as a single agent for treating recurrent or progressive atypical teratoid/rhabdoid tumor","abstract":"BACKGROUND: Recurrent atypical teratoid/rhabdoid tumor (AT/RT) is, most often, a fatal pediatric malignancy with limited curative options. METHODS: We conducted a phase II study of Aurora kinase A inhibitor alisertib in patients aged <22 years with recurrent AT/RT. Patients received alisertib once daily (80 mg/m2 as enteric-coated tablets or 60 mg/m2 as liquid formulation) on Days 1-7 of a 21-day cycle until progressive disease (PD) occurred. Alisertib plasma concentrations were measured in cycle 1 on Days 1 (single dose) and 7 (steady state) and analyzed with noncompartmental pharmacokinetics. Trial efficacy end point was ≥10 participants with stable disease (SD) or better at 12 weeks. RESULTS: SD (n = 8) and partial response (PR) (n = 1) were observed among 30 evaluable patients. Progression-free survival (PFS) was 30.0% ± 7.9% at 6 months and 13.3% ± 5.6% at 1 year. One-year overall survival (OS) was 36.7% ± 8.4%. Two patients continued treatment for >12 months. PFS did not differ by AT/RT molecular groups. Neutropenia was the most common adverse effect (n = 23/30, 77%). The 22 patients who received liquid formulation had a higher mean maximum concentration (Cmax) of 10.1 ± 3.0 µM and faster time to Cmax (Tmax = 1.2 ± 0.7 h) than those who received tablets (Cmax = 5.7 ± 2.4 µM, Tmax = 3.4 ± 1.4 h). CONCLUSIONS: Although the study did not meet predetermined efficacy end point, single-agent alisertib was well tolerated by children with recurrent AT/RT, and SD or PR was observed in approximately a third of the patients.","journal":"Neuro-Oncology","year":2022,"id":244212,"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":0.9607,"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":558757,"name":"Olivia Campagne","orcid":"0000-0001-6348-8910","position":1,"is_corresponding":false},{"id":288899,"name":"Catherine A. 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Lindsay","orcid":"0000-0001-5654-841X","position":10,"is_corresponding":false},{"id":288904,"name":"Lindsay Kilburn","orcid":"0000-0003-1478-3619","position":11,"is_corresponding":false},{"id":290377,"name":"Patricia Baxter","orcid":null,"position":12,"is_corresponding":false},{"id":481522,"name":"Amy Smith","orcid":"0000-0002-9892-3203","position":13,"is_corresponding":false},{"id":271180,"name":"John R. Crawford","orcid":"0000-0002-4100-7001","position":14,"is_corresponding":false},{"id":558759,"name":"Sonia Partap","orcid":"0000-0001-5615-7689","position":15,"is_corresponding":false},{"id":651186,"name":"Anne Bendel","orcid":"0000-0002-2062-0974","position":16,"is_corresponding":false},{"id":581489,"name":"Dolly Aguilera","orcid":"0000-0001-9668-8177","position":17,"is_corresponding":false},{"id":255622,"name":"Kim E. Nichols","orcid":"0000-0002-5581-6555","position":18,"is_corresponding":false},{"id":495591,"name":"Evadnie Rampersaud","orcid":"0000-0001-5195-5366","position":19,"is_corresponding":false},{"id":271073,"name":"David W. Ellison","orcid":"0000-0003-1239-7757","position":20,"is_corresponding":false},{"id":302801,"name":"Paul Klimo","orcid":"0000-0002-2385-6667","position":21,"is_corresponding":false},{"id":302804,"name":"Zoltán Patay","orcid":"0000-0003-1479-9864","position":22,"is_corresponding":false},{"id":5549,"name":"Giles Robinson","orcid":"0000-0001-7441-9486","position":23,"is_corresponding":false},{"id":284694,"name":"Alberto Broniscer","orcid":"0000-0001-9646-0463","position":24,"is_corresponding":false},{"id":558760,"name":"Clinton F. Stewart","orcid":"0000-0002-1546-9720","position":25,"is_corresponding":false},{"id":300542,"name":"Cynthia Wetmore","orcid":null,"position":26,"is_corresponding":false},{"id":5546,"name":"Amar Gajjar","orcid":"0000-0001-5019-0699","position":27,"is_corresponding":false},{"id":558756,"name":"Santhosh A. Upadhyaya","orcid":"0000-0001-6101-149X","position":0,"is_corresponding":true}],"reference_count":31,"raw_metadata":null,"created_at":"2026-07-19T00:23:25.718788Z","pmid":"35652336","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":[]}