{"doi":"10.1093/neuonc/noaf065","title":"A phase 2 PBTC study of selumetinib for recurrent/progressive pediatric low-grade glioma: Strata 2, 5, and 6 with long-term outcomes on strata 1, 3, and 4","abstract":"BACKGROUND: PBTC-029B was a phase 2 trial evaluating efficacy of selumetinib in children with recurrent/progressive low-grade glioma. We report results of strata 2, 5, and 6 with updated survivals for strata 1, 3, and 4. METHODS: Stratum 2 included recurrent/progressive pilocytic astrocytoma (PA) not associated with neurofibromatosis type-1 (NF1) that screened negative for the BRAF-KIAA1549 fusion and BRAFV600E mutation. Stratum 5 enrolled non-PA that screened positive for one of the BRAF aberrations. Stratum 6 enrolled children who consented to tissue screening, but there was an assay failure. For long-term survivals, stratum 1 included non-NF1 PA positive for one of the BRAF aberrations; stratum 3 included NF1-associated pLGG; and stratum 4 included non-NF1 optic pathway/hypothalamic tumors. RESULTS: Stratum 2: among 14 evaluable patients, there was 1 partial response (PR), 7 stable disease (SD), and 6 progressive disease (PD); the overall response rate (ORR) was 7.1%. Two-year progression-free survival (PFS)/overall survival (OS) were 57.1%/100%, respectively. Stratum 5: among 23 evaluable patients, there was 1 complete response (CR), 4 PR, 12 SD, and 6 PD; ORR was 21.7%. Two-year PFS/OS were 74.8%/100%, respectively. Stratum 6: among 26 evaluable patients, there were 7 PR, 14 SD, and 5 PD; ORR was 26.9%. Two-year PFS/OS were 72.0%/100%, respectively. The median follow-up for patients on strata 1, 3, and 4 without events are 60.4, 60.4, and 58.1 months, and 5-year PFS/OS were 30.8%/88.9%, 54.2%/100%, and 51.0%/100%, respectively. CONCLUSIONS: Selumetinib provided stability and responses across many pLGG subgroups, and some patients achieved prolonged disease control without additional therapy.","journal":"Neuro-Oncology","year":2025,"id":512079,"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":13,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.7983,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2025-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":288898,"name":"Arzu Onar‐Thomas","orcid":"0000-0003-1005-2413","position":1,"is_corresponding":false},{"id":260741,"name":"Tina Young Poussaint","orcid":"0000-0002-0814-0791","position":2,"is_corresponding":false},{"id":607882,"name":"Shelly Lensing","orcid":"0000-0002-6231-9362","position":3,"is_corresponding":false},{"id":35266,"name":"Azra H. Ligon","orcid":"0000-0001-7585-2441","position":4,"is_corresponding":false},{"id":86332,"name":"Neal Lindeman","orcid":null,"position":5,"is_corresponding":false},{"id":1370966,"name":"Anuradha Banerjee","orcid":"0000-0002-1694-4319","position":6,"is_corresponding":false},{"id":288904,"name":"Lindsay Kilburn","orcid":"0000-0003-1478-3619","position":7,"is_corresponding":false},{"id":1025833,"name":"Alicia Lenzen","orcid":"0000-0002-9123-419X","position":8,"is_corresponding":false},{"id":1143901,"name":"Natasha Pillay‐Smiley","orcid":null,"position":9,"is_corresponding":false},{"id":306061,"name":"Ian F. Pollack","orcid":"0000-0003-1765-357X","position":10,"is_corresponding":false},{"id":295595,"name":"Nathan Robison","orcid":"0000-0001-8613-9772","position":11,"is_corresponding":false},{"id":558759,"name":"Sonia Partap","orcid":"0000-0001-5615-7689","position":12,"is_corresponding":false},{"id":577364,"name":"Ibrahim Qaddoumi","orcid":"0000-0002-6645-4895","position":13,"is_corresponding":false},{"id":605820,"name":"Daniel Landi","orcid":"0000-0002-1487-1136","position":14,"is_corresponding":false},{"id":14376,"name":"David Jones","orcid":"0000-0002-2036-5141","position":15,"is_corresponding":false},{"id":558760,"name":"Clinton F. Stewart","orcid":"0000-0002-1546-9720","position":16,"is_corresponding":false},{"id":288906,"name":"Maryam Fouladi","orcid":"0000-0002-5693-035X","position":17,"is_corresponding":false},{"id":236601,"name":"Ira J. Dunkel","orcid":"0000-0001-8091-6067","position":18,"is_corresponding":false},{"id":496271,"name":"Jason Fangusaro","orcid":"0000-0003-3099-5259","position":0,"is_corresponding":true}],"reference_count":30,"raw_metadata":null,"created_at":"2026-07-19T02:48:01.269605Z","pmid":"40241281","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":[]}