%0 Journal Article
%A Fangusaro, Jason
%A Onar-Thomas, Arzu
%A Young Poussaint, Tina
%A Lensing, Shelly
%A Ligon, Azra H
%A Lindeman, Neal
%A Banerjee, Anuradha
%A Kilburn, Lindsay B
%A Lenzen, Alicia
%A Pillay-Smiley, Natasha
%A Pollack, Ian F
%A Robison, Nathan J
%A Partap, Sonia
%A Qaddoumi, Ibrahim
%A Landi, Daniel
%A Jones, David
%A Stewart, Clinton F
%A Fouladi, Maryam
%A Dunkel, Ira J
%T 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.
%J Neuro-Oncology
%V nn
%@ 1522-8517
%C Oxford
%I Oxford Univ. Press
%M DKFZ-2025-00808
%P nn
%D 2025
%Z epub
%X 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.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.Stratum 2: among 14 evaluable patients, there was 1 partial response (PR), 7 stable disease (SD) and 6 progressive disease (PD); overall response rate (ORR) was 7.1
%K Recurrent (Other)
%K and MEK inhibitor (Other)
%K pediatric low-grade glioma (pLGG) (Other)
%K selumetinib (Other)
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:40241281
%R 10.1093/neuonc/noaf065
%U https://inrepo02.dkfz.de/record/300575