TY  - JOUR
AU  - Fangusaro, Jason
AU  - Onar-Thomas, Arzu
AU  - Young Poussaint, Tina
AU  - Lensing, Shelly
AU  - Ligon, Azra H
AU  - Lindeman, Neal
AU  - Banerjee, Anuradha
AU  - Kilburn, Lindsay B
AU  - Lenzen, Alicia
AU  - Pillay-Smiley, Natasha
AU  - Pollack, Ian F
AU  - Robison, Nathan J
AU  - Partap, Sonia
AU  - Qaddoumi, Ibrahim
AU  - Landi, Daniel
AU  - Jones, David
AU  - Stewart, Clinton F
AU  - Fouladi, Maryam
AU  - Dunkel, Ira J
TI  - 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.
JO  - Neuro-Oncology
VL  - nn
SN  - 1522-8517
CY  - Oxford
PB  - Oxford Univ. Press
M1  - DKFZ-2025-00808
SP  - nn
PY  - 2025
N1  - epub
AB  - 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
KW  - Recurrent (Other)
KW  - and MEK inhibitor (Other)
KW  - pediatric low-grade glioma (pLGG) (Other)
KW  - selumetinib (Other)
LB  - PUB:(DE-HGF)16
C6  - pmid:40241281
DO  - DOI:10.1093/neuonc/noaf065
UR  - https://inrepo02.dkfz.de/record/300575
ER  -