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| Home > Publications database > 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. > print |
| 001 | 300575 | ||
| 005 | 20250420020252.0 | ||
| 024 | 7 | _ | |a 10.1093/neuonc/noaf065 |2 doi |
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| 041 | _ | _ | |a English |
| 082 | _ | _ | |a 610 |
| 100 | 1 | _ | |a Fangusaro, Jason |0 0000-0003-3099-5259 |b 0 |
| 245 | _ | _ | |a 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. |
| 260 | _ | _ | |a Oxford |c 2025 |b Oxford Univ. Press |
| 336 | 7 | _ | |a article |2 DRIVER |
| 336 | 7 | _ | |a Output Types/Journal article |2 DataCite |
| 336 | 7 | _ | |a Journal Article |b journal |m journal |0 PUB:(DE-HGF)16 |s 1744896405_22798 |2 PUB:(DE-HGF) |
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| 336 | 7 | _ | |a Journal Article |0 0 |2 EndNote |
| 500 | _ | _ | |a epub |
| 520 | _ | _ | |a 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%. 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.Selumetinib provided stability and responses across many pLGG subgroups, and some patients achieved prolonged disease control without additional therapy. |
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| 650 | _ | 7 | |a Recurrent |2 Other |
| 650 | _ | 7 | |a and MEK inhibitor |2 Other |
| 650 | _ | 7 | |a pediatric low-grade glioma (pLGG) |2 Other |
| 650 | _ | 7 | |a selumetinib |2 Other |
| 700 | 1 | _ | |a Onar-Thomas, Arzu |0 0000-0003-1005-2413 |b 1 |
| 700 | 1 | _ | |a Young Poussaint, Tina |b 2 |
| 700 | 1 | _ | |a Lensing, Shelly |b 3 |
| 700 | 1 | _ | |a Ligon, Azra H |b 4 |
| 700 | 1 | _ | |a Lindeman, Neal |b 5 |
| 700 | 1 | _ | |a Banerjee, Anuradha |b 6 |
| 700 | 1 | _ | |a Kilburn, Lindsay B |b 7 |
| 700 | 1 | _ | |a Lenzen, Alicia |b 8 |
| 700 | 1 | _ | |a Pillay-Smiley, Natasha |b 9 |
| 700 | 1 | _ | |a Pollack, Ian F |b 10 |
| 700 | 1 | _ | |a Robison, Nathan J |b 11 |
| 700 | 1 | _ | |a Partap, Sonia |0 0000-0001-5615-7689 |b 12 |
| 700 | 1 | _ | |a Qaddoumi, Ibrahim |b 13 |
| 700 | 1 | _ | |a Landi, Daniel |b 14 |
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| 700 | 1 | _ | |a Stewart, Clinton F |b 16 |
| 700 | 1 | _ | |a Fouladi, Maryam |0 0000-0002-5693-035X |b 17 |
| 700 | 1 | _ | |a Dunkel, Ira J |0 0000-0001-8091-6067 |b 18 |
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