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000300575 1001_ $$00000-0003-3099-5259$$aFangusaro, Jason$$b0
000300575 245__ $$aA 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.
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000300575 520__ $$aPBTC-029B was a phase 2 trial evaluating efficacy of selumetinib in children with recurrent/progressive low-grade glioma. We report results of strata 2, 5, and 6 with updated survivals for strata 1, 3, and 4.Stratum 2 included recurrent/progressive pilocytic astrocytoma (PA) not associated with neurofibromatosis type-1 (NF1) that screened negative for the BRAF-KIAA1549 fusion and BRAFV600E mutation. Stratum 5 enrolled non-PA that screened positive for one of the BRAF aberrations. Stratum 6 enrolled children who consented to tissue screening, but there was an assay failure. For long-term survivals, stratum 1 included non-NF1 PA positive for one of the BRAF aberrations; stratum 3 included NF1-associated pLGG; and stratum 4 included non-NF1 optic pathway/hypothalamic tumors.Stratum 2: among 14 evaluable patients, there was 1 partial response (PR), 7 stable disease (SD) and 6 progressive disease (PD); overall response rate (ORR) was 7.1%. Two-year progression-free survival (PFS)/overall survival (OS) were 57.1%/100%, respectively. Stratum 5: among 23 evaluable patients, there was 1 complete response (CR), 4 PR, 12 SD, and 6 PD; ORR was 21.7%. Two-year PFS/OS were 74.8%/100%, respectively. Stratum 6: among 26 evaluable patients, there were 7 PR, 14 SD, and 5 PD; ORR was 26.9%. Two-year PFS/OS were 72.0%/100%, respectively. The median follow-up for patients on strata 1, 3, and 4 without events are 60.4, 60.4, and 58.1 months, and 5-year PFS/OS were 30.8%/88.9%, 54.2%/100%, and 51.0%/100%, respectively.Selumetinib provided stability and responses across many pLGG subgroups, and some patients achieved prolonged disease control without additional therapy.
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000300575 650_7 $$2Other$$aRecurrent
000300575 650_7 $$2Other$$aand MEK inhibitor
000300575 650_7 $$2Other$$apediatric low-grade glioma (pLGG)
000300575 650_7 $$2Other$$aselumetinib
000300575 7001_ $$00000-0003-1005-2413$$aOnar-Thomas, Arzu$$b1
000300575 7001_ $$aYoung Poussaint, Tina$$b2
000300575 7001_ $$aLensing, Shelly$$b3
000300575 7001_ $$aLigon, Azra H$$b4
000300575 7001_ $$aLindeman, Neal$$b5
000300575 7001_ $$aBanerjee, Anuradha$$b6
000300575 7001_ $$aKilburn, Lindsay B$$b7
000300575 7001_ $$aLenzen, Alicia$$b8
000300575 7001_ $$aPillay-Smiley, Natasha$$b9
000300575 7001_ $$aPollack, Ian F$$b10
000300575 7001_ $$aRobison, Nathan J$$b11
000300575 7001_ $$00000-0001-5615-7689$$aPartap, Sonia$$b12
000300575 7001_ $$aQaddoumi, Ibrahim$$b13
000300575 7001_ $$aLandi, Daniel$$b14
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000300575 7001_ $$aStewart, Clinton F$$b16
000300575 7001_ $$00000-0002-5693-035X$$aFouladi, Maryam$$b17
000300575 7001_ $$00000-0001-8091-6067$$aDunkel, Ira J$$b18
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