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 Journal Article
| Authors: | Fangusaro, J.; Onar-Thomas, A.; Poussaint, T. Y.; Lensing, S.; Ligon, A. H.; Lindeman, N.; Banerjee, A.; Kilburn, L. B.; Lenzen, A.; Pillay-Smiley, N.; Pollack, I. F.; Robison, N. J.; Partap, S.; Qaddoumi, I.; Landi, D.; Jones, D. T. W.; Stewart, C. F.; Fouladi, M.; Dunkel, I. J. |
| Article 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. |
| Keywords: | children; recurrent; selumetinib; mek inhibitor; pediatric low-grade glioma (plgg) |
| Journal Title: | Neuro-Oncology |
| ISSN: | 1522-8517 |
| Publisher: | Oxford University Press |
| Publication status: | Online ahead of print |
| Date Published: | 2025-01-01 |
| Online Publication Date: | 2025-01-01 |
| Language: | English |
| ACCESSION: | WOS:001481090300001 |
| DOI: | 10.1093/neuonc/noaf065 |
| PROVIDER: | wos |
| Notes: | Article; Early Access -- Source: Wos |
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