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| Molecular Profile | BRAF V600E |
| Therapy | Vemurafenib |
| Indication/Tumor Type | childhood pilocytic astrocytoma |
| Response Type | sensitive |
| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| BRAF V600E | childhood pilocytic astrocytoma | sensitive | Vemurafenib | Phase I | Actionable | In a Phase I trial (PNOC-002), Zelboraf (vemurafenib) treatment was tolerable and resulted in 1 complete response, 5 partial responses, and 13 stable disease in 19 pediatric patients with recurrent low grade gliomas harboring BRAF V600E, including pilocytic astrocytoma (n=10), ganglioglioma (n=5), pleomorphic xanthroastrocytoma (n=2), fibrillary astrocytoma (n=1), and astrocytoma NOS (n=1) (PMID: 32523649; NCT01748149). | 32523649 |
| BRAF V600E | childhood pilocytic astrocytoma | sensitive | Vemurafenib | Case Reports/Case Series | Actionable | In a clinical case study, Zelboraf (vemurafenib) treatment resulted in a partial response in a pediatric patient with pilocytic astrocytoma harboring BRAF V600E (PMID: 38976815). | 38976815 |
| PubMed Id | Reference Title | Details |
|---|---|---|
| (38976815) | Midline Low-Grade Gliomas of Early Childhood: Focus on Targeted Therapies. | Full reference... |
| (32523649) | Phase I study of vemurafenib in children with recurrent or progressive BRAFV600E mutant brain tumors: Pacific Pediatric Neuro-Oncology Consortium study (PNOC-002). | Full reference... |