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| Molecular Profile | BRAF V600X |
| Therapy | Vemurafenib |
| Indication/Tumor Type | Erdheim-Chester disease |
| Response Type | sensitive |
| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| BRAF V600X | Erdheim-Chester disease | sensitive | Vemurafenib | FDA approved | Actionable | In a Phase II trial (VE-BASKET) that supported FDA approval, Zelboraf (vemurafenib) treatment resulted in an objective response rate of 54.5% (12/22, 1 complete and 11 partial responses) in patients with Erdheim-Chester disease harboring BRAF V600 mutations, with a 2-year progression-free survival rate of 83% and 2-year overall survival rate of 95% (PMID: 29188284; NCT01524978). | detail... 29188284 |
| PubMed Id | Reference Title | Details |
|---|---|---|
| Zelboraf (vemurafenib) FDA Drug Label | Full reference... | |
| (29188284) | Vemurafenib for BRAF V600-Mutant Erdheim-Chester Disease and Langerhans Cell Histiocytosis: Analysis of Data From the Histology-Independent, Phase 2, Open-label VE-BASKET Study. | Full reference... |