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| Ref Type | Journal Article | ||||||||||||
| PMID | (41066726) | ||||||||||||
| Authors | Yi JS, McCarthy KS, Mazur K, Kudlaty R, Armstrong T, Desai N, Shepherd SP, Zinn D, Velazquez J, Scull B, Allen C, McClain K | ||||||||||||
| Title | Sustained Response to Pan-BRAF Inhibitor Plixorafenib (FORE8394, PLX8394) in a Young Adult With Neurodegenerative Langerhans Cell Histiocytosis. | ||||||||||||
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| Molecular Profile | Treatment Approach |
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| Gene Name | Source | Synonyms | Protein Domains | Gene Description | Gene Role |
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| Therapy Name | Drugs | Efficacy Evidence | Clinical Trials |
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| Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
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| Gene | Variant | Impact | Protein Effect | Variant Description | Associated with drug Resistance |
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| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
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| BRAF V600E | histiocytosis | predicted - sensitive | PLX8394 | Case Reports/Case Series | Actionable | In a Phase I/II trial (PLX120-03), PLX8394 treatment resulted in clinical symptom improvement and stable disease ongoing for at least 6.5 years in a young adult patient with recurrent neurodegenerative Langerhans cell histiocytosis harboring BRAF V600E (PMID: 41066726; NCT02428712). | 41066726 |