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| Ref Type | Journal Article | ||||||||||||
| PMID | (35507888) | ||||||||||||
| Authors | Capone S, Ketonen L, Weathers SP, Subbiah V | ||||||||||||
| Title | Activity of Pemigatinib in Pilocytic Astrocytoma and FGFR1N546K Mutation. | ||||||||||||
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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 |
|---|---|---|---|---|---|---|---|
| FGFR1 N546K | pilocytic astrocytoma | predicted - sensitive | Pemigatinib | Case Reports/Case Series | Actionable | In a Phase I trial (FIGHT-101), Pemazyre (pemigatinib) treatment resulted in a partial response with a 52% decrease in tumor size at first restaging and a 91% tumor reduction after 13 cycles in a patient with pilocytic astrocytoma harboring FGFR1 N546K, with the response lasting 18 months (PMID: 35507888; NCT02393248). | 35507888 |