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| Molecular Profile | TSC1 inact mut |
| Therapy | Nab-rapamycin |
| Indication/Tumor Type | perivascular epithelioid cell tumor |
| Response Type | predicted - sensitive |
| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| TSC1 inact mut | perivascular epithelioid cell tumor | predicted - sensitive | Nab-rapamycin | Phase II | Actionable | In a Phase II trial (AMPECT), Fyarro (nab-rapamycin) treatment in patients with perivascular epithelioid cell tumors (PEComas) resulted in partial response in 20% (1/5) of patients with TSC1 mutations, 89% (8/9) of patients with TSC2 mutations, and 9% (1/11) of patients without a TSC1 or TSC2 mutation (PMID: 34637337; NCT02494570). | 34637337 |
| PubMed Id | Reference Title | Details |
|---|---|---|
| (34637337) | nab -Sirolimus for Patients With Malignant Perivascular Epithelioid Cell Tumors. | Full reference... |