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• W4319748049 abstract "TSC-mutated sarcomas are rare molecular and histologic types of sarcoma. Due to the presence of their specific oncogenic driver mutation, these sarcomas are particularly sensitive to mTOR inhibitors. Recently, nab-sirolimus, an albumin-bound mTOR inhibitor, was approved by the Food and Drug Administration (FDA) for PEComas, which harbor a TSC mutation, and this drug remains the only FDA-approved systemic treatment for these tumors. We report on two cases of patients with TSC-mutated sarcomas who experienced significant responses to the combination of gemcitabine and sirolimus, after progression on prior gemcitabine-based chemotherapy and single agent mTOR inhibition with nab-sirolimus. Preclinical and clinical data support rationale for a synergistic effect of the combination. This combination may represent a valid therapeutic option after failure of nab-sirolimus in these patients, with no standard-of-care treatment options." @default.
• W4319748049 created "2023-02-11" @default.
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• W4319748049 date "2023-02-09" @default.
• W4319748049 modified "2023-09-30" @default.
• W4319748049 title "Case report: Responses to the combination of gemcitabine with sirolimus in two patients with TSC-mutated sarcomas" @default.
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• W4319748049 doi "https://doi.org/10.3389/fonc.2023.1046442" @default.
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