Phase II Trial of Sirolimus in Patients With Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT): Long-Term Efficacy and Safety Update.

JCO -Sirolimus is approved in the United States for the treatment of metastatic or locally advanced malignant perivascular epithelioid cell tumor (PEComa) on the basis of the primary analysis results of the phase II Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT) trial (ClinicalTrials.gov identifier: NCT02494570). Results from the primary analysis were previously published; however, the median duration of response (mDOR) had not been reached at that time.

-Sirolimus for Patients With Malignant Perivascular Epithelioid Cell Tumors.

Purpose: Malignant perivascular epithelioid cell tumor (PEComa) is a rare aggressive sarcoma, with no approved treatment. To our knowledge, this phase II, single-arm, registration trial is the first prospective clinical trial in this disease, investigating the safety and efficacy of the mammalian target of rapamycin inhibitor -sirolimus (AMPECT, NCT02494570).

Patients And Methods: Patients with malignant PEComa were treated with -sirolimus 100 mg/m intravenously once weekly for 2 weeks in 3-week cycles.

nab-Paclitaxel in patients with advanced solid tumors and hepatic dysfunction: a pilot study.

Objective: This pilot open-label clinical study evaluated the safety and pharmacokinetics of albumin-bound paclitaxel (nab-paclitaxel) in patients with advanced solid tumors and hepatic dysfunction.

Research Design/methods: Dosing was determined according to baseline bilirubin levels as described in the package insert for Taxol((R)) (paclitaxel), and patients received 130, 200 or 260 mg/m(2) nab-paclitaxel every 3 weeks.

Results: Thirty patients with elevated baseline bilirubin and aspartate aminotransferase levels received nab-paclitaxel.