Long-term follow-up of phase 2 AMPECT trial of nab-sirolimus for advanced malignant perivascular epithelioid cell tumors

In the United States, nab-sirolimus is approved for the treatment of locally advanced or metastatic malignant perivascular epithelioid cell tumor (PEComa) on the basis of the primary analysis of the phase 2 AMPECT trial. At the time of publication of the primary analysis, the median duration of response (mDOR) had not been reached. Long-term follow-up of the trial has now been published, 3 years after the primary analysis. The confirmed overall response rate was 38.7% (95% CI 21.8-57.8) and the mDOR was reached at 39.7 months. The median progression-free survival was 10.6 months (95% CI 5.5-41.2) and the median overall survival was 53.1 months (22.2-NR). There were no new or unexpected treatment-related adverse events, and there were no grade 4 or 5 TRAEs. The authors conclude that these results highlight the long-term clinical benefit of nab-sirolimus in patients with advanced malignant PEComa.