A plain language summary of the AMPECT study: nab-sirolimus for advanced malignant perivascular epithelioid cell tumors

Plain Language Summary: What is this summary about? This summary describes the results from a clinical study called AMPECT, which stands for Advanced Malignant Perivascular Epithelioid Cell Tumors. The study evaluated treatment with nab-sirolimus in patients with a rare form of advanced cancer called malignant perivascular epithelioid cell tumors, or malignant PEComa, which can form in various parts of the body. Researchers wanted to see if nab-sirolimus could shrink the size of tumors or stop the cancer from growing or spreading, and if there were any side effects with nab-sirolimus that might stop patients from taking it. What were the results? Tumors shrank in 39% of the patients who received treatment with nab-sirolimus. Additionally, 52% of patients had stable disease, which means their tumor neither increased nor decreased significantly in size. Tumors continued to respond to treatment without growing or spreading for a median of about 40 months. Patients who took the medicine lived for a median of about 53 months. Clinicians considered the side effects of treatment to be manageable by adjusting the dose of nab-sirolimus or treating the side effects. What do the results of the study mean? The results support the use of nab-sirolimus for treating patients with advanced malignant PEComa. nab-Sirolimus was the first medicine approved for treating adults with malignant PEComa that cannot be removed with surgery or that has spread to different parts of the body. Clinical trial number: NCT02494570.