{"entity":{"id":"paper-ampect-nab-sirolimus-pecoma-wagner-jco-2021","kind":"paper","name":"AMPECT: nab-sirolimus for malignant perivascular epithelioid cell tumours","aka":[],"tldr":"Albumin-bound sirolimus shrank tumours in about four in ten patients with malignant PEComa, with responses lasting years and best results in tumours with TSC2 mutations, and became the first approved treatment for the disease.","summary":"Phase 2 study of 34 patients with malignant PEComa treated with intravenous nab-sirolimus weekly for two of every three weeks.\n\nObjective response by independent review was 39 percent with a median duration of response not reached (over 2.5 years), median progression-free survival 10.6 months; 89 percent of TSC2-mutant tumours responded. Stomatitis, myelosuppression and hyperglycaemia were manageable.","asOf":"2026-09-17","links":[{"label":"J Clin Oncol 2021","url":"https://doi.org/10.1200/JCO.21.01728"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/34637337/"}],"tags":[],"related":[],"cancers":["pecoma"],"sections":[],"technologies":[],"targets":[],"drugs":["sirolimus-albumin-bound"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["ampect"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["jco"],"dependsOn":[],"notes":[],"journal":"Journal of Clinical Oncology","year":2021,"doi":"10.1200/JCO.21.01728","pmid":"34637337","authors":"Wagner AJ, Ravi V, Riedel RF, et al.","paperType":"observational","findings":["Objective response 39 percent; 89 percent in TSC2-mutant tumours.","Median duration of response not reached at 2.5 years."],"whatItMeans":"Nab-sirolimus is the approved first-line treatment for advanced malignant PEComa, and TSC1/TSC2 testing helps predict response.","caveats":["Single-arm study in a rare tumour; comparison with oral mTOR inhibitors is indirect."],"changedPractice":true,"participants":34},"route":"/key-papers/paper-ampect-nab-sirolimus-pecoma-wagner-jco-2021/","neighbours":{"cancer":[{"id":"pecoma","kind":"cancer","name":"Perivascular epithelioid cell tumour (PEComa)","route":"/cancers/pecoma/"}],"drug":[{"id":"sirolimus-albumin-bound","kind":"drug","name":"Sirolimus protein-bound particles","route":"/drugs/sirolimus-albumin-bound/"}],"trial":[{"id":"ampect","kind":"trial","name":"AMPECT","route":"/trials/ampect/"}],"journal":[{"id":"jco","kind":"journal","name":"Journal of Clinical Oncology","route":"/journals/jco/"}]}}