Plain Language Summary of Publication - AMPECT study: nab-sirolimus for advanced malignant perivascular epithelioid cell tumors

Sep 29, 2026

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Plain language summary describing results from the AMPECT clinical study of nab-sirolimus in adults with advanced malignant perivascular epithelioid cell tumors (malignant PEComa). The study assessed tumor shrinkage, disease stability, duration of response (median ~40 months), and overall survival (median ~53 months). Side effects were reported as manageable through dose adjustment and supportive treatment. Findings support nab-sirolimus use for malignant PEComa not removable by surgery or that has metastasized to other body parts.

LanguageEnglish

CategoryHealthcare

FormatPDF

Pages13

Reading Time33 min

AccessFree to download

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Future ONCOLOGY Plain Language Summary of Publication A plain language summary of the AMPECT study: nab-sirolimus for advanced malignant perivascular epithelioid cell tumors Andrew J. Wagnera, Vinod Ravib, Richard F. Riedelc, Kristen Ganjood, Brian A. Van Tinee, Rashmi Chughf, Lee Cranmerg, Erlinda M. Gordonh, Jason L. Hornicki, Heng Dui, Kathleen M. Polsona, Sudha Yalamanchilib, Cristina DeBoerj; Li Dingk*; Anita N. Schmidk*, Willis H. Navarrok*, David J. Kwiatkowskii & Mark A. Dicksonl Author affiliations can be found at the end of this article First draft submitted: 25 July 2025; Accepted for publication: 10 October 2025 Where can I find the original article on which this summary is based? The original article titled ‘Phase II trial of nab-sirolimus in patients with advanced malignant perivascular epithelioid cell tumors (AMPECT): long-term efficacy and safety update’ was published in the Journal of Clinical Oncology and is free to access at: [https://](https://)[ ](https://)[ascopubs.org/doi/10.1200/JCO.23.02266](ascopubs.org/doi/10.1200/JCO.23.02266) 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. How to say (download PDF and double click sound icon to play sound) ... • Epithelioid: ep-UH-thee-lee-oid • mTOR inhibitor: em-TORE in-HIB-i-ter • nab‑Sirolimus: nab-see-ROH-lim-us • PEComa: peh-KOH-meh • Perivascular: pair-ee-VASS-kyuh-luhr • Retroperitoneum: ret-roh-perr-uh-tuh-NEE-uhm 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. 10. 1080/14796694 .2025.2574811 © 2025 The Author(s) . Published by Informa UK Limited, trading as Taylor & Francis Group This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License ([http://creativecommons.org/licenses/by-nc-nd/4.0/](http://creativecommons.org/licenses/by-nc-nd/4.0/)), which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly cited, and is not altered, transformed, or built upon in any way. The terms on which this article has been published allow the posting of the Accepted Manuscript in a repository by the author(s) or with their consent. For reprints and re-use requests, please contact: [t&freprints@tandf.co.uk](t&freprints@tandf.co.uk) ISSN 1744-8301 Future Oncol. (2026) 22(1), 1–13 1 Plain Language Summary of Publication Wagner, Ravi, Riedel and co-authors nab‑Sirolimus: A medicine that is used to treat adults with an aggressive type of perivascular epithelioid cell tumors (malignant PEComa) that either: • Cannot be removed by surgery (also known a

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What is the AMPECT study and what treatment did it test?

AMPECT is a clinical study of nab-sirolimus in advanced malignant perivascular epithelioid cell tumors (malignant PEComa). The goal was to see whether nab-sirolimus could shrink tumors or stop growth and spread, while also evaluating side effects.

What results did the study report for patients taking nab-sirolimus?

Tumor shrinkage occurred in 39% of treated patients, and 52% had stable disease. Responses continued without tumor growth or spread for a median of about 40 months.

How were side effects handled in the study?

Clinicians considered side effects manageable by adjusting the dose of nab-sirolimus or by treating the side effects directly.

Sep 29, 2026

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