A retrospective study of patients with malignant PEComa receiving treatment with sirolimus or temsirolimus: the Royal Marsden Hospital experience.

Abstract

UNLABELLED:

Perivascular epithelioid cell tumors (PEComas) are rare tumors driven by tuberous sclerosis complex gene mutations causing up-regulation of mTOR. We report the outcome of ten consecutive patients treated with sirolimus or temsirolimus.

PATIENTS AND METHODS:

A retrospective analysis was performed on patients seen between 2007 and 2013. Demographic and treatment data were collected and radiological response was assessed.

RESULTS:

Ten patients were investigated, eight females, with median age of 47.5 years. Nine patients received sirolimus, one temsirolimus. The median treatment duration was 128 (range=7-1,366 days). Temsirolimus was given at 25 mg IV weekly and median starting dose of sirolimus was 3 mg daily. Seven patients were evaluable for response by RECIST: 5 showed partial response (50%), 1 stable disease (10%) and 1 progressive disease (10%). Two patients progressed rapidly on treatment. One patient stopped due to grade 3 hyperlipidaemia although CT scan shows maintained response. Three patients continue on treatment while the remainder stopped due to disease progression.

CONCLUSION:

Our study confirms that mTOR inhibition with sirolimus/temsirolimus is well-tolerated with good radiological responses, albeit short-lived, and supports its use in PEComas.

• Authors: Charlotte Benson, Joanna Vitfell-Rasmussen, Marco Maruzzo, Cyril Fisher, Nina Tunariu, Scott Mitchell, Omar Al-Muderis, Khin Thway, James Larkin, Ian Judson
• Journal: Anticancer research (Anticancer Res) Vol. 34 Issue 7 Pg. 3663-8 (Jul 2014) ISSN: 1791-7530 [Electronic] Greece
• PMID: 24982384 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: Copyright© 2014 International Institute of Anticancer Research (Dr. John G. Delinassios), All rights reserved.

Chemical References

• Antibiotics, Antineoplastic
• Antineoplastic Agents
• temsirolimus
• Sirolimus

Topics

• Antibiotics, Antineoplastic (therapeutic use)
• Antineoplastic Agents (therapeutic use)
• Female
• Humans
• Male
• Middle Aged
• Perivascular Epithelioid Cell Neoplasms (drug therapy)
• Retrospective Studies
• Sirolimus (analogs & derivatives, therapeutic use)
• Treatment Outcome