A Retrospective Study of Patients with Malignant PEComa Receiving Treatment with Sirolimus or Temsirolimus: The Royal Marsden Hospital Experience

被引:2
|
作者
Benson, Charlotte [1 ]
Vitfell-Rasmussen, Joanna [5 ]
Maruzzo, Marco [1 ]
Fisher, Cyril [2 ]
Tunariu, Nina [3 ]
Mitchell, Scott [1 ]
Al-Muderis, Omar [1 ]
Thway, Khin [2 ]
Larkin, James [4 ]
Judson, Ian [1 ]
机构
[1] Royal Marsden NHS Fdn Trust, Sarcoma Unit, London SW3 6JJ, England
[2] Royal Marsden NHS Fdn Trust, Dept Pathol, London SW3 6JJ, England
[3] Royal Marsden NHS Fdn Trust, Dept Radiol, London SW3 6JJ, England
[4] Royal Marsden NHS Fdn Trust, Renal & Melanoma Unit, London SW3 6JJ, England
[5] Herlev Hosp, Dept Oncol, DK-2730 Herlev, Denmark
关键词
PEComa; mTOR inhibitor; sirolimus; temsirolimus; EPITHELIOID CELL TUMORS; MTOR INHIBITION; ACTIVATION; RAPAMYCIN;
D O I
暂无
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
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.
引用
收藏
页码:3663 / 3668
页数:6
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