Charlotte Benson1, Joanna Vitfell-Rasmussen2, Marco Maruzzo3, Cyril Fisher4, Nina Tunariu5, Scott Mitchell3, Omar Al-Muderis3, Khin Thway4, James Larkin6, Ian Judson3. 1. Sarcoma Unit, The Royal Marsden NHS Foundation Trust, London, UK Charlotte.Benson@rmh.nhs.uk. 2. Oncology Department, Herlev Hospital, Herlev, Denmark. 3. Sarcoma Unit, The Royal Marsden NHS Foundation Trust, London, UK. 4. Pathology Department, The Royal Marsden NHS Foundation Trust, London, UK. 5. Radiology Department, The Royal Marsden NHS Foundation Trust, London, UK. 6. Renal and Melanoma Unit, The Royal Marsden NHS Foundation Trust, London, UK.
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. Copyright
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. Copyright
Authors: Andrew J Wagner; Vinod Ravi; Richard F Riedel; Kristen Ganjoo; Brian A Van Tine; Rashmi Chugh; Lee Cranmer; Erlinda M Gordon; Jason L Hornick; Heng Du; Berta Grigorian; Anita N Schmid; Shihe Hou; Katherine Harris; David J Kwiatkowski; Neil P Desai; Mark A Dickson Journal: J Clin Oncol Date: 2021-10-12 Impact factor: 50.717