title: A retrospective study of patients with malignant PEComa receiving treatment with sirolimus or temsirolimus: the Royal Marsden Hospital experience.
abstract: 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.
  
  
  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.
  
  
  CONCLUSIONS
  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: Sarcoma Unit, The Royal Marsden NHS Foundation Trust, London, UK Charlotte.Benson@rmh.nhs.uk.
authors: Charlotte
authors: C
authors: Benson
authors: Oncology Department, Herlev Hospital, Herlev, Denmark.
authors: Joanna
authors: J
authors: Vitfell-Rasmussen
authors: Sarcoma Unit, The Royal Marsden NHS Foundation Trust, London, UK.
authors: Marco
authors: M
authors: Maruzzo
authors: Pathology Department, The Royal Marsden NHS Foundation Trust, London, UK.
authors: Cyril
authors: C
authors: Fisher
authors: Radiology Department, The Royal Marsden NHS Foundation Trust, London, UK.
authors: Nina
authors: N
authors: Tunariu
authors: Sarcoma Unit, The Royal Marsden NHS Foundation Trust, London, UK.
authors: Scott
authors: S
authors: Mitchell
authors: Sarcoma Unit, The Royal Marsden NHS Foundation Trust, London, UK.
authors: Omar
authors: O
authors: Al-Muderis
authors: Pathology Department, The Royal Marsden NHS Foundation Trust, London, UK.
authors: Khin
authors: K
authors: Thway
authors: Renal and Melanoma Unit, The Royal Marsden NHS Foundation Trust, London, UK.
authors: James
authors: J
authors: Larkin
authors: Sarcoma Unit, The Royal Marsden NHS Foundation Trust, London, UK.
authors: Ian
authors: I
authors: Judson
mesh_terms: D000903:Antibiotics, Antineoplastic / Q000627:therapeutic use*; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D054973:Perivascular Epithelioid Cell Neoplasms / Q000188:drug therapy*; D012189:Retrospective Studies; D020123:Sirolimus / Q000031:analogs & derivatives* / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D000903:Antibiotics, Antineoplastic; D000970:Antineoplastic Agents; C401859:temsirolimus; D020123:Sirolimus
keywords: PEComa; mTOR inhibitor; sirolimus; temsirolimus