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Poster Discussion session - Sarcoma

5047 - mTOR inhibitors in uterine and extra-uterine malignant PEComas: a multicenter international case series retrospective analysis.


22 Oct 2018


Poster Discussion session - Sarcoma


Cytotoxic Therapy

Tumour Site



Roberta Sanfilippo


Annals of Oncology (2018) 29 (suppl_8): viii576-viii595. 10.1093/annonc/mdy299


R. Sanfilippo1, R.L. Jones2, S. Provenzano3, G. Antoniou4, J. Blay5, E. Fumagalli3, S. Stacchiotti6, R.M. Bertulli3, O. Mir7, N. Hindi8, M. Brahmi9, A. Dufresne10, A..P. Dei Tos11, P.G. Casali12

Author affiliations

  • 1 Oncologia Medica, Istituto Nazionale dei Tumori di Milano - Fondazione IRCCS, 20133 - Milan/IT
  • 2 Medical Oncology, Royal Marsden Hospital NHS Foundation Trust, SW3 6JJ - London/GB
  • 3 Oncologia Medica, Fondazione IRCCS - Istituto Nazionale dei Tumori, 20133 - Milan/IT
  • 4 Sarcoma Unit, Royal Marsden Hospital, SW3 6JJ - London/GB
  • 5 Medicine, Centre Léon Bérard, 69008 - Lyon/FR
  • 6 Adult Mesenchymal Tumor Medical Oncology Unit, Cancer Medicine Dpt, Istituto Nazionale dei Tumori di Milano - Fondazione IRCCS, 20133 - Milan/IT
  • 7 Cancer Medicine, Institut Gustave Roussy, 94800 - Villejuif/FR
  • 8 Oncology Unit, Hospital Universitario Virgen del Rocio, 41013 - Sevilla/ES
  • 9 Department Of Medical Oncology, Centre Léon Bérard, 69008 - Lyon/FR
  • 10 Medical Oncology, Centre Léon Bérard, 69008 - Lyon/FR
  • 11 Pathology, Istituto Oncologico Veneto IRCCS, 35128 - Padova/IT
  • 12 Medical Oncology Unit 2, Istituto Nazionale dei Tumori di Milano - Fondazione IRCCS, 20133 - Milan/IT


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Abstract 5047


Perivascular epithelioid cell tumors (PEComa) are exceedingly rare mesenchymal neoplasms arising from a broad variety of anatomic sites. Within the female genital tract, most frequently PEComas affect the uterus. Over the last few years, the activity of mTOR inhibitors in these neoplasms has been convincingly shown. The aim of this multicenter retrospective analysis was to compare the activity of mTOR inhibitors in uterine versus extra-uterine malignant PEComas.


We retrospectively identified pts with advanced PEComa treated with mTOR inhibitors since January 2002 at Fondazione IRCCS Istituto Nazionale dei Tumori, Milan - Italy, and within the Italian Rare Cancer Network (RTR); at the Royal Marsden Hospital, London – UK; within centers of the French and Spanish Sarcoma Groups. We stratified patients based on uterine and extra-uterine primary site and collected data on the safety and efficacy of mTOR inhibitors, including PFS and OS.


A total of 41 pts with advanced PEComa treated mTOR inhibitors were analyzed. We identified 8 (20%) pts with uterine PEcomas and 33 (80%) with extra-uterine. In the group of uterine PEComas 1 out of 8 patients had a PR (12%), and 3/8 (37%) had SD with a median PFS of 4 months. In the group of extra-uterine PEComas, 16/33 (48%) had a PR, 10/33 (30%) stable disease, with a median PFS of 14 months. Two patients out of 33 were not evaluable by RECIST.


In our retrospective series, uterine PEComas had a numerically lower response rate to mTOR inhibitor therapy compared to those with extra-uterine primary tumors. Further work is required to confirm these preliminary data and to investigate the reasons for this observation. Our study provides a benchmark for further research and suggests that uterine PEComas may need to be analysed separately with regard to anti-tumor activity of novel agents.

Clinical trial identification

Legal entity responsible for the study

R. Sanfilippo.


Has not received any funding.

Editorial Acknowledgement


All authors have declared no conflicts of interest.

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