1450P - Systemic therapy in perivascular epithelioid cell tumors (PEComa)

Date 29 September 2014
Event ESMO 2014
Session Poster Display session
Topics Anticancer Agents
Soft Tissue Sarcomas
Biological Therapy
Presenter Nadia Hindi
Citation Annals of Oncology (2014) 25 (suppl_4): iv494-iv510. 10.1093/annonc/mdu354
Authors N. Hindi1, R. Sanfilippo2, S. Stacchiotti1, E. Fumagalli1, M. Libertini1, S. Provenzano3, E. Palassini1, R.M. Bertulli1, A. Marrari1, L. Galli4, V. Formica5, V.E. Chiuri6, D. Natale7, P. Collini8, A.P. Dei Tos9, P.G. Casali1
  • 1Sarcoma Unit, Fondazione IRCCS - Istituto Nazionale dei Tumori, 20133 - Milano/IT
  • 2Cancer Medicine Department, Istituto Nazionale dei Tumori, 20133 - Milano/IT
  • 3Oncology, Fondazione IRCCS Istituto Nazionale Tumori, Milan, 20133 - Milano/IT
  • 4Azienda Ospedaliero-universitaria Pisana, Istituto Toscano Tumori, Division of Medical Oncology, 55100 - Pisa/IT
  • 5Medical Oncology Unit, Internal Medicine Department, 'Tor Vergata' University Hospital, 00133 - Rome/IT
  • 6Medical Oncology Unit, Ospedale Vito Fazzi, IT-73100 - Lecce/IT
  • 7Oncology, Ospedale Civile, Pescara/IT
  • 8Anatomic Pathology, Fondazione IRCCS - Istituto Nazionale dei Tumori, Milan/IT
  • 9Pathology, General Hospital of Treviso, IT-31100 - Treviso/IT



Perivascular epithelioid cell tumors (PEComa) are a family of rare mesenchymal neoplasms with a variable biological behaviour. Surgical resection is the mainstay of therapy. However, approximately one third of patients have an advanced disease d'emblée or develop metastases later on. The role of chemotherapy is controversial, with anecdotal responses reported including some with mTOR inhibitors.


Cases diagnosed with PEComa family tumors from January 2002 at Istituto Nazionale Tumori, Milan, or within the Italian Rare Cancer Network (RTR) were reviewed.


We identified 49 pts with PEComa. Nineteen pts (median age: 54.8 years; F/M= 8/11) received systemic therapy for advance disease (1-7 lines). Site of primary tumor was soft-tissues in 6/19pts (32%), retroperitoneum in 5/19pts (26%), kidney in 4/19pts (21%). Site of metastatic disease was lung (6/19pts), liver (6/19pts), peritoneum (3/19pts), bone (2/19pts) and lymph nodes (2/19pts). Eight pts received gemcitabine-based chemotherapy and were evaluable for response: 2 had a PR (25%) and 6 a PD (75%); median PFS was 2.8 mos (0.9-5). Nine pts received an antracyclin-based combination and were evaluable for response: 3 had SD (33%) and 6 a PD (66%); median PFS was 1.5 mos (0.7-3.3). Twelve pts received a mTOR inhibitor and were evaluable for response: 4 had a PR (33%), 5 a SD (41%) and 3 a PD (25%); median PFS was 4.5 mos (1.5-14). Four patients received an antiangiogenic therapy (pazopanib or sunitinib), obtaining 1 PR and 2 SD, with a median PFS 7.3 mos (0.9-9.6).


In this series of advanced PEComa patients treated with medical therapy, chemotherapy seems to have played a limited role. However, chemotherapy with gemcitabine-based combinations allowed some objective responses, although their duration was short. We confirm the activity of mTOR inhibitors, again with a short duration.


N. Hindi: Clinical Research grant from the Spanish Society of Medical Oncology; S. Stacchiotti: Silvia Stacchiotti has received research funds from Glaxo and Pfizer; P.G. Casali: Consultant / Advisory:Amgen, ARIAD,Bayer GSK,Merck SD,Novartis,Pfizer,PharmaMar, Sanofi-Aventis. Research funds:Amgen,Bayer,GSK ImClone,Infinity,Janssen Cilag,Lilly,Merck Molmed,Novartis,Pfizer,PharmaMar,Sanofi-Aventis Schering Plough. All other authors have declared no conflicts of interest.