Neoadjuvant sirolimus for a large hepatic perivascular epithelioid cell tumor (PEComa)

World J Surg Oncol. 2014 Feb 27:12:46. doi: 10.1186/1477-7819-12-46.

Abstract

Perivascular epithelioid cell tumors (PEComas) are rare soft-tissue tumors with an extremely heterogeneous clinical behavior. They may arise in different organs and may behave indolently or sometimes metastasize with different grades of biological aggressiveness. We report the case of a young woman with a primary inoperable PEComa of the liver with malignant histological features. Since the mTOR pathway is often altered in PEComas and responses have been reported with mTOR-inhibitors such as sirolimus or temsirolimus, we decided to start a neoadjuvant treatment with sirolimus. The patient tolerated the treatment fairly well and after 8 months a favorable tumor shrinkage was obtained. The patient then stopped sirolimus and 2 weeks later underwent partial liver resection, with complete clinical recovery and normal liver function. The histological report confirmed a malignant PEComa with vascular invasion and negative margins. Then 6 additional months of post-operative sirolimus treatment were administered, followed by regular radiological follow-up. For patients with a large and histologically aggressive PEComa, we think that neoadjuvant treatment with mTOR-inhibitor sirolimus may be considered to facilitate surgery and allow early control of a potentially metastatic disease. For selected high-risk patients, the option of adjuvant treatment may be discussed.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Combined Modality Therapy
  • Female
  • Hepatectomy
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Liver Neoplasms / drug therapy*
  • Liver Neoplasms / pathology
  • Liver Neoplasms / surgery
  • Neoadjuvant Therapy*
  • Perivascular Epithelioid Cell Neoplasms / drug therapy*
  • Perivascular Epithelioid Cell Neoplasms / pathology
  • Perivascular Epithelioid Cell Neoplasms / surgery
  • Prognosis
  • Sirolimus / therapeutic use*

Substances

  • Immunosuppressive Agents
  • Sirolimus