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Efficacy of Sirolimus Treatment in PEComa-10 Years of Practice Perspective
Tomasz Świtaj1, Aleksandra Sobiborowicz1,2, Paweł Teterycz1,3
1Department of Soft Tissue/Bone, Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, 02-781 Warsaw, Poland.
Abstract:
Perivascular epithelioid cell tumors (PEComa) represent a family of rare mesenchymal tumors resultant from deregulation in mTOR pathway activity. The aim of this study is to evaluate the long-term efficacy of targeted PEComa treatment. We reviewed all consecutive patients with PEComa who started systemic treatment with sirolimus in our reference sarcoma center between January 2011 and August 2020. Histopathology of PEComa was reviewed and confirmed in all cases by a designated sarcoma pathologist. Any surviving progression-free patients were censored at the last follow-up (31 March 2021). Survival curves were calculated according to Kaplan-Meier method and compared with the log-rank test or a Cox proportional hazard model. Fifteen (12 females and 3 males) consecutive PEComa patients were treated. The median age of patients treated systemically was 50 years. Median progression-free survival (PFS) was 4.9 months (95% CI: 3.8-NA) for first-line chemotherapy and was not reached (95% CI: 42.0-NA) for sirolimus as first-line therapy. There was one objective response (OR) in the chemotherapy group. The OR rate reached 73% (11/15 cases) for sirolimus regardless of the treatment line. All patients archived disease control. Three patients died due to disease progression after 55, 32, and 32 months since metastatic disease diagnosis. After a median follow-up of 55.7 (range: 3.2-220) months, the 5 yr OS was 65% (CI 95% 39-100). Our study is the largest single-institution report on PEComa systemic targeted therapy and fills the gap in the field of advanced PEComa care since the FDA/EMEA approval of sirolimus.
Insights
Sirolimus demonstrates superior efficacy in treating perivascular epithelioid cell tumors (PEComa), significantly improving progression-free survival and objective response rates compared to chemotherapy. This study highlights sirolimus as a key targeted therapy for advanced PEComa.
Area of Science:
- Oncology
- Medical Science
- Sarcoma Research
Background:
- Perivascular epithelioid cell tumors (PEComa) are rare mesenchymal neoplasms linked to mTOR pathway dysregulation.
- Limited data exists on the long-term efficacy of systemic therapies for advanced PEComa.
- Sirolimus, an mTOR inhibitor, has shown promise for PEComa treatment.
Purpose of the Study:
- To evaluate the long-term efficacy and safety of sirolimus as a targeted systemic therapy for PEComa.
- To compare sirolimus treatment outcomes with traditional chemotherapy in PEComa patients.
Main Methods:
- Retrospective review of 15 consecutive PEComa patients treated with sirolimus at a reference sarcoma center (Jan 2011 - Aug 2020).
- Histopathology confirmation by a sarcoma pathologist.
- Survival analysis using Kaplan-Meier method, log-rank test, and Cox proportional hazard model; progression-free survival (PFS) and overall survival (OS) were assessed.
Main Results:
- Sirolimus as first-line therapy showed a median PFS not reached (95% CI: 42.0-NA), contrasting with 4.9 months for chemotherapy.
- Objective response rate (ORR) for sirolimus was 73% (11/15), with all patients achieving disease control.
- Five-year OS was 65% (CI 95% 39-100) after a median follow-up of 55.7 months.
Conclusions:
- Sirolimus is an effective targeted therapy for PEComa, offering significant improvements in PFS and ORR compared to chemotherapy.
- This study provides the largest single-institution evidence supporting sirolimus for advanced PEComa, informing clinical practice.
- Sirolimus represents a valuable therapeutic option for patients with advanced PEComa, addressing a critical unmet need.
