Efficacy of Sirolimus Treatment in PEComa–10 Years of Practice Perspective
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 sys...
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MDPI AG
2021-08-01
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| Series: | Journal of Clinical Medicine |
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| Online Access: | https://www.mdpi.com/2077-0383/10/16/3705 |
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| author | Tomasz Świtaj Aleksandra Sobiborowicz Paweł Teterycz Anna Klimczak Donata Makuła Michał Wągrodzki Anna Szumera-Ciećkiewicz Piotr Rutkowski Anna M. Czarnecka |
| author_facet | Tomasz Świtaj Aleksandra Sobiborowicz Paweł Teterycz Anna Klimczak Donata Makuła Michał Wągrodzki Anna Szumera-Ciećkiewicz Piotr Rutkowski Anna M. Czarnecka |
| author_sort | Tomasz Świtaj |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-10T08:42:07Z |
| format | Article |
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| institution | Directory Open Access Journal |
| issn | 2077-0383 |
| language | English |
| last_indexed | 2024-03-10T08:42:07Z |
| publishDate | 2021-08-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Clinical Medicine |
| spelling | doaj.art-8cefaa9ce1f24307a2d642510aae42632023-11-22T08:12:23ZengMDPI AGJournal of Clinical Medicine2077-03832021-08-011016370510.3390/jcm10163705Efficacy of Sirolimus Treatment in PEComa–10 Years of Practice PerspectiveTomasz Świtaj0Aleksandra Sobiborowicz1Paweł Teterycz2Anna Klimczak3Donata Makuła4Michał Wągrodzki5Anna Szumera-Ciećkiewicz6Piotr Rutkowski7Anna M. Czarnecka8Department of Soft Tissue/Bone, Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, 02-781 Warsaw, PolandDepartment of Soft Tissue/Bone, Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, 02-781 Warsaw, PolandDepartment of Soft Tissue/Bone, Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, 02-781 Warsaw, PolandDepartment of Soft Tissue/Bone, Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, 02-781 Warsaw, PolandDepartment of Radiology I, Maria Sklodowska-Curie National Research Institute of Oncology, 02-781 Warsaw, PolandDepartment of Pathology and Laboratory Diagnostics, Maria Skłodowska-Curie Institute-Oncology Center, 02-781 Warsaw, PolandDepartment of Pathology and Laboratory Diagnostics, Maria Skłodowska-Curie Institute-Oncology Center, 02-781 Warsaw, PolandDepartment of Soft Tissue/Bone, Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, 02-781 Warsaw, PolandDepartment of Soft Tissue/Bone, Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, 02-781 Warsaw, PolandPerivascular 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.https://www.mdpi.com/2077-0383/10/16/3705perivascular epithelioid cell tumorPEComalymphangioleiomyomatosissarcomasirolimusmTOR inhibitors |
| spellingShingle | Tomasz Świtaj Aleksandra Sobiborowicz Paweł Teterycz Anna Klimczak Donata Makuła Michał Wągrodzki Anna Szumera-Ciećkiewicz Piotr Rutkowski Anna M. Czarnecka Efficacy of Sirolimus Treatment in PEComa–10 Years of Practice Perspective Journal of Clinical Medicine perivascular epithelioid cell tumor PEComa lymphangioleiomyomatosis sarcoma sirolimus mTOR inhibitors |
| title | Efficacy of Sirolimus Treatment in PEComa–10 Years of Practice Perspective |
| title_full | Efficacy of Sirolimus Treatment in PEComa–10 Years of Practice Perspective |
| title_fullStr | Efficacy of Sirolimus Treatment in PEComa–10 Years of Practice Perspective |
| title_full_unstemmed | Efficacy of Sirolimus Treatment in PEComa–10 Years of Practice Perspective |
| title_short | Efficacy of Sirolimus Treatment in PEComa–10 Years of Practice Perspective |
| title_sort | efficacy of sirolimus treatment in pecoma 10 years of practice perspective |
| topic | perivascular epithelioid cell tumor PEComa lymphangioleiomyomatosis sarcoma sirolimus mTOR inhibitors |
| url | https://www.mdpi.com/2077-0383/10/16/3705 |
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