First-line pazopanib in patients with metastatic epithelioid hemangioendothelioma: a retrospective single-center analysis
Abstract Purpose Epithelioid hemangioendothelioma (EHE) represents an ultra-rare, translocated vascular sarcoma with a heterogeneous course of disease. The optimal systemic treatment for patients with advanced EHE remains unclear. We sought to evaluate the value of pazopanib (PAZ) as a first-line tr...
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2025-04-01
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| Series: | Journal of Cancer Research and Clinical Oncology |
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| Online Access: | https://doi.org/10.1007/s00432-025-06208-8 |
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| author | Anton Burkhard-Meier Vera Valerie Rechenauer Vindi Jurinovic Markus Albertsmeier Michael Hoberger Hans Roland Dürr Alexander Klein Thomas Knösel Wolfgang G. Kunz Andreas Mock Ada Pusztai Michael Völkl Michael von Bergwelt-Baildon Lars H. Lindner Dorit Di Gioia Luc M. Berclaz |
| author_facet | Anton Burkhard-Meier Vera Valerie Rechenauer Vindi Jurinovic Markus Albertsmeier Michael Hoberger Hans Roland Dürr Alexander Klein Thomas Knösel Wolfgang G. Kunz Andreas Mock Ada Pusztai Michael Völkl Michael von Bergwelt-Baildon Lars H. Lindner Dorit Di Gioia Luc M. Berclaz |
| author_sort | Anton Burkhard-Meier |
| collection | DOAJ |
| description | Abstract Purpose Epithelioid hemangioendothelioma (EHE) represents an ultra-rare, translocated vascular sarcoma with a heterogeneous course of disease. The optimal systemic treatment for patients with advanced EHE remains unclear. We sought to evaluate the value of pazopanib (PAZ) as a first-line treatment in metastatic EHE. Methods Thirteen patients with metastatic EHE and PAZ as a first-line treatment at our institution between 2012 und 2023 were reviewed and analyzed with regard to clinical outcomes. Results At a median follow-up of 51.4 months, the median progression-free survival (PFS) and overall survival (OS) were 35.1 and 53.8 months, respectively. In patients with documented prior tumor progression (n = 10), the median PFS and OS were 12.6 and 105 months, respectively. In patients with serosal effusion/ systemic symptoms (n = 4), the median PFS and OS were 6.1 and 10.3 months. The clinical benefit rate of the overall cohort was 62% with no complete or partial responses. Two of four patients experienced a reduction of symptoms (pain and ascites reduction/hemoptysis, respectively) under treatment with PAZ. Toxicity was mainly gastrointestinal and manageable with dose reductions. Permanent treatment interruption due to toxicity was necessary in one patient. Conclusion This is the first study to systematically report survival outcomes for PAZ as a first-line treatment in patients with metastatic EHE. PAZ is active and safe in patients with metastatic EHE and may be considered as an alternative to sirolimus for specific patient subgroups. RECIST criteria should be questioned for evaluation of treatment response in EHE. |
| format | Article |
| id | doaj-art-e6fc0e644a58413ead71cd807701d601 |
| institution | OA Journals |
| issn | 1432-1335 |
| language | English |
| publishDate | 2025-04-01 |
| publisher | Springer |
| record_format | Article |
| series | Journal of Cancer Research and Clinical Oncology |
| spelling | doaj-art-e6fc0e644a58413ead71cd807701d6012025-08-20T02:32:05ZengSpringerJournal of Cancer Research and Clinical Oncology1432-13352025-04-0115141810.1007/s00432-025-06208-8First-line pazopanib in patients with metastatic epithelioid hemangioendothelioma: a retrospective single-center analysisAnton Burkhard-Meier0Vera Valerie Rechenauer1Vindi Jurinovic2Markus Albertsmeier3Michael Hoberger4Hans Roland Dürr5Alexander Klein6Thomas Knösel7Wolfgang G. Kunz8Andreas Mock9Ada Pusztai10Michael Völkl11Michael von Bergwelt-Baildon12Lars H. Lindner13Dorit Di Gioia14Luc M. Berclaz15Department of Medicine III, University Hospital, LMU MunichDepartment of Medicine III, University Hospital, LMU MunichDepartment of Medicine III, University Hospital, LMU MunichBavarian Cancer Research Center (BZKF)Department of Medicine III, University Hospital, LMU MunichOrthopedic Oncology, Department of Orthopedics and Trauma Surgery, University Hospital, LMU MunichOrthopedic Oncology, Department of Orthopedics and Trauma Surgery, University Hospital, LMU MunichInstitute of Pathology, LMU MunichBavarian Cancer Research Center (BZKF)Institute of Pathology, LMU MunichInstitute of Pathology, LMU MunichDepartment of Medicine III, University Hospital, LMU MunichDepartment of Medicine III, University Hospital, LMU MunichDepartment of Medicine III, University Hospital, LMU MunichDepartment of Medicine III, University Hospital, LMU MunichDepartment of Medicine III, University Hospital, LMU MunichAbstract Purpose Epithelioid hemangioendothelioma (EHE) represents an ultra-rare, translocated vascular sarcoma with a heterogeneous course of disease. The optimal systemic treatment for patients with advanced EHE remains unclear. We sought to evaluate the value of pazopanib (PAZ) as a first-line treatment in metastatic EHE. Methods Thirteen patients with metastatic EHE and PAZ as a first-line treatment at our institution between 2012 und 2023 were reviewed and analyzed with regard to clinical outcomes. Results At a median follow-up of 51.4 months, the median progression-free survival (PFS) and overall survival (OS) were 35.1 and 53.8 months, respectively. In patients with documented prior tumor progression (n = 10), the median PFS and OS were 12.6 and 105 months, respectively. In patients with serosal effusion/ systemic symptoms (n = 4), the median PFS and OS were 6.1 and 10.3 months. The clinical benefit rate of the overall cohort was 62% with no complete or partial responses. Two of four patients experienced a reduction of symptoms (pain and ascites reduction/hemoptysis, respectively) under treatment with PAZ. Toxicity was mainly gastrointestinal and manageable with dose reductions. Permanent treatment interruption due to toxicity was necessary in one patient. Conclusion This is the first study to systematically report survival outcomes for PAZ as a first-line treatment in patients with metastatic EHE. PAZ is active and safe in patients with metastatic EHE and may be considered as an alternative to sirolimus for specific patient subgroups. RECIST criteria should be questioned for evaluation of treatment response in EHE.https://doi.org/10.1007/s00432-025-06208-8Epithelioid hemangioendotheliomaRare cancersMetastasisPazopanibTargeted therapy |
| spellingShingle | Anton Burkhard-Meier Vera Valerie Rechenauer Vindi Jurinovic Markus Albertsmeier Michael Hoberger Hans Roland Dürr Alexander Klein Thomas Knösel Wolfgang G. Kunz Andreas Mock Ada Pusztai Michael Völkl Michael von Bergwelt-Baildon Lars H. Lindner Dorit Di Gioia Luc M. Berclaz First-line pazopanib in patients with metastatic epithelioid hemangioendothelioma: a retrospective single-center analysis Journal of Cancer Research and Clinical Oncology Epithelioid hemangioendothelioma Rare cancers Metastasis Pazopanib Targeted therapy |
| title | First-line pazopanib in patients with metastatic epithelioid hemangioendothelioma: a retrospective single-center analysis |
| title_full | First-line pazopanib in patients with metastatic epithelioid hemangioendothelioma: a retrospective single-center analysis |
| title_fullStr | First-line pazopanib in patients with metastatic epithelioid hemangioendothelioma: a retrospective single-center analysis |
| title_full_unstemmed | First-line pazopanib in patients with metastatic epithelioid hemangioendothelioma: a retrospective single-center analysis |
| title_short | First-line pazopanib in patients with metastatic epithelioid hemangioendothelioma: a retrospective single-center analysis |
| title_sort | first line pazopanib in patients with metastatic epithelioid hemangioendothelioma a retrospective single center analysis |
| topic | Epithelioid hemangioendothelioma Rare cancers Metastasis Pazopanib Targeted therapy |
| url | https://doi.org/10.1007/s00432-025-06208-8 |
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