{
    "abstract": "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.\nThirteen 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.\nAt 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\u2009=\u200910), the median PFS and OS were 12.6 and 105 months, respectively. In patients with serosal effusion/ systemic symptoms (n\u2009=\u20094), 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.\nThis 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.",
    "authors": [
        {
            "affiliation": "Department of Medicine III, University Hospital, LMU Munich, Munich, Germany. anton.burkhardmeier@med.uni-muenchen.de.\nBavarian Cancer Research Center (BZKF), Munich, Germany. anton.burkhardmeier@med.uni-muenchen.de.\nDepartment of Medicine III, LMU University Hospital, Marchioninistr. 15, 81377, Munich, Germany. anton.burkhardmeier@med.uni-muenchen.de.",
            "firstname": "Anton",
            "initials": "A",
            "lastname": "Burkhard-Meier"
        },
        {
            "affiliation": "Department of Medicine III, University Hospital, LMU Munich, Munich, Germany.",
            "firstname": "Vera Valerie",
            "initials": "VV",
            "lastname": "Rechenauer"
        },
        {
            "affiliation": "Department of Medicine III, University Hospital, LMU Munich, Munich, Germany.\nInstitute for Medical Information Processing, Biometry, and Epidemiology, University Hospital, LMU Munich, Munich, Germany.",
            "firstname": "Vindi",
            "initials": "V",
            "lastname": "Jurinovic"
        },
        {
            "affiliation": "Bavarian Cancer Research Center (BZKF), Munich, Germany.\nDepartment of General, Visceral and Transplantation Surgery, University Hospital, LMU Munich, Munich, Germany.",
            "firstname": "Markus",
            "initials": "M",
            "lastname": "Albertsmeier"
        },
        {
            "affiliation": "Department of Medicine III, University Hospital, LMU Munich, Munich, Germany.",
            "firstname": "Michael",
            "initials": "M",
            "lastname": "Hoberger"
        },
        {
            "affiliation": "Orthopedic Oncology, Department of Orthopedics and Trauma Surgery, University Hospital, LMU Munich, Munich, Germany.",
            "firstname": "Hans Roland",
            "initials": "HR",
            "lastname": "D\u00fcrr"
        },
        {
            "affiliation": "Orthopedic Oncology, Department of Orthopedics and Trauma Surgery, University Hospital, LMU Munich, Munich, Germany.",
            "firstname": "Alexander",
            "initials": "A",
            "lastname": "Klein"
        },
        {
            "affiliation": "Institute of Pathology, LMU Munich, Munich, Germany.",
            "firstname": "Thomas",
            "initials": "T",
            "lastname": "Kn\u00f6sel"
        },
        {
            "affiliation": "Bavarian Cancer Research Center (BZKF), Munich, Germany.\nDepartment of Radiology, University Hospital, LMU Munich, Munich, Germany.",
            "firstname": "Wolfgang G",
            "initials": "WG",
            "lastname": "Kunz"
        },
        {
            "affiliation": "Institute of Pathology, LMU Munich, Munich, Germany.",
            "firstname": "Andreas",
            "initials": "A",
            "lastname": "Mock"
        },
        {
            "affiliation": "Institute of Pathology, LMU Munich, Munich, Germany.",
            "firstname": "Ada",
            "initials": "A",
            "lastname": "Pusztai"
        },
        {
            "affiliation": "Department of Medicine III, University Hospital, LMU Munich, Munich, Germany.",
            "firstname": "Michael",
            "initials": "M",
            "lastname": "V\u00f6lkl"
        },
        {
            "affiliation": "Department of Medicine III, University Hospital, LMU Munich, Munich, Germany.\nBavarian Cancer Research Center (BZKF), Munich, Germany.\nGerman Cancer Consortium (DKTK), Partner Site Munich, Munich, Germany.",
            "firstname": "Michael",
            "initials": "M",
            "lastname": "von Bergwelt-Baildon"
        },
        {
            "affiliation": "Department of Medicine III, University Hospital, LMU Munich, Munich, Germany.\nBavarian Cancer Research Center (BZKF), Munich, Germany.",
            "firstname": "Lars H",
            "initials": "LH",
            "lastname": "Lindner"
        },
        {
            "affiliation": "Department of Medicine III, University Hospital, LMU Munich, Munich, Germany.",
            "firstname": "Dorit",
            "initials": "D",
            "lastname": "Di Gioia"
        },
        {
            "affiliation": "Department of Medicine III, University Hospital, LMU Munich, Munich, Germany.\nGerman Cancer Consortium (DKTK), Partner Site Munich, Munich, Germany.",
            "firstname": "Luc M",
            "initials": "LM",
            "lastname": "Berclaz"
        }
    ],
    "conclusions": "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.",
    "copyrights": "\u00a9 2025. The Author(s).",
    "doi": "10.1007/s00432-025-06208-8",
    "journal": "Journal of cancer research and clinical oncology",
    "keywords": [
        "Epithelioid hemangioendothelioma",
        "Metastasis",
        "Pazopanib",
        "Rare cancers",
        "Targeted therapy"
    ],
    "methods": null,
    "publication_date": "2025-04-26",
    "pubmed_id": "40285941\n22910841\n33179614\n23737213\n37625241\n23589078\n21584898\n33713582\n32840417\n27838944\n21546438\n31123606\n18043096\n32271190\n31537895\n25992243\n33179614\n38446990\n27334221\n34090171\n33107985\n22595799\n39141513",
    "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\u2009=\u200910), the median PFS and OS were 12.6 and 105 months, respectively. In patients with serosal effusion/ systemic symptoms (n\u2009=\u20094), 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.",
    "title": "First-line pazopanib in patients with metastatic epithelioid hemangioendothelioma: a retrospective single-center analysis.",
    "xml": "<Element 'PubmedArticle' at 0x788504c771a0>"
}