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. 2025 Apr 26;151(4):150.
doi: 10.1007/s00432-025-06208-8.

First-line pazopanib in patients with metastatic epithelioid hemangioendothelioma: a retrospective single-center analysis

Anton Burkhard-Meier  1   2   3 , Vera Valerie Rechenauer  4 , Vindi Jurinovic  4   5 , Markus Albertsmeier  6   7 , Michael Hoberger  4 , Hans Roland Dürr  8 , Alexander Klein  8 , Thomas Knösel  9 , Wolfgang G Kunz  6   10 , Andreas Mock  9 , Ada Pusztai  9 , Michael Völkl  4 , Michael von Bergwelt-Baildon  4   6   11 , Lars H Lindner  4   6 , Dorit Di Gioia #  4 , Luc M Berclaz #  4   11
Affiliations

First-line pazopanib in patients with metastatic epithelioid hemangioendothelioma: a retrospective single-center analysis

Anton Burkhard-Meier et al. J Cancer Res Clin Oncol. .

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.

Keywords: Epithelioid hemangioendothelioma; Metastasis; Pazopanib; Rare cancers; Targeted therapy.

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Conflict of interest statement

Declarations. Ethical approval: The Internal Review Board and the Ethical Review Committee at the Ludwig Maximilians University (LMU) Hospital, Munich, Germany, approved the protocol of this research project (Protocol Nr. 23–0998). The study was performed in accordance with the ethical standards as laid down in the 1964 Declaration of Helsinki. Consent to participate: Not applicable due to the irreversibly anonymized dataset. Competing interests: The authors declare no competing interests.

Figures

Fig. 1
Fig. 1
Epithelioid hemangioendothelioma with trabecular arrangement and nests of epithelioid cells within a sclerotic matrix (HE, 20x magnification)
Fig. 2
Fig. 2
Baseline CT imaging of a patient with metastatic epithelioid hemangioendothelioma (EHE) involving liver and lungs
Fig. 3
Fig. 3
The most common toxicities in patients with epithelioid hemangioendothelioma (EHE) during treatment with pazopanib (PAZ). *Transaminases and/or gamma-glutamyl transferase (gGT) CTCAE: Common Terminology Criteria for Adverse Events
Fig. 4
Fig. 4
Progression-free survival (PFS) and overall survival (OS) in patients with epithelioid hemangioendothelioma (EHE) after start of pazopanib (PAZ)
Fig. 5
Fig. 5
Patients with metastatic epithelioid hemangioendothelioma (EHE) and pazopanib (PAZ) as a first-line treatment
See this image and copyright information in PMC

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