Systemic Therapies in Advanced Epithelioid Haemangioendothelioma: A Retrospective International Case Series from the World Sarcoma Network and a Review of Literature

Overview

Journal Cancer Med

Specialty Oncology

Date 2021 Mar 13

PMID 33713582

Citations 19

Authors

Anna M Frezza

Vinod Ravi

Salvatore Lo Vullo

Bruno Vincenzi

Francesco Tolomeo

Tom Wei-Wu Chen

Pawel Teterycz

Giacomo G Baldi

Antoine Italiano

Nicolas Penel

Antonella Brunello

Florance Duffaud

Nadia Hindi

Shintaro Iwata

Alannah Smrke

Alexander Fedenko

Hans Gelderblom

Winette van der Graaf

Aurore Vozy

Elizabeth Connolly

Massimiliano Grassi

Robert S Benjamin

Javier-Martin Broto

Giovanni Grignani

Robin L Jones

Akira Kawai

Andrzej Tysarowski

Luigi Mariani

Paolo G Casali

Silvia Stacchiotti

Affiliations

Soon will be listed here.

Abstract

Background: This observational, retrospective effort across Europe, US, Australia, and Asia aimed to assess the activity of systemic therapies in EHE, an ultra-rare sarcoma, marked by WWTR1-CAMTA1 or YAP1-TFE3 fusions.

Methods: Twenty sarcoma reference centres contributed data. Patients with advanced EHE diagnosed from 2000 onwards and treated with systemic therapies, were selected. Local pathologic review and molecular confirmation were required. Radiological response was retrospectively assessed by local investigators according to RECIST. Progression free survival (PFS) and overall survival (OS) were estimated by Kaplan-Meier method.

Results: Overall, 73 patients were included; 21 had more than one treatment. Thirty-three patients received anthracyclines regimens, achieving 1 (3%) partial response (PR), 25 (76%) stable disease (SD), 7 (21%) progressive disease (PD). The median (m-) PFS and m-OS were 5.5 and 14.3 months respectively. Eleven patients received paclitaxel, achieving 1 (9%) PR, 6 (55%) SD, 4 (36%) PD. The m-PFS and m-OS were 2.9 and 18.6 months, respectively. Twelve patients received pazopanib, achieving 3 (25%) SD, 9 (75%) PD. The m-PFS and m-OS were.2.9 and 8.5 months, respectively. Fifteen patients received INF-α 2b, achieving 1 (7%) PR, 11 (73%) SD, 3 (20%) PD. The m-PFS and m-OS were 8.9 months and 64.3, respectively. Among 27 patients treated with other regimens, 1 PR (ifosfamide) and 9 SD (5 gemcitabine +docetaxel, 2 oral cyclophosphamide, 2 others) were reported.

Conclusion: Systemic therapies available for advanced sarcomas have limited activity in EHE. The identification of new active compounds, especially for rapidly progressive cases, is acutely needed.

Citing Articles

Nab-Paclitaxel Based Chemotherapy in the Treatment of Advanced Epithelioid Hemangioendothelioma: A Single-Institution Experience.

Liu X, Yang P, Liu L, Si S, Zhou R, Liu T Cancer Manag Res. 2025; 17:373-381.

PMID: 40007912 PMC: 11853918. DOI: 10.2147/CMAR.S508673.

The challenges of hepatic epithelioid hemangioendothelioma: the diagnosis and current treatments of a problematic tumor.

Atyah M, Sun Y, Yang Z Orphanet J Rare Dis. 2024; 19(1):449.

PMID: 39616351 PMC: 11608485. DOI: 10.1186/s13023-024-03354-z.

Comprehensive evaluation of clinical outcomes in hepatic epithelioid hemangioendothelioma subsets: insights from SEER Database and departmental cohort analysis.

Wang B, Chen X, Li R, Ai B, Ye F, Zhao J Front Immunol. 2024; 15:1491922.

PMID: 39502705 PMC: 11534872. DOI: 10.3389/fimmu.2024.1491922.

Long-term prognosis and treatment modalities of hepatic epithelioid hemangioendothelioma: a retrospective study of 228 patients.

Liu X, Yang P, Liu L, Si S, Zhou R, Liu T BMC Cancer. 2024; 24(1):1285.

PMID: 39415114 PMC: 11481724. DOI: 10.1186/s12885-024-13053-4.

The observational EURACAN prospective clinical registry dedicated to epithelioid hemangioendothelioma: The protocol of an international and collaborative effort on an ultra-rare entity.

Frezza A, Leonard H, Aggerholm-Pedersen N, Badalamenti G, Baili P, Baldi G PLoS One. 2024; 19(8):e0308387.

PMID: 39133752 PMC: 11318912. DOI: 10.1371/journal.pone.0308387.

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