1627MO - Systemic therapies in advanced epithelioid haemangioendothelioma (EHE): A retrospective international series from the World Sarcoma Network

Background

This observational, retrospective effort across Europe, US and Asia aims to assess the activity of systemic therapies in EHE, a ultra rare sarcoma, marked by WWTR1-CAMTA1 or YAP1-TFE3 fusion, typically metastatic at presentation and with a unpredictable clinical course.

Methods

18 sarcoma reference centres contributed data. Patients with advanced EHE diagnosed 2000 onwords, treated with systemic therapies, were selected. Local pathological review and molecular confirmation was 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

72 patients were included (characteristics in the table), 21 had more than one treatment. 33 patients received anthracycline-based regimens, achieving 1 (3%) partial response (PR), 25 (76%) stable disease (SD, 14 with prior PD), 7 (21%) progressive disease (PD). The median (m-) PFS and m-OS were 5 and 14 months respectively. 11 patients received weekly paclitaxel, achieving 1 (9%) PR, 6 (55%, 2 with prior PD) SD, 4 (36%) PD. The m-PFS and m-OS were 3 and 19 months respectively. 12 patients received pazopanib, achieving 3 (25%, 2 with prior PD) SD, 9 (75%) PD. The m-PFS and m-OS were.3 and 9 months respectively. 15 patients received INF-α 2b, achieving 1 (7%) PR, 11 (73%, 8 with prior PD) SD, 3 (20%) PD. The m-PFS and m-OS were 9 months and unreached respectively. Among 27 patients treated with other regimens, 1 PR (ifosfamide) and 9 SD (all with prior PD, 5 gemcitabine+docetaxel, 2 cyclophosphamide, 2 others) were reported. Table: 1627MO

Population characteristics

Conclusions

Systemic therapies available for advanced sarcomas exhibited limited activity in EHE. Although PFS should be interpreted with caution as non-progressive cases were included, m-PFS with chemotherapy and pazopanib was short (<6 months). The identification of new active compounds, especially for rapidly progressive cases, is needed.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

Fondazione IRCCS Istituto Nazionale Tumori.

Funding

Has not received any funding.

Disclosure

A.M. Frezza: Research grant/Funding (institution): Amgen Dompè; Research grant/Funding (institution): Bayer; Research grant/Funding (institution): Daiichi Sankyo; Research grant/Funding (institution): Eli Lilly; Research grant/Funding (institution): Epizyme; Research grant/Funding (institution): Novartis; Research grant/Funding (institution), Travel/Accommodation/Expenses: Pharmamar; Research grant/Funding (institution): Pfizer; Research grant/Funding (institution): Eisai. G.G. Baldi: Honoraria (self), Travel/Accommodation/Expenses: Pharmamar; Honoraria (self), Travel/Accommodation/Expenses: Eli Lilly; Honoraria (self): Eisai. F. Duffaud: Honoraria (self): Bayer. N. Hindi: Honoraria (self), Research grant/Funding (institution), Travel/Accommodation/Expenses: Pharmamar; Honoraria (self), Research grant/Funding (institution): Eli Lilly; Research grant/Funding (institution): Eisai; Honoraria (institution): Novartis; Research grant/Funding (institution): Deciphera; Research grant/Funding (institution): Blueprint; Research grant/Funding (institution): Karyopharm; Research grant/Funding (institution): Daychii; Research grant/Funding (institution): GSK; Research grant/Funding (institution): Adaptimmune; Research grant/Funding (institution): Amgen. W. Van Der Graaf: Research grant/Funding (institution): Novartis; Honoraria (institution): Bayer; Honoraria (institution): Springworks. S. Stacchiotti: Advisory/Consultancy: Adaptimmune; Advisory/Consultancy, Research grant/Funding (institution): Bayer; Advisory/Consultancy, Research grant/Funding (institution): Daiichi Sankyo; Advisory/Consultancy, Research grant/Funding (institution): Eli Lilly; Advisory/Consultancy, Research grant/Funding (institution): Epizyme; Advisory/Consultancy: Immunodesign; Advisory/Consultancy: Karyopharm; Advisory/Consultancy: Maxivax; Advisory/Consultancy, Research grant/Funding (institution): Pharmamar; Research grant/Funding (institution): Amgen Dompè; Advisory/Consultancy: Takeda; Research grant/Funding (institution): Novartis; Research grant/Funding (institution): Pfizer. All other authors have declared no conflicts of interest.