Mini Oral - Sarcoma Mini Oral session

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

Presentation Number
1627MO
Speakers
  • Anna Maria Frezza (Milan, Italy)
Session Name
Mini Oral - Sarcoma

Abstract

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.

Population characteristics

Anthracycline based regimens Paclitaxel Pazopanib INFalfa2b Others
Patients 33 11 12 15 27
Median follow up 35.8 on 72 patients - - - -
Marker CAMTA1-WWTR1* YAP1-TFE3 * IHC o molecular testing 69 (95%) 4 (5%) on 72 patients - - - -
Median age 49 38 43 46 48
Gender M F 15 (45%) 18 (55%) 4 (36%) 7 (64%) 4 (33%) 8 (67%) 7 (47%) 8 (53%) 12 (44%) 15 (56%)
Stage (treatment start) Locally advanced Metastatic 6 (18%) 27 (82%) 1 (9%) 10 (91%) 1 (8%) 12 (92%) 3 (20%) 12 (80%) 4 (14%) 23 (86%)
Evidence of prior PD: Yes No 19 (58%) 14 (42%) 6 (55%) 5 (45%) 10 (83%) 2 (17%) 12 (80%) 3 (20%) 24 (89%) 3 (11%)
Previous line 0 1 ≥ 2 29 (88%) 4 (12%) 0 8 (73%) 3 (27%) 0 5 (42%) 6 (50%) 1 (8%) 13 (86%) 1 (7%) 1 (7%) Not applicable

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.

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.

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