TY - JOUR
T1 - Systemic therapies in advanced epithelioid haemangioendothelioma
T2 - A retrospective international case series from the World Sarcoma Network and a review of literature
AU - Frezza, Anna M.
AU - Ravi, Vinod
AU - Lo Vullo, Salvatore
AU - Vincenzi, Bruno
AU - Tolomeo, Francesco
AU - Chen, Tom Wei Wu
AU - Teterycz, Pawel
AU - Baldi, Giacomo G.
AU - Italiano, Antoine
AU - Penel, Nicolas
AU - Brunello, Antonella
AU - Duffaud, Florance
AU - Hindi, Nadia
AU - Iwata, Shintaro
AU - Smrke, Alannah
AU - Fedenko, Alexander
AU - Gelderblom, Hans
AU - Van Der Graaf, Winette
AU - Vozy, Aurore
AU - Connolly, Elizabeth
AU - Grassi, Massimiliano
AU - Benjamin, Robert S.
AU - Broto, Javier Martin
AU - Grignani, Giovanni
AU - Jones, Robin L.
AU - Kawai, Akira
AU - Tysarowski, Andrzej
AU - Mariani, Luigi
AU - Casali, Paolo G.
AU - Stacchiotti, Silvia
N1 - Publisher Copyright:
© 2021 The Authors. Cancer Medicine published by John Wiley & Sons Ltd.
PY - 2021/4
Y1 - 2021/4
N2 - 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.
AB - 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.
KW - anthracycline
KW - chemotherapy
KW - epithelioid haemangioendothelioma
KW - interferon
KW - paclitaxel
KW - pazopanib
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U2 - 10.1002/cam4.3807
DO - 10.1002/cam4.3807
M3 - Article
C2 - 33713582
AN - SCOPUS:85102469138
SN - 2045-7634
VL - 10
SP - 2645
EP - 2659
JO - Cancer medicine
JF - Cancer medicine
IS - 8
ER -