RT Journal Article
T1 Systemic therapies in advanced epithelioid haemangioendothelioma: A retrospective international case series from the World Sarcoma Network and a review of literature.
A1 Frezza, Anna M
A1 Ravi, Vinod
A1 Lo Vullo, Salvatore
A1 Vincenzi, Bruno
A1 Tolomeo, Francesco
A1 Chen, Tom Wei-Wu
A1 Teterycz, Pawel
A1 Baldi, Giacomo G
A1 Italiano, Antoine
A1 Penel, Nicolas
A1 Brunello, Antonella
A1 Duffaud, Florance
A1 Hindi, Nadia
A1 Iwata, Shintaro
A1 Smrke, Alannah
A1 Fedenko, Alexander
A1 Gelderblom, Hans
A1 Van Der Graaf, Winette
A1 Vozy, Aurore
A1 Connolly, Elizabeth
A1 Grassi, Massimiliano
A1 Benjamin, Robert S
A1 Broto, Javier-Martin
A1 Grignani, Giovanni
A1 Jones, Robin L
A1 Kawai, Akira
A1 Tysarowski, Andrzej
A1 Mariani, Luigi
A1 Casali, Paolo G
A1 Stacchiotti, Silvia
K1 anthracycline
K1 chemotherapy
K1 epithelioid haemangioendothelioma
K1 interferon
K1 paclitaxel
K1 pazopanib
AB 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. 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. 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. 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.
YR 2021
FD 2021-03-13
LK http://hdl.handle.net/10668/17345
UL http://hdl.handle.net/10668/17345
LA en
DS RISalud
RD Apr 6, 2025