|Title:||Systemic therapies in advanced epithelioid haemangioendothelioma: A retrospective international case series from the World Sarcoma Network and a review of literature||Authors:||Frezza A.M.
Lo Vullo S.
Van Der Graaf W.
|Keywords:||anthracycline; chemotherapy; epithelioid haemangioendothelioma; interferon; paclitaxel; pazopanib||Issue Date:||2021||Publisher:||Blackwell Publishing Ltd||Journal Volume:||10||Journal Issue:||8||Start page/Pages:||2645-2659||Source:||Cancer Medicine||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. ? 2021 The Authors. Cancer Medicine published by John Wiley & Sons Ltd.
|ISSN:||2045-7634||DOI:||10.1002/cam4.3807||metadata.dc.subject.other:||alpha2b interferon; anthracycline; cyclophosphamide; docetaxel; gemcitabine; ifosfamide; paclitaxel; pazopanib; antineoplastic agent; adult; advanced cancer; aged; Article; Asia; Australia; CAMTA1 gene; cancer chemotherapy; cancer growth; case study; clinical outcome; controlled study; drug megadose; Europe; female; gene; gene fusion; hemangioendothelioma; human; major clinical study; male; middle aged; observational study; overall survival; priority journal; progression free survival; radiological parameters; response evaluation criteria in solid tumors; retrospective study; systemic therapy; TFE3 gene; United States; WWTR1 gene; YAP1 gene; follow up; hemangioendothelioma; international cooperation; pathology; prognosis; sarcoma; survival rate; Adult; Antineoplastic Combined Chemotherapy Protocols; Female; Follow-Up Studies; Hemangioendothelioma, Epithelioid; Humans; International Agencies; Male; Middle Aged; Prognosis; Retrospective Studies; Sarcoma; Survival Rate
|Appears in Collections:||醫學院附設醫院 (臺大醫院)|
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