TY - JOUR
T1 - An open-label single-arm phase II study of regorafenib for the treatment of angiosarcoma
AU - Agulnik, Mark
AU - Schulte, Brian
AU - Robinson, Steven
AU - Hirbe, Angela C.
AU - Kozak, Kevin
AU - Chawla, Sant P.
AU - Attia, Steven
AU - Rademaker, Alfred
AU - Zhang, Hui
AU - Abbinanti, Susan
AU - Cehic, Rasima
AU - Monga, Varun
AU - Milhem, Mohammed
AU - Okuno, Scott
AU - Van Tine, Brian A.
N1 - Funding Information:
The trial was sponsored by Northwestern University and led by the MWSTP in collaboration with Bayer , which provided funding and the drug for the study. The funder collaborated with the co-authors in study design only. The funder had no role in data collection, data analysis, data interpretation or manuscript approval. All authors had full access to all the data reported in the study and the corresponding author had final responsibility for the decision to submit for publication.
Publisher Copyright:
© 2021 Elsevier Ltd
PY - 2021/9
Y1 - 2021/9
N2 - Purpose: Angiosarcomas represents a diverse group of aggressive high-grade vascular tumours with limited therapeutic options. We sought to determine the safety and efficacy of regorafenib, a small-molecule multikinase inhibitor, in the treatment of metastatic or locally advanced unresectable angiosarcoma. Patients and methods: In this single-arm multicentre, open-label phase II clinical trial, 31 patients were enrolled and received regorafenib 160 mg PO daily for 21 days of a 28-day cycle. The primary endpoint for the study was progression-free survival at 4 months. Secondary endpoints included overall survival, response rate, and safety. Patients (≥18 years) with an Eastern Cooperative Oncology Group (ECOG) score of 0–1, a life expectancy of at least 4 months who had progressed on at least one but no more than 4 prior lines of therapy were eligible. Results: Of the 23 patients evaluable for efficacy, 2 had a complete response (8.7%), and 2 had a partial response (8.7%), for a total overall response rate of 17.4%. Median PFS was 5.5 months, and 12/23 patients (52.2%) had a PFS of greater than 4 months. 10/31 (32.3%) patients evaluable for toxicity had a grade 3 or higher adverse events. Conclusions: Regorafenib is a safe and active treatment for refractory metastatic and unresectable angiosarcoma. Rates of adverse events were comparable to prior studies of regorafenib for other tumour types. Regorafenib, the single agent, could be considered as therapy for patients with metastatic or unresectable AS.
AB - Purpose: Angiosarcomas represents a diverse group of aggressive high-grade vascular tumours with limited therapeutic options. We sought to determine the safety and efficacy of regorafenib, a small-molecule multikinase inhibitor, in the treatment of metastatic or locally advanced unresectable angiosarcoma. Patients and methods: In this single-arm multicentre, open-label phase II clinical trial, 31 patients were enrolled and received regorafenib 160 mg PO daily for 21 days of a 28-day cycle. The primary endpoint for the study was progression-free survival at 4 months. Secondary endpoints included overall survival, response rate, and safety. Patients (≥18 years) with an Eastern Cooperative Oncology Group (ECOG) score of 0–1, a life expectancy of at least 4 months who had progressed on at least one but no more than 4 prior lines of therapy were eligible. Results: Of the 23 patients evaluable for efficacy, 2 had a complete response (8.7%), and 2 had a partial response (8.7%), for a total overall response rate of 17.4%. Median PFS was 5.5 months, and 12/23 patients (52.2%) had a PFS of greater than 4 months. 10/31 (32.3%) patients evaluable for toxicity had a grade 3 or higher adverse events. Conclusions: Regorafenib is a safe and active treatment for refractory metastatic and unresectable angiosarcoma. Rates of adverse events were comparable to prior studies of regorafenib for other tumour types. Regorafenib, the single agent, could be considered as therapy for patients with metastatic or unresectable AS.
KW - Angiosarcoma
KW - Phase 2
KW - Regorafenib
KW - Sarcoma
KW - TKI
UR - http://www.scopus.com/inward/record.url?scp=85110267246&partnerID=8YFLogxK
U2 - 10.1016/j.ejca.2021.06.027
DO - 10.1016/j.ejca.2021.06.027
M3 - Article
C2 - 34284255
AN - SCOPUS:85110267246
SN - 0959-8049
VL - 154
SP - 201
EP - 208
JO - European Journal of Cancer
JF - European Journal of Cancer
ER -