An open-label single-arm phase II study of regorafenib for the treatment of angiosarcoma.

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. 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. 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. 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.

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Déclaration de conflit d'intérêts

Conflict of interest statement M.A. reports receiving consultation fees from Lilly, Adaptimmune, Regeneron, AstraZeneca; Speaker Bureau: BMS, Bayer, Deciphera, B.V.T. has received Basic Science Grant Funding from Pfizer, Tracon and Merck; has received consulting fees from Epizyme, Lilly, CytRx, Janssen, Immune Design, Daiichi Sankyo, Plexxikon and Adaptimmune; has received speaking fees from Caris, Janseen and Lilly; and has received travel support from Adaptimmune and Lilly, S.A. has received research funding from Desmoid Tumor Research Foundation; has also received research funding to Institution from AB Science, TRACON Pharma, CytRx Corporation, Bayer, Novartis, Daiichi Sankyo, Lilly, Immune Design, Karyopharm Therapeutics, Epizyme, Blueprint Medicines, Genmab, CBA Pharma, Merck, Philogen, Gradalis, Deciphera, Takeda, Incyte, Springworks, Adaptimmune, Advenchen Laboratories, Bavarian Nordic, BTG, PTC Therapeutics, GlaxoSmithKline, FORMA Therapeutics; has received travel fees, accommodation expenses and general expenses from Immune Design. M.M. reports being a member of the consultant/advisory boards with Amgen, Trieza, Biontech, Blueprint Medicine, Immunocore, Array BioPharma, INC. S.R. reports receiving research support from TRACON Pharmaceutical; has been a member of the advisory boards with BTG International and the Society of Interventional Radiology Foundation; has received honoraria to the institution; all outside the submitted work. All the authors have no conflict of interest to disclose.