Activity of regorafenib in advanced pretreated soft tissue sarcoma: Results of a single-center phase II study

Andrea Marrari, Alexia Bertuzzi, Silvia Bozzarelli, Nicolò Gennaro, Laura Giordano, Vittorio Quagliuolo, Rita De Sanctis, Simona Sala, Luca Balzarini, Armando Santoro

Research output: Contribution to journalArticlepeer-review


BACKGROUND: Regorafenib, a multitargeted tyrosine kinase inhibitor, proved to be active in patients with soft tissue sarcomas (STS).

METHODS: We conducted an open-label, non-randomized, single-center phase II study in advanced pretreated STS patients. Patients received regorafenib 160 mg daily on days 1 enrule 21 of a 28-day cycle. The primary endpoint was the progression-free survival (PFS) at 8 weeks. Toxicity was registered.

RESULTS: Between April 2015 and November 2016, 21 patients were enrolled in the trial. A total of 13 out of 21 evaluable patients (61.9%) were progression-free at 8 weeks. Median PFS was 3.8 months (95% CI: 2.1-9.4). Median overall survival was 14.8 months (95% CI: 7.7-27.8). In the intention-to-treat population, we reported a PFS of 66.7% at 3 months (95% CI: 40.4-83.4) and 16.7% at 12 months (95% CI: 4.1-36.5). As per the RECIST criteria, the response rate was 4.7% (1 partial response out of 21 evaluable patients) with a clinical benefit rate of 61.9%; no complete response was observed. Treatment was well tolerated.

CONCLUSION: Regorafenib shows signs of clinical activity in patients with advanced STS.


Original languageEnglish
Pages (from-to)e20719
Issue number26
Publication statusPublished - Jun 26 2020


  • Antineoplastic Agents/administration & dosage
  • Drug Monitoring/methods
  • Enzyme Inhibitors/administration & dosage
  • Female
  • Humans
  • Male
  • Middle Aged
  • Ovarian Neoplasms/drug therapy
  • Pancreatic Neoplasms/drug therapy
  • Phenylurea Compounds/administration & dosage
  • Progression-Free Survival
  • Pyridines/administration & dosage
  • Response Evaluation Criteria in Solid Tumors
  • Sarcoma/drug therapy
  • Thymoma/drug therapy
  • Treatment Outcome


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