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Updated: Dec 17, 2025

Establishment of a Primary Culture of Patient-derived Soft Tissue Sarcoma
Published on: April 11, 2018
Activity of regorafenib in advanced pretreated soft tissue sarcoma: Results of a single-center phase II study
Andrea Marrari1,2, Alexia Bertuzzi1, Silvia Bozzarelli1
1Medical Oncology and Hematology Unit.
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.
Clinical Trial Registration:
ClinicalTrials.gov NCT02307500.
Insights
Regorafenib demonstrated clinical activity in advanced soft tissue sarcomas (STS). This phase II trial showed a 61.9% progression-free survival at 8 weeks, indicating potential therapeutic benefit in pretreated patients.
Area of Science:
- Oncology
- Medical Oncology
- Clinical Pharmacology
Background:
- Regorafenib, a tyrosine kinase inhibitor, has shown activity in soft tissue sarcomas (STS).
- Previous research suggests potential efficacy in this patient population.
Purpose of the Study:
- To evaluate the clinical activity of regorafenib in advanced, pretreated soft tissue sarcoma (STS) patients.
- To determine the progression-free survival (PFS) at 8 weeks as the primary endpoint.
Main Methods:
- An open-label, non-randomized, single-center phase II study was conducted.
- Patients received regorafenib 160 mg daily for 21 days of a 28-day cycle.
- Progression-free survival (PFS) at 8 weeks and toxicity were assessed.
Main Results:
- 61.9% of 21 evaluable patients were progression-free at 8 weeks.
- Median PFS was 3.8 months; median overall survival was 14.8 months.
- A 4.7% response rate and a 61.9% clinical benefit rate were observed; treatment was well tolerated.
Conclusions:
- Regorafenib exhibits clinical activity in patients with advanced soft tissue sarcomas (STS).
- The drug is a potential treatment option for pretreated STS patients.
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