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.

Medicine
|June 27, 2020
PubMed
Abstract

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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