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Updated: Jul 16, 2025
A Mouse Model of Incompletely Resected Soft Tissue Sarcoma for Testing Neoadjuvant Therapies
Published on: July 28, 2020
Axitinib in patients with advanced/metastatic soft tissue sarcoma (Axi-STS): an open-label, multicentre, phase II
Penella J Woll1, Piers Gaunt2, Charlotte Gaskell2
1University of Sheffield, Sheffield UK and Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, S10 2JF, UK.
Background:
Axitinib is an oral vascular endothelial growth factor receptor inhibitor with anti-tumour activity in renal, thyroid, and pancreatic cancer.
Methods:
Axi-STS was a pathologically-stratified, non-randomised, open-label, multi-centre, phase II trial of continuous axitinib treatment in patients ≥16 years, performance status ≤2, with pathologically-confirmed advanced/metastatic soft tissue sarcoma (STS). Patients were recruited within four tumour strata, each analysed separately: angiosarcoma, leiomyosarcoma, synovial sarcoma, or other eligible STSs. The primary outcome was progression-free survival at 12 weeks (PFS12). A Simon's two-stage design with activity defined as PFS12 rate of 40% determined a sample size of 33 patients per strata.
Results:
Between 31-August-2010 and 29-January-2016, 145 patients were recruited: 38 angiosarcoma, 37 leiomyosarcoma, 36 synovial sarcoma, and 34 other subtypes. PFS12 rate for each stratum analysed was 42% (95% lower confidence interval (LCI); 29), 45% (95% LCI; 32), 57% (95% LCI; 42), and 33% (95% LCI; 21), respectively. There were 74 serious adverse events including two treatment-related deaths of pulmonary haemorrhage and gastrointestinal bleeding. Fatigue and hypertension were the most common grade 3 adverse events.
Conclusions:
Axitinib showed clinical activity in all STS strata investigated. The adverse event profile was acceptable, supporting further investigation in phase III trials.
Clinical Trial Registration:
ISRCTN 60791336.
Insights
Axitinib demonstrated anti-tumour activity in advanced soft tissue sarcoma (STS) patients. This phase II trial found acceptable safety, supporting further investigation in advanced cancer treatment.
Area of Science:
- Oncology
- Medical research
- Clinical trials
Background:
- Axitinib is a targeted therapy inhibiting vascular endothelial growth factor receptors.
- It has shown efficacy in renal, thyroid, and pancreatic cancers.
- Soft tissue sarcoma (STS) is a rare cancer with limited treatment options.
Purpose of the Study:
- To evaluate the efficacy and safety of axitinib in patients with advanced/metastatic soft tissue sarcoma (STS).
- To assess progression-free survival at 12 weeks (PFS12) across different STS subtypes.
Main Methods:
- A phase II, open-label, multi-centre trial (Axi-STS) enrolled 145 patients aged ≥16 years with advanced/metastatic STS.
- Patients were stratified into four groups: angiosarcoma, leiomyosarcoma, synovial sarcoma, and other STS subtypes.
- The primary endpoint was the PFS12 rate, with activity defined as a 40% PFS12 rate.
Main Results:
- The PFS12 rates were 42% for angiosarcoma, 45% for leiomyosarcoma, 57% for synovial sarcoma, and 33% for other STS subtypes.
- Serious adverse events occurred in 74 patients, including two treatment-related deaths.
- Fatigue and hypertension were the most frequent grade 3 adverse events.
Conclusions:
- Axitinib exhibited clinical activity across all investigated STS subtypes.
- The safety profile of axitinib was deemed acceptable for further clinical development.
- These findings support the progression of axitinib to phase III trials for advanced STS treatment.
