Axitinib in patients with advanced/metastatic soft tissue sarcoma (Axi-STS): an open-label, multicentre, phase II trial in four histological strata.

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Embargo End Date

ICR Authors

Judson, Ian

Authors

Woll, PJ
Gaunt, P
Gaskell, C
Young, R
Benson, C
Judson, IR
Seddon, BM
Marples, M
Ali, N
Strauss, SJ
Lee, A
Hughes, A
Kaur, B
Hughes, D
Billingham, L

Document Type

Journal Article

Date

2023-10-26

Date Accepted

2023-08-23

Abstract

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.

Citation

British Journal of Cancer, 2023, 129 (9), pp. 1490 - 1499

DOI

10.1038/s41416-023-02416-6
10.1038/s41416-023-02416-6

URI

https://repository.icr.ac.uk/handle/internal/6096

Rights

http://creativecommons.org/licenses/by/4.0/

Source Title

British Journal of Cancer

Publisher

SPRINGERNATURE

ISSN

0007-0920

eISSN

1532-1827
1532-1827

Collections

Clinical Studies

Research Team

Sarcoma Clinical Trials

Notes