A Timely Oral Option: Single Agent Vinorelbine in Desmoid Tumours

Introduction: Desmoid tumours (DT) are rare collagen-forming tumours which can exhibit locally aggressive patterns of behaviour. The aim of this study was to evaluate the efficacy and safety of treatment of DT with single agent oral vinorelbine.

Methods: A retrospective review of patients treated with vinorelbine 90mg orally on Days 1, 8 and 15 of a 28-day cycle from January 2004-July 2019 was performed. Response was assessed using Response Evaluation Criteria in Solid Tumours version 1.1. Descriptive statistics were employed.

Results: 29 patients were included. Response rate was 20.7% (6/29) and clinical benefit rate (response by RECIST 1.1 and/or clinical symptom improvement) was 65.5% (19/29).. No patient experienced grade 3 or above toxicity. Common toxicities were grade 1-2 nausea (14/26, 48.3%), fatigue (9/26, 31.0%) and diarrhoea (4/26, 13.8%).

Conclusion: Single agent oral vinorelbine is an effective, safe and well tolerated treatment for DT. It represents a new oral alternative for management of DT.

Keywords: Desmoid tumour; Desmoid-type fibromatosis; Efficacy; Fibromatosis; Safety; Systemic therapy; Vinorelbine.