Efficacy and safety of a recombinant von Willebrand factor treatment in acquired von Willebrand syndrome in case of bleeding and surgical procedures

Abstract

Introduction

Acquired von Willebrand syndrome (AVWS) is a rare haemorrhagic disorder. The prophylaxis and treatment of bleeding before surgery are complex. Since 2018, a new recombinant VWF (rVWF) concentrate that contains no factor VIII (FVIII) but a high amount of high molecular weight VWF multimers has been available in France.

Aim

To describe the real-world experience of using rVWF in non-surgical bleeding and surgical procedures in patients with AVWS.

Methods

Fifteen bleeding episodes in seven patients and 16 surgeries in 10 patients were retrospectively analysed in t French haemostasis centres.

Results

During bleeding, the median number of infusions was only 1 (range 1–27) with a median loading dose of 58 IU/kg (range 17–116) rVWF and a total median dose of 65 IU/kg (range 35–1488) rVWF. Bleeding control was rated markedly effective in 73% (11/15) of the cases and ineffective in 27% (4/15).

During surgeries, the median number of infusions was 3 (range 1–8) with a preoperative loading dose of 60 IU/kg (range 23–118) rVWF and a total median dose of 123 IU/kg (range 31–542). The overall clinical efficacy was qualified as excellent, good and poor (ISTH criteria) in respectively 7 (43%), 6 (38%) and 3 (19%) procedures.

There was no accumulation of VWF or FVIII during postoperative monitoring. No thromboembolic events nor adverse events were reported.

Conclusion

This French ‘real-world’ experience shows that rVWF could be of interest in the treatment and prophylaxis of bleeding in patients with AVWS, with no clinically significant safety concern.