Successful and safe response to ibrutinib alone in treating relapsed Waldenström macrogobulinemia and related acquired von Willebrand syndrome: an option to consider

Abstract

Ibrutinib, a first-class Bruton tyrosine kinase inhibitor, is known to be associated with adverse bleeding events and has been recently approved for the treatment of relapse Waldenström macroglobulinemia (WM). Here, we report the exhaustive clinical and biological follow-up of 2 patients treated by ibrutinib alone in the context of relapsed WM with an acquired von Willebrand syndrome (AVWS) complication. In two cases, ibrutinib has been shown to be quickly efficient and safe for treating both AVWS and its underlying condition the WM, without bleeding complications. Interestingly, ibrutinib treatment brings a rapid and extended over time normalization of von Willebrand factor clearance. These observations show that ibrutinib is a valuable therapeutic option in relapsed WM patients associated with AVWS and highlighting the need for further cohort studies with long-term follow-up of patients to confirm the efficacy and safety of a treatment by ibrutinib for WM patients with AVWS complication.

Keywords:

• Waldenström macroglobulinemia
• acquired von Willebrand syndrome
• ibrutinib
• bleeding risk
• platelet function

Consent

Written informed consent was obtained from 2 patients.

Author contributions

MC, AD, SP and EJ designed the project, ABu, EJ, ABa performed the analysis, ABu, SP, EJ, ABa analyzed the data; ABu, SP, AD and MC wrote the paper, MS, MC, PC, MN and SS managed the patients and all authors critically reviewed the manuscript and approved the final version of the manuscript.

Disclosure statement

The authors have no competing interests to declare.