Different bleeding risk in type 2A and 2M von Willebrand disease: a 2-year prospective study in 107 patients
Article first published online: 30 MAR 2012
© 2012 International Society on Thrombosis and Haemostasis
Journal of Thrombosis and Haemostasis
Volume 10, Issue 4, pages 632–638, April 2012
How to Cite
CASTAMAN, G., FEDERICI, A. B., TOSETTO, A., LA MARCA, S., STUFANO, F., MANNUCCI, P. M. and RODEGHIERO, F. (2012), Different bleeding risk in type 2A and 2M von Willebrand disease: a 2-year prospective study in 107 patients. Journal of Thrombosis and Haemostasis, 10: 632–638. doi: 10.1111/j.1538-7836.2012.04661.x
- Issue published online: 30 MAR 2012
- Article first published online: 30 MAR 2012
- Accepted manuscript online: 13 FEB 2012 12:31PM EST
- Received 31 October 2011, accepted 6 February 2012
- gene mutation;
- inherited bleeding disorder;
- von Willebrand disease;
- von Willebrand factor
Summary. Background: Type 2A and 2M von Willebrand disease (VWD2A and VWD2M) are characterized by the presence of a dysfunctional von Willebrand factor (VWF) and a variable bleeding tendency. So far, a head-to-head comparison of the clinical history and bleeding risk between VWD2A and VWD2M has never been provided in a prospective manner.
Aim of the study: We assessed the bleeding incidence rate and clinical characteristics in two cohorts of 17 families (46 patients) with VWD2A and 15 families (61 patients) with VWD2M prospectively followed-up for 24 months. VWF gene mutations were characterized in all of them.
Results: Mean bleeding score (BS) and VWF antigen at enrollment were significantly higher in VWD2A patients (P = 0.007). No correlation between VWF activity or factor VIII levels and the severity of BS was observed. The incidence rate of spontaneous bleeding requiring treatment was 107/100 patient-years (95% CI, 88.3–131) in VWD2A compared with 40/100 patient-years (95% CI, 30–53) in VWD2M (P < 0.001). The risk of bleeding was significantly higher in patients with BS ≥ 10 at enrollment compared with those with BS 0–2. Furthermore, 54 episodes of gastrointestinal bleeding occurred in 17/46 (36.9%) VWD2A patients and seven in 2/61 (3.3%) VWD2M patients (P < 0.0001).
Conclusion: Bleeding tendency in VWD2A is greater than that of VWD2M, is not explained by factor VIII or VWF levels and is mainly due to an increased incidence of gastrointestinal bleeding.