Findings from a new study published in the journal Blood Transfusion, suggests that patients with severe von Willebrand disease (VWD) may benefit from secondary long-term prophylaxis with von Willebrand factor (VWF)/factor VIII (FVIII) therapy. Secondary prophylaxis is utilized after a patient has had several bleeds into a single joint. The lead author of the study was Dr. Flora Peyvandi, Medical Director of the Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre in Milan, Italy.
Peyvandi and her colleagues enrolled 22 patients with severe VWD, 19 of whom were randomly assigned to receive either on-demand treatment (9 patients) or prophylaxis (10 patients) with VWF/FVIII factor products. Twelve patients completed the full study. Patients in the on-demand group received factor infusions at the onset of each bleeding episode, while patients in the prophylaxis group received scheduled infusions once every two-to-three days.
The results showed that patients receiving on-demand therapy experienced a significantly greater number of bleeds than those patients in the prophylaxis group (172 vs. 32). It should be noted that 112 of the bleeds in the on-demand group occurred in a single patient. The authors go on to explain that even after excluding that patient, “the number of bleeding episodes in the on-demand treatment group was still 2-fold higher” compared to the prophylaxis group.
While adverse events were reported in three patients in the on-demand group and six patients in the prophylaxis group, the investigators established that none of the events were linked to factor use.
“Despite the small sample size and the heterogeneity of the study population, patients on vWF/FVIII prophylaxis showed a reduction in bleeding risk and rate compared to on-demand treatment,” concluded the authors.
Additional larger studies are clearly needed to further explore the use of prophylaxis with VWF/FVIII therapies in VWD patients.
Source: Hematology Advisor, February 21, 2019