Prophylaxis with recombinant von Willebrand factor in patients with type 3 von Willebrand disease: Results of a post hoc analysis from a phase 3 trial

Abstract

Objectives: To describe efficacy/safety of recombinant von Willebrand factor (rVWF) prophylaxis in patients with type 3 von Willebrand disease (VWD).Methods: This post hoc analysis of a phase 3 open-label trial provides a more detailed analysis of adults with type 3 VWD, categorized based on prior treatment at screening: "Prior On-Demand (OD)" (OD VWF; ≥3 documented spontaneous bleeding events [BEs] requiring VWF in previous 12 months) or "Switch" (plasma-derived[pd] VWF prophylaxis for ≥12 months). Annualized bleeding rates (ABRs) were evaluated during 12 months of rVWF prophylaxis versus historical data from medical records.Results: In the Prior OD group (n = 10), mean spontaneous ABR (sABR) for treated BEs was reduced by 91.6% (ratio, 0.08; 95% CI, 0.02-0.45) versus mean historical sABR. In the Switch group (n = 8), mean sABR for treated BEs was reduced by 47% (ratio, 0.53; 95% CI, 0.08-3.62). One non-serious adverse event (AE) was considered

von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease

“…In adult patients (≥17 years old), Wilate reduced the treated SABR by 85%, whereas Vonvendi reduced the treated SABR by 92% in the patients on prior on-demand treatment. 14 Interestingly, the reduction was only 49% with Vonvendi in a post-hoc analysis of type 3 patients with VWD when untreated BEs were included for the analysis, 16 whereas with Wilate the reduction in untreated SABR in type 3 patients with VWD was 89%. Of the 31 treated breakthrough BEs in the study with Vonvendi, 7 (22.6%) BEs required recombinant FVIII infusions in addition to treatment with VWF, 14 supporting the clinical relevance of a preparation containing both VWF and FVIII.…”

Emicizumab in Type 3 von Willebrand Disease: Report of a Case with an Alloantibody and Literature Review

“…19 20 Recombinant and plasma-derived VWF products that contain no FVIII have been approved and are effective in the treatment of bleeding and during surgery in type 3 VWD as well as in other forms of VWD. 26 27…”