PB0225 - Evaluation of Real-world Treatment Patterns and Outcomes in Patients with Von Willebrand Disease Treated Prophylactically with recombinant Von Willebrand Factor in the United States: Interim Analysis

Background: VWD is the most common inherited bleeding disorder, characterized by a wide range of bleeding symptoms with different treatment options. rVWF is approved for treatment of VWD (on-demand, perioperative, and routine prophylaxis). Guidelines conditionally suggest long-term prophylaxis for patients with history of severe and frequent bleeds. However, due to wide variation in treatment practices, there is a need to describe the real-world characteristics of patients with VWD and the prophylactic use of rVWF in the United States.

Aims: To describe demographic/clinical characteristics, treatment patterns, clinical outcomes, and healthcare resource utilization (HCRU) of patients with VWD before, during, and after prophylactic treatment with rVWF.

Methods: This retrospective observational chart review study used data from US healthcare centers, including patients aged ≥12 years with a confirmed diagnosis of VWD who received rVWF for prophylactic treatment (Figure 1). Patient histories and detailed treatment information were collected from medical charts (Figure 2).

Results: Interim results to date include four patients who initiated prophylaxis with rVWF (one with Type 2B and three with Type 3). Male and female patients were equally represented. All patients received routine rVWF prophylaxis with dosing frequencies of one to 3x weekly. Three of four patients had anemia or iron deficiency. Reasons for initiating rVWF prophylaxis were: patient preference, caregiver preference, to change dose and frequency of infusions, and to improve bleed control. The mean (SD) duration of routine prophylaxis was 3.7 (0.7) years.

Conclusion(s): This study describes real-world characteristics of patients with VWD who received rVWF prophylaxis. Clinical outcomes, treatment patterns, and HCRU data from the full data set will compare real-world prophylactic use of rVWF with previous treatment regimens. Results from this study may identify the potential role of routine rVWF prophylactic treatment in mitigating clinical burden and HCRU of patients with VWD.