PB0825 - Burden of Illness in Patients With von Willebrand Disease Receiving Prophylaxis Versus Potential Prophylaxis-Eligible Patients: A Post-Hoc Analysis of A European Socioeconomic Study

Background: Limited real-world evidence is available for the health benefits of prophylaxis in patients with von Willebrand disease (VWD).

Aims: To compare patient characteristics and disease burden in patients treated with prophylaxis with those identified as eligible for prophylaxis according to current VWD management guidelines but not currently receiving it.

Methods: Post-hoc analysis of “Cost of VWD Across Europe, A Socioeconomic Study” (CVESS), a retrospective, descriptive, cross-sectional study conducted in 2018 in France, Germany, Italy, Spain, and the UK. Patients aged >1 year, with a laboratory diagnosis of hereditary VWD and known VWD classification were eligible. Data were collected by clinicians using medical records and an optional patient questionnaire. The prophylaxis group had received intermittent or continuous prophylaxis to prevent bleeding in the previous 12 months. Prophylaxis-eligible patients were defined in this analysis as those with severe (resulting in hospitalization or involving critical organs) and/or frequent (≥5 total, ≥3 joint, or ≥2 gastrointestinal) bleeds in the 12 months before the index date (ie, time of data collection), and were not prescribed prophylaxis. Ethical approval for CVESS and patient informed consent were obtained.

Results: Demographics and VWD severity/type for the prophylaxis (n=229) and prophylaxis-eligible (n=102) groups are presented in Table 1. Lower levels of pain were reported in the prophylaxis versus prophylaxis-eligible groups. Patients in the prophylaxis group were less likely to have ≥1 target joints (10.5% vs 23.5%; p=0.002), and/or ≥1 chronically damaged joints (19.2% vs 35.3%, p=0.002). Mental/behavioral disorders were the most common concomitant conditions; anxiety was reported in 12.2% of prophylaxis versus 27.5% of the prophylaxis-eligible groups (p=0.001). Rates of hospitalization were lower in the prophylaxis versus prophylaxis-eligible groups (Table 2).

Conclusion(s): This European-based real-world study highlights the increased disease burden and unmet needs in patients with VWD and severe and/or frequent bleeds who had not received prophylaxis in the previous 12 months.