OBJECTIVES: International guidelines recommend long-term prophylaxis for patients with von Willebrand disease (VWD) and severe/frequent bleeding, but this is not currently approved in the US. We assessed healthcare resource utilization (HRU) in patients with VWD including those eligible for prophylaxis.

METHODS: This retrospective cohort study used data from an integrated US healthcare system (Geisinger Health). Two cohorts were analyzed: (1) patients with ≥2 diagnoses of VWD from 01/2004–12/2020, and (2) the subset of these patients who were eligible for prophylaxis (history of ≥3 joint bleeds, ≥2 gastrointestinal bleeds, ≥5 total bleeds in the last 12 months, or ≥1 major bleed). Patients with other bleeding disorders were excluded. Patients eligible for analysis had ≥6 months of available data before the index date (defined as first date of VWD diagnosis or date meeting prophylaxis eligibility, respectively). HRU was measured during the 6-month period after the index date using linked claims data.

RESULTS: Linked data were available from 110 VWD patients overall and 23 prophylaxis-eligible VWD patients (none received long-term prophylaxis). The proportion of patients with all-cause hospitalizations was similar in prophylaxis-eligible VWD patients and the overall VWD population (22% vs 20%). Among those with ≥1 hospitalization, prophylaxis-eligible VWD patients experienced a higher mean±SD number of hospitalizations (2.2±2.7 vs 1.1±0.3) and total mean hospital days (10.0±14.5 vs 4.8±3.0 days). Nearly all patients had ≥1 outpatient visit, with a mean±SD of 9.7±7.9 and 11.0±11.6 visits, respectively. Almost half (48%) of prophylaxis-eligible VWD patients had an all-cause emergency department visit versus 28% in the overall VWD population. The proportion of patients with ≥1 VWD-related outpatient (91% vs 81%) or emergency department (39% vs 13%) visit was also higher for prophylaxis-eligible VWD patients.

CONCLUSIONS: Prophylaxis-eligible VWD patients experienced a higher HRU burden than the overall VWD population, suggesting a potential benefit from long-term prophylaxis.