Background: Although international guidelines recommend long-term prophylaxis for patients with von Willebrand disease (VWD) who experience severe/frequent bleeding, it is not currently approved in the US.

Aims: Assess unmet needs by describing the real-world characteristics and clinical burden among patients with VWD overall and those eligible for prophylaxis (i.e., with a history of severe/frequent bleeds).

Methods: This retrospective cohort study used de-identified electronic medical records (EMR) data from an integrated US healthcare system. Two cohorts were analyzed: (1) patients with ≥2 diagnoses of VWD in the EMR (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). The index dates of these two cohorts were defined as the date of first VWD diagnosis and the date that a patient first met prophylaxis eligibility criteria, respectively. Patients were required to have ≥6 months of data before (baseline period) and after the index date (study period). Clinical characteristics and bleeding events were described.

Results: Overall, 396 patients with VWD, including a subset of 75 prophylaxis-eligible patients, were included for analysis; none received long-term prophylaxis. Patient demographics were similar between cohorts, except for age (Table 1). In both cohorts, the proportions of patients with comorbidities or bleeds increased between the baseline and study periods, and the proportions appeared higher in prophylaxis-eligible patients. The most common comorbidities were chronic pulmonary disease, obesity, anemia, anxiety, and depression. Patients experienced more new bleeds than recurrent bleeds from the same anatomical location (Table 2).

Conclusion(s): In this retrospective US cohort study, patients with VWD experienced a high burden of bleeding-related illness and other comorbidities. Nearly 20% of patients were eligible for prophylaxis owing to severe or frequent bleeds, and thus may have benefited from prophylactic treatment.

Table 1

Table 1. Patient baseline characteristics for all patients with VWD and those eligible for prophylaxis

Table 2

Table 2. Clinical burden among all patients with VWD and those eligible for prophylaxis

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/characteristics-and-clinical-burden-among-patients-with-von-willebrand-disease-eligible-for-prophylaxis-results-from-a-retrospective-us-cohort-study/