Personalizing Care in von Willebrand Disease Is Essential

Since the growth of specialty pharmacies, genetically acquired bleeding disorders such as von Willebrand disease (VWD) have been among the most common conditions addressed at these practice locations. VWD usually occurs as 1 of 3 types, based on the number and quality of inherited defects. From 60% to 80% of patients have autosomal dominant type 1, the mildest form. Between 15% and 30% have type 2, which also presents with mild to moderate symptoms. Fewer patients have type 3 and, typically, experience severe spontaneous bleeds, usually in joints and muscles. A fourth type, acquired VWD, is not inherited but it is usually associated with underlying disease.

Attendees at the Asembia Specialty Pharmacy Summit 2019 learned that although the disease affects both sexes, women bear a disproportionate burden because of menorrhagia. Patients with VWD must overcome social, clinical, and economic barriers.

Sheh-Li Chen, PharmD, BCOP, a clinical pharmacy specialist in benign hematology at the University of North Carolina Medical Center in Chapel Hill, and Tim Affeldt, PharmD, director of Specialty/Infusion Operations at Fairview Pharmacy Services in Minneapolis, Minnesota, teamed up to discuss clinical and specialty pharmacy concerns. Although a personal and familial history of excessive bleeding is telling, it is not definitive for diagnosis. Each patient needs a thorough workup—ideally, led by a skilled hematologist.

Treatment revolves around 2 principles. The first is elevating plasma levels of von Willebrand factor (vWF) and factor VIII to promote hemostasis and wound healing. For decades, specialty pharmacists have worked to help patients secure and use desmopressin. Replacement therapy is the second strategy and is accomplished using factor products.

Available human plasma—derived VWF concentrate products contain different ratios of vWF to factor VIII and include 3 products available in the United States, Chen said. She noted that products are not interchangeable and patients are at risk of thrombotic complications. For this reason, the specialty pharmacies must be vigilant and monitor patients closely. The recently approved recombinant vWF is indicated for adults with VWD. Since it does not rely on human donors, it decreases the risk of infectious transfer.

Chen also covered adjunctive treatments for managing mucosal bleeds, gastrointestinal bleeds, menorrhagia, and other types of acute episodes. Treatments include antifibrinolytics such as tranexamic acid and aminocaproic acid. In women, hormonal treatment with a combined progestin/estrogen oral contraceptive or a levonorgestrel intrauterine device can be helpful.

Affeldt highlighted the role of the pharmacist and important counseling points. In addition to offering patient education and monitoring, specialty pharmacists need to step in and take the lead when patients experience difficulties. Every specialty pharmacist should know the patient’s specific VWD subtype, bleeding history, and typical plasma levels of vWF and factor VIII. Specialty pharmacists also must ensure that they have an adequate supply of medications available because these patients cannot wait for treatment. The session’s takehome message was that patients with VWD need considerably more personalized care to help improve outcomes and manage costs.