Von Willebrand factor for menorrhagia: a survey and literature review

Abstract

Background von Willebrand disease (VWD) is the most common congenital bleeding disorder. In women, menorrhagia is the most common bleeding symptom, and is disabling with iron deficiency anemia, high health cost, and poor quality of life. Current hormonal and non-hormonal therapies are limited by ineffectiveness and intolerance. Few data exist regarding Von Willebrand factor (VWF), typically prescribed when other treatments fail. The lack of effective therapy for menorrhagia remains the greatest unmet healthcare need in women with VWD. Better therapies are needed to treat women with menorrhagia. Methods We conducted a survey of U.S. hemophilia treatment centers (HTCs) and a literature review using medical subject heading (MeSH) search terms “von Willebrand factor,” “menorrhagia,” and “von Willebrand disease” to assess the use of VWF in menorrhagia. Analysis was by descriptive statistics. Results Of 83 surveys distributed to HTC MDs, 20 (24.1%) provided sufficient data for analysis. Of 1,321 women with VWD seen during 2011-2014, 816 (61.8%) had menorrhagia, for which combined oral contraceptives, tranexamic acid, and desmopressin were the most common first-line therapies for menorrhagia, while VWF was third-line therapy reported in 13 women (1.6%). Together with data from 88 women from six published studies, VWF safely reduced menorrhagia in 101 women at a dose of 33-100 IU/kg on day 1-6 of menstrual cycle. Conclusions This represents the largest VWD menorrhagia treatment experience to date. VWF safely and effectively reduces menorrhagia in women with VWD. A prospective clinical trial is planned to confirm these findings.

Prevalence of Bleeding Symptoms among Adolescents and Young Adults in the Capital City of Saudi Arabia

“…There are additional inherited platelet disorders with a generally unknown prevalence. Inherited thrombocytopathies are a heterogeneous group of platelet disorders present mainly with mucocutaneous bleeding of variable severity caused by defects in platelet adhesion, aggregation, granules, and signal transduction [ 15 ]. The diagnosis of more prevalent mild forms of inherited thrombocytopathies is difficult, even with extensive laboratory testing [ 16 ].…”

Clinical Issues in Women with Inherited Bleeding Disorders

“…HMB occurs in approximately 80% of cases and is associated with significant co-morbidity, namely iron deficiency anemia, stress, and reduction in the quality of life affecting daily activities; in addition, it entails higher costs in medical care [31]. There is evidence that women with VWD have higher rates of postpartum hemorrhage and transfusions at the time of delivery compared to healthy women [32].…”

Case-based discussion on the implications of exogenous estrogens in hemostasis and thrombosis: the hematologist’s view

“…This is a challenging question because treatment for menorrhagia that is effective and well tolerated is a major unmet health need in WBD (Table 2). 24 The currently recommended nonhormonal agent, tranexamic acid (Lysteda), an antifibrinolytic agent dosed at 1300 mg 3 times daily during the first 5 days of the cycle, reduces menstrual loss by 50% but is limited by nasal congestion, headache, and nausea. 25 Desmopressin (1-deamino-8-D-arginine vasopressin [DDAVP]), a synthetic vasopressin analog that stimulates VWF release from the vascular endothelium at the site of injury, is limited by hyponatremia, tachyphylaxis, and local infusion reaction, and the intranasal form, Stimate, although convenient, is less potent.…”

“…25 Desmopressin (1-deamino-8-D-arginine vasopressin [DDAVP]), a synthetic vasopressin analog that stimulates VWF release from the vascular endothelium at the site of injury, is limited by hyponatremia, tachyphylaxis, and local infusion reaction, and the intranasal form, Stimate, although convenient, is less potent. 24 Combined oral contraceptives (COCs) stimulate VW synthesis but are avoided in 35% of patients because of headaches and hypertension. 24 The levonorgestrel intrauterine system, Mirena, stimulates an antifibrinolytic Table 1.…”