New guidelines issued jointly by four major international hematology groups focus on the management of patients with von Willebrand disease (VWD), the most common bleeding disorder in the world.
The evidence-based guidelines, published in Blood Advances, were developed in collaboration by the American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis, the National Hemophilia Foundation, and the World Federation of Hemophilia. They outline key recommendations spanning the care of patients with a broad range of therapeutic needs.
“We addressed some of the questions that were most important to the community, but certainly there are a lot of areas that we couldn’t cover” said coauthor Veronica H. Flood, MD, of the Medical College of Wisconsin in Milwaukee.
The guidelines process began with a survey sent to the von Willebrand disease community, including patients, caregivers, nurses, physicians, and scientists. The respondents were asked to prioritize issues that they felt should be addressed in the guidelines.
“Interestingly, some of the issues were the same between patients and caregivers and physicians, and some were different, but there were obviously some areas that we just couldn’t cover,” she said in an interview.
One of the areas of greatest concern for respondents was bleeding in women, and many of the recommendations include specific considerations for management of gynecologic and obstetric patients, Dr. Flood said.
“We also tried to make the questions applicable to as many patients with von Willebrand disease as possible,” she added.
Some of the questions, such as recommendation 1, regarding prophylaxis, are geared toward management of patients with severe disease, while others, such as recommendations for treatment of menstrual bleeding, are more suited for patients with milder VWD.
All of the recommendations in the guidelines are “conditional” (suggested), due to very low certainty in the evidence of effects, the authors noted.
Prophylaxis
The guidelines suggest long-term prophylaxis for patients with a history of severe and frequent bleeds, with periodic assessment of the need for prophylaxis.
Desmopressin
For those patients who may benefit from the use of desmopressin, primarily those with type 1 VWD, and who have a baseline von Willebrand factor (VWF) level below 0.30 IU/mL, the panel issued a conditional recommendation for a desmopressin trial with treatment based on the patient’s results compared with not performing a trial and treating with tranexamic acid or factor concentrate. The guidelines also advise against treating with desmopressin in the absence of a trial. In a section of “good practice statements,” the guidelines indicate that using desmopressin in patients with type 2B VWD is generally contraindicated, because of the risk of thrombocytopenia as a result of increased platelet binding. In addition, desmopressin is generally contraindicated in patients with active cardiovascular disease, patients with seizure disorders, patients less than 2 years old, and patients with type 1C VWD in the setting of surgery.
Antithrombotic therapy
The guideline panelists conditionally recommend antithrombotic therapy with either antiplatelet agents or anticoagulants, with an emphasis on reassessing bleeding risk throughout the course of treatment.
An accompanying good practice statement calls for individualized assessments of risks and benefits of specific antithrombotic therapies by a multidisciplinary team including hematologists, cardiovascular specialists, and the patient.
