Treatment of the acquired von Willebrand syndrome

Abstract

Acquired von Willebrand syndrome (aVWS) accounts for 22% of patients with abnormal von Willebrand factor. Most patients with known pathophysiological mechanisms suffer from cardiovascular, myeloproliferative and lymphoproliferative disorders. Less frequent associations are of autoimmune origin, due to hyperfibrinolysis, adsorption to tumor cells, reduced synthesis and prolonged circulation. The mechanisms leading to aVWS is hitherto not known in patients with liver and kidney diseases, drug use, glycogen storage disease, virus infections and at least 18 other disease entities. Diagnosis is complicated by the battery of tests needed, and their inherent rather low sensitivity and specificity for aVWS. Thus, even in acute bleeding situations it may take days until a firm diagnosis is settled and specific therapies can be initiated. The main aim is to shed more light onto this, compared with inherited von Willebrand disease, rare disease which affects at least 2–3% of the older population.

Keywords:

• acquired von Willebrand syndrome
• cardiovascular diseases
• hyperfibrinolysis
• intravenous immunoglobulins
• lymphoproliferative disorders
• myeloproliferative disorders

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending or royalties.

No writing assistance was utilized in the production of this manuscript.

Key issues

• Up to now no large prospective randomized studies can be found in the literature concerning frequency of the acquired von Willebrand syndrome (aVWS) and therapeutic options in the different disease entities.

• The increasing literature on this topic may lead to an increasing awareness of the aVWS.

• A battery of tests has to be used for the correct diagnosis and classification of aVWS.

• The three main goals are to control acute bleeds, to prevent bleeding in high-risk situations and to obtain long-term remission.

• There are at least eight specific drug therapy options and only a firm diagnosis of the aVWS and the underlying disease allows a successful treatment of the patients.

• In a subset of patients with a loss of the large multimers (type 2), angiodysplasia of the colon leads a considerable morbidity, and rarely a substantial need for red cell concentrates. Anti-angiogenic drugs are available, but most of them have intolerable side effects.

• The largest groups of patients stem from patients with cardiovascular disorders, myeloproliferative diseases and lymphoproliferative disorders.

Notes