Introduction

In VWD, bleeding tendency is caused by decreased levels or by inappropriate function of VWF, and sometimes in addition low levels of coagulation FVIII. The deficiencies can in general be corrected either by stimulating the release of endogenous VWF and FVIII with desmopressin (DDAVP) in VWD type 1, or by substitution with a VWF/FVIII concentrate in type 2 and 3. DDAVP may temporarily normalize hemostasis if functional levels of VWF and FVIII can be reached by endogenous release. VWF/FVIII or purified VWF concentrates are to be used when DDAVP is not an alternative.

Plasma-derived concentrates carry a potential risk of transmission of infectious agents, but the risk has been negligible with the current safety actions, including specific protein purification steps combined with pathogen inactivation procedures. Recombinant VWF has been produced but is not yet available in all the Nordic countries. Cost is an issue, as these concentrates are expensive. On the other hand, safety may be compromised with DDAVP due to its modest or short-acting effects, side effects, mainly caused by the strong antidiuretic mechanism that may limit its use. Antifibrinolytic treatment (tranexamic acid) is an important adjuvant to DDAVP or concentrates, or as a single hemostatic agent, especially in connection with mucous membrane bleeds. Oral contraceptive pills and progesterone releasing IUD may be used as treatment of menorrhagia in females with VWD, as they typically alleviate menstrual blood loss.

VWD is a complex condition to manage, and it is important that the patient is treated at a designated European Hemophilia center with access to a multidisciplinary team of specialists. The European association for haemophilia and allied disorders (EAHAD) is a European association of hemophilia and allied disorders, operating since 2007. EUHANET is an EAHAD-affiliated network that provides uniform recommendations for management of hemophilia and allied disorders (https://www.euhanet.org). It has defined the services to be provided by comprehensive care centers (EHCCC) and hemophilia treatment centers (EHC). A multidisciplinary team should take care of the clinical decision making, typically in association with anesthesia, surgery, pregnancy, and delivery. Interaction between the clinicians and the specific laboratory needs to be available for 24/7 in the EHCCC. Also, a safety surveillance register of EUHASS is prospectively running to capture all side effects and complications, including mortality among patients with type 1 severe (<15 IU/dL VWF:RiCo), type 2, type 3 or type unknown.