Only a limited number of patients with von Willebrand disease (VWD) are likely to require treatment with von Willebrand factor (VWF) concentrates, according to results from a retrospective analysis.
The retrospective, single-center study sought to describe the need for VWF concentrates in a “real-life setting,” wrote Ana Boban, MD, PhD, of the University of Zagreb (Croatia) and colleagues in a letter to the editor published in Haemophilia. The study was conducted at the Saint‐Luc University Hospital in Brussels and included all the VWD patients listed in the hospital registry from 2000 to 2015.
The researchers evaluated the necessity for VWF concentrate therapy based on VWD severity (mild, moderate, severe) and type (1, 2A, 2B, 2M, 2N, or 3), in addition to therapeutic indication. Bleeding scores were not included in the analysis.
A total of 174 patients with VWD were included in the study, which consisted of 116 females and 61 males aged 3-81 years. The majority of study participants had type 1 VWD (n = 118, 67%).
Data collected included patient demographic information, disease type and severity, responses to specific tests, and type of treatment received.
The researchers found that just 18% (n = 32) of patients within the cohort required therapy with VWF concentrates over the study period. Additionally, the team reported that a large number of patients did not require any therapy (n = 79; 45%) or were or managed with DDAVP (desmopressin) (n = 61; 64%).
“When assessing the results according to disease severity, it clearly occurred that most patients with severe disease required VWF concentrates [100%, 75%, and 100% for type 1, type 2, and type 3 VWD, respectively],” the authors wrote.
With respect to therapeutic indication, apart from prophylaxis, contraindications to DDAVP (n = 5) and unresponsiveness to DDAVP (n = 7) were absolute indications for the use of VWF concentrates among patients within the cohort.
“Our study has confirmed that the absolute indications for using VWF concentrates in VWD patients are prophylaxis, major surgeries, and nonresponsiveness/contraindications to DDAVP,” they concluded.
No funding sources were reported. The authors reported having no conflicts of interest.
SOURCE: Boban A et al. Haemophilia. 2019 May 20. doi: 10.1111/hae.13779.