How I manage severe von Willebrand disease.

Von Willebrand disease (VWD) is the most common inherited bleeding disorder. Most patients with mild and moderate VWD can be treated effectively with desmopressin. The management of severe VWD patients, mostly affected by type 2 and type 3 disease, can be challenging. In this article we review the current diagnosis and treatment of severe VWD patients. We will also discuss the management of severe VWD patients in specific situations, such as pregnancy, delivery, patients developing alloantibodies against von Willebrand factor and VWD patients with recurrent gastrointestinal bleeding. Moreover, we review emerging treatments that may be applied in future management of patients with severe VWD.

Hemorrhagic symptoms and bleeding risk in obligatory carriers of type 3 von Willebrand disease in southern Iran.

The objective of the present study was to compare old and new bleeding scores in patients with type-3 von Willebrand disease (vWD), obligatory carriers and normal controls, and to compare the ability of bleeding scores vs. clinical and laboratory data to predict bleeding after surgery. We identified 15 patients from 12 families who had type 3 vWD. Normal controls were matched to carriers by sex and age. Two physician-administered standardized questionnaires were used to evaluate old and new bleeding symptoms. Scores for old symptoms were the same in carriers and control participants (median score 0.00 vs. 0.00, P < 0.001), and patients with vWD had a significantly higher bleeding score than carriers (median 10.00 vs. 0.00, P < 0.001). Scores for new symptoms were higher in carriers than in control participants (median score -1.00 vs. -2.00, P < 0.001), and patients had a significantly higher bleeding score than carriers (median 14.00 vs. -1.00, P < 0.001). The clinical situations associated with increased bleeding risk (old symptoms) in patients with type 3 vWD compared to obligatory carriers were epistaxis [odds ratio (OR) = 175.5; 95% confidence interval (CI) 14.55-2116.69; P < 0.001], cutaneous symptoms (OR = 108; 95% CI 10.16-1147.39; P < 0.001) and hemarthrosis (OR = 19.5%; 95% CI 4.32-156.46; P < 0.001). The clinical situations associated with increased bleeding risk according to scores for new symptoms in patients with type 3 vWD compared to obligatory carriers were epistaxis (OR = 175.5; 95% CI 14.55-2116.69; P < 0.001), cutaneous symptoms (OR = 52; 95% CI 7.65-353.09; P < 0.001) and bleeding from minor wounds (OR = 74.25; 95% CI 7.43-741.118; P < 0.001). The three groups differed significantly in the severity of epistaxis and cutaneous bleeding according to scores for new and old symptoms. The new bleeding score was more reliable than the old bleeding score in predicting bleeding after invasive procedure.

Elective surgery in patients with congenital coagulopathies and inhibitors: experience of the National Haemophilia Centre of Venezuela.

Elective surgery in patients with congenital haemophilia with inhibitors carries a high risk of bleeding. However, inhibitor patients also have a high risk of haemarthroses and other orthopaedic complications, and surgery could improve their quality of life. Successful elective surgery has been reported in inhibitor patients under haemostatic cover with plasma-derived activated prothrombin complex concentrate (pd-aPCC) or recombinant activated factor VII (rFVIIa). Recombinant FVIIa has recently become available in Venezuela and, unlike pd-aPCC, has not been associated with an anamnestic response. The aim of this study was to assess our experience using rFVIIa as a first-line and sustained treatment in elective invasive surgical procedures at the National Haemophilia Centre in Venezuela. Surgical procedures were classified as major or minor, under haemostatic cover with rFVIIa. A total of 13 patients (12 with haemophilia A with high-responding inhibitors and one with von Willebrand's disease type 3) underwent a total of 19 surgical procedures under rFVIIa cover. Thirteen procedures were classified as major surgeries. Intraoperative haemostasis was achieved in the majority of patients. Only two patients required an additional dose of rFVIIa, at 30 min and 75 min, respectively, with good results. Postoperative haemostasis was considered effective in 16 of 18 (89%) of the procedures in haemophilia A patients. Treatment was considered to be ineffective in two patients because of excessive postoperative bleeding. Data from the study provide no safety concerns, and demonstrate that rFVIIa provides effective haemostatic cover in elective surgery in patients with inhibitors; research is ongoing to determine the optimal dose for such procedures.