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

BMI is an important determinant of VWF and FVIII levels and bleeding phenotype in patients with von Willebrand disease

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Social participation is reduced in type 3 Von Willebrand disease patients and in patients with a severe bleeding phenotype

INTRODUCTION: The negative impact of haemophilia on social participation is well established in previous studies, however, the impact of Von Willebrand disease (VWD) on social participation has not been studied. AIM: To compare the social participation of a large cohort of VWD patients in the Netherlands with the general Dutch population. In addition, to identify factors associated with social participation in VWD. METHODS: Patients participating in the "Willebrand in the Netherlands" study completed an extensive questionnaire on educational level, absenteeism from school or work, and occupational disabilities. RESULTS: Seven-hundred and eighty-eight VWD patients were included (mean age 38.9 years, 59.5% females), of whom 136 children < 16 years. Adult patients with type 3 VWD more often had a low educational level (52.9%) compared to type 1 (40.2%), type 2 VWD (36.8%) and the general population (36.4%) (p = .005). Moreover, in patients aged ≥16 years the days lost from school and/or work in the year prior to study inclusion differed significantly between the VWD types (p = .011). Using negative binomial regression analysis, the occurrence of bleeding episodes requiring treatment in the year preceding study inclusion was significantly associated with the number of days lost from school and/or work among patients aged ≥16 years. Multivariable logistic regression analysis showed that a higher total bleeding score, older age and presence of at least one comorbidity were significantly associated with occupational disability in patients aged ≥16 years. CONCLUSION: Our study shows that social participation was lower in type 3 VWD and VWD patients with a more severe bleeding phenotype

THE OCCURRENCE AND IMPACT OF JOINT BLEEDS IN VON WILLEBRAND DISEASE

Clinical epidemiolog

The occurrence and impact of joint bleeds in Von Willebrand disease

Background: Von Willebrand disease (VWD) is a heterogeneous inherited bleeding disorder that affects up to 1% of the population. Joint bleeds are not predominant, but have been reported to occur in 8-45% of patients with VWD, especially in those with a more severe phenotype. Joint bleeds can lead to structural joint damage. The most severe type 3 VWD patients develop similar rates of joint range of motion limitation over time as moderate hemophilia A patients. However, the severity, onset and impact of joint bleeds and its complications in VWD patients are largely unknown. Aims: The aim of this study is to assess the incidence, onset and treatment of joint bleeds and its impact on quality of life and joint integrity in moderate and severe VWD. Methods: In the Willebrand in the Netherlands (WIN) study 804 moderate and severe VWD patients (VWF activity ≤30U/dL) completed a comprehensive questionnaire after giving informed consent. We quantified joint bleed occurrence as reported in the questionnaires and examined the medical files for documentation on joint bleeds and joint problems from all patients who had reported treatment for joint bleeds with desmopressin or clotting factor concentrate (CFC) and from as many patients without joint bleeds for comparison, matched for gender, age, FVIII and VWF activity. Results: Twenty three percent of the patients (184/804) self-reported joint bleeds in the questionnaire, mostly in the knee, followed by the ankle and elbow. These patients had more severe VWD (type 3 VWD 12% vs. 4%, p50% in 14/16 VWD patients with joint bleeds who started CFC prophylaxis. We found documented X-ray joint damage in 44% of the patients with joint bleeds compared to 11% of the controls (

Joint assessment in von Willebrand disease. Validation of the Haemophilia Joint Health score and Haemophilia Activities List

Item does not contain fulltextAssessment of clinical outcome after joint bleeding is essential to identify joint damage and optimise treatment, to prevent disability. However, disease-specific tools to assess the musculoskeletal status in patients with von Willebrand disease (VWD) are lacking. We aimed to determine validity and reliability of the Haemophilia Joint Health Score (HJHS) and Haemophilia Activities List (HAL) in patients with Von Willebrand disease (VWD). Ninety-six patients with VWD were included (mean age 46 years) of whom 27 had more than five documented joint bleeds. The HJHS was performed in all patients and all patients completed the HAL and Impact on Participation and Autonomy (IPA) questionnaires. Health-related quality of life (SF36) results were obtained from the prior 'Willebrand in the Netherlands' study. Joint X-rays of knees, elbows and ankles were scored according to Pettersson (PS). Internal consistency of the HJHS (Cronbach's alpha (alpha)=0.75) and HAL (alpha=0.89) were good. Inter-observer agreement of the HJHS was good (ICC 0.84; Limits of Agreement +/- 10.3). The HJHS showed acceptable correlation with the X-ray PS (Spearman's r (rs)>0.60 all joints) and HAL (rs=0.71). The HAL also showed acceptable correlation with the SF36 physical functioning (rs=0.65) and IPA (rs=0.69). Hypothesis testing showed adequate discriminative power of both instruments: in patients with a history of >5 versus </= 5 joint bleeds (median HJHS 10 vs 2 (p<0.01); median HAL 77 vs 98 (p<0.01)), independent from age. In conclusion, both the HJHS and HAL are feasible to assess clinical outcome after joint bleeds in VWD