Type 3 von Willebrand disease: assessment of complications and approaches to treatment -- results of a patient and Hemophilia Treatment Center Survey in the United States.

It has been reported many times that the optimal treatment of patients with von Willebrand disease (VWD) has yet to be determined. To assist in achieving this goal, the aim of this survey was as follows: to collect information on current treatment practices; to determine the impact of the disease in type 3 VWD patients in the USA and to gain insight into the perceived and actual role that prophylaxis therapy plays in patient management. Nurse coordinators of the 146 federally funded Hemophilia Treatment Centers (HTCs) in the USA were contacted and informed of the survey and its goals. Those caring for type 3 VWD patients and willing to participate in the survey were mailed two questionnaires. The first questionnaire addressed the overall VWD patient population at their centre and asked questions relating to treatment practices. The second questionnaire focused on the impact that type 3 VWD has upon a patient's life. Thirty-four centres responded to the survey, providing a 48% response rate within the available pool of those centres treating type 3 VWD patients. Seventy-nine patients, with a mean age of 24.2 years, reported their primary causes of bleeding as; epistaxis (53.9%); joint bleeds (40.9%); dental bleeds (27.6%) and heavy menstrual bleeding (23.7%). There were a mean number of 9.2 reported non-life-threatening bleeds per year and a mean of 0.7 reported life-threatening bleeds per year. Forty percent reported lost days from school or work, caused by bleeds or other VWD complications. Sixty-two percent reported no joint damage. Regarding current treatment practices, 88.6% of respondents stated that their centre treated on demand and 13.9% used a prophylactic treatment regimen. Seventy-three percent of respondents felt that prophylaxis was either very or extremely helpful. This survey helps to document current treatment practices and morbidity exhibited by type 3 VWD patients. The results highlight the discrepancy between the use of prophylactic therapy, its perceived value and the level of joint damage and other morbidity experienced in this population.