Journal of Blood Disorders & Transfusion

Abstract

Treatment and Management Patterns of Patients with Von Willebrand Disease in the United States

Cindy A Leissinger and Diane Nugent

Background: Few data exist regarding patient management strategies for von Willebrand disease (VWD), the most frequently inherited bleeding disorder. The objective of this study was to retrospectively assess patterns of VWD patient management in the United States.

Methods: 41 physicians were recruited using the Centers for Disease Control and Prevention’s list of hemophilia treatment centers (HTCs) and the American Medical Association’s physician database (16 HTC-, 25 non-HTC-affiliated). Patient charts were randomly sampled by quota method.

Results: Charts of 225 VWD patients treated from 1/2005–12/2007 were reviewed (94/74/57 for VWD types 1/2/3, respectively). The proportion of white patients was higher at non-HTCs than HTCs, and among type 1 than type 2 patients. Females comprised 65% of patients, (77% of type 1, 55% of type 2, and 58% of type 3 patients) However, bleeding episodes requiring treatment occurred in 53% of male but only 36% of female patients. The proportion of patients treated with desmopressin or von Willebrand factor/factor VIII (VWF/FVIII) concentrates for bleeding (148 events) and surgery (140 episodes) within VWD type were: 31% and 47% of type 1, 55% and 36% of type 2, 44% and 39% of type 3 patients, respectively. Of the 140 surgical events requiring desmopressin or VWF/FVIII concentrates, 61% occurred in the non-HTC setting.

Conclusion: In addition to revealing interesting disparities in VWD patient demographics, these data indicate VWD patients receive care generally aligned with treatment guidelines, with the exception of the number of surgeries performed outside of the HTC setting, where access to laboratory monitoring of factor levels may be limited.