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Von Willebrand disease in the United States: perspective from the Zimmerman program.

Annals of Blood 2018 January
This article will discuss the diagnosis and management of von Willebrand disease (VWD) in the United States and results from the Zimmerman Program, a national study of VWD. An algorithm is presented to show how we currently approach diagnostic testing for VWD, including the potential replacement of the ristocetin cofactor assay with a new von Willebrand factor (VWF)-GPIb binding assay. Results from the Zimmerman Program type 1 cohort are presented, including the findings that genetic defects in the VWF gene are most common with VWF levels <30 IU/dL, but bleeding symptoms were present across the entire cohort regardless of VWF level. Typical management of VWD patients is also discussed, including the use of desmopressin and VWF concentrates. Despite these advances, there remain several areas of VWD where more research is required to optimize treatment.

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