Bleeding symptoms and laboratory correlation in patients with severe von Willebrand disease

A. D. Metjian, C. Wang, S. L. Sood, A. Cuker, S. M. Peterson, J. M. Soucie, Barbara A. Konkle*, Marjorie Boyd, Glen Roy, Edwin Forman, Jean Marandola, Alan C. Homans, Miriam Granat, Jeanne Lusher, Patricia Fleming, Donna DiMichele, Ilene Goldberg, Parvin Saidi, Robin Schwartz, Franklin DespositoDominique Joseph, Peter A. Kouides, Jennifer La Franco, Tom Coyle, Neiha Dhar, Joanne Porter, Mia Frank, Richard Lipton, Margaret Bosch, Rosemary P. Holmberg, Lynn Menza, Alice Cohen, Ellen White, Michael Guerrera, Chris Guelcher, Craig Kessler, Helena Jacobs, Gita Massey, Mindy Nolte, Eric Werner, Kim Stewart, Margaret Ragni, Kristen Jaworski, John S. Rogers, Sylvia Webber, Steven Jubelirer, Donna Arden, Jim Casella, Trish Underland, Karen Panckeri

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

27 Scopus citations


Type 3 von Willebrand disease (VWD) is a rare bleeding disorder with markedly decreased or absent von Willebrand factor (VWF) protein, accompanied by a parallel decrease in VWF function and factor VIII (FVIII) activity. The goal of this study was to describe the population of patients enrolled in the USA Centers for Disease Control Universal Data Collection (UDC) study with type 3 VWD, defined as a VWF:Ag of <10%, and to correlate bleeding symptoms with VWF and FVIII levels. Data on 150 patients were analysed. Almost all patients experienced bleeding episodes (98%) and required blood and/or factor product treatment (92%). While oral mucosal bleeding (the site of first bleed in 54%) was most common, subsequent muscle and joint bleeds were also seen (28%, 45%, respectively), and intracranial haemorrhage occurred in 8% of individuals. Mean age of first bleed was lower in those with either a FVIII ≤5% or a VWF:Ag <1%. Univariate marginal model analysis showed lower levels of FVIII and VWF:Ag both predicted a higher risk of joint bleeding. Longitudinal multivariate analysis found a lower FVIII level (P = 0.03), increasing age (P < 0.0001), history of joint bleeding (P = 0.001), higher body mass index (BMI) (P < 0.0001), and use of home infusion (P = 0.02) were all negatively associated with joint mobility. Low levels of VWF:Ag (P = 0.003) and male sex (P = 0.007) were also negatively associated with joint function. This study documents the strong bleeding phenotype in severe VWD and provides data to help target therapy, including prophylaxis, for patients most at risk of bleeding complications.

Original languageEnglish (US)
Pages (from-to)918-925
Number of pages8
Issue number4
StatePublished - 2009


  • Arthropathy
  • Factor VIII
  • Haemorrhage
  • Von Willebrand disease
  • Von Willebrand factor

ASJC Scopus subject areas

  • Genetics(clinical)
  • Hematology


Dive into the research topics of 'Bleeding symptoms and laboratory correlation in patients with severe von Willebrand disease'. Together they form a unique fingerprint.

Cite this