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, Lisa Palumbo, Hernan Sabio, Anita Smith, Vivek Sharma, Patricia Ashby, Vlad Radulescu, Wahid Hanna, Cherys Zimmerman, Anne Neff, Mary G. Hudson, Ulrike Reiss, Robin Grant, Nigel Key, Brenda Nielsen, Amy Dunn, Karen Gutting, Raymond Watts, Mary Catherine Noa, Tom Abshire, Corinthian Bryant, Afshin Ameri, Valerie Crenshaw, Philip Kuriakose, Angela Lambing, Glenn Heggie, Sue Kovats-Bell, Susumu Inoue, JoAnn A. Ruff, Deanna Mitchell, Elizabeth Sandon-Kleiboer, Eric Kraut, Leslie Witkoff, Ralph Gruppo, Anne Chambers, Bryce Kerlin, Claudia Lupia, James French, Sandy Hibner, Amy Shapiro, Sarah May, Sarah Alexander, Steve Hopewell, Paula Bockenstedt, Steven Pipe, Diana Mathis, Anaadriana Zakarija, Sandy Harris, Alexis Thompson, Geoffrey A. Allen, Ashley T. Brummel, Michael Tarantino, Angie Riedel, Mark Reding, Susan Curoe, Rajiv Pruthi, Kirstin Schmidt, Katie Kralovetz, Jay Charles, Carol Diamond, Paulette Drozdowicz, Joan C. Gill, Jodi Haar, W. Keith Hoots, Valerie Gonzalez, Cindy Leissinger, Cecilia V. Schmidt, Kimo Stine, Glenda Eckert, Charles Sexauer, Felicia Kiplinger, Patricia Amerson, Erica Johnson, George Buchanan, Roberto Torres, Jorge DiPaola, Mike Lammer, Brian Wicklund, Debbie Nelson, William D. Haire, Elizabeth Hanlon, Marilyn Manco-Johnson, Sue Geraghty, Hassan Yaish, Jennifer Green, Guy Young, Kathy McGinty, Kenneth Herbst, Cathy Glass, Bertil Glader, JeanMarie M. Zoland, Desiree Medeiros, Dee Ann Omatsu, Jonathan M Ducore, Muriel Herr, Marion Koerper, Susan Karp, Amy E. Lovejoy, Katherine Farrow, Nadia Ewing, Lisa Pullens, Lee Meadows, Angela Tackney, Robert Mignacca, Frances Patterson, Jonathan Bernstein, Becki Berkowitz, Arthur R. Thompson, Cheryl Brower, Michael Recht, Karen Granger, Laura Schulz, Angela Stewart

Research output: Contribution to journalArticle

22 Scopus citations

Abstract

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
JournalHaemophilia
Volume15
Issue number4
DOIs
StatePublished - 2009

Keywords

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

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

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    Metjian, A. D., Wang, C., Sood, S. L., Cuker, A., Peterson, S. M., Soucie, J. M., Konkle, B. A., Boyd, M., Roy, G., Forman, E., Marandola, J., Homans, A. C., Granat, M., Lusher, J., Fleming, P., DiMichele, D., Goldberg, I., Saidi, P., Schwartz, R., ... Stewart, A. (2009). Bleeding symptoms and laboratory correlation in patients with severe von Willebrand disease. Haemophilia, 15(4), 918-925. https://doi.org/10.1111/j.1365-2516.2009.02025.x