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, Jo Ann 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, Jean Marie Zoland, Desiree Medeiros, Dee Ann Omatsu, Jonathan 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

21 Citations (Scopus)

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 - Jul 10 2009

Fingerprint

von Willebrand Diseases
Hemorrhage
von Willebrand Factor
Joints
Type 3 Von Willebrand's Disease
Factor VIII
Intracranial Hemorrhages
Centers for Disease Control and Prevention (U.S.)
Body Mass Index
Multivariate Analysis
Phenotype
Muscles
Therapeutics

All Science Journal Classification (ASJC) codes

  • Hematology
  • Genetics(clinical)

Cite this

Metjian, A. D., Wang, C., Sood, S. L., Cuker, A., Peterson, S. M., Soucie, J. M., ... 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

Bleeding symptoms and laboratory correlation in patients with severe von Willebrand disease. / Metjian, A. D.; Wang, C.; Sood, S. L.; Cuker, A.; Peterson, S. M.; Soucie, J. M.; Konkle, Barbara A.; Boyd, Marjorie; Roy, Glen; Forman, Edwin; Marandola, Jean; Homans, Alan C.; Granat, Miriam; Lusher, Jeanne; Fleming, Patricia; DiMichele, Donna; Goldberg, Ilene; Saidi, Parvin; Schwartz, Robin; Desposito, Franklin; Joseph, Dominique; Kouides, Peter A.; La Franco, Jennifer; Coyle, Tom; Dhar, Neiha; Porter, Joanne; Frank, Mia; Lipton, Richard; Bosch, Margaret; Holmberg, Rosemary P.; Menza, Lynn; Cohen, Alice; White, Ellen; Guerrera, Michael; Guelcher, Chris; Kessler, Craig; Jacobs, Helena; Massey, Gita; Nolte, Mindy; Werner, Eric; Stewart, Kim; Ragni, Margaret; Jaworski, Kristen; Rogers, John S.; Webber, Sylvia; Jubelirer, Steven; Arden, Donna; Casella, Jim; Underland, Trish; Panckeri, Karen; Palumbo, Lisa; Sabio, Hernan; Smith, Anita; Sharma, Vivek; Ashby, Patricia; Radulescu, Vlad; Hanna, Wahid; Zimmerman, Cherys; Neff, Anne; Hudson, Mary G.; Reiss, Ulrike; Grant, Robin; Key, Nigel; Nielsen, Brenda; Dunn, Amy; Gutting, Karen; Watts, Raymond; Noa, Mary Catherine; Abshire, Tom; Bryant, Corinthian; Ameri, Afshin; Crenshaw, Valerie; Kuriakose, Philip; Lambing, Angela; Heggie, Glenn; Kovats-Bell, Sue; Inoue, Susumu; Ruff, Jo Ann; Mitchell, Deanna; Sandon-Kleiboer, Elizabeth; Kraut, Eric; Witkoff, Leslie; Gruppo, Ralph; Chambers, Anne; Kerlin, Bryce; Lupia, Claudia; French, James; Hibner, Sandy; Shapiro, Amy; May, Sarah; Alexander, Sarah; Hopewell, Steve; Bockenstedt, Paula; Pipe, Steven; Mathis, Diana; Zakarija, Anaadriana; Harris, Sandy; Thompson, Alexis; Allen, Geoffrey A.; Brummel, Ashley T.; Tarantino, Michael; Riedel, Angie; Reding, Mark; Curoe, Susan; Pruthi, Rajiv; Schmidt, Kirstin; Kralovetz, Katie; Charles, Jay; Diamond, Carol; Drozdowicz, Paulette; Gill, Joan C.; Haar, Jodi; Hoots, W. Keith; Gonzalez, Valerie; Leissinger, Cindy; Schmidt, Cecilia V.; Stine, Kimo; Eckert, Glenda; Sexauer, Charles; Kiplinger, Felicia; Amerson, Patricia; Johnson, Erica; Buchanan, George; Torres, Roberto; DiPaola, Jorge; Lammer, Mike; Wicklund, Brian; Nelson, Debbie; Haire, William D.; Hanlon, Elizabeth; Manco-Johnson, Marilyn; Geraghty, Sue; Yaish, Hassan; Green, Jennifer; Young, Guy; McGinty, Kathy; Herbst, Kenneth; Glass, Cathy; Glader, Bertil; Zoland, Jean Marie; Medeiros, Desiree; Omatsu, Dee Ann; Ducore, Jonathan; Herr, Muriel; Koerper, Marion; Karp, Susan; Lovejoy, Amy E.; Farrow, Katherine; Ewing, Nadia; Pullens, Lisa; Meadows, Lee; Tackney, Angela; Mignacca, Robert; Patterson, Frances; Bernstein, Jonathan; Berkowitz, Becki; Thompson, Arthur R.; Brower, Cheryl; Recht, Michael; Granger, Karen; Schulz, Laura; Stewart, Angela.

In: Haemophilia, Vol. 15, No. 4, 10.07.2009, p. 918-925.

Research output: Contribution to journalArticle

Metjian, AD, Wang, C, Sood, SL, Cuker, A, Peterson, SM, Soucie, JM, Konkle, BA, Boyd, M, Roy, G, Forman, E, Marandola, J, Homans, AC, Granat, M, Lusher, J, Fleming, P, DiMichele, D, Goldberg, I, Saidi, P, Schwartz, R, Desposito, F, Joseph, D, Kouides, PA, La Franco, J, Coyle, T, Dhar, N, Porter, J, Frank, M, Lipton, R, Bosch, M, Holmberg, RP, Menza, L, Cohen, A, White, E, Guerrera, M, Guelcher, C, Kessler, C, Jacobs, H, Massey, G, Nolte, M, Werner, E, Stewart, K, Ragni, M, Jaworski, K, Rogers, JS, Webber, S, Jubelirer, S, Arden, D, Casella, J, Underland, T, Panckeri, K, Palumbo, L, Sabio, H, Smith, A, Sharma, V, Ashby, P, Radulescu, V, Hanna, W, Zimmerman, C, Neff, A, Hudson, MG, Reiss, U, Grant, R, Key, N, Nielsen, B, Dunn, A, Gutting, K, Watts, R, Noa, MC, Abshire, T, Bryant, C, Ameri, A, Crenshaw, V, Kuriakose, P, Lambing, A, Heggie, G, Kovats-Bell, S, Inoue, S, Ruff, JA, Mitchell, D, Sandon-Kleiboer, E, Kraut, E, Witkoff, L, Gruppo, R, Chambers, A, Kerlin, B, Lupia, C, French, J, Hibner, S, Shapiro, A, May, S, Alexander, S, Hopewell, S, Bockenstedt, P, Pipe, S, Mathis, D, Zakarija, A, Harris, S, Thompson, A, Allen, GA, Brummel, AT, Tarantino, M, Riedel, A, Reding, M, Curoe, S, Pruthi, R, Schmidt, K, Kralovetz, K, Charles, J, Diamond, C, Drozdowicz, P, Gill, JC, Haar, J, Hoots, WK, Gonzalez, V, Leissinger, C, Schmidt, CV, Stine, K, Eckert, G, Sexauer, C, Kiplinger, F, Amerson, P, Johnson, E, Buchanan, G, Torres, R, DiPaola, J, Lammer, M, Wicklund, B, Nelson, D, Haire, WD, Hanlon, E, Manco-Johnson, M, Geraghty, S, Yaish, H, Green, J, Young, G, McGinty, K, Herbst, K, Glass, C, Glader, B, Zoland, JM, Medeiros, D, Omatsu, DA, Ducore, J, Herr, M, Koerper, M, Karp, S, Lovejoy, AE, Farrow, K, Ewing, N, Pullens, L, Meadows, L, Tackney, A, Mignacca, R, Patterson, F, Bernstein, J, Berkowitz, B, Thompson, AR, Brower, C, Recht, M, Granger, K, Schulz, L & Stewart, A 2009, 'Bleeding symptoms and laboratory correlation in patients with severe von Willebrand disease', Haemophilia, vol. 15, no. 4, pp. 918-925. https://doi.org/10.1111/j.1365-2516.2009.02025.x
Metjian, A. D. ; Wang, C. ; Sood, S. L. ; Cuker, A. ; Peterson, S. M. ; Soucie, J. M. ; Konkle, Barbara A. ; Boyd, Marjorie ; Roy, Glen ; Forman, Edwin ; Marandola, Jean ; Homans, Alan C. ; Granat, Miriam ; Lusher, Jeanne ; Fleming, Patricia ; DiMichele, Donna ; Goldberg, Ilene ; Saidi, Parvin ; Schwartz, Robin ; Desposito, Franklin ; Joseph, Dominique ; Kouides, Peter A. ; La Franco, Jennifer ; Coyle, Tom ; Dhar, Neiha ; Porter, Joanne ; Frank, Mia ; Lipton, Richard ; Bosch, Margaret ; Holmberg, Rosemary P. ; Menza, Lynn ; Cohen, Alice ; White, Ellen ; Guerrera, Michael ; Guelcher, Chris ; Kessler, Craig ; Jacobs, Helena ; Massey, Gita ; Nolte, Mindy ; Werner, Eric ; Stewart, Kim ; Ragni, Margaret ; Jaworski, Kristen ; Rogers, John S. ; Webber, Sylvia ; Jubelirer, Steven ; Arden, Donna ; Casella, Jim ; Underland, Trish ; Panckeri, Karen ; Palumbo, Lisa ; Sabio, Hernan ; Smith, Anita ; Sharma, Vivek ; Ashby, Patricia ; Radulescu, Vlad ; Hanna, Wahid ; Zimmerman, Cherys ; Neff, Anne ; Hudson, Mary G. ; Reiss, Ulrike ; Grant, Robin ; Key, Nigel ; Nielsen, Brenda ; Dunn, Amy ; Gutting, Karen ; Watts, Raymond ; Noa, Mary Catherine ; Abshire, Tom ; Bryant, Corinthian ; Ameri, Afshin ; Crenshaw, Valerie ; Kuriakose, Philip ; Lambing, Angela ; Heggie, Glenn ; Kovats-Bell, Sue ; Inoue, Susumu ; Ruff, Jo Ann ; Mitchell, Deanna ; Sandon-Kleiboer, Elizabeth ; Kraut, Eric ; Witkoff, Leslie ; Gruppo, Ralph ; Chambers, Anne ; Kerlin, Bryce ; Lupia, Claudia ; French, James ; Hibner, Sandy ; Shapiro, Amy ; May, Sarah ; Alexander, Sarah ; Hopewell, Steve ; Bockenstedt, Paula ; Pipe, Steven ; Mathis, Diana ; Zakarija, Anaadriana ; Harris, Sandy ; Thompson, Alexis ; Allen, Geoffrey A. ; Brummel, Ashley T. ; Tarantino, Michael ; Riedel, Angie ; Reding, Mark ; Curoe, Susan ; Pruthi, Rajiv ; Schmidt, Kirstin ; Kralovetz, Katie ; Charles, Jay ; Diamond, Carol ; Drozdowicz, Paulette ; Gill, Joan C. ; Haar, Jodi ; Hoots, W. Keith ; Gonzalez, Valerie ; Leissinger, Cindy ; Schmidt, Cecilia V. ; Stine, Kimo ; Eckert, Glenda ; Sexauer, Charles ; Kiplinger, Felicia ; Amerson, Patricia ; Johnson, Erica ; Buchanan, George ; Torres, Roberto ; DiPaola, Jorge ; Lammer, Mike ; Wicklund, Brian ; Nelson, Debbie ; Haire, William D. ; Hanlon, Elizabeth ; Manco-Johnson, Marilyn ; Geraghty, Sue ; Yaish, Hassan ; Green, Jennifer ; Young, Guy ; McGinty, Kathy ; Herbst, Kenneth ; Glass, Cathy ; Glader, Bertil ; Zoland, Jean Marie ; Medeiros, Desiree ; Omatsu, Dee Ann ; Ducore, Jonathan ; Herr, Muriel ; Koerper, Marion ; Karp, Susan ; Lovejoy, Amy E. ; Farrow, Katherine ; Ewing, Nadia ; Pullens, Lisa ; Meadows, Lee ; Tackney, Angela ; Mignacca, Robert ; Patterson, Frances ; Bernstein, Jonathan ; Berkowitz, Becki ; Thompson, Arthur R. ; Brower, Cheryl ; Recht, Michael ; Granger, Karen ; Schulz, Laura ; Stewart, Angela. / Bleeding symptoms and laboratory correlation in patients with severe von Willebrand disease. In: Haemophilia. 2009 ; Vol. 15, No. 4. pp. 918-925.
@article{4d14d7782d7346b6985274f453bf536e,
title = "Bleeding symptoms and laboratory correlation in patients with severe von Willebrand disease",
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.",
author = "Metjian, {A. D.} and C. Wang and Sood, {S. L.} and A. Cuker and Peterson, {S. M.} and Soucie, {J. M.} and Konkle, {Barbara A.} and Marjorie Boyd and Glen Roy and Edwin Forman and Jean Marandola and Homans, {Alan C.} and Miriam Granat and Jeanne Lusher and Patricia Fleming and Donna DiMichele and Ilene Goldberg and Parvin Saidi and Robin Schwartz and Franklin Desposito and Dominique Joseph and Kouides, {Peter A.} and {La Franco}, Jennifer and Tom Coyle and Neiha Dhar and Joanne Porter and Mia Frank and Richard Lipton and Margaret Bosch and Holmberg, {Rosemary P.} and Lynn Menza and Alice Cohen and Ellen White and Michael Guerrera and Chris Guelcher and Craig Kessler and Helena Jacobs and Gita Massey and Mindy Nolte and Eric Werner and Kim Stewart and Margaret Ragni and Kristen Jaworski and Rogers, {John S.} and Sylvia Webber and Steven Jubelirer and Donna Arden and Jim Casella and Trish Underland and Karen Panckeri and Lisa Palumbo and Hernan Sabio and Anita Smith and Vivek Sharma and Patricia Ashby and Vlad Radulescu and Wahid Hanna and Cherys Zimmerman and Anne Neff and Hudson, {Mary G.} and Ulrike Reiss and Robin Grant and Nigel Key and Brenda Nielsen and Amy Dunn and Karen Gutting and Raymond Watts and Noa, {Mary Catherine} and Tom Abshire and Corinthian Bryant and Afshin Ameri and Valerie Crenshaw and Philip Kuriakose and Angela Lambing and Glenn Heggie and Sue Kovats-Bell and Susumu Inoue and Ruff, {Jo Ann} and Deanna Mitchell and Elizabeth Sandon-Kleiboer and Eric Kraut and Leslie Witkoff and Ralph Gruppo and Anne Chambers and Bryce Kerlin and Claudia Lupia and James French and Sandy Hibner and Amy Shapiro and Sarah May and Sarah Alexander and Steve Hopewell and Paula Bockenstedt and Steven Pipe and Diana Mathis and Anaadriana Zakarija and Sandy Harris and Alexis Thompson and Allen, {Geoffrey A.} and Brummel, {Ashley T.} and Michael Tarantino and Angie Riedel and Mark Reding and Susan Curoe and Rajiv Pruthi and Kirstin Schmidt and Katie Kralovetz and Jay Charles and Carol Diamond and Paulette Drozdowicz and Gill, {Joan C.} and Jodi Haar and Hoots, {W. Keith} and Valerie Gonzalez and Cindy Leissinger and Schmidt, {Cecilia V.} and Kimo Stine and Glenda Eckert and Charles Sexauer and Felicia Kiplinger and Patricia Amerson and Erica Johnson and George Buchanan and Roberto Torres and Jorge DiPaola and Mike Lammer and Brian Wicklund and Debbie Nelson and Haire, {William D.} and Elizabeth Hanlon and Marilyn Manco-Johnson and Sue Geraghty and Hassan Yaish and Jennifer Green and Guy Young and Kathy McGinty and Kenneth Herbst and Cathy Glass and Bertil Glader and Zoland, {Jean Marie} and Desiree Medeiros and Omatsu, {Dee Ann} and Jonathan Ducore and Muriel Herr and Marion Koerper and Susan Karp and Lovejoy, {Amy E.} and Katherine Farrow and Nadia Ewing and Lisa Pullens and Lee Meadows and Angela Tackney and Robert Mignacca and Frances Patterson and Jonathan Bernstein and Becki Berkowitz and Thompson, {Arthur R.} and Cheryl Brower and Michael Recht and Karen Granger and Laura Schulz and Angela Stewart",
year = "2009",
month = "7",
day = "10",
doi = "10.1111/j.1365-2516.2009.02025.x",
language = "English (US)",
volume = "15",
pages = "918--925",
journal = "Haemophilia",
issn = "1351-8216",
publisher = "Wiley-Blackwell",
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TY - JOUR

T1 - Bleeding symptoms and laboratory correlation in patients with severe von Willebrand disease

AU - Metjian, A. D.

AU - Wang, C.

AU - Sood, S. L.

AU - Cuker, A.

AU - Peterson, S. M.

AU - Soucie, J. M.

AU - Konkle, Barbara A.

AU - Boyd, Marjorie

AU - Roy, Glen

AU - Forman, Edwin

AU - Marandola, Jean

AU - Homans, Alan C.

AU - Granat, Miriam

AU - Lusher, Jeanne

AU - Fleming, Patricia

AU - DiMichele, Donna

AU - Goldberg, Ilene

AU - Saidi, Parvin

AU - Schwartz, Robin

AU - Desposito, Franklin

AU - Joseph, Dominique

AU - Kouides, Peter A.

AU - La Franco, Jennifer

AU - Coyle, Tom

AU - Dhar, Neiha

AU - Porter, Joanne

AU - Frank, Mia

AU - Lipton, Richard

AU - Bosch, Margaret

AU - Holmberg, Rosemary P.

AU - Menza, Lynn

AU - Cohen, Alice

AU - White, Ellen

AU - Guerrera, Michael

AU - Guelcher, Chris

AU - Kessler, Craig

AU - Jacobs, Helena

AU - Massey, Gita

AU - Nolte, Mindy

AU - Werner, Eric

AU - Stewart, Kim

AU - Ragni, Margaret

AU - Jaworski, Kristen

AU - Rogers, John S.

AU - Webber, Sylvia

AU - Jubelirer, Steven

AU - Arden, Donna

AU - Casella, Jim

AU - Underland, Trish

AU - Panckeri, Karen

AU - Palumbo, Lisa

AU - Sabio, Hernan

AU - Smith, Anita

AU - Sharma, Vivek

AU - Ashby, Patricia

AU - Radulescu, Vlad

AU - Hanna, Wahid

AU - Zimmerman, Cherys

AU - Neff, Anne

AU - Hudson, Mary G.

AU - Reiss, Ulrike

AU - Grant, Robin

AU - Key, Nigel

AU - Nielsen, Brenda

AU - Dunn, Amy

AU - Gutting, Karen

AU - Watts, Raymond

AU - Noa, Mary Catherine

AU - Abshire, Tom

AU - Bryant, Corinthian

AU - Ameri, Afshin

AU - Crenshaw, Valerie

AU - Kuriakose, Philip

AU - Lambing, Angela

AU - Heggie, Glenn

AU - Kovats-Bell, Sue

AU - Inoue, Susumu

AU - Ruff, Jo Ann

AU - Mitchell, Deanna

AU - Sandon-Kleiboer, Elizabeth

AU - Kraut, Eric

AU - Witkoff, Leslie

AU - Gruppo, Ralph

AU - Chambers, Anne

AU - Kerlin, Bryce

AU - Lupia, Claudia

AU - French, James

AU - Hibner, Sandy

AU - Shapiro, Amy

AU - May, Sarah

AU - Alexander, Sarah

AU - Hopewell, Steve

AU - Bockenstedt, Paula

AU - Pipe, Steven

AU - Mathis, Diana

AU - Zakarija, Anaadriana

AU - Harris, Sandy

AU - Thompson, Alexis

AU - Allen, Geoffrey A.

AU - Brummel, Ashley T.

AU - Tarantino, Michael

AU - Riedel, Angie

AU - Reding, Mark

AU - Curoe, Susan

AU - Pruthi, Rajiv

AU - Schmidt, Kirstin

AU - Kralovetz, Katie

AU - Charles, Jay

AU - Diamond, Carol

AU - Drozdowicz, Paulette

AU - Gill, Joan C.

AU - Haar, Jodi

AU - Hoots, W. Keith

AU - Gonzalez, Valerie

AU - Leissinger, Cindy

AU - Schmidt, Cecilia V.

AU - Stine, Kimo

AU - Eckert, Glenda

AU - Sexauer, Charles

AU - Kiplinger, Felicia

AU - Amerson, Patricia

AU - Johnson, Erica

AU - Buchanan, George

AU - Torres, Roberto

AU - DiPaola, Jorge

AU - Lammer, Mike

AU - Wicklund, Brian

AU - Nelson, Debbie

AU - Haire, William D.

AU - Hanlon, Elizabeth

AU - Manco-Johnson, Marilyn

AU - Geraghty, Sue

AU - Yaish, Hassan

AU - Green, Jennifer

AU - Young, Guy

AU - McGinty, Kathy

AU - Herbst, Kenneth

AU - Glass, Cathy

AU - Glader, Bertil

AU - Zoland, Jean Marie

AU - Medeiros, Desiree

AU - Omatsu, Dee Ann

AU - Ducore, Jonathan

AU - Herr, Muriel

AU - Koerper, Marion

AU - Karp, Susan

AU - Lovejoy, Amy E.

AU - Farrow, Katherine

AU - Ewing, Nadia

AU - Pullens, Lisa

AU - Meadows, Lee

AU - Tackney, Angela

AU - Mignacca, Robert

AU - Patterson, Frances

AU - Bernstein, Jonathan

AU - Berkowitz, Becki

AU - Thompson, Arthur R.

AU - Brower, Cheryl

AU - Recht, Michael

AU - Granger, Karen

AU - Schulz, Laura

AU - Stewart, Angela

PY - 2009/7/10

Y1 - 2009/7/10

N2 - 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.

AB - 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.

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U2 - 10.1111/j.1365-2516.2009.02025.x

DO - 10.1111/j.1365-2516.2009.02025.x

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JO - Haemophilia

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