The impact of bleeding history, von Willebrand factor and PFA-100 (R) on the diagnosis of type 1 von Willebrand disease: results from the European study MCMDM-1VWD

TY - JOUR

T1 - The impact of bleeding history, von Willebrand factor and PFA-100 (R) on the diagnosis of type 1 von Willebrand disease: results from the European study MCMDM-1VWD

AU - Castaman, Giancarlo

AU - Tosetto, Alberto

AU - Goodeve, Anne

AU - Federici, Augusto B.

AU - Lethagen, Stefan

AU - Budde, Ulrich

AU - Batlle, Javier

AU - Meyer, Dominique

AU - Mazurier, Claudine

AU - Goudemand, Jenny

AU - Eikenboom, Jeroen

AU - Schneppenheim, Reinhard

AU - Ingerslev, Jorgen

AU - Habart, David

AU - Hill, Frank

AU - Peake, Ian

AU - Rodeghiero, Francesco

N1 - The information about affiliations in this record was updated in December 2015. The record was previously connected to the following departments: Emergency medicine/Medicine/Surgery (013240200)

PY - 2010

Y1 - 2010

N2 - P>The relationships between the Platelet Function Analyzer (PFA)-100 and von Willebrand factor (VWF) levels and bleeding score (BS) were evaluated within a multicentre project on Molecular and Clinical Markers for the Diagnosis and Management of type 1 von Willebrand disease (MCMDM-1VWD). PFA-100 closure time, either with epinephrine (EPI) or adenosine diphosphate (ADP)-cartridges, was measured in 107 index cases, 105 affected and 71 unaffected family members, and 79 healthy controls. By regression analysis VWF levels were strongly related to both closure times, with a non-linear progression. In a multiple stepwise regression model, age- and sex-adjusted PFA-100 ADP and VWF ristocetin cofactor activity (VWF:RCo) were independently associated with BS. Most of the variation of BS was predicted by PFA-100 ADP and VWF:RCo alone. In the subgroup of patients with subtle abnormalities of the multimeric pattern, VWF was invariably reduced and closure time prolonged in almost all of them. Neither PFA-100 ADP nor EPI closure times appeared to significantly improve the diagnostic capability of VWF antigen (VWF:Ag) measurement. Thus, in an unselected population a normal PFA-100 would be useful to exclude VWD, but whether it could replace the more specific VWF assay in patients with significant mucocutaneous bleeding symptoms remains to be investigated prospectively.

AB - P>The relationships between the Platelet Function Analyzer (PFA)-100 and von Willebrand factor (VWF) levels and bleeding score (BS) were evaluated within a multicentre project on Molecular and Clinical Markers for the Diagnosis and Management of type 1 von Willebrand disease (MCMDM-1VWD). PFA-100 closure time, either with epinephrine (EPI) or adenosine diphosphate (ADP)-cartridges, was measured in 107 index cases, 105 affected and 71 unaffected family members, and 79 healthy controls. By regression analysis VWF levels were strongly related to both closure times, with a non-linear progression. In a multiple stepwise regression model, age- and sex-adjusted PFA-100 ADP and VWF ristocetin cofactor activity (VWF:RCo) were independently associated with BS. Most of the variation of BS was predicted by PFA-100 ADP and VWF:RCo alone. In the subgroup of patients with subtle abnormalities of the multimeric pattern, VWF was invariably reduced and closure time prolonged in almost all of them. Neither PFA-100 ADP nor EPI closure times appeared to significantly improve the diagnostic capability of VWF antigen (VWF:Ag) measurement. Thus, in an unselected population a normal PFA-100 would be useful to exclude VWD, but whether it could replace the more specific VWF assay in patients with significant mucocutaneous bleeding symptoms remains to be investigated prospectively.

KW - bleeding score

KW - PFA-100 closure time

KW - disorders

KW - inherited bleeding

KW - von Willebrand disease

KW - von Willebrand factor

U2 - 10.1111/j.1365-2141.2010.08333.x

DO - 10.1111/j.1365-2141.2010.08333.x

M3 - Article

VL - 151

SP - 245

EP - 251

JO - British Journal of Haematology

JF - British Journal of Haematology

SN - 0007-1048

IS - 3

ER -