PFA-100 monitoring of von Willebrand factor (VWF) responses to desmopressin (DDAVP) and factor VIII/VWF concentrate substitution in von Willebrand disease type 1 and 2

Huub H. D. M. van Vliet; Mies C. Kappers-Klunne; Frank W. G. Leebeek; Jan J. Michiels

doi:10.1160/TH07-08-0527

Thrombosis and Haemostasis, Table of Contents

Thromb Haemost 2008; 100(03): 462-468
DOI: 10.1160/TH07-08-0527

Blood Coagulation, Fibrinolysis and Cellular Haemostasis

Schattauer GmbH

PFA-100 monitoring of von Willebrand factor (VWF) responses to desmopressin (DDAVP) and factor VIII/VWF concentrate substitution in von Willebrand disease type 1 and 2

Huub H. D. M. van Vliet

¹Department of Haematology, Erasmus MC, Rotterdam, The Netherlands

,

Mies C. Kappers-Klunne

¹Department of Haematology, Erasmus MC, Rotterdam, The Netherlands

,

Frank W. G. Leebeek

¹Department of Haematology, Erasmus MC, Rotterdam, The Netherlands

,

Jan J. Michiels

²Department of Haematology, Antwerp University Hospital, University of Antwerp, Belgium

› Author Affiliations

Abstract

Summary

Dose-response relationship was studied between PFA-100 closure times (PFA CTs) and factor (F)VIII-von Willebrand factor (VWF) parameters in patients with von Willebrand disease (VWD) type 1 and type 2 before and after treatment with DDAVP (n=84) or FVIII/VWF concentrate (n=38). DDAVP treatment of patients with VWD type 1 normalised the PFA CTs by increasing VWF levels to normal. Of the 14 patients with VWD type 2, PFA CTs did not normalize in eight. Haemate-P substitution in patients with VWD type 1 induced a less favourable response as compared to DDAVP, because PFA CTs did not correct in all patients. Of 12 patients with VWD type 2 treated with Haemate-P, six showed a correction of PFA CTs (<250 sec), which correlated with the normalisation of the VWF CB/ Ag ratio. In-vitro studies were performed by using whole blood of patients with VWD and adding various amounts of FVIII/VWF concentrate. Addition of Haemate-P induced an increase of the VWF CB/Ag ratio from 0.30 to 0.70 in blood of patients with VWD type 2 with correction of the PFA CTs. Immunate did not result in an increase of VWF CB/Ag ratio in blood of VWD type 2 patients, and the PFA CTs remained prolonged. We conclude that PFA-100 might be an adequate instrument not only for diagnosis but also for monitoring of DDAVP responses and FVIII/ VWF substitution of patients with VWD type 1 and 2,but this is dependent upon the type of VWD and the concentrate used.

Keywords

von Willebrand disease - DDAVP - Haemate-P - PFA-100 - vonWille-brand factor collagen binding

Full Text

References

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