Validation of the first commercial ELISA for type 2N von Willebrand's disease diagnosis

Haemophilia. 2011 Nov;17(6):944-51. doi: 10.1111/j.1365-2516.2011.02499.x. Epub 2011 Mar 3.

Abstract

Type 2N von Willebrand's disease (VWD) is characterized by a factor VIII (FVIII) deficiency and a low FVIII/VWF ratio related to a markedly decreased affinity of von Willebrand factor (VWF) to FVIII. Type 2N VWD is diagnosed using assays allowing the measurement of plasma VWF capacity to bind FVIII (VWF:FVIIIB). These assays, crucial in order to distinguish type 2N VWD patients from mild haemophiliacs A and haemophilia A carriers, remain exclusively homemade and limited to laboratories possessing a high level of expertise in VWD. We evaluated the first commercial ELISA (Asserachrom® VWF:FVIIIB; Stago) comparated to a reference method in a multicentric study involving 205 subjects: 60 healthy volunteers, 37 haemophiliacs A, 17 haemophilia A carriers, 37 patients with type 2N VWD, 9 heterozygous carriers for a 2N mutation and 45 patients with miscellaneous other types of VWD (all previously characterized). A diluted plasma sample adjusted to 10 IU dL(-1) of VWF:Ag was incubated with a rabbit antihuman VWF polyclonal antibody. After removing the endogenous FVIII, recombinant FVIII (rFVIII) was added and bound rFVIII was quantified using a peroxydase-conjugated mouse antihuman FVIII monoclonal antibody. The intra-assay and inter-assay reproducibility was satisfactory. In all subgroups, both methods were well correlated. All type 2N VWD patients exhibited a markedly decreased VWF:FVIIIB (lower than 15%) and all heterozygous 2N carriers had a moderately decreased VWF:FVIIIB (between 30% and 65%). All controls (healthy subjects, haemophiliacs A and haemophilia A carriers) had a normal VWF:FVIIIB (higher than 80%) except one healthy volunteer and three haemophiliacs who exhibited a moderately decreased VWF:FVIIIB suggesting a heterozygous status for a 2N mutation. In conclusion, the Asserachrom® VWF:FVIIIB is easy to perform, standardized and accurate for type 2N VWD diagnosis with a 100% sensitivity and specificity.

Publication types

  • Validation Study

MeSH terms

  • Enzyme-Linked Immunosorbent Assay / methods*
  • Factor VIII / metabolism*
  • Hemophilia A / diagnosis
  • Humans
  • Mutation / genetics
  • Reagent Kits, Diagnostic
  • Reference Values
  • Reproducibility of Results
  • Sensitivity and Specificity
  • von Willebrand Disease, Type 2 / diagnosis*
  • von Willebrand Disease, Type 2 / genetics
  • von Willebrand Diseases / diagnosis
  • von Willebrand Factor / metabolism*

Substances

  • Reagent Kits, Diagnostic
  • von Willebrand Factor
  • Factor VIII