A novel nanobody that detects the gain-of-function phenotype of von Willebrand factor in ADAMTS13 deficiency and von Willebrand disease type 2B

Blood. 2005 Nov 1;106(9):3035-42. doi: 10.1182/blood-2005-03-1153. Epub 2005 Jul 12.

Abstract

Von Willebrand factor (VWF) is unable to interact spontaneously with platelets because this interaction requires a conversion of the VWF A1 domain into a glycoprotein Ibalpha (GpIbalpha) binding conformation. Here, we discuss a llama-derived antibody fragment (AU/VWFa-11) that specifically recognizes the GpIbalpha-binding conformation. AU/VWFa-11 is unable to bind VWF in solution, but efficiently interacts with ristocetin- or botrocetin-activated VWF, VWF comprising type 2B mutation R1306Q, or immobilized VWF. These unique properties allowed us to use AU/VWFa-11 for the detection of activated VWF in plasma of patients characterized by spontaneous VWF-platelet interactions: von Willebrand disease (VWD) type 2B and thrombotic thrombocytopenic purpura (TTP). For VWD type 2B, levels of activated VWF were increased 12-fold (P < .001) compared to levels in healthy volunteers. An inverse correlation between activated VWF levels and platelet count was observed (R2 = 0.74; P < .003). With regard to TTP, a 2-fold (P < .001) increase in activated VWF levels was found in plasma of patients with acquired TTP, whereas an 8-fold increase (P < .003) was found in congenital TTP. No overlap in levels of activated VWF could be detected between acquired and congenital TTP, suggesting that AU/VWFa-11 could be used to distinguish between both disorders. Furthermore, it could provide a tool to investigate the role of VWF in the development of thrombocytopenia in various diseases.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • ADAM Proteins / deficiency*
  • ADAM Proteins / metabolism
  • ADAMTS13 Protein
  • Animals
  • Antibodies / immunology*
  • Blood Platelets / metabolism
  • Camelids, New World
  • Humans
  • Nanomedicine*
  • Phenotype
  • Platelet Glycoprotein GPIb-IX Complex / metabolism
  • Purpura, Thrombotic Thrombocytopenic / metabolism
  • Solutions
  • von Willebrand Diseases / metabolism*
  • von Willebrand Factor / immunology
  • von Willebrand Factor / metabolism*

Substances

  • Antibodies
  • Platelet Glycoprotein GPIb-IX Complex
  • Solutions
  • von Willebrand Factor
  • ADAM Proteins
  • ADAMTS13 Protein
  • ADAMTS13 protein, human