Therapeutic consequences for misdiagnosis of type 2N von Willebrand disease

Pediatr Blood Cancer. 2011 Dec 1;57(6):1081-3. doi: 10.1002/pbc.23120. Epub 2011 Mar 21.

Abstract

Patients presenting with a low FVIII:C and with normal VWF levels are usually presumed to have hemophilia (males) or be carriers for hemophilia (females). Some of these patients may instead have VWD:2N. Such patients if misdiagnosed are likely to suffer from insufficiently treated bleeds. We report 2 males and 1 female who presented with a low FVIII:C (1-21%) and minimally reduced/normal VWF and were assumed to have, or be a carrier for, hemophilia A. Eventually all were found to have VWD:2N. Prior to the correct diagnosis the males had been treated with rFVIII with poor responses and ultimately adverse clinical consequences.

Publication types

  • Case Reports

MeSH terms

  • Child
  • Child, Preschool
  • Factor VIII / therapeutic use*
  • Female
  • Humans
  • Male
  • Recombinant Proteins / therapeutic use
  • von Willebrand Disease, Type 2 / diagnosis
  • von Willebrand Disease, Type 2 / drug therapy*
  • von Willebrand Disease, Type 2 / genetics

Substances

  • Recombinant Proteins
  • Factor VIII