Eighteen unrelated patients with factor XI deficiency, four novel mutations and a 100% detection rate by denaturing high-performance liquid chromatography

Br J Haematol. 2003 May;121(3):500-2. doi: 10.1046/j.1365-2141.2003.04302.x.

Abstract

Factor XI (FXI) deficiency is an autosomal bleeding disorder of variable severity. Inheritance is not completely recessive as heterozygotes may display a distinct, if mild, bleeding tendency. Eighteen unrelated FXI-deficient patients were screened blind by fluorescent single-stranded conformation polymorphism (F-SSCP) analysis and denaturing high-performance liquid chromatography (dHPLC). Mutations were detected in 14 of the 18 patients ( approximately 78%) by F-SSCP and in all 18 patients by dHPLC. Dideoxy sequencing confirmed the mutations in all 18 patients: eight of the mutations being novel (four of which were in previously reported patients). This showed dHPLC to be a highly sensitive, reliable technique for mutation screening in heterogeneous disorders.

MeSH terms

  • Chromatography, High Pressure Liquid / methods
  • DNA Mutational Analysis
  • Factor XI Deficiency / diagnosis*
  • Factor XI Deficiency / genetics*
  • Humans
  • Jews
  • Mutation*
  • Polymorphism, Single-Stranded Conformational