Complement factor I deficiency in a family with recurrent infections

Immunopharmacology. 1997 Dec;38(1-2):207-13. doi: 10.1016/s0162-3109(97)00080-5.

Abstract

Factor I deficiency causes a permanent, uncontrolled activation of the alternative pathway resulting in an increased turnover of C3 and consumption of factor B, factor H and properdin. Factor I deficiency is clinically associated with recurrent bacterial infections already in early infancy, mainly affecting the upper and lower respiratory tract, or presenting as meningitis or septicemia. We here report on a Brazilian family (n = 9) with known consanguinity, where in 3/7 children, suffering from chronic otitis, meningitis, and respiratory infections, a complete factor I deficiency was recognized. One of the patients died after fulminant sepsis. Hemolytic activity of the alternative pathway was not detectable in the patients' sera due to decreased plasma concentrations of C3, factor B and properdin. As a consequence of factor I deficiency, C3b could not be metabolized with the result that no C3-derived split products (C3dg/C3d) were detectable in the patients' sera. In vitro reconstitution with purified factor I restored the regulatory function in the patients' sera with the subsequent cleavage of C3b to C3c and C3dg. Factor H levels were decreased in all patients' sera and found to be tightly complexed with C3b resulting in a modified electrophoretic mobility. Upon factor I reconstitution, factor H was released from C3b regaining its beta 1 electrophoretic mobility. Complement-mediated biological functions like opsonization of bacteria, chemotactic activity and phagocytosis in these patients were impaired. The parents (cousins, 2nd degree) and 3/4 siblings had significantly reduced factor I plasma levels without further alteration in their complement profile. 3 of these obviously heterozygously deficient family members suffered from recurrent bacterial infections of different frequency and severity.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Bacterial Infections / immunology*
  • Brazil
  • Child
  • Child, Preschool
  • Complement Activation / immunology
  • Complement C3 / immunology
  • Complement C3 / metabolism
  • Complement Factor B / immunology
  • Complement Factor B / metabolism
  • Complement Factor H / immunology
  • Complement Factor H / metabolism
  • Complement Factor I / deficiency*
  • Complement Factor I / immunology
  • Consanguinity
  • Female
  • Humans
  • Male
  • Meningitis, Bacterial / immunology*
  • Middle Aged
  • Otitis Media / immunology*
  • Pedigree
  • Properdin / immunology
  • Properdin / metabolism
  • Respiratory Tract Infections / immunology*
  • Sepsis / immunology

Substances

  • CFH protein, human
  • Complement C3
  • Properdin
  • Complement Factor H
  • Complement Factor I
  • Complement Factor B