Attempted treatment of factor H deficiency by liver transplantation

Pediatr Nephrol. 2004 Apr;19(4):454-8. doi: 10.1007/s00467-003-1371-2. Epub 2004 Feb 24.

Abstract

Complement factor H (FH) deficiency is one of the causes of atypical hemolytic uremic syndrome (HUS). Most patients with FH deficiency associated HUS progress to end-stage renal disease despite plasma therapy. Moreover, the disease invariably recurs in the graft kidney and causes graft failure. We confirmed FH deficiency in a 30-month-old boy with recurrent HUS of 2 years duration, and attempted an auxiliary partial orthotopic liver transplantation (APOLT) to overcome the sustained intractable dependency on plasma therapy. APOLT restored the plasma FH level, without HUS recurrence, for 7 months. However, thereafter he suffered from serious infectious complications associated with immunosuppression and finally died 11 months after APOLT. In conclusion, although APOLT showed clinical and laboratory improvement for some period in this patient, the final fatal outcome suggests that liver transplantation should be cautiously applied to patients with HUS associated with FH deficiency.

Publication types

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

MeSH terms

  • Bacterial Infections / immunology*
  • Blotting, Western
  • Child, Preschool
  • Complement Factor H / analysis
  • Complement Factor H / deficiency*
  • Complement Factor H / genetics
  • Epstein-Barr Virus Infections / immunology
  • Fatal Outcome
  • Hemolytic-Uremic Syndrome / etiology
  • Hemolytic-Uremic Syndrome / surgery*
  • Humans
  • Immunocompromised Host*
  • Immunosuppressive Agents / therapeutic use
  • Infant
  • Liver Failure / etiology
  • Liver Failure / surgery
  • Liver Transplantation / adverse effects*
  • Lymphoproliferative Disorders / drug therapy
  • Lymphoproliferative Disorders / etiology
  • Male
  • Tacrolimus / therapeutic use

Substances

  • CFH protein, human
  • Immunosuppressive Agents
  • Complement Factor H
  • Tacrolimus