Attempted treatment of factor H deficiency by liver transplantation.
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.
Department of Pediatrics, Seoul National University Children's Hospital, Seoul, Korea. cheonghi@plaza.snu.ac.kr
Hae Il
HI
Cheong
Byong Sop
BS
Lee
Hee-Gyung
HG
Kang
Hyewon
H
Hahn
Kyung-Suk
KS
Suh
Il Soo
IS
Ha
Yong
Y
Choi
D001424:Bacterial Infections / Q000276:immunology*; D015153:Blotting, Western; D002675:Child, Preschool; D017242:Complement Factor H / Q000032:analysis / Q000172:deficiency* / Q000235:genetics; D020031:Epstein-Barr Virus Infections / Q000276:immunology; D017809:Fatal Outcome; D006463:Hemolytic-Uremic Syndrome / Q000209:etiology / Q000601:surgery*; D006801:Humans; D016867:Immunocompromised Host; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D017093:Liver Failure / Q000209:etiology / Q000601:surgery; D016031:Liver Transplantation / Q000009:adverse effects*; D008232:Lymphoproliferative Disorders / Q000188:drug therapy / Q000209:etiology; D008297:Male; D016559:Tacrolimus / Q000627:therapeutic use
C493495:CFH protein, human; D007166:Immunosuppressive Agents; D017242:Complement Factor H; D016559:Tacrolimus