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.
Cheong
Hae Il
HI
Department of Pediatrics, Seoul National University Children's Hospital, Seoul, Korea. cheonghi@plaza.snu.ac.kr
Lee
Byong Sop
BS
Kang
Hee-Gyung
HG
Hahn
Hyewon
H
Suh
Kyung-Suk
KS
Ha
Il Soo
IS
Choi
Yong
Y
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