Maintenance of kidney function following treatment with eculizumab and discontinuation of plasma exchange after a third kidney transplant for atypical hemolytic uremic syndrome associated with a CFH mutation.
Kidney transplant in patients with atypical hemolytic uremic syndrome (aHUS) is associated with a poor outcome because of recurrent disease, especially in patients known to have a factor H mutation. Long-term prophylactic plasma exchange and combined liver-kidney transplant have prevented graft loss caused by recurrence. However, the mortality associated with liver transplant is not negligible, and prophylactic plasma exchange requires permanent vascular access and regular hospitalization and exposes the patient to potential allergic reactions to plasma. Eculizumab is a high-affinity humanized monoclonal antibody that binds to C5 and thus prevents generation of C5a and the membrane attack complex. We report the case of a 17-year-old girl with aHUS associated with a mutation in the gene for complement factor H (CFH; c.3572C>T, Ser1191Leu) who was highly dependent on plasma exchange. Because of severe allergic reactions to plasma after the third renal graft, eculizumab was introduced in place of plasma exchange without problems. This and other reports suggest that the promise of complement inhibitors in the management of aHUS is going to be fulfilled.
Davin
Jean-Claude
JC
Paediatric Nephrology Department, Emma Children's Hospital/Academic Medical Centre of Amsterdam, Amsterdam Zuid-Oost, The Netherlands. j.c.davin@amc.nl
Gracchi
Valentina
V
Bouts
Antonia
A
Groothoff
Jaap
J
Strain
Lisa
L
Goodship
Tim
T
D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized; D002675:Child, Preschool; D017242:Complement Factor H / Q000235:genetics; D005260:Female; D006463:Hemolytic-Uremic Syndrome / Q000188:drug therapy* / Q000235:genetics / Q000601:surgery* / Q000628:therapy; D006801:Humans; D016030:Kidney Transplantation / Q000502:physiology*; D009154:Mutation; D010951:Plasma Exchange; D012086:Reoperation
D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D017242:Complement Factor H; C481642:eculizumab