Long-term remission with eculizumab in atypical haemolytic uraemic syndrome.
The understanding of the role of complement dysregulation in atypical haemolytic uraemic syndrome (aHUS) has led to major changes in therapeutic approaches and outcomes. Eculizumab is a humanized anti-C5 monoclonal antibody that inhibits the terminal complement pathway and has revolutionized the treatment and prognosis of aHUS. However, published reports to date have had relatively short-term follow-up. We report two paediatric cases of aHUS successfully treated with eculizumab longer than 6 years with no serious adverse events and preservation of renal function.
The Royal Children's Hospital, Melbourne, Victoria, Australia.
Simon
S
Carter
Princess Margaret Hospital, Perth, Western Australia, Australia.
Ian
I
Hewitt
The Royal Children's Hospital, Melbourne, Victoria, Australia.|Murdoch Children's Research Institute, Melbourne, Victoria, Australia.|University of Melbourne, Melbourne, Victoria, Australia.
Joshua
J
Kausman
D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D065766:Atypical Hemolytic Uremic Syndrome / Q000188:drug therapy* / Q000473:pathology; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D016896:Treatment Outcome
D061067:Antibodies, Monoclonal, Humanized; C481642:eculizumab
atypical haemolytic uraemic syndrome; eculizumab; long term outcome; paediatric; safety profile