Pediatric Nephrology

, Volume 26, Issue 4, pp 613–619

Eculizumab induces long-term remission in recurrent post-transplant HUS associated with C3 gene mutation

Authors

    • Division of Pediatric NephrologyDriscoll Children’s Hospital
  • P. Stephen Almond
    • Department of Surgery and TransplantationDriscoll Children’s Hospital
  • Van H. SavellJr
    • Department of Pathology and Laboratory MedicineDriscoll Children’s Hospital
  • Salam I. Gharaybeh
    • Division of Pediatric NephrologyDriscoll Children’s Hospital
  • Cris Hogue
    • Pharmacy DepartmentDriscoll Children’s Hospital
Brief Report

DOI: 10.1007/s00467-010-1708-6

Cite this article as:
Al-Akash, S.I., Almond, P.S., Savell, V.H. et al. Pediatr Nephrol (2011) 26: 613. doi:10.1007/s00467-010-1708-6

Abstract

A 15-year-old male patient developed atypical hemolytic uremic syndrome (aHUS) at 16 months of age leading to end-stage renal disease. The family history was suggestive of autosomal dominant aHUS, and he was more recently found to have a C3 heterozygous gene mutation (1835C>T mutation in exon 14, which determines the amino-acidic substitution R570W) with no other complement abnormalities. He had two renal transplants, the first at 2.5 years, and the second at 8 years of age, but allograft dysfunction developed in both transplants leading to graft failure due to recurrent HUS at 5 years and 18 months post-transplantation respectively. At 15 years of age he received a third transplant from a deceased donor with pre-emptive plasmapheresis. He had immediate graft function and nadir serum creatinine was 1.3–1.4 mg/dl. Severe allograft dysfunction and hypertension developed 2 months after transplantation following influenza infection. Renal allograft biopsy showed thrombotic microangiopathy. He received plasmapheresis followed by eculizumab therapy. Allograft function returned to baseline 3 weeks after starting therapy, and post-treatment allograft biopsies showed improvement in thrombotic microangiopathy. He continues to receive eculizumab every 2 weeks with stable graft function 13 months after transplantation.

Keywords

Atypical hemolytic uremic syndromeRenal transplantationEculizumabPediatricPlasma exchangeComplement

Copyright information

© IPNA 2010