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Prophylactic eculizumab for kidney transplantation in a child with atypical hemolytic uremic syndrome due to
Daniel Ranch1, Barrett Crowther, Mazen Arar
1Department of Pediatrics, University of Texas Health Science Center, San Antonio, TX, USA.
Insights
A child with atypical hemolytic uremic syndrome (aHUS) underwent successful kidney transplant. Despite initial post-operative aHUS exacerbation, additional eculizumab doses controlled the disease and maintained graft function, highlighting the need for optimized treatment protocols.
Area of Science:
- Nephrology
- Transplantation Immunology
- Rare Diseases
Background:
- Atypical hemolytic uremic syndrome (aHUS) is a rare, life-threatening condition characterized by thrombotic microangiopathy.
- Complement factor H mutations are a common genetic cause of aHUS.
- Kidney transplantation is a potential treatment for end-stage renal disease in aHUS patients, but carries a risk of disease recurrence.
Abstract:
We present a case of successful deceased-donor kidney transplantation in a three-yr-old child with aHUS due to complement factor H mutation, using only prophylactic eculizumab treatment prior to transplant. She developed disease exacerbation in the immediate post-operative period despite having therapeutic eculizumab concentrations and evidence for complete complement pathway blockade. The patient responded well to additional doses of eculizumab and has maintained excellent graft function and disease control in the first year post-transplantation. The optimal dosing scheme for eculizumab in the perioperative period remains to be determined. More sensitive biomarkers of early disease activity are needed to improve disease monitoring. Finally, the duration of eculizumab therapy in patients with aHUS remains to be determined.
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