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Abstract: SA-PO261

Ideal Clinical Outcome and Potential Cost Savings with Individualized Eculizumab Dosing using Bayesian Modeling in Atypical Hemolytic Uremic Syndrome

Session Information

  • Trainee Case Reports - VI
    October 27, 2018 | Location: Exhibit Hall, San Diego Convention Center
    Abstract Time: 10:00 AM - 12:00 PM

Category: Trainee Case Reports

  • 1600 Pediatric Nephrology


  • Benoit, Stefanie W., Cincinnati Children''s Hospital Medical Center, Cincinnati, Ohio, United States
  • Drake, Keri A., UT Southwestern Medical Center, Dallas, Texas, United States
  • Fukuda, Tsuyoshi, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States
  • Dixon, Bradley P., Children's Hospital of Colorado, Aurora, Colorado, United States

Ecullizumab (Ecu) acquisition cost is approximately $7,000 per 300mg vial. There is limited literature regarding individualized Ecu dosing in atypical hemolytic uremic syndrome (aHUS), particularly in pediatric patients and as disease activity varies.

Case Description

A previously healthy 4 year old male presented with acute onset muscle weakness, elevated creatine kinase, and elevated liver enzymes. He subsequently developed anemia, thrombocytopenia, hypertension, and anuric acute kidney injury requiring dialysis. A muscle biopsy revealed microthrombi in skeletal muscle vessels, and a renal biopsy confirmed thrombotic microangiopathy. He showed clinical improvement with Ecu, although he required an increase in dose amount and frequency to maintain clinical stability and Ecu level >100mcg/mL. With increased dosing, he achieved remission with eventually normalized kidney function. Dosing was adjusted based on Ecu levels to maintenance on standard Ecu dosing. Pharmacokinetic (PK) analysis using Bayesian fitting with population PK parameters (Fig.1) now indicates a sufficient Ecu level could be maintained with every 3 week dosing. The cost of the Ecu doses required to suppress his early, severe disease was $84,000. Standard dosing costs $14,000 every 2 weeks; $364,000 per year. Spacing to every 3 weeks would cost $242,200, a savings of $121,800 per year.


This report describes an unusual presentation of aHUS with severe multisystem involvement and highlights the potential for improved clinical outcomes and cost savings by individualizing Ecu dosing in aHUS patients.