Utilization Pattern for Eculizumab Among Children With Hemolytic Uremic Syndrome

Area of Science:

• Pediatric Nephrology
• Hematology
• Pharmacology

Background:

• Hemolytic uremic syndrome (HUS) is a severe, multi-organ disease.
• Eculizumab is recommended for complement-mediated HUS (cHUS).
• Limited data exists on eculizumab use in pediatric HUS.

Purpose of the Study:

• To describe eculizumab therapy patterns in children with HUS.
• To identify factors associated with eculizumab use in pediatric HUS.

Main Methods:

• Retrospective, multi-center cohort study using the PHIS database.
• Included patients ≤18 years hospitalized with HUS from 2011-2018.
• Multivariate analysis identified factors associated with eculizumab therapy.

Main Results:

• 1,885 children with HUS identified; 167 (8.9%) received eculizumab.
• Eculizumab was given early (within 7 days) in 65% of treated children.
• Encephalopathy, seizure disorder, and cardiac involvement were associated with eculizumab use.

Conclusions:

• Eculizumab use in pediatric HUS is low but often initiated early.
• Children with severe neurological and cardiac complications are more likely to receive eculizumab.
• Further prospective studies are needed to validate these findings.