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Eculizumab is Associated With Increased Infection Rates and Infection Related Mortality in Children With Thrombotic
Michelle L Schoettler1, Robert Lisac1, Joel Ofori1
1Children's Healthcare of Atlanta, Aflac Cancer and Blood Disorders Center, Emory University, Atlanta, Georgia, USA.
American Journal of Hematology
|July 30, 2025
Summary
Eculizumab treatment for hematopoietic cell transplant-associated thrombotic microangiopathy (TA-TMA) significantly increases infection risks and mortality in children. This finding highlights the need for careful monitoring and further research in HCT patients receiving eculizumab.
Area of Science:
- Hematology
- Immunology
- Transplant Medicine
Background:
- Complement C5 inhibition, using eculizumab, treats hematopoietic cell transplant-associated thrombotic microangiopathy (TA-TMA).
- However, C5 inhibition may impair infectious organism clearance, increasing infection susceptibility post-hematopoietic cell transplant (HCT).
- The specific infectious risks associated with eculizumab use after HCT remain largely unknown.
Purpose of the Study:
- To investigate the infectious complications and outcomes in pediatric patients treated with eculizumab for TA-TMA post-HCT.
- To compare infection rates and mortality between eculizumab-exposed patients and matched controls.
Main Methods:
- A single-center, retrospective case-control study included allogeneic HCT recipients from January 2019 to January 2023.
- Cases were TA-TMA patients treated with eculizumab (days 14-180 post-HCT); controls were matched for acute GVHD and ICU admission.
- Infections were captured for one year post-index date (first eculizumab dose).
Main Results:
- Eculizumab-exposed patients showed significantly higher rates of bacteremia (8.49x), tissue-specific bacterial infections (6.22x), and viral infections (3.16x) compared to controls.
- These increased infection rates persisted after adjusting for immunosuppressive medications and GVHD severity.
- Infection-related mortality was substantially higher in the eculizumab group (45% vs. 19% at 1-year).
Conclusions:
- Eculizumab treatment in pediatric HCT recipients for TA-TMA is associated with significantly increased infectious complications.
- The findings suggest a heightened risk of bacterial and viral infections and increased infection-related mortality.
- Further research is warranted to validate these findings and explore risk mitigation strategies.
