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. 2025 Apr 4:16:1535407.
doi: 10.3389/fphar.2025.1535407. eCollection 2025.

Eculizumab is efficacious and safe in pediatric patients with various forms of hemolytic uremic syndrome: a retrospective clinical experience of a tertiary center

Naama Lax  1 , Miriam Davidovits  2   3 , Gabriel Chodick  3   4 , Yael Bernfeld  1 , Orit Peled  1   5
Affiliations

Eculizumab is efficacious and safe in pediatric patients with various forms of hemolytic uremic syndrome: a retrospective clinical experience of a tertiary center

Naama Lax et al. Front Pharmacol. .

Abstract

Background: Eculizumab, a terminal complement inhibitor, prevents thrombotic microangiopathy (TMA) and multiorgan damage in hemolytic uremic syndrome (HUS). We evaluated its efficacy and safety in pediatric patients with TMA sub-types: atypical HUS (aHUS), Shiga toxin-producing Escherichia coli (STEC)-HUS, and transplant-associated TMA (TA-TMA).

Methods: This retrospective study included all pediatric patients treated with eculizumab for HUS at Schneider Children's Medical Center (2011-2020), including those with pre-existing end-stage kidney disease. Clinical and laboratory parameters were analyzed over 28 weeks. The primary endpoint was achievement of complete TMA response, defined by sustained normalization of hematologic parameters and renal function. Secondary endpoints included TMA event-free status and additional clinical improvements.

Results: Twenty-four pediatric patients (median age 5.8 years) were included: 13 with aHUS, 5 with STEC-HUS, and 6 with TA-TMA. A complete TMA response was achieved in 12 (50%) of the patients overall: 7 (54%) with aHUS, 3 (60%) with STEC-HUS, and 2 (33%) with TA-TMA. TMA event-free status was reached in 15 (63%) patients. Significant improvements were observed in platelet count (63%), lactate dehydrogenase levels (76% within the first week), hemoglobin (60%), and estimated glomerular filtration rate (79%); while CH-50 levels decreased. No severe adverse events were attributed to eculizumab. Chronic kidney disease stage improved for 17 (90%).

Conclusion: The efficacy and safety of eculizumab for three TMA subtypes in pediatric patients potentially expands its therapeutic applications. The complete TMA response rate in aHUS supports eculizumab as a first-line use, while the response rate in STEC-HUS suggests potential efficacy beyond eculizumab's primary indication. The early hematologic responses and reduced CH-50 levels confirm the role of eculizumab complement-mediated HUS and underscore the need for further research in TA-TMA.

Keywords: STEC-HUS; TA-TMA; aHUS; eculizumab; hemolytic uremic syndrome; pediatrics.

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Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Figures

FIGURE 1
FIGURE 1
Mean change in platelets (PLT) during the 28 weeks from baseline.
FIGURE 2
FIGURE 2
Mean change in lactate dehydrogenase levels (LDH) during the 28 weeks from baseline.
FIGURE 3
FIGURE 3
Mean change in hemoglobin (Hb) during the 28 weeks from baseline.
FIGURE 4
FIGURE 4
Mean change in eGFR during the 28 weeks from baseline.
See this image and copyright information in PMC

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References

    1. Aklilu A. M., Shirali A. C. (2023). Chemotherapy-associated thrombotic microangiopathy. Kidney360 4 (3), 409–422. 10.34067/KID.0000000000000061 - DOI - PMC - PubMed
    1. Alfandary H., Rinat C., Gurevich E., Eisenstein I., Goldberg O., Kropach N., et al. (2020). Hemolytic uremic syndrome: a contemporary pediatric experience. Nephron 144 (3), 109–117. 10.1159/000505401 - DOI - PubMed
    1. Benz K., Amann K. (2010). Thrombotic microangiopathy: new insights. Curr. Opin. Nephrol. Hypertens. 19 (3), 242–247. 10.1097/MNH.0b013e3283378f25 - DOI - PubMed
    1. Buelli S., Zoja C., Remuzzi G., Morigi M. (2019). Complement activation contributes to the pathophysiology of Shiga toxin-associated hemolytic uremic syndrome. Microorganisms 7 (1), 15. 10.3390/microorganisms7010015 - DOI - PMC - PubMed
    1. Campistol J. M., Arias M., Ariceta G., Blasco M., Espinosa L., Espinosa M., et al. (2013). An update for atypical haemolytic uraemic syndrome: diagnosis and treatment. A consensus document. Nefrologia 35, 421–447. 10.3265/Nefrologia.pre2012.Nov.11781 - DOI - PubMed

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