Work

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

Year: 2025

Type: article

Abstract: Background: (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.

Source: Frontiers in Pharmacology

DOI: 10.3389/fphar.2025.1535407

Authors: Naama Lax, Miriam Davidovits, Gabriel Chodick, Yael Bernfeld, Orit Peled

Institutions: Schneider Children's Medical Center, Tel Aviv University, Maccabi Healthcare Services, Ariel University

Language: English

Topic: Complement system in diseases

Subfield: Immunology

Field: Immunology and Microbiology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,801