ArticlePediatric nephrology (Berlin, Germany)2021
Eculizumab exposure in children and young adults: indications, practice patterns, and outcomes-a Pediatric Nephrology Research Consortium study.
Article in Pediatric nephrology (Berlin, Germany), 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers, 1 of them a synthesis that pooled it.
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
8 citing papers in PubMed, 1 synthesis or guideline pooled it, 11 citations in OpenAlex.
- Eculizumab in Shiga toxin-producing Escherichia coli hemolytic uremic syndrome: a systematic review.Pediatric nephrology (Berlin, Germany) · 2024Pooled it
- Delayed eculizumab therapy guided by markedly elevated soluble C5b-9 in a child with dialysis-requiring acute poststreptococcal glomerulonephritis: a case report.CEN case reports · 2026Article
- Article
- Therapeutic advances in eculizumab for atypical hemolytic uremic syndrome: a narrative review.Frontiers in pharmacology · 2026Review
- Article
- HUS with mutations in CFH and STEC infection treated with eculizumab in a 4-year-old girl.Pediatric nephrology (Berlin, Germany) · 2023Article
- Diagnosis and Treatment for Shiga Toxin-ProducingToxins · 2022Review
- Second and Third Generational Advances in Therapies of the Immune-Mediated Kidney Diseases in Children and Adolescents.Children (Basel, Switzerland) · 2022Review
Corrections and comments
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Authors and funding
25 authors at 20 institutions in 1 country.
Funding
Abstract
backgroundEculizumab is approved for the treatment of atypical hemolytic uremic syndrome (aHUS). Its use off-label is frequently reported. The aim of this study was to describe the broader use and outcomes of a cohort of pediatric patients exposed to eculizumab.
methodsA retrospective, cohort analysis was performed on the clinical and biomarker characteristics of eculizumab-exposed patients < 25 years of age seen across 21 centers of the Pediatric Nephrology Research Consortium. Patients were included if they received at least one dose of eculizumab between 2008 and 2015. Traditional summary statistics were applied to demographic and clinical data.
resultsA total of 152 patients were identified, mean age 9.1 (+/-6.8) years. Eculizumab was used "off-label" in 44% of cases. The most common diagnoses were aHUS (47.4%), Shiga toxin-producing Escherichia coli HUS (12%), unspecified thrombotic microangiopathies (9%), and glomerulonephritis (9%). Genetic testing was available for 60% of patients; 20% had gene variants. Dosing regimens were variable. Kidney outcomes tended to vary according to diagnosis. Infectious adverse events were the most common adverse event (33.5%). No cases of meningitis were reported. Nine patients died of noninfectious causes while on therapy.
conclusionsThis multi-center retrospective cohort analysis indicates that a significant number of children and young adults are being exposed to C5 blockade for off-label indications. Dosing schedules were highly variable, limiting outcome conclusions. Attributable adverse events appeared to be low. Cohort mortality (6.6%) was not insignificant. Prospective studies in homogenous disease cohorts are needed to support the role of C5 blockade in kidney outcomes.
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