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Eculizumab exposure in children and young adults: indications, practice patterns, and outcomes—a Pediatric Nephrology Research Consortium study
Journal article   Open access   Peer reviewed

Eculizumab exposure in children and young adults: indications, practice patterns, and outcomes—a Pediatric Nephrology Research Consortium study

Melissa Muff-Luett, Keia R. Sanderson, Rachel M. Engen, Rima S. Zahr, Scott E. Wenderfer, Cheryl L. Tran, Sheena Sharma, Yi Cai, Susan Ingraham, Erica Winnicki, …
Pediatric nephrology (Berlin, West), Vol.36(8), pp.2349-2360
2021
DOI: 10.1007/s00467-021-04965-5
PMCID: PMC8263513
PMID: 33693990
url
https://corescholar.libraries.wright.edu/pediatrics/710View
Open Access

Abstract

Background Eculizumab 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. Methods A 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. Results A 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. Conclusions This 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.
 Medicine Medicine & Public Health Nephrology Original Article Pediatrics Urology

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