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Eculizumab exposure in children and young adults: indications, practice patterns, and outcomes-a Pediatric Nephrology
Melissa Muff-Luett1, Keia R Sanderson2, Rachel M Engen3
1Department of Pediatrics, Pediatric Nephrology, University of Nebraska Medical School, Children's Hospital and Medical Center, 8200 Dodge St., Omaha, NE, 68114-4113, USA. mluett@childrensomaha.org.
Insights
Eculizumab, approved for atypical hemolytic uremic syndrome (aHUS), was frequently used off-label in 152 pediatric patients. Outcomes varied by diagnosis, with low attributable adverse events but significant mortality, necessitating further research.
Area of Science:
- Pediatric Nephrology
- Rare Diseases
- Pharmacology
Background:
- Eculizumab is approved for atypical hemolytic uremic syndrome (aHUS).
- Off-label use of eculizumab is common in pediatric populations.
- This study examines the broader use and outcomes of eculizumab in children and young adults.
Purpose of the Study:
- To describe the utilization patterns and clinical outcomes of eculizumab in pediatric patients.
- To analyze eculizumab's off-label use across various diagnoses in a multi-center cohort.
- To assess adverse events and mortality associated with eculizumab therapy in young patients.
Main Methods:
- Retrospective cohort analysis of 152 patients under 25 years old treated between 2008-2015.
- Inclusion criteria: at least one dose of eculizumab.
- Data collected on clinical characteristics, diagnoses, genetic testing, dosing, outcomes, and adverse events across 21 centers.
Main Results:
- Eculizumab was used off-label in 44% of cases, with common diagnoses including aHUS, Shiga toxin-producing E. coli HUS, and glomerulonephritis.
- Variable dosing regimens were observed.
- Infectious adverse events were most common (33.5%), and overall cohort mortality was 6.6% (non-infectious causes).
Conclusions:
- A significant number of pediatric patients receive eculizumab for off-label indications.
- Variable dosing limits definitive outcome conclusions.
- While attributable adverse events appear low, cohort mortality is notable, highlighting the need for prospective studies in homogenous disease cohorts to clarify C5 blockade's role in kidney outcomes.
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.
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