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

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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.

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Data Availability

Not applicable.

Code availability

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Acknowledgements

We would like to thank the following additional centers for contributing patients to this study: Seattle Children’s Hospital, Children’s Healthcare of Atlanta, Children’s Hospital of Richmond at Virginia Commonwealth University, UT Southwestern Children’s Hospital, and Buffalo Children’s Hospital.

Funding

Dr. Sanderson is supported by the National Center for Advancing Translational Sciences, National Institutes of Health, through Grant KL2TR002490 and Grant 2015213 from the Doris Duke Charitable Foundation. Dr. Nester is supported by the National Institutes of Health, through Grant 1R01DK110023-01A1.

Author information

Author notes

  1. Melissa Muff-Luett and Keia R. Sanderson contributed equally to this work.

Authors and Affiliations

  1. Department of Pediatrics, Pediatric Nephrology, University of Nebraska Medical School, Children’s Hospital and Medical Center, 8200 Dodge St., Omaha, NE, 68114-4113, USA

    Melissa Muff-Luett

  2. Department of Medicine-Nephrology, University of North Carolina, Chapel Hill, NC, USA

    Keia R. Sanderson & Yichun Yu

  3. Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, USA

    Rachel M. Engen

  4. Division of Pediatric Nephrology and Hypertension, University of Tennessee Health Science Center, Memphis, TN, USA

    Rima S. Zahr

  5. Pediatric Nephrology, Department of Pediatrics, Baylor College of Medicine, Texas Children’s Hospital, Houston, TX, USA

    Scott E. Wenderfer

  6. Division of Pediatric Nephrology, Mayo Clinic, Rochester, MN, USA

    Cheryl L. Tran

  7. Division of Nephrology, The Children’s Hospital of Philadelphia, Philadelphia, PA, USA

    Sheena Sharma

  8. Division of Nephrology, Helen DeVos Children’s Hospital, Grand Rapids, MI, USA

    Yi Cai

  9. Kapi’olani Medical Center for Women and Children, Honolulu, HI, USA

    Susan Ingraham

  10. Department of Pediatrics, University of California Davis, Sacramento, CA, USA

    Erica Winnicki

  11. Division of Pediatric Nephrology and Hypertension, Atrium Health Levine Children’s Hospital, Charlotte, NC, USA

    Donald J. Weaver

  12. Vanderbilt University School of Medicine, Nashville, TN, USA

    Tracy E. Hunley

  13. Division of Pediatric Nephrology, Department of Pediatrics, University of Kentucky, Lexington, KY, USA

    Stefan G. Kiessling

  14. University of Utah, Salt Lake City, UT, USA

    Meredith Seamon

  15. Pediatric Nephrology and Hypertension, Stony Brook Children’s Hospital, Stony Brook, NY, USA

    Robert Woroniecki

  16. Department of Pediatrics, Division of Pediatric Nephrology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA

    Yosuke Miyashita

  17. Valley Children’s Healthcare, Madera, CA, USA

    Nianzhou Xiao

  18. Nephrology Department, The Children’s Medical Center, Dayton, OH, USA

    Abiodun A. Omoloja

  19. Department of Pediatric Nephrology, University of Minnesota, Minneapolis, MN, USA

    Sarah J. Kizilbash

  20. Children’s Hospital of Georgia, Augusta University, Augusta, GA, USA

    Asif Mansuri

  21. Division of Nephrology, Nationwide Children’s Hospital, The Ohio State University College of Medicine, Columbus, OH, USA

    Mahmoud Kallash

  22. Division of Health Services and Outcomes Research, Children’s Mercy Kansas City, Kansas City, MO, USA

    Ashley K. Sherman

  23. Division of Pediatric Nephrology, Children’s Mercy Kansas City, Kansas City, MO, USA

    Tarak Srivastava

  24. Departments of Internal Medicine and Pediatrics, Division of Nephrology, Carver College of Medicine, University of Iowa, Iowa City, IA, USA

    Carla M. Nester

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  1. Melissa Muff-Luett
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  2. Keia R. Sanderson
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  3. Rachel M. Engen
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  4. Rima S. Zahr
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  5. Scott E. Wenderfer
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  6. Cheryl L. Tran
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  7. Sheena Sharma
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  8. Yi Cai
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  9. Susan Ingraham
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  10. Erica Winnicki
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  11. Donald J. Weaver
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  13. Stefan G. Kiessling
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  14. Meredith Seamon
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Corresponding author

Correspondence to Melissa Muff-Luett.

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IRB approval was obtained at all participating centers.

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This was a retrospective study, and no participant consent was required.

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Not applicable.

Competing interests

Carla Nester is a member of the Alexion advisory board.

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Muff-Luett, M., Sanderson, K.R., Engen, R.M. et al. Eculizumab exposure in children and young adults: indications, practice patterns, and outcomes—a Pediatric Nephrology Research Consortium study. Pediatr Nephrol 36, 2349–2360 (2021). https://doi.org/10.1007/s00467-021-04965-5

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  • DOI: https://doi.org/10.1007/s00467-021-04965-5

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