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Eculizumab in Pediatric Dense Deposit Disease
Michiel J S Oosterveld1, Mark R Garrelfs2, Bernd Hoppe3
1Department of Pediatric Nephrology and m.oosterveld@amc.nl.
Clinical Journal of the American Society of Nephrology : CJASN
|August 29, 2015
Summary
Eculizumab effectively treats dense deposit disease (DDD) in children, reducing proteinuria and improving kidney function. This rare condition, a C3 glomerulopathy, showed significant improvement in pediatric patients within 12 weeks of treatment.
Area of Science:
- Nephrology
- Complement System Biology
- Pediatric Nephrology
Background:
- Dense deposit disease (DDD) is a rare C3 glomerulopathy primarily affecting children.
- Limited treatment options exist for DDD, with ongoing debate regarding eculizumab's efficacy.
- Data on eculizumab use in pediatric DDD cases are scarce.
Purpose of the Study:
- To evaluate the effectiveness of eculizumab in pediatric patients with dense deposit disease.
- To assess changes in kidney function and proteinuria following eculizumab treatment.
- To report on the resolution of urinary abnormalities, such as leukocyturia.
Main Methods:
- Retrospective analysis of clinical and histological data from five pediatric DDD patients.
- Eculizumab administered as a last resort for severe nephritic or nephrotic syndrome unresponsive to immunosuppressants.
- Outcomes measured included kidney function (eGFR) and proteinuria (urine protein-to-creatinine ratio).
Main Results:
- Seven disease episodes in five children were treated with eculizumab.
- Significant reductions in proteinuria (median 8.5 to 1.1 g/g) and improvements in eGFR (median 58 to 77 ml/min) were observed within 12 weeks.
- Leukocyturia resolved within one week in all patients who presented with it.
Conclusions:
- Eculizumab treatment in pediatric DDD patients is associated with reduced proteinuria and improved kidney function.
- Rapid resolution of leukocyturia suggests a direct impact on renal inflammation.
- These findings support the need for a randomized controlled trial to confirm eculizumab's efficacy in DDD.
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