Eculizumab in Pediatric Dense Deposit Disease

Dense deposit disease (DDD), a subtype of C3 glomerulopathy, is a rare disease affecting mostly children. Treatment options are limited. Debate exists whether eculizumab, a monoclonal antibody against complement factor C5, is effective in DDD. Reported data are scarce, especially in children. The au...

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Bibliographic Details
Published in:Clinical journal of the American Society of Nephrology 2015-10, Vol.10 (10), p.1773-1782
Main Authors: Oosterveld, Michiel J S, Garrelfs, Mark R, Hoppe, Bernd, Florquin, Sandrine, Roelofs, Joris J T H, van den Heuvel, L P, Amann, Kerstin, Davin, Jean-Claude, Bouts, Antonia H M, Schriemer, Pietrik J, Groothoff, Jaap W
Format: Article
Language:English
Subjects:
Adolescent
Antibodies, Monoclonal, Humanized - therapeutic use
Child
Child, Preschool
Complement C5 - antagonists & inhibitors
Complement Inactivating Agents - therapeutic use
Creatinine - blood
Creatinine - urine
Female
Glomerular Filtration Rate
Glomerulonephritis, Membranoproliferative - drug therapy
Glomerulonephritis, Membranoproliferative - pathology
Glomerulonephritis, Membranoproliferative - physiopathology
Humans
Leukocytes
Male
Nephrotic Syndrome - drug therapy
Nephrotic Syndrome - etiology
Original
Proteinuria - drug therapy
Proteinuria - etiology
Urine - cytology
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Summary:Dense deposit disease (DDD), a subtype of C3 glomerulopathy, is a rare disease affecting mostly children. Treatment options are limited. Debate exists whether eculizumab, a monoclonal antibody against complement factor C5, is effective in DDD. Reported data are scarce, especially in children. The authors analyzed clinical and histologic data of five pediatric patients with a native kidney biopsy diagnosis of DDD. Patients received eculizumab as therapy of last resort for severe nephritic or nephrotic syndrome with alternative pathway complement activation; this therapy was given only when the patients had not or only marginally responded to immunosuppressive therapy. Outcome measures were kidney function, proteinuria, and urine analysis. In all, seven disease episodes were treated with eculizumab (six episodes of severe nephritic syndrome [two of which required dialysis] and one nephrotic syndrome episode). Median age at treatment start was 8.4 (range, 5.9-13) years. For three treatment episodes, eculizumab was the sole immunosuppressive treatment. In all patients, both proteinuria and renal function improved significantly within 12 weeks of treatment (median urinary protein-to-creatinine ratio of 8.5 [range, 2.2-17] versus 1.1 [range, 0.2-2.0] g/g, P
ISSN:1555-9041
1555-905X
DOI:10.2215/CJN.01360215