Eculizumab in Pediatric Dense Deposit Disease.

ABSTRACT:

Background and objectives

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.

Design, setting, participants, & measurements

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

SUBMITTER: Oosterveld MJ 

PROVIDER: S-EPMC4594061 | biostudies-literature | 2015 Oct

REPOSITORIES: biostudies-literature