New approaches to the treatment of dense deposit disease

Richard J.H. Smith, Jessy Alexander, Paul N. Barlow, Marina Botto, Thomas L. Cassavant, H. Terence Cook, Santiago Rodriguez De Córdoba, Gregory S. Hageman, T. Sakari Jokiranta, William J. Kimberling, John D. Lambris, Lynne D. Lanning, Vicki Levidiotis, Christoph Licht, Hans U. Lutz, Seppo Meri, Matthew C. Pickering, Richard J. Quigg, Angelique L. Rops, David J. SalantSanjeev Sethi, Joshua M. Thurman, Hope F. Tully, Sean P. Tully, Johan Van Der Vlag, Patrick D. Walker, Reinhard Würzner, Peter F. Zipfel

Research output: Contribution to journalReview articlepeer-review

196 Scopus citations


The development of clinical treatment protocols usually relies on evidence-based guidelines that focus on randomized, controlled trials. For rare renal diseases, such stringent requirements can represent a significant challenge. Dense deposit disease (DDD; also known as membranoproliferative glomerulonephritis type II) is a prototypical rare disease. It affects only two to three people per million and leads to renal failure within 10 yr in 50% of affected children. On the basis of pathophysiology, this article presents a diagnostic and treatment algorithm for patients with DDD. Diagnostic tests should assess the alternative pathway of complement for abnormalities. Treatment options include aggressive BP control and reduction of proteinuria, and on the basis of pathophysiology, animal data, and human studies, plasma infusion or exchange, rituximab, sulodexide, and eculizumab are additional options. Criteria for treatment success should be prevention of progression as determined by maintenance or improvement in renal function. A secondary criterion should be normalization of activity levels of the alternative complement pathway as measured by C3/C3d ratios and C3NeF levels. Outcomes should be reported to a central repository that is now accessible to all clinicians. As the understanding of DDD increases, novel therapies should be integrated into existing protocols for DDD and evaluated using an open-label Bayesian study design.

Original languageEnglish (US)
Pages (from-to)2447-2456
Number of pages10
JournalJournal of the American Society of Nephrology
Issue number9
StatePublished - Sep 2007

ASJC Scopus subject areas

  • General Medicine


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