Journal article
New Approaches to the Treatment of Dense Deposit Disease
Journal of the American Society of Nephrology, Vol.18(9), pp.2447-2456
09/2007
DOI: 10.1681/ASN.2007030356
PMCID: PMC4853920
PMID: 17675665
Abstract
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.
Details
- Title: Subtitle
- New Approaches to the Treatment of Dense Deposit Disease
- Creators
- Richard J.H Smith - Department of Internal Medicine and Otolaryngology, University of Iowa Carver College of Medicine, Iowa City, IowaJessy Alexander - Department of Internal Medicine, University of Chicago Hospitals, Chicago, IllinoisPaul N Barlow - Institute of Structural and Molecular Biology, University of Edinburgh, King’s Buildings, Edinburgh, United KingdomMarina Botto - Imperial College, Faculty of Medicine, London, EnglandThomas L Cassavant - Center for Bioinformatics and Computational Biology, University of Iowa, Iowa City, IowaH. Terence Cook - Imperial College, Faculty of Medicine, London, EnglandSantiago Rodriguez de Córdoba - Centro de Investigaciones Biologicas, Madrid, SpainGregory S Hageman - Department of Ophthalmology and Visual Sciences, University of Iowa Carver College of Medicine, Iowa City, IowaT. Sakari Jokiranta - Department of Bacteriology and Immunology, University of Helsinki and Helsinki University Central Hospital, Helsinki, FinlandWilliam J Kimberling - Boys Town National Research Hospital, Omaha, NebraskaJohn D Lambris - Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PennsylvaniaLynne D Lanning - Kidneeds, Greater Cedar Rapids Community Foundation, Cedar Rapids, IowaVicki Levidiotis - Royal Prince Alfred Hospital, Sydney, AustraliaChristoph Licht - Department of Pediatrics, Hospital for Sick Children, Toronto, CanadaHans U Lutz - Institute of Biochemistry, Swiss Federal Institute of Technology, Zurich, SwitzerlandSeppo Meri - Department of Bacteriology and Immunology, University of Helsinki and Helsinki University Central Hospital, Helsinki, FinlandMatthew C Pickering - Imperial College, Faculty of Medicine, London, EnglandRichard J Quigg - Department of Internal Medicine, University of Chicago Hospitals, Chicago, IllinoisAngelique L Rops - Nephrology Research Laboratory, Division of Nephrology, Nijmegen Centre for Molecular Life Sciences, Radboud University Nijmegen Medical Centre, Nijmegen, NetherlandsDavid J Salant - Department of Medicine, Boston University Medical Center, Boston, MassachusettsSanjeev Sethi - Department of Pathology, The Mayo Clinic, Rochester, MinnesotaJoshua M Thurman - Department of Medicine, University of Colorado Health Sciences Center, Denver, ColoradoHope F Tully - Milagros Research Fund, Chappaqua, New York, New YorkSean P Tully - Milagros Research Fund, Chappaqua, New York, New YorkJohan van der Vlag - Institute of Biochemistry, Swiss Federal Institute of Technology, Zurich, SwitzerlandPatrick D Walker - Nephropathology Associates, Little Rock, ArkansasReinhard Würzner - Department of Hygiene, Microbiology and Social Medicine, Innsbruck Medical University, Innsbruck, AustriaPeter F Zipfel - Leibniz Institute for Natural Product Research and Infection Biology and Friedrich Schiller University, Jena, GermanyDense Deposit Dis Focus Grp
- Resource Type
- Journal article
- Publication Details
- Journal of the American Society of Nephrology, Vol.18(9), pp.2447-2456
- DOI
- 10.1681/ASN.2007030356
- PMID
- 17675665
- PMCID
- PMC4853920
- NLM abbreviation
- J Am Soc Nephrol
- ISSN
- 1046-6673
- eISSN
- 1533-3450
- Language
- English
- Date published
- 09/2007
- Academic Unit
- Roy J. Carver Department of Biomedical Engineering; Molecular Physiology and Biophysics; Anatomy and Cell Biology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Otolaryngology; Internal Medicine
- Record Identifier
- 9984006323402771
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