Complement factor H-antibody-associated hemolytic uremic syndrome: pathogenesis, clinical presentation, and treatment.

Abstract

The presence of circulating autoantibodies, primarily to complement factor H antibodies (CFH-Abs) in plasma characterizes the autoimmune form of atypical hemolytic uremic syndrome (aHUS). This acquired form of aHUS defines a distinct subgroup of aHUS patients, which requires diagnostic and treatment approaches in part different from those of the genetically defined forms. The mechanisms leading to CFH-Ab production and disease onset are not completely understood, but CFH-Ab HUS seems to be secondary to a combination of genetic predisposition and environmental factors. Early diagnosis of this specific aHUS entity is important, as prompt induction of plasma exchange and concomitant immunosuppression leads to a favorable outcome. Nevertheless, information on clinical features and outcome in children is limited. Here, we review the literature on the biological and clinical features of CFH-Ab HUS and discuss therapeutic options.

• Authors: Johannes Hofer, Thomas Giner, Mihály Józsi
• Journal: Seminars in thrombosis and hemostasis (Semin Thromb Hemost) Vol. 40 Issue 4 Pg. 431-43 (Jun 2014) ISSN: 1098-9064 [Electronic] United States
• PMID: 24799303 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)
• Copyright: Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.

Chemical References

• Antibodies, Monoclonal, Humanized
• Autoantibodies
• Complement C3b Inactivator Proteins
• Complement Factor H
• eculizumab

Topics

• Antibodies, Monoclonal, Humanized (therapeutic use)
• Atypical Hemolytic Uremic Syndrome (diagnosis, immunology, therapy)
• Autoantibodies (blood)
• Binding Sites
• Clinical Trials as Topic
• Complement C3b Inactivator Proteins (immunology)
• Complement Factor H (immunology)
• Genetic Predisposition to Disease
• Humans
• Kidney Transplantation
• Protein Structure, Tertiary
• Treatment Outcome