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Atypical Hemolytic Uremic Syndrome

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Pediatric Kidney Disease

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Abstract

The hemolytic uremic syndrome (HUS) is a thrombotic microangiopathy characterized by the triad of thrombocytopenia, nonimmune microangiopathic hemolytic anemia and acute kidney injury. The most frequent form of HUS in children is secondary to Shiga toxin producing Escherichia coli and the term atypical HUS was initially used to designate any HUS not caused by these organisms. It is now clear that within the umbrella of atypical HUS are a number of specific causes of HUS (e.g., Streptococcus pneumoniae infection, cobalamin C defect and diacylglycerol kinase ε defect). Atypical HUS without these causes is mostly a disease of complement alternative pathway overactivation, due to hereditary mutations in complement genes or acquired autoantibodies against complement factor H. The prognosis of atypical HUS was poor, with high mortality, progression to permanent kidney failure and recurrence after transplantation. Since 2009, terminal complement blockade therapy by eculizumab has dramatically changed the hitherto dismal outcome of the disease. The aims of this chapter are to summarize the previous era of treatment, to review new knowledge in the domain of atypical HUS and to scan the horizon for future developments in the management of atypical HUS.

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  1. National Renal Complement Therapeutics Centre and Great North Children’s Hospital, Royal Victoria Infirmary, Newcastle Upon Tyne Hospitals NHS Foundation Trust, Newcastle, UK

    Michal Malina & Sally Johnson

  2. Translation and Clinical Research Institute, Newcastle University, Tyne, UK

    Michal Malina & Sally Johnson

  3. Assistance Publique-Hôpitaux de Paris, Hôpital Européen Georges Pompidou, Laboratory of Immunology, Paris, France

    Veronique Fremeaux-Bacchi

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    Franz Schaefer

  2. Emory School of Medicine, Emory University, Atlanta, GA, USA

    Larry A. Greenbaum

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Malina, M., Fremeaux-Bacchi, V., Johnson, S. (2023). Atypical Hemolytic Uremic Syndrome. In: Schaefer, F., Greenbaum, L.A. (eds) Pediatric Kidney Disease. Springer, Cham. https://doi.org/10.1007/978-3-031-11665-0_22

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