An international consensus approach to the management of atypical hemolytic uremic syndrome in children

• Published in: Pediatric Nephrology Vol. 31; no. 1; pp. 15 - 39
• Main Authors: Loirat, Chantal, Fakhouri, Fadi, Ariceta, Gema, Besbas, Nesrin, Bitzan, Martin, Bjerre, Anna, Coppo, Rosanna, Emma, Francesco, Johnson, Sally, Karpman, Diana, Landau, Daniel, Langman, Craig B, Lapeyraque, Anne-Laure, Licht, Christoph, Nester, Carla, Pecoraro, Carmine, Riedl, Magdalena, van de Kar, Nicole C. A. J., Van de Walle, Johan, Vivarelli, Marina, Frémeaux-Bacchi, Véronique
• Format: Journal Article Book Review
• Language: English
• Published: Berlin/Heidelberg Springer Berlin Heidelberg 01.01.2016; Springer; Springer Nature B.V; Springer Verlag
• Subjects: Adolescent; Age Factors; Anti-factor H antibody; Antibodies, Monoclonal, Humanized - therapeutic use; Atypical hemolytic uremic syndrome; Atypical Hemolytic Uremic Syndrome - diagnosis; Atypical Hemolytic Uremic Syndrome - epidemiology; Atypical Hemolytic Uremic Syndrome - immunology; Atypical Hemolytic Uremic Syndrome - therapy; Care and treatment; Child; Child, Preschool; Children; Clinical Medicine; Combined liver-kidney transplantation; Combined Modality Therapy; Complement; Complement Activation - drug effects; Consensus; Cooperative Behavior; Drug Monitoring; Eculizumab; Hemolytic; Hemolytic-uremic syndrome; Human health and pathology; Humans; Immunologic Factors - therapeutic use; Infant; Infant, Newborn; infusion; International Cooperation; Kidney Transplantation; Klinisk medicin; Life Sciences; Liver Transplantation; Medical and Health Sciences; Medicin och hälsovetenskap; Medicine; Medicine & Public Health; Monitoring, Immunologic; Nephrology; Nephrology - standards; Patient Selection; Pediatrics; Pediatrik; Plasma; Plasma Exchange; Predictive Value of Tests; Review; Risk Factors; Thrombotic microangiopathy; Treatment Outcome; uremic syndrome; Urologi och njurmedicin; Urology; Urology and Nephrology; France; Plasma exchange; Thrombotic microangiopathy; Plasma infusion; Hemolytic uremic syndrome; Anti-factor H antibody; Combined liver–kidney transplantation; Atypical hemolytic uremic syndrome; Eculizumab; Complement; Children; Kidney transplantation
• ISSN: 0931-041X; 1432-198X; 1432-198X
• DOI: 10.1007/s00467-015-3076-8

Atypical hemolytic uremic syndrome (aHUS) emerged during the last decade as a disease largely of complement dysregulation. This advance facilitated the development of novel, rational treatment options targeting terminal complement activation, e.g., using an anti-C5 antibody (eculizumab). We review treatment and patient management issues related to this therapeutic approach. We present consensus clinical practice recommendations generated by HUS International, an international expert group of clinicians and basic scientists with a focused interest in HUS. We aim to address the following questions of high relevance to daily clinical practice: Which complement investigations should be done and when? What is the importance of anti-factor H antibody detection? Who should be treated with eculizumab? Is plasma exchange therapy still needed? When should eculizumab therapy be initiated? How and when should complement blockade be monitored? Can the approved treatment schedule be modified? What approach should be taken to kidney and/or combined liver–kidney transplantation? How should we limit the risk of meningococcal infection under complement blockade therapy? A pressing question today regards the treatment duration. We discuss the need for prospective studies to establish evidence-based criteria for the continuation or cessation of anticomplement therapy in patients with and without identified complement mutations.