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

Loirat, Chantal; Fakhouri, Fadi; Ariceta, Gema; Besbas, Nesrin; Bitzan, Martin; Bjerre, Anna; Coppo, Rosanna; Emma, Francesco; Johnson, Sally and Karpman, Diana LU , et al. (2016) In Pediatric Nephrology 31(1). p.15-39
Abstract
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... (More)
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. (Less)
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@article{8cfe72a5-b3e3-4407-bf7d-ecef6ba76c2b,
  abstract     = {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.},
  author       = {Loirat, Chantal and Fakhouri, Fadi and Ariceta, Gema and Besbas, Nesrin and Bitzan, Martin and Bjerre, Anna and Coppo, Rosanna and Emma, Francesco and Johnson, Sally and Karpman, Diana and Landau, Daniel and Langman, Craig B. and Lapeyraque, Anne-Laure and Licht, Christoph and Nester, Carla and Pecoraro, Carmine and Riedl, Magdalena and van de Kar, Nicole C. A. J. and Van de Walle, Johan and Vivarelli, Marina and Fremeaux-Bacchi, Veronique},
  issn         = {1432-198X},
  keyword      = {Thrombotic microangiopathy,infusion,Plasma,Plasma exchange,Kidney transplantation,uremic syndrome,Hemolytic,Eculizumab,Complement,Combined liver-kidney transplantation,Children,Atypical hemolytic uremic syndrome,Anti-factor H antibody},
  language     = {eng},
  number       = {1},
  pages        = {15--39},
  publisher    = {Springer},
  series       = {Pediatric Nephrology},
  title        = {An international consensus approach to the management of atypical hemolytic uremic syndrome in children},
  url          = {http://dx.doi.org/10.1007/s00467-015-3076-8},
  volume       = {31},
  year         = {2016},
}