Abstract

Atypical hemolytic uremic syndrome is the result of a spectrum of diseases. Disorders of complement regulation are the most important reasons in the etiology. It is associated with defective regulation of the alternative complement pathway in over 50% of the cases. Clinical abnormalities are related with the presence of thrombotic microangiopathy. Patients with atypical hemolytic uremic syndrome have a poor prognosis with a high mortality and morbidity in the acute phase of the disease and progression to end-stage renal disease in 50% of the cases. Various extra renal complications due to systemic thrombotic microangiopathy may occur in HUS, including neurological, pancreatic and cardiac involvement. Eculizumab is a humanized monoclonal anti-C5 antibody. It blocks the alternative complement pathway at the level of proinflammatory C5a and lytic C5b-9 complex generation. With the increase in experience, eculizumab therapy may be the first-line treatment. We do not know the optimal duration of eculizumab therapy. We also do not know in which patient a severe relapse could develop. At this moment we can suggest that eculizumab is life-saving and enhances the quality of life

Keywords: Atypical hemolytic uremic syndrome, Eculizumab, Thrombotic microangiopathy

References

  1. Schmidtko J, Peine S, El-Housseini Y, Pascual M, Meier P. Treatment of atypical hemolytic uremic syndrome and thrombotic microangiopathies: A focus on eculizumab. Am J Kidney Dis 2013; 61: 289-99.
  2. Ariceta G, Besbas N, Johnson S, Karpman D, Landau D, Licht C, Loirat C, et al. Guideline for the investigation and initial therapy of diarrhea-negative hemolytic uremic syndrome. Pediatr Nephrol 2009;24:687-96.
  3. Fremeaux-Bacchi V, Fakhouri F, Garnier A, Bienaimé F, Dragon- Durey MA, Ngo S, et al. Genetics and outcome of atypical hemolytic uremic syndrome: A nationwide French series comparing children and adults. Clin J Am Soc Nephrol 2013; 8: 554-62.
  4. Waters AM, Licht C. aHUS caused by complement dysregulation: New therapies on the horizon. Pediatr Nephrol 2011; 26:41-57.
  5. Kavanagh D, Goodship TH, Richards A. Atypical hemolytic uremic syndrome. Seminars in Nephrology 2013; 33:508-30.
  6. Kavanagh D, Goodship T. Genetics and complement in atypical HUS. Pediatr Nephrol 2010; 25:2431-42.
  7. Geerdink LM, Westra D, Van Wijk JAE, Dorresteijn EM, Lilien MR, Davin JC, et al. Atypical hemolytic uremic syndrome in children: Complement mutations and clinical characteristics. Pediatr Nephrol 2012; 27:1283-91.
  8. Tsai HM. Thrombotic thrombocytopenic purpura and the atypical hemolytic uremic syndrome. Hematol Oncol Clin N Am 2013; 27: 565-84.
  9. Kavanagh D, Goodship TH, Richards A. Atypical haemolytic uraemic syndrome. Br Med Bull 2006; 77-78:5-22.
  10. Koehl B, Boyer O, Biebuyck-Gougé N, Kossorotoff M, Frémeaux- Bacchi V, Boddaert N, et al. Neurological involvement in a child with atypical hemolytic uremic syndrome. Pediatr Nephrol 2010; 25:2539–42.
  11. Gulleroglu K, Fidan K, Hancer VS, Bayrakci U, Baskin E, Soylemezoglu O. Neurologic involvement in atypical hemolytic uremic syndrome and successful treatment with eculizumab. Pediatr Nephrol 2013; 28:827-30.
  12. Nathanson S, Kwon T, Elmaleh M, Charbit M, Launay EA, Harambat J, et al. Acute neurological involvement in diarrhea-associated hemolytic uremic syndrome. Clin J Am Soc Nephrol, 2010;5:1218- 28.
  13. Woodruff TM, Ager RR, Tenner AJ, Noakes PG, Taylor SM. The role of the complement system and the activation fragment C5a in the central nervous system. Neuromolecular Med 2010; 12:179–92.
  14. Waters AM, Licht C. aHUS caused by complement dysregulation: New therapies on the horizon. Pediatr Nephrol 2011; 26: 41-57.
  15. Rizvi MA, Vesely SK, George JN, Chandler L, Duvall D, Smith JW, et al. Complications of plasma exchange in 71 consecutive patients treated for clinically suspected thrombotic thrombocytopenic purpura-hemolytic uremic syndrome. Transfusion 2000; 40: 896-901.
  16. Noris M, Remuzzi G. Hemolytic uremic syndrome. J Am Soc Nephrol 2005;16: 1035-50.
  17. Tschumi S, Gugger M, Bucher BS, Riedl M, Simonetti GD. Eculizumab in atypical hemolytic uremic syndrome: Long-term clinical course and histological findings. Pediatr Nephrol 2011; 26: 2085-8.
  18. Legendre CM, Licht C, Muus P, Greenbaum LA, Babu S, Bedrosian C, et al. Terminal complement inhibitor eculizumab in atypical hemolytic-uremic syndrome. N Engl J Med 2013; 368: 2169-81.
  19. Weitz M, Amon O, Bassler D, Koenigsrainer A, Nadalin S. Prophylactic eculizumab prior to kidney transplantation for atypical hemolytic uremic syndrome. Pediatr Nephrol 2011; 26: 1325-9.
  20. hemolytic uremic syndrome J Am Soc Nephrol 2010; 21:2180-7.

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How to cite

1.
Gülleroğlu K, Gülleroğlu K, Baskın E. Atypical Hemolytic Uremic Syndrome. Turk J Pediatr Dis [Internet]. 2015 Dec. 1 [cited 2025 May 24];9(4):286-91. Available from: https://turkjpediatrdis.org/article/view/313