A complicated case of atypical hemolytic uremic syndrome with frequent relapses under eculizumab.

Pediatric Nephrology : Journal of the International Pediatric Nephrology Association
Gesa SchalkLutz T Weber

Abstract

Atypical hemolytic uremic syndrome (aHUS) is a form of thrombotic microangiopathy characterized by uncontrolled activation of the alternative complement pathway with consecutive generation of the terminal complement complex. Mortality is increased, particularly in the first year of the disease. Therapeutic options include plasma therapy and terminal complement blockade using the anti-C5 monoclonal antibody eculizumab. Eculizumab prevents activation of the terminal sequence of the complement cascade and formation of the potentially lytic terminal complement complex (C5b-9). We report a 3-year-old boy with aHUS due to a novel heterozygous truncating complement Factor H mutation in combination with other changes known to be associated with an increased risk for aHUS. Despite eculizumab treatment and maximal suppression of the classical and alternative complement pathways, C3d and sC5b-9 remained consistently elevated and the patient showed repeated relapses. Not every patient with aHUS and uncontrolled complement activation shows optimal therapeutic response to eculizumab with the recommended or even increased dosing regimen. Reliable outcome measures to determine the efficacy of treatment have to be defined.

References

Apr 20, 2006·Blood·Jessica CaprioliUNKNOWN International Registry of Recurrent and Familial HUS/TTP
Jun 8, 2010·Pediatric Nephrology : Journal of the International Pediatric Nephrology Association·David Kavanagh, Tim Goodship
Aug 15, 2012·Pediatric Nephrology : Journal of the International Pediatric Nephrology Association·Ramon VilaltaJose Nieto
Sep 19, 2012·Nature Reviews. Nephrology·Marina NorisGiuseppe Remuzzi
Oct 3, 2012·Nature Reviews. Nephrology·Julien ZuberUNKNOWN French Study Group for aHUS/C3G
Apr 30, 2013·Pediatric Nephrology : Journal of the International Pediatric Nephrology Association·Rodney D GilbertTimothy H Goodship
Jun 7, 2013·The New England Journal of Medicine·C M LegendreC Loirat
Aug 1, 2014·Clinical and Experimental Immunology·E B VolokhinaL P van den Heuvel

❮ Previous
Next ❯

Citations

Nov 26, 2015·Pediatric Nephrology : Journal of the International Pediatric Nephrology Association·Rosanna CoppoGiuseppe Remuzzi
Oct 27, 2016·Clinical and Experimental Immunology·C WehlingM Kirschfink
Jan 18, 2018·Journal of the American Society of Nephrology : JASN·Stefan MichelfelderKarsten Häffner
Dec 28, 2016·Case Reports in Nephrology·Camino García MonteavaroMaría Teresa Visus-Fernández de Manzanos
Feb 18, 2017·Pediatric Nephrology : Journal of the International Pediatric Nephrology Association·Maria Helena VaisbichFernando Kok

❮ Previous
Next ❯

Related Concepts

Related Feeds

Alternative Complement Pathway

The Alternative Complement Pathway is part of the innate immune system, and activation generates membrane attack complexes that kill pathogenic cells. Discover the latest research on the Alternative Complement Pathway.

Related Papers

Pediatric Nephrology : Journal of the International Pediatric Nephrology Association
Sibylle TschumiGiacomo D Simonetti
Transfusion and Apheresis Science : Official Journal of the World Apheresis Association : Official Journal of the European Society for Haemapheresis
Nilay Sengul SamanciSavas Ozturk
American Journal of Nephrology
Kavita S HodgkinsCraig B Langman
© 2022 Meta ULC. All rights reserved