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Is eculizumab efficacious in Shigatoxin-associated hemolytic uremic syndrome? : a narrative review of current evidence

Werner Keenswijk (UGent) , Ann Raes (UGent) and Johan Vande Walle (UGent)
(2018) EUROPEAN JOURNAL OF PEDIATRICS. 177(3). p.311-318
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Abstract
Severe complications due to Shigatoxin-associated hemolytic uremic syndrome (STEC-HUS) currently present a serious challenge since no specific treatment for this condition is available. Eculizumab, a terminal complement inhibitor, has been used especially in STEC-HUS patients with severe neurological involvement, but the efficacy remains undetermined. In order to determine its efficacy, we searched the databases Pubmed, Web of Science, Embase, and LiLACS for reports describing outcomes of eculizumab administration in STEC-HUS. We retrieved 11 reports ranging from case reports to cohort studies with the largest study population emanating from the 2011 German outbreak. Outcomes were variable and difficult to interpret in light of the absence of high-quality studies but seemed to point towards potential efficacy of eculizumab if administered early in the course. Conclusion: The efficacy of eculizumab in STEC-HUS could not be established nor disproven based on current data, and there is a desperate need for randomized controlled trials. What is known? . Eculizumab has been used in complicated cases of Shigatoxin-associated hemolytic uremic syndrome but the efficacy remains unknown? What is new? . Eculizumab might be efficacious if given early in selected cases of Shigatoxin-associated hemolytic uremic syndrome; however, randomized trials are needed to assess this.
Keywords
Shigatoxin-associated hemolytic uremic syndrome, Eculizumab, Neurologic dysfunction, Efficacy, TOXIN-ASSOCIATED HUS, NEUROLOGICAL INVOLVEMENT, COMPLEMENT, CHILDREN, ACTIVATION, OUTBREAK

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MLA
Keenswijk, Werner, Ann Raes, and Johan Vande Walle. “Is Eculizumab Efficacious in Shigatoxin-associated Hemolytic Uremic Syndrome? : a Narrative Review of Current Evidence.” EUROPEAN JOURNAL OF PEDIATRICS 177.3 (2018): 311–318. Print.
APA
Keenswijk, W., Raes, A., & Vande Walle, J. (2018). Is eculizumab efficacious in Shigatoxin-associated hemolytic uremic syndrome? : a narrative review of current evidence. EUROPEAN JOURNAL OF PEDIATRICS, 177(3), 311–318.
Chicago author-date
Keenswijk, Werner, Ann Raes, and Johan Vande Walle. 2018. “Is Eculizumab Efficacious in Shigatoxin-associated Hemolytic Uremic Syndrome? : a Narrative Review of Current Evidence.” European Journal of Pediatrics 177 (3): 311–318.
Chicago author-date (all authors)
Keenswijk, Werner, Ann Raes, and Johan Vande Walle. 2018. “Is Eculizumab Efficacious in Shigatoxin-associated Hemolytic Uremic Syndrome? : a Narrative Review of Current Evidence.” European Journal of Pediatrics 177 (3): 311–318.
Vancouver
1.
Keenswijk W, Raes A, Vande Walle J. Is eculizumab efficacious in Shigatoxin-associated hemolytic uremic syndrome? : a narrative review of current evidence. EUROPEAN JOURNAL OF PEDIATRICS. 2018;177(3):311–8.
IEEE
[1]
W. Keenswijk, A. Raes, and J. Vande Walle, “Is eculizumab efficacious in Shigatoxin-associated hemolytic uremic syndrome? : a narrative review of current evidence,” EUROPEAN JOURNAL OF PEDIATRICS, vol. 177, no. 3, pp. 311–318, 2018.
@article{8576112,
  abstract     = {Severe complications due to Shigatoxin-associated hemolytic uremic syndrome (STEC-HUS) currently present a serious challenge since no specific treatment for this condition is available. Eculizumab, a terminal complement inhibitor, has been used especially in STEC-HUS patients with severe neurological involvement, but the efficacy remains undetermined. In order to determine its efficacy, we searched the databases Pubmed, Web of Science, Embase, and LiLACS for reports describing outcomes of eculizumab administration in STEC-HUS. We retrieved 11 reports ranging from case reports to cohort studies with the largest study population emanating from the 2011 German outbreak. Outcomes were variable and difficult to interpret in light of the absence of high-quality studies but seemed to point towards potential efficacy of eculizumab if administered early in the course. 
Conclusion: The efficacy of eculizumab in STEC-HUS could not be established nor disproven based on current data, and there is a desperate need for randomized controlled trials. 
What is known? . Eculizumab has been used in complicated cases of Shigatoxin-associated hemolytic uremic syndrome but the efficacy remains unknown? What is new? . Eculizumab might be efficacious if given early in selected cases of Shigatoxin-associated hemolytic uremic syndrome; however, randomized trials are needed to assess this.},
  author       = {Keenswijk, Werner and Raes, Ann and Vande Walle, Johan},
  issn         = {0340-6199},
  journal      = {EUROPEAN JOURNAL OF PEDIATRICS},
  keywords     = {Shigatoxin-associated hemolytic uremic syndrome,Eculizumab,Neurologic dysfunction,Efficacy,TOXIN-ASSOCIATED HUS,NEUROLOGICAL INVOLVEMENT,COMPLEMENT,CHILDREN,ACTIVATION,OUTBREAK},
  language     = {eng},
  number       = {3},
  pages        = {311--318},
  title        = {Is eculizumab efficacious in Shigatoxin-associated hemolytic uremic syndrome? : a narrative review of current evidence},
  url          = {http://dx.doi.org/10.1007/s00431-017-3077-7},
  volume       = {177},
  year         = {2018},
}

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