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

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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.

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Abbreviations

EHEC:

Enterohemorrhagic E. coli

HUS:

Hemolytic Uremic Syndrome

PMN:

Polymorphonuclear leucocytes

STEC-HUS:

Shigatoxin-associated hemolytic uremic syndrome

TMA:

Thrombotic microangiopathy

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Authors and Affiliations

  1. Department of Pediatrics, Pediatric Nephrology, Ghent University Hospital, Ghent, Belgium

    Werner Keenswijk, Ann Raes & Johan Vande Walle

  2. Department of Pediatrics, Diakonessenhuis , Paramaribo, Suriname

    Werner Keenswijk

Authors
  1. Werner Keenswijk
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  2. Ann Raes
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  3. Johan Vande Walle
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Contributions

Research idea and study design: Werner Keenswijk; data acquisition: Werner Keenswijk, Ann Raes, Johan Vande Walle; Data analysis/interpretation: Werner Keenswijk, Ann Raes, Johan Vande Walle All authors contributed to the overall work by ensuring that questions pertaining to the accuracy or integrity of any portion of the work are appropriately investigated and resolved.

Corresponding author

Correspondence to Werner Keenswijk.

Ethics declarations

This review was performed in accordance with the Declaration of Helsinki. No funding was provided at any stage for writing this paper. No informed consent was obtained since no patient information was directly used.

Conflict of interest

JVW has been a consultant and member of the speakers’bureaus of Alexion, Astellas, Ferring and Novartis. The remaining authors have no conflicts of interest or financial relationships to declare.

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Communicated by Mario Bianchetti

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Keenswijk, W., Raes, A. & Vande Walle, J. Is eculizumab efficacious in Shigatoxin-associated hemolytic uremic syndrome? A narrative review of current evidence. Eur J Pediatr 177, 311–318 (2018). https://doi.org/10.1007/s00431-017-3077-7

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  • DOI: https://doi.org/10.1007/s00431-017-3077-7

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