Extra-Renal manifestations of atypical hemolytic uremic syndrome in children

Abstract

Background

Atypical hemolytic uremic syndrome (aHUS) is a chronic disease characterized by thrombotic microangiopathy and a high risk of end-stage kidney disease. Dysregulation and/or excessive activation of the complement system results in thrombotic microangiopathy. Interest in extrarenal manifestations of aHUS is increasing. This study aimed to determine the clinical characteristics of patients with extrarenal manifestations of aHUS in childhood.

Methods

This study included 70 children with extrarenal manifestations of HUS from the national Turkish aHUS Registry. The demographics, clinical characteristics, genetic test results, all treatments, and renal/hematologic status of aHUS patients with extrarenal involvement were recorded.

Results

The most common extrarenal manifestation was neurological system involvement (n = 46 [27.2%]), followed by gastrointestinal (n = 20 [11.8%]), cardiovascular (n = 12 [7%]), and respiratory (n = 12 [7%]) involvement. The patients with neurological involvement had a higher mortality rate and a lower estimated glomerular filtration rate (eGFR) than the other patients at last follow-up. Eculizumab (with or without plasma exchange/plasma infusion) treatment increased the renal and hematologic recovery rates.

Conclusions

The most common and serious extrarenal manifestation of aHUS is neurological involvement and treatment outcome findings presented herein are important to all relevant clinicians.

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Fig. 1

Abbreviations

aHUS:

Atypical hemolytic uremic syndrome

TMA:

Thrombotic microangiopathy

DGKE:

Diacylglycerol kinase-ε

THBD:

Thrombomodulin

CNS:

Central nervous system

PRES:

Posterior reversible encephalopathy syndrome

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Acknowledgements

This study was supported by the Hacettepe University Scientific Research and Development Office (grant no. 010A101009).

Author information

Affiliations

Authors

Contributions

Kibriya Fidan, Nilufer Goknar, and Oguz Soylemezoglu participated in conception, design, analysis and interpretation of data, drafting the article, and revising. Kaan Gülleroğlu, Esra Baskın, Engin Melek, Zeynep Y. Yıldırım, Bora Gülhan, and Zeynep Birsin Özçakar participated in data collection. Fatih Özaltın participated in study design and evaluation of genetic results. All authors contributed to manuscript development and approved the final manuscript.

Corresponding author

Correspondence to Kibriya Fidan.

Ethics declarations

The study protocol was approved by the Hacettepe University Ethics Committee (FON10/03-22) and written informed consent was obtained from the parents of each patient.

Conflict of interest

The authors declare that they have no conflict of interest.

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Fidan, K., Göknar, N., Gülhan, B. et al. Extra-Renal manifestations of atypical hemolytic uremic syndrome in children. Pediatr Nephrol 33, 1395–1403 (2018). https://doi.org/10.1007/s00467-018-3933-3

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Keywords

  • Atypical hemolytic uremic syndrome
  • Children
  • Neurological involvement
  • Extrarenal involvement
  • Eculizumab