Extra-Renal manifestations of atypical hemolytic uremic syndrome in children

Pediatr Nephrol. 2018 Aug;33(8):1395-1403. doi: 10.1007/s00467-018-3933-3. Epub 2018 Apr 2.


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.

Keywords: Atypical hemolytic uremic syndrome; Children; Eculizumab; Extrarenal involvement; Neurological involvement.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Antibodies, Monoclonal, Humanized / therapeutic use
  • Atypical Hemolytic Uremic Syndrome / complications*
  • Atypical Hemolytic Uremic Syndrome / genetics
  • Atypical Hemolytic Uremic Syndrome / therapy
  • Cardiovascular Diseases / diagnosis
  • Cardiovascular Diseases / epidemiology*
  • Cardiovascular Diseases / etiology
  • Cardiovascular Diseases / prevention & control
  • Central Nervous System Diseases / diagnosis
  • Central Nervous System Diseases / epidemiology*
  • Central Nervous System Diseases / etiology
  • Central Nervous System Diseases / prevention & control
  • Child
  • Child, Preschool
  • Female
  • Follow-Up Studies
  • Gastrointestinal Diseases / diagnosis
  • Gastrointestinal Diseases / epidemiology*
  • Gastrointestinal Diseases / etiology
  • Gastrointestinal Diseases / prevention & control
  • Glomerular Filtration Rate
  • Humans
  • Infant
  • Male
  • Plasma Exchange
  • Prognosis
  • Registries / statistics & numerical data
  • Respiratory Tract Diseases / diagnosis
  • Respiratory Tract Diseases / epidemiology*
  • Respiratory Tract Diseases / etiology
  • Respiratory Tract Diseases / prevention & control
  • Retrospective Studies
  • Survival Rate
  • Treatment Outcome
  • Turkey / epidemiology


  • Antibodies, Monoclonal, Humanized
  • eculizumab