title: Neurologic involvement in atypical hemolytic uremic syndrome and successful treatment with eculizumab.
abstract: BACKGROUND
  Atypical hemolytic uremic syndrome (aHUS) is associated with defective regulation of the complement pathway. Neurological involvement is the most common extrarenal complication and represents a major cause of mortality and morbidity.
  
  
  METHODS
  Two girls aged 11 and 6 years, respectively, developed aHUS and were treated immediately with plasma exchange (PE) and fresh frozen plasma infusion (PI). Although initial improvement in renal function was seen in both cases, the first patient showed progressing thrombotic microangiopathy (TMA) despite daily PE, and neurological manifestations (seizures, vision loss, loss of balance, and confusion) developed after 1 month. The second patient developed cerebral TMA (seizures, vision loss, and nystagmus) 6 days after initial presentation and remained unresponsive to PE/PI. Neurological symptoms were similar in both patients, even though they had different complement protein mutations. Treatment with eculizumab achieved complete control of neurological symptoms within 24 h and gradually normalized hematological and renal parameters in both children.
  
  
  CONCLUSIONS
  Based on our two cases, we conclude that eculizumab is a rapid-acting, effective, and life-saving treatment for pediatric patients with aHUS and severe neurological involvement, which works by inhibiting complement-mediated TMA in the kidney and other organs, such as the brain.
authors: Department of Pediatric Nephrology, Baskent University, Ankara, Turkey.
authors: Kaan
authors: K
authors: Gulleroglu
authors: Kibriya
authors: K
authors: Fidan
authors: Veysel S
authors: VS
authors: Hançer
authors: Umut
authors: U
authors: Bayrakci
authors: Esra
authors: E
authors: Baskin
authors: Oguz
authors: O
authors: Soylemezoglu
mesh_terms: D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D065766:Atypical Hemolytic Uremic Syndrome; D002648:Child; D003167:Complement Activation / Q000187:drug effects / Q000235:genetics; D003221:Confusion / Q000188:drug therapy / Q000209:etiology; D005260:Female; D006463:Hemolytic-Uremic Syndrome / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy* / Q000235:genetics / Q000276:immunology; D006801:Humans; D009422:Nervous System Diseases / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology / Q000276:immunology; D009460:Neurologic Examination; D009759:Nystagmus, Pathologic / Q000188:drug therapy / Q000209:etiology; D004856:Postural Balance / Q000187:drug effects; D012640:Seizures / Q000188:drug therapy / Q000209:etiology; D012678:Sensation Disorders / Q000188:drug therapy / Q000209:etiology; D016896:Treatment Outcome; D014786:Vision Disorders / Q000188:drug therapy / Q000209:etiology
chemical_list: D061067:Antibodies, Monoclonal, Humanized; C481642:eculizumab
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