title: Efficacy and safety of eculizumab in adult patients with atypical hemolytic uremic syndrome: A single center experience from Turkey.
abstract: BACKGROUND
  Atypical hemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy, which develops as a result of defective activity of the alternative complement pathway and excessive complement activation due to genetic or acquired factors. No satisfactory responses were obtained by plasmapheresis, corticosteroids and fresh frozen plasma (FFP) transfusion. However, promising results are obtained in recent years by eculuzimab treatment, which inhibits C5 activation.
  
  
  OBJECTIVE
  To evaluate the efficacy, safety and effect of eculizumab on quality of life of adult aHUS patients followed in our center.
  
  
  METHODS
  Seven patients who received eculizumab treatment in single center between the years 2012 and 2016 due to aHUS diagnosis were retrospectively evaluated. Patients were diagnosed with aHUS in accordance with certain criteria, after eliminating all the other factors caused by thrombotic microangiopathy. Complement gene mutations were completed in six patients. All patients received eculizumab as recommended (900 mg/per two weeks) following plasmapheresis, FFP, corticosteroid and hemodialysis (HD) treatments.
  
  
  RESULTS
  Four out of seven patients were men and three were women; average patient age was 51.1 (26-69) years and average duration of disease was 25.3 (2-45) months. Average period from the initial complaints of the patients up to aHUS diagnosis was 4.2 (2-13) months. Tests were implemented on six patients for complement gene mutations, and complement factor H (CFH) homozygous mutation was identified in three patients. Complete remission was observed in four patients and partial remission in two patients after eculizumab; however, one patient died. Plasmapheresis was discontinued in patients with complete remission, whereas two patients with partial remission continued the HD program, despite normalization in hematologic parameters. Significant improvement was observed in post-treatment quality of life in all six patients who currently continue eculuzimab treatment. No transfusion reaction and/or no serious infections were observed in any of the patients, while URTI (upper respiratory tract infection) was observed in one patient.
  
  
  CONCLUSIONS
  Eculizumab is an effective and safety treatment option in adult aHUS patients. Early diagnosis and initializing eculizumab therapy at an early stage may decrease mortality and morbidity in patients with aHUS. New studies are required on this topic.
authors.affiliation: Department of Hematology, Izmir Katip Celebi University, Ataturk Training and Research Hospital, Izmir, Turkey. Electronic address: mdfusun2014@gmail.com.
authors.forename: Fusun
authors.initials: F
authors.lastname: Gediz
authors.affiliation: Department of Hematology, Izmir Katip Celebi University, Ataturk Training and Research Hospital, Izmir, Turkey.
authors.forename: Bahriye Kadriye
authors.initials: BK
authors.lastname: Payzin
authors.affiliation: Department of Internal Medicine, Izmir Katip Celebi University, Ataturk Training and Research Hospital, Izmir, Turkey.
authors.forename: Sertac
authors.initials: S
authors.lastname: Ecemis
authors.affiliation: Department of Hematology, Izmir Katip Celebi University, Ataturk Training and Research Hospital, Izmir, Turkey.
authors.forename: Naile
authors.initials: N
authors.lastname: Güler
authors.affiliation: Department of Hematology, Izmir Katip Celebi University, Ataturk Training and Research Hospital, Izmir, Turkey.
authors.forename: Asu Fergun
authors.initials: AF
authors.lastname: Yilmaz
authors.affiliation: Department of Internal Medicine, Izmir Katip Celebi University, Ataturk Training and Research Hospital, Izmir, Turkey.
authors.forename: Fusun
authors.initials: F
authors.lastname: Topcugil
authors.affiliation: Department of Molecular Genetics, Ege University Faculty of Medicine, Izmir, Turkey.
authors.forename: Afig
authors.initials: A
authors.lastname: Berdeli
mesh_terms: D000328:Adult; D000368:Aged; D061067:Antibodies, Monoclonal, Humanized / Q000009:adverse effects* / Q000627:therapeutic use*; D065766:Atypical Hemolytic Uremic Syndrome / Q000188:drug therapy* / Q000235:genetics; D003710:Demography; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D016896:Treatment Outcome; D014421:Turkey
chemical_list: D061067:Antibodies, Monoclonal, Humanized; C481642:eculizumab
keywords: Atypical hemolytic uremic syndrome; Eculizumab; Efficacy; Quality of life; Safety