title: Turkish pediatric atypical hemolytic uremic syndrome registry: initial analysis of 146 patients.
abstract: BACKGROUND
  Atypical hemolytic uremic syndrome (aHUS) is a devastating disease with significant morbidity and mortality. Its genetic heterogeneity impacts its clinical presentation, progress, and outcome, and there is no consensus on its clinical management.
  
  
  METHODS
  To identify the characteristics of aHUS in Turkish children, an industry-independent registry was established for data collection that includes both retrospective and prospective patients.
  
  
  RESULTS
  In total, 146 patients (62 boys, 84 girls) were enrolled; 53 patients (36.3%) were less than 2 years old at initial presentation. Among the 42 patients (37.1%) whose mutation screening was complete for CFH, CFI, MCP, CFB, C3, DGKE, and CHFR5 genes, underlying genetic abnormalities were uncovered in 34 patients (80.9%). Sixty-one patients (41.7%) had extrarenal involvement. During the acute stage, 33 patients (22.6%) received plasma therapy alone, among them 17 patients (51.5%) required dialysis, and 4 patients (12.1%) were still on dialysis at the time of discharge. In total, 103 patients (70.5%) received eculizumab therapy, 16 of whom (15.5%) received eculizumab as a first-line therapy. Plasma therapy was administered to 84.5% of the patients prior to eculizumab. In this group, renal replacement therapy was administered to 80 patients (77.7%) during the acute period. A total of 3 patients died during the acute stage. A total of 101 patients (77.7%) had a glomerular filtration rate >90 mL/min/1.73 m2at the 2-year follow-up.
  
  
  CONCLUSIONS
  The Turkish aHUS registry will increase our knowledge of patients with aHUS who have different genetic backgrounds and will enable evaluation of the different treatment options and outcomes.
authors.affiliation: Department of Pediatric Nephrology, Nephrogenetics Laboratory, Hacettepe University Faculty of Medicine, Sihhiye, 06100, Ankara, Turkey.
authors.forename: Nesrin
authors.initials: N
authors.lastname: Besbas
authors.affiliation: Department of Pediatric Nephrology, Nephrogenetics Laboratory, Hacettepe University Faculty of Medicine, Sihhiye, 06100, Ankara, Turkey.
authors.forename: Bora
authors.initials: B
authors.lastname: Gulhan
authors.affiliation: Department of Pediatric Nephrology, Gazi University Faculty of Medicine, Ankara, Turkey.
authors.forename: Oguz
authors.initials: O
authors.lastname: Soylemezoglu
authors.affiliation: Department of Pediatric Nephrology, Ankara University Faculty of Medicine, Ankara, Turkey.
authors.forename: Z Birsin
authors.initials: ZB
authors.lastname: Ozcakar
authors.affiliation: Department of Pediatric Nephrology, Nephrogenetics Laboratory, Hacettepe University Faculty of Medicine, Sihhiye, 06100, Ankara, Turkey.
authors.forename: Emine
authors.initials: E
authors.lastname: Korkmaz
authors.affiliation: Department of Preventive Oncology, Hacettepe University, Ankara, Turkey.
authors.forename: Mutlu
authors.initials: M
authors.lastname: Hayran
authors.affiliation: Department of Pediatric Nephrology, Nephrogenetics Laboratory, Hacettepe University Faculty of Medicine, Sihhiye, 06100, Ankara, Turkey. fozaltin@hacettepe.edu.tr.
authors.forename: Fatih
authors.identifier: http://orcid.org/0000-0003-1194-0164
authors.initials: F
authors.lastname: Ozaltin
mesh_terms: D000293:Adolescent; D065766:Atypical Hemolytic Uremic Syndrome / Q000235:genetics / Q000401:mortality* / Q000628:therapy*; D001803:Blood Transfusion / Q000401:mortality* / Q000706:statistics & numerical data; D002648:Child; D002675:Child, Preschool; D015897:Comorbidity; D005260:Female; D020022:Genetic Predisposition to Disease / Q000235:genetics; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D010865:Pilot Projects; D015995:Prevalence; D012042:Registries; D006435:Renal Dialysis / Q000401:mortality* / Q000706:statistics & numerical data; D012307:Risk Factors; D015996:Survival Rate; D016896:Treatment Outcome; D014421:Turkey / Q000453:epidemiology
chemical_list: D007166:Immunosuppressive Agents
keywords: Atypical hemolytic uremic syndrome; Outcome; Prognosis; Treatment; Turkish registry