title: Eculizumab in neonatal hemolytic uremic syndrome with homozygous factor H deficiency.
abstract: BACKGROUND
  Neonatal atypical hemolytic uremic syndrome (aHUS) is a rare but severe disease that is mainly due to methylmalonic aciduria or genetic complement abnormalities. Traditional management of aHUS includes plasma infusion/exchange, but in small or unstable infants, plasma exchange can be challenging because of high extracorporeal volume and difficulty to obtain an adequate venous access. The C5 complement blocker eculizumab has become a cornerstone of first-line management of aHUS due to complement deregulation in older patients. However, little data are available on its use in neonatal aHUS.
  
  
  METHODS
  We report on an 11-day-old neonate with severe aHUS (myocardial impairment, respiratory failure, acute kidney disease requiring hemodiafiltration) due to homozygous factor-H deficiency. She received early treatment with eculizumab as first-line therapy and completely recovered within 5 days. A second dose of eculizumab was administered 7 days after the first infusion, followed by a dose every 2 weeks for 2 months and then every 3 weeks, at the same dosage (300 mg). With more than 24 months of follow-up, renal function remains normal.
  
  
  CONCLUSIONS
  We report on the long-term efficacy and safety of eculizumab as first-line therapy in neonatal aHUS. However its use still requires optimization in terms of indications and administration (frequency, dosage).
authors.affiliation: Centre de Référence des Maladies Rénales Rares, Hôpital Femme Mère Enfant, Hospices Civils de Lyon, 59 boulevard Pinel Bron, 69677, Bron Cedex, France, katell.michaux@gmail.com.
authors.forename: Katell
authors.initials: K
authors.lastname: Michaux
authors.forename: Justine
authors.initials: J
authors.lastname: Bacchetta
authors.forename: Etienne
authors.initials: E
authors.lastname: Javouhey
authors.forename: Pierre
authors.initials: P
authors.lastname: Cochat
authors.forename: Véronique
authors.initials: V
authors.lastname: Frémaux-Bacchi
authors.forename: Anne-Laure
authors.initials: AL
authors.lastname: Sellier-Leclerc
mesh_terms: D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D065766:Atypical Hemolytic Uremic Syndrome / Q000188:drug therapy* / Q000209:etiology*; D017242:Complement Factor H / Q000172:deficiency*; D005260:Female; D000081208:Hereditary Complement Deficiency Diseases; D006801:Humans; D007231:Infant, Newborn; D007674:Kidney Diseases / Q000150:complications* / Q000188:drug therapy*; D015432:Glomerulonephritis, Membranoproliferative
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D017242:Complement Factor H; C481642:eculizumab