title: New insights into childhood autoimmune hemolytic anemia: a French national observational study of 265 children.
abstract: BACKGROUND
  Autoimmune hemolytic anemia is a rare condition in children. Little is known about its initial presentation and the subsequent progression of the disease.
  
  
  METHODS
  Since 2004, a national observational study has been aiming to thoroughly describe cases and identify prognostic factors. Patients from all French hematologic pediatric units have been included if they had a hemoglobin concentration less than 11 g/dL, a positive direct antiglobulin test and hemolysis. Evans' syndrome was defined by the association of autoimmune hemolytic anemia and immunological thrombocytopenic purpura. Data from patients' medical records were registered from birth to last follow-up. Autoimmune hemolytic anemia was classified as primary or secondary. Remission criteria, qualifying the status of anemia at last follow-up, were used with the aim of identifying a subgroup with a favorable prognosis in continuous complete remission.
  
  
  RESULTS
  The first 265 patients had a median age of 3.8 years at diagnosis. In 74% of cases the direct antiglobulin test was IgG/IgG+C3d. Consanguinity was reported in 8% of cases and first degree familial immunological diseases in 15% of cases. Evans' syndrome was diagnosed in 37% of cases. Autoimmune hemolytic anemia was post-infectious in 10%, immunological in 53% and primary in 37% of cases. After a median follow-up of 3 years, 4% of children had died, 28% were still treatment-dependent and 39% were in continuous complete remission. In multivariate analysis, IgG and IgG+C3d direct antiglobulin tests were associated with a lower rate of survival with continuous complete remission (adjusted hazard ratio, 0.43; 95% confidence interval, 0.21-0.86).
  
  
  CONCLUSIONS
  This nationwide French cohort is the largest reported study of childhood autoimmune hemolytic anemia. The rarity of this condition is confirmed. Subgroups with genetic predisposition and underlying immune disorders were identified.
authors.affiliation: Pediatric Hematology Unit, Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE) Hôpital des Enfants, Hôpital Pellegrin, CHU Bordeaux, Place Amelie Raba Leon, 33 000 Bordeaux, France. nathalie.aladjidi@chu-bordeaux.fr
authors.forename: Nathalie
authors.initials: N
authors.lastname: Aladjidi
authors.forename: Guy
authors.initials: G
authors.lastname: Leverger
authors.forename: Thierry
authors.initials: T
authors.lastname: Leblanc
authors.forename: Marie Quitterie
authors.initials: MQ
authors.lastname: Picat
authors.forename: Gérard
authors.initials: G
authors.lastname: Michel
authors.forename: Yves
authors.initials: Y
authors.lastname: Bertrand
authors.forename: Brigitte
authors.initials: B
authors.lastname: Bader-Meunier
authors.forename: Alain
authors.initials: A
authors.lastname: Robert
authors.forename: Brigitte
authors.initials: B
authors.lastname: Nelken
authors.forename: Virginie
authors.initials: V
authors.lastname: Gandemer
authors.forename: Hélène
authors.initials: H
authors.lastname: Savel
authors.forename: Jean Louis
authors.initials: JL
authors.lastname: Stephan
authors.forename: Fanny
authors.initials: F
authors.lastname: Fouyssac
authors.forename: Julien
authors.initials: J
authors.lastname: Jeanpetit
authors.forename: Caroline
authors.initials: C
authors.lastname: Thomas
authors.forename: Pierre
authors.initials: P
authors.lastname: Rohrlich
authors.forename: André
authors.initials: A
authors.lastname: Baruchel
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
authors.forename: Geneviève
authors.initials: G
authors.lastname: Chêne
authors.forename: Y
authors.initials: Y
authors.lastname: Perel
mesh_terms: D000293:Adolescent; D000744:Anemia, Hemolytic, Autoimmune / Q000175:diagnosis* / Q000453:epidemiology / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005602:France / Q000453:epidemiology; D006306:Health Surveys / Q000706:statistics & numerical data; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D015999:Multivariate Analysis; D017063:Outcome Assessment, Health Care / Q000706:statistics & numerical data; D011379:Prognosis; D012042:Registries / Q000706:statistics & numerical data