title: Childhood immune thrombocytopenia: A nationwide cohort study on condition management and outcomes.
abstract: OBJECTIVE
  Nationwide prospective cohort study exploring (i) the factors associated with treatment initiation (vs. watchful waiting) in children with primary immune thrombocytopenia (ITP) followed in routine clinical practice and (ii) the predictors of chronicity at 12 months.
  
  
  METHODS
  Between 2008 and 2013, 23 centers throughout France consecutively included 257 children aged 6 months-18 years and diagnosed with primary ITP over a 5-year period. Data on ITP clinical features along with medical management were collected at baseline and 12 months. Multivariate logistic regressions were used to determine (i) and (ii) as defined above, providing odds ratio (OR) with 95% confidence interval (95% CI).
  
  
  RESULTS
  One hundred thirty-seven (53%) children were males, median age was 4.6 years, median platelet count was 7 × 109/l, and 214 (81%) patients initiated medication. Factors independently associated with treatment initiation included platelet counts <10 × 109/l (P < 0.0001) and mucocutaneous bleeding symptoms at baseline (P < 0.001). At 12 months, data were available for 211 (82%) children, of whom 160 (74%) had recovered. Predictors of chronicity included female gender (OR = 2.2; 95% CI = 1.0-4.8), age ≥10 years (OR = 2.6; 95% CI = 1.1-6.0), and platelet counts ≥10 × 109/l (OR = 3.2; 95% CI = 1.5-6.9).
  
  
  CONCLUSIONS
  In routine clinical practice, the decision to apply a watchful waiting strategy seems to be driven by platelet counts even in the absence of bleeding symptoms, resulting in treatment being initiated in more than 80% of the children surveyed. Overall, younger children with ITP showed good prognosis, with lower platelet counts and, to a lesser extent, male gender predicting more favorable outcomes.
authors.affiliation: LASER, Paris, France.
authors.forename: Lamiae
authors.initials: L
authors.lastname: Grimaldi-Bensouda
authors.affiliation: LASER, Paris, France.
authors.forename: Clémentine
authors.initials: C
authors.lastname: Nordon
authors.affiliation: Department of Pediatric Hematology, Robert-Debré Hospital, AP-HP, Paris, France.|French Reference Center for Rare Diseases and Autoimmune Cytopenias of Childhood (CEREVANCE), Pellegrin Hospital, Bordeaux, France.
authors.forename: Thierry
authors.initials: T
authors.lastname: Leblanc
authors.affiliation: LASER, Paris, France.
authors.forename: Lucien
authors.initials: L
authors.lastname: Abenhaim
authors.affiliation: Department of General Pediatrics, Necker Hospital, AP-HP and Paris Descartes University, Paris, France.
authors.forename: Slimane
authors.initials: S
authors.lastname: Allali
authors.affiliation: Department of Pediatric Hematology, Grenoble University Hospital, Grenoble, France.
authors.forename: Corinne
authors.initials: C
authors.lastname: Armari-Alla
authors.affiliation: Department of Pediatric Hematology, Saint-Etienne University Hospital, Saint-Etienne, France.
authors.forename: Claire
authors.initials: C
authors.lastname: Berger
authors.affiliation: Department of Immunology and Hematology, Armand-Trousseau Hospital, Paris, France.
authors.forename: Mary-France
authors.initials: MF
authors.lastname: Courcoux
authors.affiliation: Department of Pediatric Hematology, Nancy University Hospital, Vandoeuvre-les-Nancy, France.
authors.forename: Fanny
authors.initials: F
authors.lastname: Fouyssac
authors.affiliation: Department of General Pediatrics, Sud-Francilien Hospital, Corbeil-Essonnes, France.
authors.forename: Cécile
authors.initials: C
authors.lastname: Guillaumat
authors.affiliation: Department of Pediatric Rheumatology and Hematology, Bicêtre Hospital, Le Kremlin-Bicêtre, France.
authors.forename: Corinne
authors.initials: C
authors.lastname: Guitton
authors.affiliation: Department of Pediatrics and Clinical Genetics, Morvan University Hospital, Brest, France.
authors.forename: Philippe
authors.initials: P
authors.lastname: Le Moine
authors.affiliation: Department of Pediatric Hematology, Lille University Hospital, Lille, France.
authors.forename: Françoise
authors.initials: F
authors.lastname: Mazingue
authors.affiliation: Department of Pediatrics, Intercommunal Hospital, Creteil, France.
authors.forename: Corinne
authors.initials: C
authors.lastname: Pondarré
authors.affiliation: Pediatric Intensive Care and Onco-Hematology Units, Nantes University Hospital, Nantes, France.
authors.forename: Caroline
authors.initials: C
authors.lastname: Thomas
authors.affiliation: Department of Pediatric Hematology and Immunology, Purpan University Hospital and INSERM U1037, Toulouse, France.
authors.forename: Marlène
authors.initials: M
authors.lastname: Pasquet
authors.affiliation: French Reference Center for Rare Diseases and Autoimmune Cytopenias of Childhood (CEREVANCE), Pellegrin Hospital, Bordeaux, France.|Department of Pediatric Hematology, CIC 1401, INSERM CICP, Pellegrin Hospital, Bordeaux, France.
authors.forename: Yves
authors.initials: Y
authors.lastname: Perel
authors.affiliation: French Reference Center for Rare Diseases and Autoimmune Cytopenias of Childhood (CEREVANCE), Pellegrin Hospital, Bordeaux, France.|Department of Immunology and Hematology, Armand-Trousseau Hospital, Paris, France.
authors.forename: Guy
authors.initials: G
authors.lastname: Leverger
authors.affiliation: French Reference Center for Rare Diseases and Autoimmune Cytopenias of Childhood (CEREVANCE), Pellegrin Hospital, Bordeaux, France.|Department of Pediatric Hematology, CIC 1401, INSERM CICP, Pellegrin Hospital, Bordeaux, France.
authors.forename: Nathalie
authors.initials: N
authors.lastname: Aladjidi
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D005260:Female; D005602:France; D006801:Humans; D007223:Infant; D008297:Male; D016017:Odds Ratio; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000473:pathology* / Q000628:therapy*; D016896:Treatment Outcome
keywords: idiopathic thrombocytopenic purpura; predictors of chronicity; prospective cohort; routine clinical practice