title: Tolerance and efficacy of azathioprine in pediatric Crohn's disease.
abstract: BACKGROUND
  Thiopurines are considered first-line immunomodulators for the prevention of relapse in moderate to severe pediatric Crohn's disease (CD). Early introduction of thiopurines was shown in a pediatric trial to maintain steroid-free remission in 90% of patients for 18 months. In the present study we analyzed the tolerance and efficacy of azathioprine (AZA) to maintain remission in a homogenous single-center observational cohort of children with CD.
  
  
  METHODS
  In all, 105 pediatric CD patients (male/female 68/37) were retrospectively evaluated for the efficacy of AZA (doses 1.4-4 mg/kg) to maintain remission at 6, 12, 18, and 24 months of follow-up. Overall, 93 children were included with active disease (pediatric Crohn's disease activity index [PCDAI] >30), steroid/enteral-nutrition dependency, or postileocecal resection. Remission was defined as PCDAI ≤10 without steroids. Patients requiring antitumor necrosis factor (TNF) medication, other immunomodulators, or surgery were considered to experience a relapse.
  
  
  RESULTS
  Based on PCDAI, steroid-free remission was achieved in 56/93 (60.2%), 37/93 (39.8%), 31/93 (33.3%), and 29/93 (31.2%) at visits month (M)6, M12, M18, and M24, respectively. Within the first 4 weeks, AZA was stopped in 10/93 patients due to adverse reactions (pancreatitis, nausea, vomiting, skin reactions, general weakness), or not introduced due to low thiopurine methyl transferase (TPMT) activity (n = 3). No neutropenia occurred in patients with normal TPMT activity. Three infectious episodes were documented requiring temporary AZA suspension.
  
  
  CONCLUSIONS
  AZA is efficacious in maintaining remission in pediatric CD patients, but to a lesser extent than previously suggested. The majority of patients who are in steroid-free remission at 12 months remained in prolonged remission. Overall tolerance of AZA was excellent.
authors: Assistance Publique-Hôpitaux de Paris, Hôpital Necker-Enfants Malades, Paediatric Gastroenterology Unit, Paris, France.
authors: Laura
authors: L
authors: Riello
authors: Cécile
authors: C
authors: Talbotec
authors: Hélène
authors: H
authors: Garnier-Lengliné
authors: Bénédicte
authors: B
authors: Pigneur
authors: Johan
authors: J
authors: Svahn
authors: Danielle
authors: D
authors: Canioni
authors: Olivier
authors: O
authors: Goulet
authors: Jacques
authors: J
authors: Schmitz
authors: Frank M
authors: FM
authors: Ruemmele
mesh_terms: D000293:Adolescent; D001379:Azathioprine / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D003424:Crohn Disease / Q000188:drug therapy*; D004750:Enteral Nutrition; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D020714:Maximum Tolerated Dose; D012074:Remission Induction; D012189:Retrospective Studies; D012720:Severity of Illness Index; D015996:Survival Rate; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents; D001379:Azathioprine