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Published on: October 14, 2025
Tolerance and efficacy of azathioprine in pediatric Crohn's disease
Laura Riello1, Cécile Talbotec, Hélène Garnier-Lengliné
1Assistance Publique-Hôpitaux de Paris, Hôpital Necker-Enfants Malades, Paediatric Gastroenterology Unit, Paris, France.
Insights
Azathioprine (AZA) maintains remission in pediatric Crohn's disease (CD) patients, though less effectively than previously thought. Most patients achieving remission at 12 months sustained it, with excellent overall tolerance observed.
Area of Science:
- Pediatric Gastroenterology
- Immunomodulatory Therapy
- Inflammatory Bowel Disease Research
Background:
- Thiopurines are first-line treatments for moderate to severe pediatric Crohn's disease (CD) to prevent relapse.
- Early thiopurine introduction demonstrated high steroid-free remission rates (90%) for 18 months in a prior pediatric trial.
- This study evaluates azathioprine (AZA) efficacy and tolerance for maintaining remission in pediatric CD.
Purpose of the Study:
- To assess the efficacy of azathioprine (AZA) in maintaining steroid-free remission in pediatric Crohn's disease (CD) patients.
- To evaluate the tolerance and safety profile of AZA in this pediatric cohort.
- To compare real-world outcomes with previous trial data on thiopurine therapy for pediatric CD.
Main Methods:
- Retrospective evaluation of 105 pediatric CD patients treated with AZA (1.4-4 mg/kg).
- Efficacy assessed by Pediatric Crohn's Disease Activity Index (PCDAI) at 6, 12, 18, and 24 months; remission defined as PCDAI ≤10 without steroids.
- Relapse criteria included need for anti-TNF medication, other immunomodulators, or surgery.
Main Results:
- Steroid-free remission rates were 60.2% at 6 months, decreasing to 31.2% by 24 months.
- AZA was discontinued in 10/93 patients within 4 weeks due to adverse reactions or low TPMT activity.
- No neutropenia occurred in patients with normal TPMT; three infectious episodes required temporary AZA suspension.
Conclusions:
- AZA demonstrates efficacy in maintaining remission for pediatric CD, albeit at lower rates than previously suggested.
- Patients in steroid-free remission at 12 months generally maintained prolonged remission.
- The overall tolerance of AZA in this pediatric cohort was excellent.
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.
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Drugs for Treatment of Crohn's Disease in IBD Using Biologic Agents: Anti-TNF
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Pharmacokinetics in Pediatric Patients: Drug Metabolism
Pharmacokinetics in Pediatric Patients: Drug Excretion
