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.

Inflammatory Bowel Diseases
|September 13, 2011
PubMed

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.
Abstract

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