Oral tacrolimus treatment of severe colitis in children

J Pediatr. 2000 Dec;137(6):794-9. doi: 10.1067/mpd.2000.109193.

Abstract

Objective: To evaluate the efficacy of oral tacrolimus as an induction agent in steroid-refractory severe colitis.

Study design: Open-label, multicenter trial of oral tacrolimus in patients with severe colitis. Patients not responding to conventional therapy received tacrolimus, 0.1 mg/kg/dose given twice a day, and the dosage was adjusted to achieve blood levels between 10 and 15 ng/mL. Response was defined as improvement in a number of clinical parameters (including abdominal pain, diarrhea, rectal bleeding, and cessation of transfusions). Patients who responded by 14 days continued to receive tacrolimus, and 6-mercaptopurine or azathioprine was added as a steroid-sparing agent 4 to 6 weeks after the tacrolimus was instituted.

Results: Fourteen patients were enrolled in the study. One patient elected to withdraw after 48 hours. Of the 13 remaining, 9 (69%) responded and were discharged. Tacrolimus was continued for 2 to 3 months in the responders, except for 1 patient who was given tacrolimus for 11 months. After 1 year of follow-up, only 5 (38%) patients were receiving maintenance therapy; the other 4 responders had undergone colectomy.

Conclusion: Although tacrolimus is effective induction therapy for severe ulcerative or Crohn's colitis, fewer than 50% of patients treated will successfully achieve a long-term remission.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adolescent
  • Adult
  • Azathioprine / administration & dosage
  • Azathioprine / therapeutic use
  • Child
  • Child, Preschool
  • Colitis, Ulcerative / drug therapy*
  • Crohn Disease / drug therapy*
  • Drug Therapy, Combination
  • Female
  • Humans
  • Immunosuppressive Agents / administration & dosage
  • Immunosuppressive Agents / therapeutic use*
  • Infant
  • Male
  • Mercaptopurine / administration & dosage
  • Mercaptopurine / therapeutic use
  • Prospective Studies
  • Remission Induction
  • Severity of Illness Index
  • Tacrolimus / administration & dosage
  • Tacrolimus / therapeutic use*

Substances

  • Immunosuppressive Agents
  • Mercaptopurine
  • Azathioprine
  • Tacrolimus