Oral tacrolimus treatment of severe colitis in children.
OBJECTIVE
To evaluate the efficacy of oral tacrolimus as an induction agent in steroid-refractory severe colitis.


METHODS
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.


CONCLUSIONS
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.
Bousvaros
A
A
Combined Program in Pediatric Gastroenterology and Nutrition, Boston Children's Hospital, Boston, Massachusetts 02115, USA.
Kirschner
B S
BS
Werlin
S L
SL
Parker-Hartigan
L
L
Daum
F
F
Freeman
K B
KB
Balint
J P
JP
Day
A S
AS
Griffiths
A M
AM
Zurakowski
D
D
Ferry
G D
GD
Leichtner
A M
AM
D000293:Adolescent; D000328:Adult; D001379:Azathioprine / Q000008:administration & dosage / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003093:Colitis, Ulcerative / Q000188:drug therapy*; D003424:Crohn Disease / Q000188:drug therapy*; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D015122:Mercaptopurine / Q000008:administration & dosage / Q000627:therapeutic use; D011446:Prospective Studies; D012074:Remission Induction; D012720:Severity of Illness Index; D016559:Tacrolimus / Q000008:administration & dosage / Q000627:therapeutic use*
D007166:Immunosuppressive Agents; D015122:Mercaptopurine; D001379:Azathioprine; D016559:Tacrolimus