Methotrexate in the Treatment of Inflammatory Bowel Disease: An 8-Year Retrospective Study in a Canadian Pediatric IBD Center

Background: Methotrexate (MTX) is used as an alternative immunosuppressive treatment for patients with inflammatory bowel disease (IBD). The aim of the study was to evaluate effectiveness and tolerance of MTX for children with IBD. Methods: A retrospective study was conducted in our pediatric IBD center of all children having received MTX for the treatment of their IBD between 2000 and 2008. Remission was defined as discontinuation of steroids and Harvey-Bradshaw Index <4 for Crohn's disease (CD) patients or Pediatric Ulcerative Colitis Activity Index (PUCAI) <10 for ulcerative (UC) or indeterminate colitis (IC) patients. Results: Seventy-five patients had CD, 5 UC, and 13 IC. Mean age at diagnosis was 11 (0.6-17.4) years. Ninety patients were previously treated with purine analogs and 26 with anti-tumor necrosis factor (TNF). Among patients assessed for effectiveness of MTX (n = 79), clinical remission was observed in 29, 37, 25, and 16% of CD patients (n = 63) and 19, 25, 13, and 7% of patients with UC or IC (n = 16), respectively, 3, 6,12, and 24 months after initiation of MTX. The 1-year remission rate for CD patients was significantly higher in patients with colonic disease. Forty-six patients (49%) experienced side effects but only 13 (14%) had to discontinue treatment. Conclusions: The long-term remission rate with MTX in our pediatric IBD population was low. However, MTX was generally well tolerated and induced and maintained remission in some patients who previously had failed a purine analog and/or anti-TNF. Prospective controlled trials are indicated to determine the place of MTX in the management of pediatric IBD. (Inflamm Bowel Dis 2011;17:2521-2526)