Methotrexate for Primary Maintenance Therapy in Mild-to-Moderate Crohn Disease in Children
Katherine Baldwin1,2, Alexa Goldfarb2, Michael Brimacombe2,3
1From the Division of Digestive Diseases, Hepatology, and Nutrition, Connecticut Children's Medical Center, Hartford, CT.
Insights
Methotrexate (MTX) shows promise as an initial maintenance therapy for mild to moderate pediatric Crohn disease (CD), with over half of patients remaining on MTX monotherapy and achieving remission after one year.
Area of Science:
- Pediatric Gastroenterology
- Immunomodulatory Therapy
- Inflammatory Bowel Disease Research
Background:
- Methotrexate (MTX) is frequently used for maintenance therapy in pediatric Crohn disease (CD), despite limited supporting data.
- The effectiveness of MTX as an initial primary maintenance therapy in newly diagnosed mild to moderate pediatric CD requires further investigation.
Purpose of the Study:
- To assess the efficacy of methotrexate (MTX) as an initial primary maintenance therapy in pediatric patients newly diagnosed with mild to moderate Crohn disease (CD).
- To identify baseline factors that predict treatment response or failure in this patient population.
Main Methods:
- A retrospective review of 65 pediatric CD patients treated with MTX as primary maintenance therapy within 4 months of diagnosis.
- Comparison of baseline characteristics between patients achieving sustained remission and those requiring escalation to anti-TNF therapy within one year.
- Remission defined as Pediatric Crohn Disease Activity Index (PCDAI) ≤ 10.
Main Results:
- 55% of patients remained on MTX monotherapy at one year, with 89% of those in clinical remission (81% steroid-free).
- 39% of patients on MTX at one year demonstrated gross mucosal healing, with an additional 22% showing mucosal improvement.
- No baseline factors predicted treatment outcomes; MTX was well-tolerated, with no surgeries required within the follow-up period.
Conclusions:
- Methotrexate (MTX) may serve a valuable role as an initial primary maintenance therapy for mild to moderate pediatric Crohn disease (CD).
- Further prospective studies are warranted to confirm these findings and optimize MTX use in pediatric CD management.
Objectives:
Despite limited data, methotrexate (MTX) is often used as primary maintenance therapy in pediatric Crohn disease (CD). We sought to assess the effectiveness of MTX as "initial" primary maintenance therapy in newly diagnosed mild/moderate pediatric CD and ascertain baseline predictive factors.
Methods:
Single-center 10-year retrospective review of newly diagnosed CD patients treated with MTX as primary maintenance therapy. We compared baseline characteristics of those patients with sustained response/clinical remission to those patients who escalated to anti-TNF therapy within 1 year. Pediatric Crohn Disease Activity Index (PCDAI) ≤ 10 defined remission.
Results:
We identified 65 patients (mean age, 11.8 years; 72 % male; mean ± SD PCDAI, 17.8 ± 10.5) who started MTX ≤4 months of diagnosis as their primary maintenance therapy. Initial therapy prior to MTX was corticosteroids (CS) (54/65), defined diet (4/65), and combination CS/diet (6/65). Oral dosing was used in 55%; mean dose was 11.4 mg/m 2 orally and 12.5 mg/m 2 subcutaneously. At 1 year, 36 of 65 (55%) were on MTX monotherapy, and of those, 32 of 36 were in clinical remission; 81% were in steroid-free remission for the year following induction. For the 36 patients on MTX at 1 year, 14 (39%) had gross mucosal healing (22% of the original cohort). Ten additional patients had mucosal improvement (37% of total healed/improved). Fifteen patients (23%) were early failures, transitioning to anti-TNF ≤4 months. Baseline PCDAI, hemoglobin, ESR, albumin, and route of administration were not predictive of outcome. MTX was well tolerated in our cohort, with only 1 patient stopping due to elevated aminotransferases. No patient required CD surgery in the 1-year follow-up.
Conclusions:
MTX may have a primary maintenance role in mild/moderate CD.
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