Methotrexate for Pediatric Crohn Disease Viable Primary Maintenance Therapy

crohn disease
Pediatrician touch tummy of kid in clinic couch. Little boy at medical checkup.
Researchers investigated whether methotrexate could be used as primary maintenance therapy among children newly diagnosed with mild to moderate Crohn disease.

Methotrexate is an efficacious primary maintenance therapy for children newly diagnosed with mild to moderate Crohn disease (CD), according to study results published in the Journal of Pediatric Gastroenterology and Nutrition.

For the study, researchers conducted a single-center, retrospective review of children who had been diagnosed with CD and received treatment with methotrexate as a primary maintenance therapy within 4 months of their diagnosis. All patients (N=65; mean age, 11.8 [range, 5-17] years; male, 72%; mean Pediatric Crohn Disease Activity Index [PCDAI], 17.8±10.5) were newly diagnosed with CD between January 2010 and February 2020. Prior to methotrexate initiation, corticosteroids (n=54), a specific diet (n=4), and a combination of corticosteroids/diet (n=6) were the first treatments used by patients.

Researchers collected patient data, including demographics, disease severity, clinical characteristics, Paris classification, endoscopic findings, inflammatory markers, and more. Disease activity was measured using the PCDAI, which scored remission (≤10), moderate disease (11-29), and moderate to severe disease (≥30). Researchers assessed data between patients with clinical remission vs patients who required anti-TNF therapy by 1 year.

For methotrexate therapy, a mean dosage of 11.4 mg/m2 orally and 12.5 mg/m2 subcutaneously was administered. In the study, 55% of patients received an oral dose.

The primary outcome was disease activity 1 year after being diagnosed with the PCDAI.

By 1 year, 36 of 65 patients were receiving treatment with methotrexate. Of those 36 patients, 32 achieved clinical remission, 1 achieved clinical response, 1 had neither clinical remission nor response, 14 had gross mucosal healing, and 10 had mucosal improvement.  Researchers also assessed early failures, defined as transitioning from methotrexate to infliximab within 4 months. A total of 15 patients were early failures.

Study limitations include not conducting a double-blind placebo-controlled trial and a lack of central blinded review of endoscopic videos and histology. Instead, researchers relied on written reports and photographs.

The study authors concluded, “This study demonstrates the efficacy of MTX [methotrexate] for mild-to-moderate CD, with over half of the patients in clinical remission on 1 year of MTX [methotrexate] and almost a quarter of patients with mucosal healing.”


Baldwin K, Goldfarb A, Brimacombe M, Hopkins D, Hyans JS. Methotrexate for primary maintence therapy in mild-to-moderate Crohn disease in children. J Pediatr Gastroenterol Nutr. 2022;75(3):320-324. doi:10.1097/MPG.0000000000003543