Results Summary

What was the research about?

Crohn’s disease causes inflammation in the intestines and stomach, leading to long-term diarrhea and stomach pain. It can also lead to growth problems and lower quality of life. For children, the illness can get worse over time.

Doctors treat Crohn’s disease with antibiotics, steroids, or anti-TNF medicines. Anti-TNF medicines help reduce inflammation. Along with anti-TNF medicines, doctors may also prescribe another type of medicine called methotrexate. This medicine lowers inflammation. But it may cause problems like infections.

In this study, the research team wanted to learn whether anti-TNF medicines and methotrexate work better together than anti-TNF medicines alone. The team compared:

  • Anti-TNF medicine plus methotrexate. Patients received an anti-TNF medicine that their doctors prescribed, plus methotrexate. The anti-TNF medicines were adalimumab or infliximab.
  • Anti-TNF medicine alone. Patients received adalimumab or infliximab, which their doctors prescribed.

The research team looked at time to treatment failure. They defined treatment failure as having uncontrolled symptoms, having a hospital stay, having to use a steroid, or stopping the medicine because it wasn’t working or due to side effects.

What were the results?

After one year, patients who received an anti-TNF medicine plus methotrexate and patients who received an anti-TNF medicine alone didn’t differ in the time to treatment failure, pain, or fatigue.

Compared with patients who received adalimumab plus methotrexate, patients who received adalimumab alone had a shorter time until treatment failure. Time to treatment failure didn’t differ between patients who received infliximab plus methotrexate or infliximab alone.

Who was in the study?

The study included 297 children and adolescents under 21 years of age with Crohn’s disease who were starting anti-TNF medicines. Of these patients, 82 percent were White, 11 percent were Black, 1 percent were Asian, and 4 percent identified as multiple or other races. The average age was 14, and 65 percent were male. All were receiving care from one of 35 clinics across the United States.

What did the research team do?

The research team assigned patients by chance to one of the two groups. The team looked at health records up to one year after patients started anti-TNF medicines. The team also called patients one and two years after they started anti-TNF medicines to ask about their pain and fatigue.

Parents of children with Crohn’s disease and doctors helped design the study.

What were the limits of the study?

Fewer children enrolled in the study than planned, making it hard to find differences in study outcomes. Also, the study didn’t look at other health outcomes that show how well medicines work, like healing of the stomach and intestines.

Future research could compare how well medicines treat other health outcomes from Crohn’s disease.

How can people use the results?

Parents and doctors can use the results when considering treatments for children who have Crohn’s disease.

Final Research Report

This project's final research report is expected to be available by June 2024.

Peer-Review Summary

The Peer-Review Summary for this project will be posted here soon.

Conflict of Interest Disclosures

Project Information

Michael D. Kappelman, MD, MPH
The University of North Carolina at Chapel Hill
Anti-TNF Monotherapy versus Combination Therapy with Low Dose Methotrexate in Pediatric Crohn’s Disease

Key Dates

April 2015
September 2023

Study Registration Information


Has Results
Health Conditions Health Conditions These are the broad terms we use to categorize our funded research studies; specific diseases or conditions are included within the appropriate larger category. Note: not all of our funded projects focus on a single disease or condition; some touch on multiple diseases or conditions, research methods, or broader health system interventions. Such projects won’t be listed by a primary disease/condition and so won’t appear if you use this filter tool to find them. View Glossary
Populations Populations PCORI is interested in research that seeks to better understand how different clinical and health system options work for different people. These populations are frequently studied in our portfolio or identified as being of interest by our stakeholders. View Glossary
Intervention Strategy Intervention Strategies PCORI funds comparative clinical effectiveness research (CER) studies that compare two or more options or approaches to health care, or that compare different ways of delivering or receiving care. View Glossary
State State The state where the project originates, or where the primary institution or organization is located. View Glossary
Last updated: November 2, 2023