Results Summary

What was the research about?

Ulcerative colitis, or UC, and Crohn’s disease, or CD, are types of inflammatory bowel disease, or IBD. UC and CD cause long-term diarrhea and stomach pain. Medicines called anti-tumor necrosis factors, or anti-TNFs, are the first option for treating UC and CD. But sometimes anti-TNFs don’t work on IBD symptoms.

In this study, the research team compared how well other medicines worked to treat UC and CD when anti-TNFs didn’t work. For patients with UC, the team compared vedolizumab and tofacitinib. For patients with CD, the team compared vedolizumab and ustekinumab.

To compare medicines, the research team tracked pain and fatigue among patients starting these medicines. To see if patients continued taking their medicine, the team looked at a larger group of patients who took these medicines in the past. Stopping a medicine can be a sign that patients have side effects or difficulty taking their medicine.

What were the results?

In patients with UC, patients who took vedolizumab and those who took tofacitinib didn’t differ in fatigue or how much pain interfered with their daily life. Patients who took vedolizumab were more likely to continue taking their medicine than patients who took tofacitinib.

In patients with CD, patients who took vedolizumab and those who took ustekinumab didn’t differ in fatigue or how much pain interfered with their daily life. Patients who took the two medicines didn’t differ in how likely they were to continue their medicine.

Who was in the study?

To look at fatigue and pain, the study included 465 patients with IBD. Of these, 87 percent were White, 3 percent were Black, and 10 percent were another race. The average age was 45, and 73 percent were women. 

To look at how long patients continued taking their medicine, the study included health insurance records for 2,116 patients with IBD; the average age was 43 and 53 percent were women. The research team didn’t collect information about race.

What did the research team do?

To look at fatigue and pain, the research team reviewed surveys from when patients started a medicine and 10 months later. Surveys were from IBD Partners, a patient-powered research network, or PPRN. In PPRNs, patients, families, caregivers, and community members share health data with the network. They work closely with researchers to plan and conduct research. 

To look at how long patients took their medicine, the research team looked at insurance records from a large national health insurer to find patients who started one of the medicines after anti-TNFs didn’t work. The team looked at records 15 months after patients started the second medicines.

Patients with IBD helped design the study.

What were the limits of the study?

Fewer patients completed surveys than expected. Also, most of the patients surveyed were White. Results may have differed if more patients and more diverse patients completed surveys.

Future research could look at why some patients stop taking medicines more often than others.

How can people use the results?

Doctors and patients can use these results when considering treatment for UC and CD when anti-TNFs don’t work.

Final Research Report

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

Peer-Review Summary

Peer review of PCORI-funded research helps make sure the report presents complete, balanced, and useful information about the research. It also assesses how the project addressed PCORI’s Methodology Standards. During peer review, experts read a draft report of the research and provide comments about the report. These experts may include a scientist focused on the research topic, a specialist in research methods, a patient or caregiver, and a healthcare professional. These reviewers cannot have conflicts of interest with the study.

The peer reviewers point out where the draft report may need revision. For example, they may suggest ways to improve descriptions of the conduct of the study or to clarify the connection between results and conclusions. Sometimes, awardees revise their draft reports twice or more to address all of the reviewers’ comments.

Peer reviewers commented and the researchers made changes or provided responses. Those comments and responses included the following:

  • The reviewers noted that the original plan for managing missing data was to use multiple imputation to account for missing data with the assumption that data would be missing at random, but this apparently was not done as described. The researchers explained that they did not impute data for baseline symptoms because participants missing some of the symptom measures typically were missing all of them because of the timing of baseline assessment. The researchers pointed out that they did use multiple imputation for missing covariates in the inverse probability of treatment weighting (IPTW) model unless variables had extensive missing data.
  • The reviewers felt that a limitation of the study was using unadjusted analyses in measuring subgroup differences. The researchers explained in the report that because there was general consistency between adjusted and unadjusted analyses for the main analyses, and sample sizes for the subgroups were small, they could only conduct unadjusted analyses. The researchers did identify this as a limitation in their discussion section.
  • The reviewers requested that the researchers describe how their study limitations might have affected their results. Responding to this request, the researchers posited that issues like underreporting or misclassification of covariates was possible in the claims data, but such errors were likely to bias the results toward the null hypothesis. Similarly, they noted that loss to follow-up was relatively high but likely to be more prevalent among participants with more favorable outcomes, again potentially biasing the results towards the null.

Conflict of Interest Disclosures

Project Information

Michael D. Kappelman, MD, MPH^
The University of North Carolina at Chapel Hill^
$2,794,763
Comparative Effectiveness of Biologic or Small Molecule Therapies in IBD

Key Dates

April 2018
June 2023
2018
2024

Study Registration Information

^Angela Dobes, MPH, was the original principal investigator of this study, and the original organization was the Crohn's & Colitis Foundation in New York.

Tags

Has Results
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: March 15, 2024