Patients and Researchers Join Forces to Fight Inflammatory Bowel Disease

I remember first learning about the immune system in elementary school. “Think of it like a very powerful army,” my teacher told me. “Different soldiers have different jobs, but everyone fights together against germs, who are the bad guys.” The lesson faded in my memory until a few years later, when, as a young teen, I quietly endured a 40-pound weight loss and several months of pain, nausea, and severe rectal bleeding. A dramatic collapse after a ski trip and subsequent hospitalization revealed my “army’s” new target: me. A kind nurse explained, “We don’t know why it happens, but some people’s immune systems attack their intestines as if they were germs.”

A Lifelong Illness

As many as 70,000 new cases of Crohn’s disease and ulcerative colitis, collectively known as inflammatory bowel diseases (IBD), are diagnosed in the United States each year. In 2011, the US Senate voted to support a week dedicated to raising awareness of the diseases. The Senate expressed appreciation for families, caregivers, and healthcare professionals who support people living with Crohn's disease and ulcerative colitis, and it commended biomedical researchers whose work aims to develop new treatments.

PCORI is supporting several projects that aim to help patients and those who care for them make informed decisions about treatment options.

Patient Partner Jessica Burris and others talk about CCFA Partners and how this PPRN is supporting those living with IBD. (Video provided courtesy of CCFA Partners PPRN)

As my nurse explained years ago, IBD involves autoimmune inflammation and damage to the gastrointestinal tract. It affects approximately 1.6 million people in the United States, causing illness and diminishing quality of life. The annual financial burden of IBD in the United States is estimated at more than $31 billion. Most patients are diagnosed before age 35. The diseases can be treated, but there is no cure.

What does this disease look like on an individual level? Its clinical course is highly variable, with many patients experiencing periods of remission and relapse. Some patients need surgery to remove severely inflamed organs; it took months of inpatient treatment and 10 surgeries during my teens and 20s to relieve my symptoms.

There is a critical need for research that gives patients and clinicians more evidence so that they can make informed decisions about care. Researchers are rapidly making discoveries about how genetic, immunological, and microbiological factors are involved in the development of IBD. So more treatment options are becoming available. However, not all treatments are effective for all people, and IBD patients, caregivers, and clinicians need to be able to collaboratively and confidently choose the best option for each patient.

Evidence for Selecting Among Options

PCORI is addressing the challenge of helping patients obtain effective treatments. Below are some examples of that work. Two of them come from PCORnet, PCORI’s national patient-centered clinical research network. These projects are both Patient-Powered Research Networks (PPRNs), operated and governed by groups of patients interested in sharing health information and participating in research. PPRNs also include partners who are researchers.

I am on the Patient Governance Committee of one of these networks: the CCFA (Crohn’s and Colitis Foundation of America) Partners PPRN. It’s an innovative project that brings together CCFA, which serves more than 300,000 patients, and researchers at the University of North Carolina School of Medicine. In CCFA Partners, more than 14,000 patients are providing information about their health and experiences living with IBD. They can also submit and vote on research questions for researchers to answer. CCFA Partners also connects patients with research opportunities, and the network plans to collaborate in research studies, including a PCORI-funded pragmatic clinical study. A better understanding of how health behaviors and treatment therapies affect IBD in different people will lead to more effective, personalized treatments.

Another PPRN is the ImproveCareNow Network (ICN), which aims to advance the health and care for children and youth with IBD. Currently, 84 ICN centers in 36 states, the District of Columbia, and the United Kingdom provide care for more than 24,000 patients. ICN has engaged patients, families, and clinicians to generate a prioritized list of research topics. In response, the network is planning three studies over the next three years. The first is an online observational study of the natural history of IBD and the relationship between disease activity, symptoms, and health-related quality of life. The second is a historical study focused on treatment safety that requires a large sample to document rare outcomes. Finally, the network will participate in a comparison of two treatments for moderate to severe Crohn’s disease. ICN has been featured on PCORI’s website.

PCORI recently funded a pragmatic clinical study that looks at treatments targeting a natural protein—called tumor necrosis factor (TNF)—that promotes inflammation. In the study, all patients will receive an anti-TNF drug, and some will add a low dose of methotrexate, another medication that tempers immune system activities. North Carolina researchers will recruit about 450 patients younger than 18 years old through ICN and follow them for two years. The researchers have also partnered with CCFA, children’s hospitals, healthcare payers, and pharmaceutical companies.

A separate study, begun in 2013, compares the safety and effectiveness of anti-TNF therapy with intermittent use of corticosteroids, the previous treatment strategy. Fear of complications from long-term suppression of the immune system often deters patients from pursuing anti-TNF therapies. The Pennsylvania team is examining how patients choose a therapy. It is also analyzing data previously collected on more than 14,000 Medicare beneficiaries with IBD. Using the two sets of results, the team plans to take into account patient preferences for different outcomes as it computes the relative benefits and risks of the treatment strategies.

I look forward to the findings of these and other projects. They will add to the evidence useful to IBD patients and those who take care of them as they make decisions about treatments. Please let PCORI know your ideas for other projects in the comment area below.

The views expressed here are those of the author and not necessarily those of PCORI.