Ulcerative Colitis Clinical Trial End Points and Metrics


Experts in gastroenterology share the clinical trial end points and metrics they use to guide their treatment of patients with ulcerative colitis.

Maia Kayal, MD: In addition to the normalization of stool frequency and a decrease in rectal bleeding, a very important metric that I look for in patients on therapy for moderate to severe ulcerative colitis, and in clinical trial data as well, is the improvement of their quality of life. The symptoms of ulcerative colitis are significant and can be quite debilitating, and it is important that our therapies improve quality of life for patients such that they can return to their baseline quality of life. An additional metric I’m interested in looking at for patients with moderate to severe ulcerative colitis, and that I am hopeful clinical trials will incorporate, is the use of intestinal ultrasound. We’re increasingly understanding that ulcerative colitis is not only a mucosal disease, but is also a transmural disease similar to Crohn disease. We are increasingly using intestinal ultrasound in the clinic as a noninvasive point-of-care diagnostic and prognostic plan. Intestinal ultrasound has been used in Europe for decades for the assessment of patients with ulcerative colitis and Crohn disease…. Incorporating intestinal ultrasound into your follow-up and monitoring of patients is adjunctive to the use of C-reactive protein and fecal calprotectin.

Increasingly, clinical trials have been using histologic healing as a metric and a treatment goal, which I think is very exciting. There have been conflicting data to suggest how far in remission we need to get our patients. Our treatment guidelines indicate that remission includes both clinical and endoscopic end points. However, histologic end points have not been fully understood or studied. Increasingly, clinical trial data are incorporating histologic healing as an end point. It’s important because it will impact treatment decisions. For example, in a patient who is in clinical and endoscopic remission, but still has histologic activity in their colon, is it worth optimizing or escalating therapy, or can we maintain the patient on the current regimen? In the future, increasing data will support either escalating therapy or maintaining the current regimen based on the disease activity on the histological level.

David P. Hudesman, MD: Over the past 7 years or so now, we’ve moved from treating just to symptom improvement or clinical remission, to making sure a patient is healed inside. Our goal of therapy is to lead to that endoscopic remission. Many of our trials, if not all of them, now have these combined end points looking at both symptoms and endoscopic activity. What our unmet need is, and what we’re starting to see in clinical trials, is that quality of life part. A lot of times we’re seeing patients in the office and I’m focusing on how many times a day they’re going to the bathroom, how much pain they’re having. But there are other factors: whether they’re making it to work, they’re going out and seeing friends and family, their energy levels and so forth, that aren’t really captured. I think some end points we’d like to see in clinical trials, and we’re starting to see them, are surveys looking at fatigue, work productivity, as well as anxiety and depression. This is how we could take that next step in our care and really improve our patients’ quality of life. We’re not only focusing on symptoms, but again work productivity, fatigue, and underlying anxiety.

Transcript Edited for Clarity

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