Jeffrey Hyams, MD, of the University of Connecticut and Connecticut Children’s Hospital in Hartford, Connecticut, shares results from the UNIFI JR trial evaluating ustekinumab in pediatric patients with ulcerative colitis who did not respond to conventional therapies.
From these results with ustekinumab, Dr. Hyams stated, “We hopefully will have regulatory approval of an additional agent for the treatment of pediatric ulcerative colitis.”
Transcript:
This transcript has been edited for clarity.
Jeffrey Hyams, MD: My name is Jeffrey Hyams. I’m a Professor of Pediatrics at the University of Connecticut and the Head of the Division of Digestive Diseases, Hepatology, and Nutrition at Connecticut Children’s Medical Center in Hartford, Connecticut.
Gastro Today: What is the rationale for the UNIFI JR study?
As of now, the number of advanced therapies that we have available for pediatric ulcerative colitis are very few. In fact, they’re limited to anti-TNF [tumor necrosis factor] agents. There is a crying need for additional therapies for children with ulcerative colitis that is unresponsive to conventional therapies such as 5-ASA [5-aminosalicylic acid], steroids, etc. The UNIFI JR trial was started to get more information on the safety and effectiveness of ustekinumab in children with ulcerative colitis.
What was the trial design?
The trial itself was one in which children under the age of 17 years were given a single IV induction dose, which if you were over 40 kg was weight-based, and if you were less than 40 kg was body surface area based. That was an attempt to have similar serum levels of the drug, no matter how big you were. Everyone got an IV induction and then eight weeks later, maintenance therapy started. That was given subcutaneously either every eight weeks or every 12 weeks. In order to maintain the blind, placebo subcutaneous injections were given, so we didn’t know which group was which. That was followed out to week 52. People who were responders at week eight were allowed to move forward in the trial.
What were the results of the trial?
Very similar to what was seen in the adult trials, in the sense that clinical remission at week eight was in about 25% of patients, clinical response was in around 70% of patients. Of the week eight responders, 49% of those who got drug every eight weeks were in clinical remission at week 52. If you take off the original cohort, it’s 49% of around 70%, it’s around 35%.
What is the significance of these results?
The significance is that we hopefully will have regulatory approval of an additional agent for the treatment of pediatric ulcerative colitis. These numbers are quite similar to the adult UNITI trials, so there really is not much of a difference in response and remission between children and adults.
Why is it so important to have another agent available for this patient population?
Unfortunately, I wish everybody responded to an anti-TNF, they don’t. And just as in adults, where there’s a therapeutic ceiling, not more than 50% or 60% are getting better.
This gives us another option. The safety of this drug is excellent, and therefore I think it’ll be a very attractive therapy for individuals who have failed in anti-TNF. A bigger question moving forward is whether or not it will be used as primary advanced therapy. We do know, and even in this limited trial, those who were bio-naive, which really meant anti-TNF–naive, did better than those who were anti-TNF–experienced.