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Early response to Stelara linked to better outcomes in pediatric IBD
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WASHINGTON — Children with inflammatory bowel disease who achieve an early response to Stelara tend to have better long-term outcomes, according to research presented at Digestive Disease Week.
In his presentation, Steven J. Fusillo, MD, of the Children’s Hospital of Philadelphia IBD Center discussed unique challenges that differ from what is seen in adult IBD, including disease extent and risk for surgery, particularly those centered on Stelara (ustekinumab, Janssen).
“There are few therapeutic options for pediatric IBD beyond the anti-TNFs,” Fusillo said in his presentation. “Ustekinumab has been shown to have efficacy in treating adults with moderate-to-severe Crohn’s disease. ... While the adult literature continues to expand, there has been very little published in the pediatric population.”
Fusillo and colleagues conducted a single-center, prospective cohort study to determine the response rate to ustekinumab and assess potential differences between responders and non-responders to therapy in refractory pediatric IBD.
Investigators enrolled 20 patients younger than 21 years with Crohn’s disease or indeterminate colitis receiving ustekinumab therapy at the Children’s Hospital of Philadelphia. They included patients with prior anti-TNF failure and clinically active disease.
Patients received an intravenous injection at induction and subcutaneous injections every 8 weeks. The primary outcome for the trial was clinical response defined as decrease in pediatric disease index scores.
Fusillo and colleagues found that 25 patients achieved clinical response by week 6 (52%), and 20 achieved clinical response by their last follow-up (45%).
Investigators found that initial responders at week 6 had better outcomes regarding long-term clinical remission compared with non-responders at week 6 (64% vs. 11%, P < .028). Non-responders appeared to have higher rates of surgery and ustekinumab discontinuation, however Fusillo said neither reached statistical significance.
“There’s clearly some patients that demonstrate response at least in terms of clinical response, but there’s a subset of patients who do not respond favorably, and many of them undergo surgery within a matter of months,” Fusillo said. “There’s a promising role for ustekinumab in pediatric IBD, even in a medically complex and refractory population.” - by Alex Young
Reference:
Fusillo SJ, et al. Abstract 329. Presented at: Digestive Disease Week; June 2-5, 2018; Washington, D.C.
Disclosures: Fusillo reports no relevant financial disclosures. Please see the DDW faculty disclosure index for a list of all other authors’ relevant financial disclosures.
Perspective
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Joel R. Rosh, MD, FAAP, FACG, AGAF
This study included a very small group of patients who were given a medication that is currently licensed for patients aged older than 18 years. It can be challenging for us to know the optimal dosing, and to obtain such a medication for our pediatric patients. So, the authors did a very good job of pulling together 20 patients from their own center, but we recognize that this is very preliminary data. The researchers looked at two time points: they looked at 8 weeks and they looked at 24 weeks.
Of the 65% of patients who responded by 8weeks, only about half (38%) were still in clinical response at 24 weeks, so about half who responded at 8 weeks fell out of response. None of the patients who had not responded by 8 weeks had had a response by 24 weeks. The researchers then sliced the data a little bit more and said that of the patients who had already been on two anti-TNFs, none of them responded and the Crohn’s patients seemed to do better than the colitis patients. The researchers are suggesting that further studies on pediatric use of Stelara look to see if there’s a difference between colonic patients and small bowel patients, and that our highest-risk patients, those who have been failed by multiple biologics, are probably the hardest to get to respond to this medication. So, that, again is a lot of findings from a very small group of patients. To me the most positive finding is that 65% had early response. The fact that 65% responded to the intravenous load gives us a clear signal that we should not be surprised that this drug will work for pediatric patients and now we must do the work of figuring out correct dosing and how to optimize maintenance. It is unfortunate that with this medication, as with all new medications in IBD, we’re only first starting to figure out pediatric dosing after adult labeling. Such a scenario has been recognized as a barrier to performing pediatric clinical trials since the same agent can be given off label.
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