Maintenance treatment with upadacitinib helps sustain remission in children with refractory ulcerative colitis (UC), according to a retrospective study.
In a cohort of patients who experienced treatment failure on advanced therapies, the primary endpoint of sustained corticosteroid-free clinical remission (CFR) through week 52 was achieved in 60 percent. [ESPGHAN 2026, abstract OP100]
Presenting study author Dr Anat Yerushalmy-Feler from Tel Aviv Sourasky Medical Center, Tel Aviv, Israel, defined the primary endpoint as a Pediatric Ulcerative Colitis Activity Index (PUCAI) score of <10 at both weeks 26 and 52.
Half of the patients who had sustained CFR also had faecal calprotectin levels below 150 mcg/g, Yerushalmy-Feler added.
Among patients who underwent endoscopic or radiologic evaluation, 41 percent had endoscopic remission (ie, Mayo endoscopic score of 0) and 71 percent had radiologic remission (ie, complete normalization of findings on MRE or intestinal ultrasound).
Failure of three to four biologic agents was a negative predictor of sustained corticosteroid-free clinical remission (vs one to two: odds ratio [OR], 0.15, 95 percent confidence interval [CI], 0.05–0.61; p=0.008), whereas achieving CFR by the end of induction at week 8 was a positive predictor (OR, 29.7, 95 percent CI, 7.86–112.3; p<0.001).
Week-8 PUCAI was the best predictor of sustained CFR, noted Yerushalmy-Feler.
At a cutoff of ≤5, week-8 PUCAI score predicted sustained CFR with an area under the curve of 80 percent, sensitivity of 72 percent, specificity of 82 percent, positive predictive value of 87 percent, and negative predictive value of 63 percent.
As for adverse events (AEs), the most common were hyperlipidaemia (19 percent), infections (17 percent), and acne (13 percent). Two serious AEs were reported, including an incidental finding of a neuroendocrine tumour in the appendix and cytomegalovirus colitis. There were no incidence of thromboembolic events and other malignancies.
“This is the largest and the first multicentre study on upadacitinib for maintenance therapy in paediatric UC,” Yerushalmy-Feler said.
The findings provide evidence of the drug’s efficacy, she added. “However, as always, when we use off-label therapies, the efficacy should be weighed against the potential risk of AEs.”
The study included 105 children and adolescents with UC who received treatment with upadacitinib for the maintenance of remission at 35 centres affiliated with the Pediatric IBD Interest and Porto groups of the ESPGHAN, as well as those in North America.
The mean age of the patients was 14.6 years, and 65 percent had pancolitis. The median disease duration was 2.3 years. All patients had failed previous advanced therapies, mainly biologics, with 31 percent having failed more than two biologics.
In terms of upadacitinib dosing, patients weighing >40 kg received the adult recommended dose of 45 mg for induction and 30 mg for maintenance. For those weighing <40 kg, dose was adjusted to body surface area and weight, with 1.3 mg/kg used for induction and 1 mg/kg used for maintenance.
“Generally, the outcomes of children who weighed <40 kg were comparable to those of children who weighed >40 kg, meaning that the drug was effective in young children,” Yerushalmy-Feler pointed out.
Among patients weighing <40 kg, those who did vs did not achieve postinduction remission received numerically higher dose of upadacitinib, she said. “The difference was not statistically significant due to the very small number of patients in this group, but it might be important to some patients.”