Severe-onset pediatric ulcerative colitis (UC) is significantly more likely to result in colectomy and the need for potent treatments such as calcineurin inhibitors (CI) and biologic agents, according to a study published in the Journal of Gastroenterology.
The prospective observational study evaluated the management and prognosis for severe-onset pediatric UC using the Japanese Pediatric Inflammatory Bowel Disease Registry (JPIBD-R), which enrolls newly diagnosed pediatric patients with inflammatory bowel disease (IBD) aged less than 18 years.
Participants were enrolled from October 1, 2012 to March 31, 2020 and were classified by Pediatric Ulcerative Colitis Activity Index (PUCAI) scores at diagnosis as severe onset (S1 group, PUCAI ≥65) or nonsevere onset (S0 group, PUCAI <65).
A total of 301 patients were included in the study (mean [SD] age at diagnosis, 11.8[3.4] years; men, 154); 75 patients were in the S1 group, and 226 were in the S0 group. At diagnosis, 70 patients (93%) in the S1 group presented with pancolitis (E4), compared with 68% in the S0 group (P <.0001). The mean observation period in the S1 group was 3.0[1.9] years vs 3.8[1.9] years in the S0 group.
During the observation period, the colectomy rate in the S1 group was 29%, which was significantly higher than the rate in the S0 group (6.2%, P <.001). A colectomy-free rate of 89% was observed in the S1 group within 1 year, 79% within 2 years, and 74% within 5 years, which was significantly lower compared with the S0 group (P =.0003). When analysis only included patients observed for over 18 months after diagnosis without colectomy, colectomy after at least 18 months postdiagnosis was conducted in 2 of 45 participants (4.4%) in the S1 group vs 9 of 182 in the S0 group (4.9%).
In the S1 group, 5-aminosalicylic acid was used in 74 (99%) patients, corticosteroids in 68 (91%), an immunomodulator in 51 (68%), CI in 41 (53%), and a biologic agent in 42 (56%). The proportion of patients receiving CI increased over time more in the S1 group (44% within 6 months vs 52% within 1 year) compared with the S0 group (P <.00001). Use of biologic agents also increased significantly to a greater degree over time in the S1 group (24% within 6 months and 41% within 1 year) vs in the S0 group (P <.00001).
For the S1 group, 35 patients received a CI as second-line therapy, and 19 patients received biologic agents as second-line therapy. Among those treated with CI as second-line therapy, 23 (66%) needed a biologic agent after discontinuation of CI and 12 (34%) required colectomy. No significant difference was found between the S1 and S0 groups (P =.72) in Kaplan–Meier analysis of the cumulative incidence of escalation to biologic agents or colectomy after CI treatment.
Among S1 patients receiving CI (23%), 8 did not need biologic agents or colectomy. In addition, 32% of S0 group participants who received CI as second-line therapy required neither of those escalations, and no difference was observed between the 2 groups (P =.46).
Researchers noted that some mild cases of IBD may have been treated at adult facilities or regional core hospitals without a pediatric gastroenterologist, and so the finding that 25% of patients had severe-onset UC may be an overestimate. In addition, medication doses and dosage intervals were not included in the registry, and the analysis could not assess the reasons for discontinuation of each medication.
“Considering increasing costs related to IBD, a therapeutic trial of CI in steroid-refractory cases rather than turning to biologic agents or colectomy immediately might reduce overall need for biologic agents as a maintenance therapy,” the study authors noted. “This likely would apply as well to cases with a less severe onset.”
Disclosure: Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.
Nambu R, Arai K, Kudo T, et al. Clinical outcome of ulcerative colitis with severe onset in children: a multicenter prospective cohort study. J Gastroenterol. Published online March 8, 2023. doi:10.1007/s00535-023-01972-1
This article originally appeared on Gastroenterology Advisor