Clinically meaningful rates of response and remission were observed in pediatric patients with ulcerative colitis receiving treatment with adalimumab. These results suggest adalimumab treatment is safe and effective for these children, according to research results published in The Lancet Gastroenterology & Hepatology.
This randomized, double-blind trial included 93 children with active, moderate to severe ulcerative colitis, aged 4 to 17 years old (ClinicalTrials.gov Identifier: NCT02065557). The study began with an 8-week induction period followed by a 44-week maintenance period.
During the induction phase, participants were randomly assigned to receive either high-dose induction adalimumab (2.4 mg/kg [maximum 160 mg] at weeks 0 and 1) or standard-dose induction adalimumab (2.4 mg/kg [maximum 160 mg] at week 0 and placebo at week 1). At Week 2, both groups received 1.2 mg/kg (maximum 80 mg) and 0.6 mg/kg (maximum 40 mg) at weeks 4 and 6.
During the 44-week maintenance period, the high-dose group received maintenance adalimumab at 0.6 mg/kg (maximum 40 mg) weekly and the standard-dose group received the same dosage, but on an every other week schedule.
Of the 93 children included in the study, 77 were randomly assigned to receive either high-dose or standard-dose induction adalimumab, while 16 received high-dose induction adalimumab following a change in study design. There were 47 patients within the high-dose induction group and 30 in the standard-dose induction group.
By week 8 of the 8-week induction period, 77 (83%) patients were partial Mayo score responders (a decrease of 2 points or more and ≥ 30% decrease from baseline partial Mayo score). In total, 80% of the responders continued adalimumab as maintenance therapy. During the maintenance period, 62 patients were randomly assigned to receive either high-dose or standard-dose maintenance adalimumab while 12 patients received placebo.
Compared with the placebo group, the adalimumab groups achieved notably higher remission rates. At week 8, 53% of the 77 patients receiving adalimumab were in partial Mayo score remission (P <.0001). At week 52, 37% of 62 patients in the adalimumab group were in full Mayo score remission (P =.0001).
During exposure to adalimumab, 21 (23%) patients reported 1 or more treatment-emergent serious adverse events. While most reported serious adverse events were not in fact treatment related and did not lead to study discontinuation, 5 (5%) patients experienced adverse events that were potentially linked to the study-drug. These included: headache, loss of consciousness, and injection site inflammation. These events resulted in drug interruption.
This study was limited by its low number of patients included in the cohort. Additionally, the overall study population was racially homogenous, as most patients were from Eastern Europe.
The researchers concluded, “Clinically meaningful rates of clinical remission and response, including steroid-free remission and mucosal healing, were reported in children with moderate-to-severe ulcerative colitis who received adalimumab. No new safety signals were observed, suggesting that adalimumab is an efficacious and safe treatment option for children with moderate-to-severe ulcerative colitis.”
Disclosure: This clinical trial was supported by AbbVie. Please see the original reference for a full list of authors’ disclosures.
Croft NM, Faubion WA, Kugathasan S, et al. Efficacy and safety of adalimumab in pediatric patients with moderate-to-severe ulcerative colitis (ENVISION I): a randomised, controlled, phase 3 study. Lancet Gastroenterol Hepatol. Published online June 18, 2021. doi: 10.1016/S2468-1253(21)00142-4