Abstract
BackgroundPediatric ulcerative colitis (UC) is typically more extensive and has a more active disease course than adult UC, and requires early treatment augmentation to achieve and maintain disease remission. The present study aimed to investigate the efficacy, safety, and pharmacokinetic profile of infliximab (IFX) in pediatric patients with moderate-to-severe UC and inadequate response to existing treatment.MethodsThis open-label, uncontrolled, multicenter, Phase 3 trial was conducted at 17 centers in Japan between April 2012 and September 2014. Pediatric patients (aged 6–17 years) diagnosed with moderate-to-severe UC received a treatment protocol comprising 5 mg/kg IFX at Weeks 0, 2, and 6, and Clinical Activity Index (CAI)-based responders at Week 8 also received treatment at 8-week intervals at Weeks 14 and 22, with a final evaluation at Week 30.ResultsA total of 21 patients were treated in this study. IFX therapy rapidly improved clinical symptoms, and this effect was maintained for up to 30 weeks. Overall CAI-based remission rate was 42.9% and overall Pediatric Ulcerative Colitis Activity Index (PUCAI)-based remission rate was 19.0%. Median partial Mayo score was 6.0 at baseline and 4.0 at Week 30 (overall). Among the eight patients who underwent sigmoidoscopy, Mayo response was achieved at Week 30 (overall) in three patients (37.5%). Trough serum IFX concentrations in Week 8 CAI-based responders were maintained throughout the study period. Adverse events and serious adverse events were observed in 95.2 and 14.3% of patients, respectively.ConclusionsThese results support the use of IFX in the treatment of pediatric patients with UC with inadequate response to existing treatment.Trial registrationClinicalTrials.gov, registration number: NCT01585155.
Highlights
Pediatric ulcerative colitis (UC) is typically more extensive and has a more active disease course than adult UC, and requires early treatment augmentation to achieve and maintain disease remission
We examined a treatment protocol comprising 5 mg/kg IFX administered at Weeks 0, 2, and 6, with Week 8 responders receiving treatment at 8-week intervals at Weeks 14 and 22, with a final evaluation at Week 30. This is the first Phase 3 study of IFX in Asian pediatric patients with UC. This multicenter, open-label, uncontrolled, Phase 3 trial was conducted at 17 centers in Japan between April 2012 and September 2014, in accordance with the ethical principles described in the Declaration of Helsinki and in compliance with Good Clinical Practice guidelines
Efficacy Marked improvements in the median Clinical Activity Index (CAI) score were observed from baseline (9.0 [8.0, 11.0]) as early as Week 2 of treatment (4.0 [1.0, 5.5])
Summary
Pediatric ulcerative colitis (UC) is typically more extensive and has a more active disease course than adult UC, and requires early treatment augmentation to achieve and maintain disease remission. The present study aimed to investigate the efficacy, safety, and pharmacokinetic profile of infliximab (IFX) in pediatric patients with moderate-to-severe UC and inadequate response to existing treatment. Ulcerative colitis (UC) is an inflammatory disease of unknown etiology that is characterized by repeating periods of relapse and remission. When age of presentation is considered, 20.0% of Patients with moderate-to-severe UC typically receive corticosteroid therapy as primary treatment [5, 6, 8]; as corticosteroid therapy affects growth and bone density, it is important to utilize corticosteroidsparing treatment strategies in pediatric UC [5, 6]. Administration of appropriate immunomodulators is recommended for those with corticosteroiddependent disease [4, 6]
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