Infliximab for pediatric patients with ulcerative colitis: a phase 3, open-label, uncontrolled, multicenter trial in
Hitoshi Tajiri1, Katsuhiro Arai2, Seiichi Kagimoto3
1Department of Pediatrics, Osaka General Medical Center, Osaka, Japan. tajiriji@gh.opho.jp.
Insights
Infliximab (IFX) effectively improved symptoms in pediatric ulcerative colitis (UC) patients. This study supports IFX as a treatment option for moderate-to-severe pediatric UC unresponsive to other therapies.
Area of Science:
- Gastroenterology
- Pediatric Gastroenterology
- Immunology
Background:
- Pediatric ulcerative colitis (UC) often presents with more severe disease activity than adult UC.
- Early treatment intensification is crucial for achieving and maintaining remission in pediatric UC.
- Existing treatments may be inadequate for some pediatric UC patients.
Purpose of the Study:
- To evaluate the efficacy and safety of infliximab (IFX) in pediatric patients with moderate-to-severe UC.
- To assess the pharmacokinetic profile of IFX in this patient population.
- To determine IFX effectiveness in patients with an inadequate response to current therapies.
Main Methods:
- An open-label, uncontrolled, multicenter Phase 3 trial involving 21 pediatric patients (aged 6-17 years) in Japan.
- Patients received IFX at 5 mg/kg at Weeks 0, 2, and 6.
- Responders at Week 8 received IFX every 8 weeks through Week 22, with evaluation at Week 30.
Main Results:
- IFX rapidly improved clinical symptoms, with sustained effects up to 30 weeks.
- Remission rates were 42.9% (CAI-based) and 19.0% (PUCAI-based).
- Adverse events occurred in 95.2% of patients, with serious adverse events in 14.3%.
Conclusions:
- Infliximab (IFX) demonstrates efficacy in treating pediatric ulcerative colitis (UC) patients.
- The study supports IFX as a viable treatment option for pediatric UC with inadequate response to existing therapies.
- Sustained IFX concentrations were observed in responders.
Background:
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.
Methods:
This 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.
Results:
A 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.
Conclusions:
These results support the use of IFX in the treatment of pediatric patients with UC with inadequate response to existing treatment.
Trial Registration:
ClinicalTrials.gov, registration number: NCT01585155 .
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