Ustekinumab in paediatric patients with moderately to severely active Crohn's disease: UniStar study long-term extension results

Dan Turner*, Joel R. Rosh, Stanley A. Cohen, Anne M. Griffiths, Jeffrey S. Hyams, Jarosław Kierkuś, Omoniyi J. Adedokun, Richard Strauss, Lilianne Kim, Sheri Volger

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

Abstract

Objectives: To assess the efficacy, safety, immunogenicity, and pharmacokinetics through 240 weeks of ustekinumab treatment in paediatric patients from the long-term extension (LTE) of the phase 1, double-blind UniStar trial. Methods: Paediatric patients with moderately to severely active Crohn's disease (CD) were randomised 1:1 and stratified by body weight (<40 or ≥40 kg) to low- or high-dose intravenous ustekinumab followed by a subcutaneous maintenance dose at Week 8. At Week 16, patients were eligible to enter the LTE at the discretion of the investigator and continued maintenance dosing every 8 weeks up to Week 240. Results: Of the 34 patients who entered the LTE, 25 patients with evaluable data completed Week 48, and 41.2% (14/34) achieved clinical remission at Week 48. Among the 24 patients with Week-0 C-reactive protein (CRP) levels ≥3 mg/L, 29.2% (7/24) achieved normalisation of CRP at Week 48, while imputing missing data as failures. Through Week 240, the most common adverse events were infections (n = 28) and gastrointestinal disorders (n = 26). The most common serious adverse event was worsening of CD (n = 6). Only one patient had detectable antibodies to ustekinumab. Median serum ustekinumab concentrations remained consistent through Week 48, were detectable through Week 224, and trended lower in patients <40 kg. Conclusions: Efficacy and pharmacokinetics through 1 year and safety and immunogenicity through 4 years of ustekinumab treatment in paediatric patients with CD were generally comparable to those previously reported in adults.

Original languageAmerican English
JournalJournal of Pediatric Gastroenterology and Nutrition
DOIs
StateAccepted/In press - 2024

Bibliographical note

Publisher Copyright:
© 2024 European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition.

Keywords

  • biologics
  • Crohn's disease (CD)
  • inflammatory bowel disease (IBD)
  • paediatric gastroenterology

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