Ustekinumab Use in Pediatric Inflammatory Bowel Disease: A French Multicenter Study From the Pediatric GETAID

Objectives: Ustekinumab is known to be efficient in adult patients suffering from moderate to severe Crohn disease (CD) and ulcerative colitis (UC) resistant to anti-tumor necrosis factor-alpha (TNF-alpha). Here, we described the clinical course of treatment with ustekinumab in French pediatric inflammatory bowel disease (IBD) patients treated with ustekinumab. Methods: This study includes all pediatric patients treated by ustekinumab injection for IBD (CD and UC), between January 2016 and December 2019. Results: Fifty-three patients were enrolled, 15 males and 38 females. Forty-eight patients (90%) had a diagnosis of CD and 5 (9.4%) had UC. Sixty-five percent of CD patients presented an ileocolitis. Perineal disease was observed in 20 out of 48 CD patients (41.7%), among them 9 were treated surgically. All patients included were resistant to anti-TNF-alpha treatment. Fifty-one percent had presented side effects linked to anti-TNF-alpha, including psoriasis and anaphylactic reaction. The average Pediatric Crohn Disease Activity Index (PCDAI) at induction was 28.7 (5-85), 18.7 (0-75) at 3 months of treatment and 10 (0-35) at the last follow-up. The average Pediatric Ulcerative Colitis Activity Index at induction was 47 (25-65), 25 (15-40) at 3 months of treatment and 18.3 (0-35) at the last follow-up. No severe side effects were observed. Conclusion: In this retrospective, multicentral study, ustekinumab proved to be efficient in pediatric patients resistant to anti-TNF-alpha. PCDAI has been significantly improved in patients with severe disease, treated with ustekinumab.