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

Abstract

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-α). 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-α treatment. Fifty-one percent had presented side effects linked to anti-TNF-α, 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-α. PCDAI has been significantly improved in patients with severe disease, treated with ustekinumab.

Ustekinumab for anti-tumor necrosis factor refractory pediatric ulcerative colitis: a promising approach towards endoscopic healing

“…This is higher than that reported in other large pediatric studies where SFCR was achieved by 44% of patients at week 52, and 62% of patients at last follow-up visit. 9,10 Possible explanations include different regimen of prescribing and tapering steroids by the providers or difference in the disease phenotype. In all these studies, failure of prior biologic therapy was associated with decreased likelihood of achieving remission.…”

“…In all these studies, failure of prior biologic therapy was associated with decreased likelihood of achieving remission. [8][9][10] We did not have any biological naïve group for comparison, so this could not be fully assessed. However, prior biological failure did not seem to affect the primary outcome of SFCR in our study.…”

Ustekinumab Offers Long-Term Clinical Remission With Safety in Very Early-Onset Inflammatory Bowel Disease

“… 1 , 2 Ustekinumab (UST), a human monoclonal antibody directed against P40, a subunit of interleukin-12 and interleukin-23, is approved for treatment of refractory ulcerative colitis (UC) and Crohn’s disease (CD). The efficacy and safety of UST in IBD have been reported in adults and older children 3-5 but not yet in VEO-IBD patients.…”