Ustekinumab Use in Pediatric Inflammatory Bowel Disease: A French Multicenter Study From the Pediatric GETAID
Mounzer Koudsi1, Christine Martinez-Vinson2, Bénédicte Pigneur3,4
1From the Service de gastroentérologie, hépatologie et nutrition pédiatriques, Hospices Civils de Lyon, Lyon, France.
Insights
Ustekinumab effectively treated pediatric patients with moderate to severe Crohn disease and ulcerative colitis resistant to anti-tumor necrosis factor-alpha (TNF-α) therapy. The treatment significantly improved disease activity scores with no severe side effects observed in this study.
Area of Science:
- Pediatric Gastroenterology
- Immunology
- Pharmacology
Background:
- Ustekinumab is an established treatment for moderate to severe Crohn disease (CD) and ulcerative colitis (UC) in adults resistant to anti-tumor necrosis factor-alpha (TNF-α) therapies.
- Data on its efficacy and safety in pediatric populations, particularly those refractory to anti-TNF-α, remain limited.
- This study addresses the clinical outcomes of ustekinumab in French pediatric inflammatory bowel disease (IBD) patients.
Purpose of the Study:
- To evaluate the clinical course and effectiveness of ustekinumab treatment in pediatric patients diagnosed with Crohn disease (CD) or ulcerative colitis (UC).
- To assess the safety profile of ustekinumab in this specific pediatric cohort.
- To determine the impact of ustekinumab on disease activity indices in pediatric IBD patients previously treated with anti-TNF-α agents.
Main Methods:
- A retrospective, multicenter study involving pediatric patients treated with ustekinumab for IBD (CD and UC) between January 2016 and December 2019.
- Inclusion criteria comprised pediatric patients with CD or UC resistant to anti-TNF-α therapy.
- Disease activity was assessed using the Pediatric Crohn Disease Activity Index (PCDAI) and the Pediatric Ulcerative Colitis Activity Index (PUCAI) at induction, 3 months, and last follow-up.
Main Results:
- Fifty-three pediatric patients were enrolled: 48 with CD and 5 with UC. All patients were refractory to prior anti-TNF-α treatment.
- Significant improvements were observed in both CD and UC activity indices: PCDAI decreased from an average of 28.7 at induction to 10 at last follow-up, and PUCAI decreased from 47 to 18.3.
- While 51% of patients experienced side effects from previous anti-TNF-α treatments (e.g., psoriasis, anaphylaxis), no severe adverse events were reported with ustekinumab.
Conclusions:
- Ustekinumab demonstrates significant efficacy in treating pediatric patients with moderate to severe Crohn disease and ulcerative colitis who are resistant to anti-TNF-α therapy.
- The treatment led to a substantial improvement in disease activity scores, as measured by PCDAI and PUCAI.
- Ustekinumab is a safe and effective therapeutic option for pediatric IBD patients with a history of anti-TNF-α failure.
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.
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