Pediatr Gastroenterol Nutr. 2023 Mar 10. doi: 10.1097/MPG.0000000000003758.Online ahead of print.

Mounzer Koudsi ¹, Christine Martinez-Vinson ², Bénédicte Pigneur ^3 4, Stéphanie Willot ⁵, Djeddi Djamal ⁶, Raphael Enaud ⁷, Julie Rebeuh ⁸, Claire Dupont ⁹, Alain Dabadie ¹⁰, Valérie Bertrand ¹¹, Jean-Pierre Hugot ², Alain Lachaux ^1 12 13, Franck Ruemmele ³, Jérôme Viala ², Rémi Duclaux-Loras ^1 12 13

Author information

¹Service de gastroentérologie, hépatologie et nutrition pédiatriques - Hospices Civils de Lyon.

²Service de gastroentérologie et nutrition pédiatrique - Hôpital Robert Debré - Paris.

³Service de Gastro-entérologie et Nutrition pédiatrique, Centre de Référence des Maladies rares digestives (MARDI), Assistance Publique - Hôpitaux de Paris, Hôpital Necker Enfants malades, Paris, France.

⁴INSERM UMR S 1139, Faculté de Pharmacie de Paris, Paris, France.

⁵Médecine pédiatrique - CHRU de Tours - Hôpital Clocheville.

⁶Pédiatrie médicale et médecine de l'adolescent - CHU Amiens-Picardie.

⁷Service de gastroentérologie, hépatologie et nutrition pédiatriques - CHU Bordeaux.

⁸Service de gastro-entérologie pédiatrique - CHU de Strasbourg - Hôpital de Hautepierre.

⁹Service de gastro-entérologie pédiatrique - CHU de Caen.

¹⁰Service de Pédiatrie - CHU Rennes. Hôpital Sud, 16 boulevard de Bulgarie, BP 90347, 35203 Rennes Cedex 2, France.

¹¹Pédiatrie médicale - CH du Havre.

¹²U INSERM 1111, Centre de Recherche International en Infectiologie, équipe « Autophagy, Infection, Immunity ».

¹³Université Claude Bernard Lyon 1.

Abstract

Objectives: Ustekinumab is known to be efficient in adult patients suffering from moderate to severe Crohn's disease (CD) and ulcerative colitis (UC) resistant to anti- Tumor Necrosis Factor-alpha (TNFαHere, 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 to December 2019.

Results: 53 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 ileo-colitis. Perineal disease was observed in 20 out of 48 CD patients (41.7%), amongst them 9 were treated surgically.All patients included were resistant to anti-TNFα reatment. 51 percent had presented side effects linked to anti-TNFα, including psoriasis and anaphylactic reaction. The average Pediatric Crohn's 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 (PUCAI) 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 paediatric patients resistant to anti-TNFα. PCDAI has been significantly improved in patients with severe disease, treated with Ustekinumab.