Long-term effects of ustekinumab in children with inflammatory bowel disease: A systematic review

Ustekinumab, a monoclonal antibody used in adults, is increasingly employed in children and adolescents with refractory inflammatory bowel disease (IBD). This review aimed to analyze its long-term efficacy and safety in the pediatric population. To this end, a systematic review was registered under PROSPERO (CRD42024555896). Articles indexed in PubMed/Medline, Cochrane Library, and Web of Science up to May 30, 2024, were screened. Quality was assessed using the Newcastle-Ottawa Scale. From 563 articles, 11 observational studies were included, analyzing 444 pediatric IBD patients. Remission rates varied: 47% at Week 16, 57%-59% at Week 26, and 40%-64% at Week 52. In addition, clinical improvements included better Z-scores, body mass index, reduced inflammation, and healing of mucosal and perianal disease. Five studies standardized doses by weight: 260 mg (<55 kg), 390 mg (55-85 kg), and 520 mg (>85 kg), with maintenance doses typically 90 mg every 8-12 weeks. Some patients received concomitant therapies (e.g., methotrexate, corticosteroids, and 5-aminosalicylic acid). While adverse effects were reported, including worsening psoriasis, cutaneous and neurological reactions, infections, elevated transaminases, and lymphopenia. Severe adverse events were rare, though anaphylaxis and one death from acute diarrhea were reported. Overall, ustekinumab shows promising clinical and laboratory outcomes in pediatric IBD. However, long-term studies are essential to solidify evidence regarding remission rates and adverse effects.