Off-label use of thalidomide in children with Crohn's disease and inflammatory bowel diseases

Thalidomide exerts immunomodulatory properties that may be used to treat various conditions. Although its effectiveness in treating Crohn's disease in pediatric patients has been reported, its safety for off-label use is not well established. This study aimed to analyze the efficacy and safety of thalidomide in children and adolescents with inflammatory bowel disease, specifically Crohn's and ulcerative colitis. A narrative review of clinical trials and observational studies published between January 2001 and May 2023 that investigated thalidomide use in pediatric patients with Crohn's or ulcerative colitis was conducted. The results of clinical trials showed statistically significant remission rates and clinical improvement in patients. The most reported adverse event was peripheral neuropathy, resulting in discontinuation of medication in some cases. Other adverse effects, such as sedation, constipation, dizziness, and allergic reactions, were observed but were not serious. Thalidomide can be considered an alternative treatment option for pediatric patients with refractory inflammatory bowel disease. However, monitoring for peripheral neuropathy and potential dose adjustments are necessary. No new or unknown adverse reactions related to the medication were identified in this review.