What is known and objective: Thalidomide is used off-label for the treatment of inflammatory bowel disease (IBD) and not as a first-line treatment option. The instructions clearly state that thalidomide is contraindicated in children because its safety and effectiveness in children are unknown. In this article, we review the efficacy and safety of thalidomide as a treatment for IBD in children and adolescents.
Methods: We searched PubMed, Embase, the Cochrane Library, CNKI, WanFang Data, CBM database [from the date of database establishment to June 2019] and clinical trials [systematic review and meta-analysis, randomized controlled trials (RCTs), cohort studies, case-control studies and case series studies] for studies concerning the use of thalidomide as a treatment for IBD in children and adolescents.
Results and discussion: Seven studies (two RCTs and five case series), which included 134 children and adolescents (32 with ulcerative colitis, 102 with Crohn's disease), met the inclusion criteria. The included studies showed that the clinical remission rate of thalidomide was 44%-100% and the steroid tapering rate was 50%-100% in children and adolescents with refractory IBD. Peripheral neuropathy was the most common major adverse reaction, and it appeared to be cumulative dose-dependent.
What is new and conclusion: Thalidomide as a treatment for refractory IBD in children and adolescents can improve clinical remission and achieve longer-term maintenance of remission. Peripheral neuropathy is the main adverse drug reaction, and it can be monitored and prevented. It is necessary to fully communicate with parents and obtain informed consent before using this drug.
Keywords: adolescents; children; inflammatory bowel disease; thalidomide.
© 2020 John Wiley & Sons Ltd.