Teduglutide 5mg powder and solvent for solution for injection vials

BACKGROUND Pediatric short bowel syndrome (SBS) poses management challenges, and teduglutide is a potential therapy. However, comprehensive data on its pediatric safety are lacking. AIM To evaluate the impact of teduglutide on infection and gastrointestinal adverse events in pediatric SBS patients via systematic review and meta-analysis. METHODS Following PRISMA 2009 guidelines and PROSPERO registration, we searched PubMed, Web of Science, and EMBASE for randomized controlled trials (RCTs) (pediatric SBS patients ≤ 18 years; teduglutide vs placebo/standard care). Two reviewers screened studies, extracted data, and assessed bias (ROB2). Meta-analyses used RevMan 5.4 (Mantel-Haenszel method, random-effects if I2 ≠ 0). Trial sequential analysis and GRADE were applied. RESULTS Three RCTs involving 115 pediatric patients were included. Pooled analysis revealed no statistically significant differences between the teduglutide and control groups for the primary outcome of infection events [RR = 0.83; (95%CI: 0.44-1.56); P = 0.57; I2 = 0%; 2 studies, n = 55]. Similarly, no significant differences were found for secondary outcomes: Upper respiratory tract infection [RR = 0.68; (95%CI: 0.32-1.47); P = 0.33; I2 = 0%], catheter site infection [RR = 1.86; (95%CI: 0.23-14.78); P = 0.56; I2 = 0%], vomiting [RR = 1.35; (95%CI: 0.10-18.23); P = 0.82; I2 = 72%], abdominal pain [RR = 2.47; (95%CI: 0.50-12.16); P = 0.27; I2 = 0%], nausea [RR = 1.31; (95%CI: 0.24-7.22); P = 0.75; I2 = 0%], diarrhea [RR = 1.02; (95%CI: 0.23-4.43); P = 0.98; I2 = 0%], and abdominal distension [RR = 1.49; (95%CI: 0.18-12.35); P = 0.71; I2 = 0%]. The overall certainty of evidence assessed by GRADE was moderate. CONCLUSION Teduglutide does not increase infection or gastrointestinal adverse event risk in pediatric SBS, but small sample sizes limit conclusions. Larger studies are needed.

Abstract licence: CC BY-NC

Abstract Background Although studies have evaluated the safety and efficacy of GLP drugs in adult patients with short bowel syndrome, pediatric applications remain contentious due to developmental divergences in drug response and safety profiles. This meta-analysis aims at systematically evaluating efficacy and safety outcomes of teduglutide in pediatric SBS and providing comprehensive evidence for clinical practitioners and families of affected children. Methods and analysis: Randomized controlled trials (RCTs) and cohort studies comparing teduglutide and placebo or regular treatments in pediatric patients with a confirmed diagnosis of short bowel syndrome will be included. Literature searches will be conducted in PubMed, Web of Science, Embase, and Cochrane Library. Two reviewers independently perform the processes of literature retrieval, screening, data extraction, and assessment of risk of bias. Risk of bias in included studies is evaluated using Revised Cochrane risk-of-bias tool (ROB 2) for RCTs and Risk Of Bias In Non-randomized Studies of Interventions (ROBINS-1) for non-RCTs. Review Manager (RevMan) was used for data pooling. Subgroup analysis, meta-regression, trial sequential analysis (TSA), and sensitivity analysis are conducted. Ethics and dissemination: Ethical approval is not required because this study is a secondary analysis of existing data. We will disseminate the findings through peer- reviewed publications. PROSPERO registration number: CRD420251047221

Abstract licence: CC BY