S1471 Hematopoietic Stem Cell Transplantation in Pediatric Inflammatory Bowel Disease: A Systematic Review of Indications, Outcomes, and Safety

Introduction: The efficacy and safety of hematopoietic stem cell transplantation (HSCT) for children and adolescents with refractory inflammatory bowel disease (IBD) remain unclear. We conducted a systematic review and meta-analysis to evaluate clinical outcomes, remission rates, and adverse events associated with HSCT. Methods: Five different databases, including PubMed, Web of Science, SCOPUS, Cochrane, and Embase, were searched up to May 24, 2025. Outcomes of interest included clinical remission, endoscopic remission, steroid-free remission, survival, relapse, acute graft-vs-host disease (GVHD), chronic GVHD, graft failure, mortality, and other adverse events. Pooled proportions and 95% confidence intervals (CIs) were calculated using random effects models in R (version 4.4.3). Results: A total of 6 studies were included with 144 participants. Using random-effects models, (HSCT) resulted in a clinical remission rate of 93% (95% CI: 85% to 97%; I² = 0%, P for heterogeneity = 0.64) and endoscopic remission was achieved in 80% of patients (95% CI: 48% to 95%; I² = 13%, P for heterogeneity = 0.29). Steroid-free remission occurred in 92% (95% CI: 81% to 97%; I² = 0%, P for heterogeneity = 0.85). Overall survival was 80% (95% CI: 68% to 88%; I² = 43%, P for heterogeneity = 0.12). Relapse occurred in 9% of patients (95% CI: 4% to 21%; I² = 39%, P for heterogeneity = 0.14). The rate of acute GVHD was 42% (95% CI: 22% to 65%; I² = 77%, P for heterogeneity < 0.01) and chronic GVHD was 13% (95% CI: 8% to 21%; I² = 0%, P for heterogeneity = 0.63). Graft failure was observed in 14% (95% CI: 7% to 26%; I² = 48%, P for heterogeneity = 0.09). Overall mortality rate of 19%, 95% CI 11%-31%, I2 = 44%, P for heterogeneity = 0.11) and other adverse events had a pooled incidence of 38% (95% CI: 25% to 54%; I2 = 53%, P for heterogeneity = 0.08. Conclusion: Hematopoietic replacement therapy leads to high remission rates and low relapse and complication rates, but mortality remains a concern. Further studies are needed to clarify its overall benefit.