Hematopoietic stem cell transplantation (HSCT) is a cornerstone in the treatment of hematological disorders. However, its application is frequently complicated by acute and chronic graft-versus-host disease (aGVHD/cGVHD), pathological conditions in which donor-derived immune cells attack host tissues. With suboptimal survival rates and limited therapeutic options, GVHD remains a major clinical challenge. Mesenchymal stem cells (MSCs) have emerged as a promising therapeutic modality due to their immunomodulatory capabilities, yet standardized protocols for their use in preventing or treating GVHD have not been established. We performed a comprehensive literature search of PubMed, Web of Science, EMBASE, and the Cochrane Library up to 10 February 2025 to identify eligible randomized controlled trials (RCTs). Study selection was based on the PICOS framework, and the risk of bias was assessed using appropriate quality appraisal tools. Outcome data were systematically extracted and synthesized via meta-analysis. A total of 15 RCTs were included. The meta-analysis revealed that MSC administration significantly reduced the incidence of aGVHD (OR: 0.47; 95% CI 0.32–0.71; p = 0.00003) and cGVHD (OR: 0.50; 95% CI 0.34–0.74; p = 0.0005) compared with controls. MSC therapy was also associated with improved response rates in steroid-refractory aGVHD (SR-aGVHD) (OR: 1.50; 95% CI 1.04–2.17; p = 0.03). MSCs demonstrate efficacy in preventing both aGVHD and cGVHD following HSCT, particularly in moderate to severe forms. A dose range of 1 × 10⁶ to < 4 × 10⁶ cells/kg was associated with optimal prophylactic outcomes. For SR-aGVHD, MSC infusion resulted in significantly higher remission rates compared to conventional treatments, especially in severe cases.
Wu et al. (Thu,) studied this question.