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Mesenchymal stromal cells for the prophylaxis of graft-versus-host disease after hematopoietic stem cell transplantation: a meta-analysis of randomized controlled trials

Expert Rev Hematol. 2025 Jul 23. doi: 10.1080/17474086.2025.2535422. Online ahead of print.

ABSTRACT

BACKGROUND: Mesenchymal stromal cells (MSCs) have emergedas a potential alternative therapeutic strategy for the prophylaxisof graft-versus-host disease (GVHD) in patients undergoinghematopoietic stem cell transplantation (HSCT).

RESEARCH DESIGN AND METHODS: This meta-analysis included eight randomizedcontrolled trials (RCTs) involving 570 patients. The primary outcomesassessed were overall survival (OS), the development of acute GVHD(aGVHD), and chronic GVHD (cGVHD). Secondary outcomes includedprimary disease relapse and adverse events. The statistical analysiswas performed using Review Manager (RevMan 5.4) with a random-effectsmodel.

RESULTS: The meta-analysis showed a significantimprovement in overall survival in the MSC group compared to thecontrol group (RR 1.12; 95% CI: 1.02-1.23), with no evidence ofheterogeneity (I² = 0%). MSC prophylaxis was associated with asignificant reduction in the incidence of aGVHD (RR 0.67; 95% CI:0.40-0.83, I² = 33%) and cGVHD (RR 0.65; 95% CI: 0.49-0.87, I² = 0%).However, no significant difference was found between the MSC andcontrol groups regarding primary disease relapse (RR 1.00; 95% CI:0.73-1.38, I² = 0%) or the incidence of infections (RR 0.80; 95% CI:0.57-1.11, I² = 0%). In terms of patients with at least one adverseevent, no statistically significant difference was observed betweenthe two groups (RR 1.10; 95% CI: 0.74-1.63, I² = 34%).

CONCLUSIONS: MSC prophylaxis significantly improves overallsurvival and reduces the incidence of both aGVHD and cGVHD in HSCTpatients, without increasing the risk of relapse, infections, oradverse events, indicating its potential as a safe and effectiveintervention for GVHD management. Further large-scale, multicenterRCTs are needed to validate or refute the current findings.

REGISTRATION: This review has been registered with theInternational Prospective Register of Systematic Reviews (PROSPERO)(CRD42024569358).

PMID:40699555 | DOI:10.1080/17474086.2025.2535422

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