Co‐transplantation of bone marrow‐derived mesenchymal stem cells with hematopoietic stem cells does not improve transplantation outcome in class III beta‐thalassemia major: A prospective cohort study with long‐term follow‐up

Bone marrow transplantation is the only curative treatment for beta‐thalassemia major. Data on the co‐transplantation of MSCs with HSCs in beta‐thalassemia major patients are scarce. We aimed to investigate the outcomes of thalassemia major patients who underwent bone marrow‐derived MSC co‐transplantation with HSCs compared with those who only received HSCs. This prospective randomized study included patients with class III thalassemia major undergoing HSCT divided randomly into two groups: Thirty‐three patients underwent co‐transplantation of bone marrow‐derived MSCs with HSCs, and 26 patients only received HSCs. Five‐year OS, TFS, TRM, graft rejection rate, and GVHD were estimated. The 5‐year OS was 66.54% (95% CI, 47.8% to 79.9%) in patients who underwent co‐transplantation of MSCs with HSCs vs 76.92% (95% CI, 55.7% to 88.9%) in patients who only received HSCs (P = .54). No significant difference was observed in the 5‐year TFS between the two groups (59.1% vs 69.2%; P = .49). The 5‐year cumulative incidence of TRM was not statistically significant among patients who underwent co‐transplantation of MSCs with HSCs (27.27%) vs those who only received HSCs (19.23%; P = .61). There was no statistically significant difference in graft rejection, acute GvHD, and chronic GvHD between the two groups. Based on our findings, the co‐transplantation of MSCs and HSCs to class III thalassemia major patients does not alter their transplantation outcomes including OS, TFS, rejection rate, transplant‐related mortality, and GvHD.