Cotransplantation of bone marrow-derived mesenchymal stem cells in haploidentical hematopoietic stem cell transplantation in patients with severe aplastic anemia: an interim summary for a multicenter phase II trial results

Liu Z, Zhang Y, Xiao H, Yao Z, Zhang H, Liu Q, Wu B, Nie D, Li Y, Pang Y, Fan Z, Li L, Jiang Z, Duan F, Li H, Zhang P, Gao Y, Ouyang L, Yue C, Xie M, Shi C, Xiao Y, Wang S.
Cotransplantation of bone marrow-derived mesenchymal stem cells in haploidentical hematopoietic stem cell transplantation in patients with severe aplastic anemia: an interim summary for a multicenter phase II trial results.
Bone Marrow Transplant. 2017 Jan 9. doi: 10.1038/bmt.2016.347.

Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for severe aplastic anemia (SAA)  has been reported to have high incidences of graft failure and GvHD. Mesenchymal stem cells (MSCs) have been shown to support hematopoiesis in vivo and to display potent immunosuppressive effects to prevent or treat GvHD after HSCT. In this  multicenter phase II trial by Liu et al,  efficacy of co-transplantation of MSCs in patients undergoing haplo-HSCT was tested  in 44 patients with SAA. The conditioning regimen included busulfan, cyclophosphamide and thymoglobulin (ATG).  Three out of 44 patients, who died early before hematopoietic engraftment, were not assessed and of the remaining 41 patients 40 (97.6%) achieved hematopoietic reconstitution and sustained full donor chimerism. The incidence of grade II-IV acute GvHD and chronic GvHD were reported as 29.3% and 14.6%, respectively. The overall survival was 77.3% with a median 12-month (range 0.9-30.8) follow-up for surviving patients. The beneficial effect of co-transplantation of MSCs seems promising in decreasing the risk of graft failure and severe GvHD in haplo-HSCT for SAA. 
This Abstract is prepared by Sule Unal, M.D. for ISH Website.

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