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Aplastic Anemia

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

A Corrigendum to this article was published on 05 July 2017

Abstract

Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for severe aplastic anemia (SAA) is mainly limited by the high incidence 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 a multicenter phase II trial, we developed an approach with co-transplantation of MSCs in patients undergoing haplo-HSCT. Forty-four patients with SAA were included. The conditioning regimen included busulfan, cyclophosphamide and thymoglobulin (ATG). The recipients received cyclosporin A (CsA), mycophenolate mofetil and short-term methotrexate for GvHD prophylaxis. Three out of 44 patients, who died early before hematopoietic engraftment, were not assessed. Evaluable patients (97.6%; 40/41) achieved hematopoietic reconstitution and sustained full donor chimerism. The median time for myeloid engraftment was 12 days (range 8–21 days) and for platelet engraftment was 19 days (range 8–154 days). The incidence was 29.3% for grade II–IV acute GvHD and 14.6% for chronic GvHD. The overall survival was 77.3% with a median 12-month (range 0.9–30.8) follow-up for surviving patients. These data suggest that co-transplantation of MSCs could reduce the risk of graft failure and severe GvHD in haplo-HSCT for SAA.

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Acknowledgements

This work was supported by funding from the Guangzhou Health and Medical Collaborative Innovation Major Program, Project number 201400000003-1 and 201400000003-4; the Army Medical, Science and Technology Research Program (12.5 Program), Project number BWS11J071; the State Natural Sciences Fund, Project number 81570107; and Natural Science Foundation of Guangdong Province, Project number 2014A030311006.

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Correspondence to Y Xiao or S Wang.

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Liu, Z., Zhang, Y., Xiao, H. et al. 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 52, 704–710 (2017). https://doi.org/10.1038/bmt.2016.347

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