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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 May; 52(5):704-710.BM

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.

Authors+Show Affiliations

Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Guangzhou First People's Hospital, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China. Department of Hematology, Dongguan Municipal People's Hospital, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Nanfang Hospital of Southern Medical University, Guangdong Province, PR China.Department of Hematology, Zhujiang Hospital of Southern Medical University, Guangdong Province, PR China.Department of Hematology, Second Affiliated Hospital, Sun Yat-Sen University, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Nanfang Hospital of Southern Medical University, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China. Guangdong Saliai Stem Cell Research Institute, Guangdong Province, PR China.Department of Hematology, Guangzhou First People's Hospital, Guangdong Province, PR China.

Pub Type(s)

Clinical Trial, Phase II
Journal Article
Multicenter Study

Language

eng

PubMed ID

28067873

Citation

Liu, Z, 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 Transplantation, vol. 52, no. 5, 2017, pp. 704-710.
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. 2017;52(5):704-710.
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., ... Wang, S. (2017). 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 Transplantation, 52(5), 704-710. https://doi.org/10.1038/bmt.2016.347
Liu Z, 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. 2017;52(5):704-710. PubMed PMID: 28067873.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - 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. AU - Liu,Z, AU - Zhang,Y, AU - Xiao,H, AU - Yao,Z, AU - Zhang,H, AU - Liu,Q, AU - Wu,B, AU - Nie,D, AU - Li,Y, AU - Pang,Y, AU - Fan,Z, AU - Li,L, AU - Jiang,Z, AU - Duan,F, AU - Li,H, AU - Zhang,P, AU - Gao,Y, AU - Ouyang,L, AU - Yue,C, AU - Xie,M, AU - Shi,C, AU - Xiao,Y, AU - Wang,S, Y1 - 2017/01/09/ PY - 2016/06/16/received PY - 2016/10/27/revised PY - 2016/10/31/accepted PY - 2017/1/10/pubmed PY - 2018/3/6/medline PY - 2017/1/10/entrez SP - 704 EP - 710 JF - Bone marrow transplantation JO - Bone Marrow Transplant. VL - 52 IS - 5 N2 - 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. SN - 1476-5365 UR - https://www.unboundmedicine.com/medline/citation/28067873/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_ L2 - http://dx.doi.org/10.1038/bmt.2016.347 DB - PRIME DP - Unbound Medicine ER -