title: 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.
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.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: Z
authors.initials: Z
authors.lastname: Liu
authors.affiliation: Department of Hematology, Guangzhou First People's Hospital, Guangdong Province, PR China.
authors.forename: Y
authors.initials: Y
authors.lastname: Zhang
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: H
authors.initials: H
authors.lastname: Xiao
authors.affiliation: 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.
authors.forename: Z
authors.initials: Z
authors.lastname: Yao
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: H
authors.initials: H
authors.lastname: Zhang
authors.affiliation: Department of Hematology, Nanfang Hospital of Southern Medical University, Guangdong Province, PR China.
authors.forename: Q
authors.initials: Q
authors.lastname: Liu
authors.affiliation: Department of Hematology, Zhujiang Hospital of Southern Medical University, Guangdong Province, PR China.
authors.forename: B
authors.initials: B
authors.lastname: Wu
authors.affiliation: Department of Hematology, Second Affiliated Hospital, Sun Yat-Sen University, Guangdong Province, PR China.
authors.forename: D
authors.initials: D
authors.lastname: Nie
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: Y
authors.initials: Y
authors.lastname: Li
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: Y
authors.initials: Y
authors.lastname: Pang
authors.affiliation: Department of Hematology, Nanfang Hospital of Southern Medical University, Guangdong Province, PR China.
authors.forename: Z
authors.initials: Z
authors.lastname: Fan
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: L
authors.initials: L
authors.lastname: Li
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: Z
authors.initials: Z
authors.lastname: Jiang
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: F
authors.initials: F
authors.lastname: Duan
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: H
authors.initials: H
authors.lastname: Li
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: P
authors.initials: P
authors.lastname: Zhang
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: Y
authors.initials: Y
authors.lastname: Gao
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: L
authors.initials: L
authors.lastname: Ouyang
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: C
authors.initials: C
authors.lastname: Yue
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: M
authors.initials: M
authors.lastname: Xie
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.
authors.forename: C
authors.initials: C
authors.lastname: Shi
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangdong Province, PR China.|Guangdong Saliai Stem Cell Research Institute, Guangdong Province, PR China.
authors.forename: Y
authors.initials: Y
authors.lastname: Xiao
authors.affiliation: Department of Hematology, Guangzhou First People's Hospital, Guangdong Province, PR China.
authors.forename: S
authors.initials: S
authors.lastname: Wang
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000401:mortality / Q000628:therapy*; D002648:Child; D046528:Chimerism; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000188:drug therapy / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D006801:Humans; D008297:Male; D045164:Mesenchymal Stem Cell Transplantation / Q000379:methods*; D008875:Middle Aged; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods; D000075442:Transplantation, Haploidentical / Q000379:methods*; D055815:Young Adult