Hematopoietic stem cell transplantation with umbilical cord multipotent stromal cell infusion for the treatment of aplastic anemia—a single-center experience

• Published in: Cytotherapy (Oxford, England) England), 2013-09, Vol.15 (9), p.1118-1125
• Main Authors: Wang, Hengxiang, Wang, Zhidong, Zheng, Xiaoli, Ding, Li, Zhu, Ling, Yan, Hongmin, Guo, Zikuan
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Advanced Basic Science; Anemia, Aplastic - immunology; Anemia, Aplastic - therapy; aplastic anemia; Bone Marrow - immunology; Bone Marrow - physiology; Child; Child, Preschool; cord multipotent stromal cells; Female; Graft vs Host Disease - immunology; Granulocyte Colony-Stimulating Factor - immunology; Granulocyte Colony-Stimulating Factor - physiology; hematopoietic stem cell transplantation; Hematopoietic Stem Cell Transplantation - adverse effects; Hematopoietic Stem Cell Transplantation - methods; Hematopoietic Stem Cell Transplantation - mortality; Hematopoietic Stem Cells - immunology; Hematopoietic Stem Cells - physiology; HLA Antigens - immunology; Humans; Infusions, Intraventricular; Male; Middle Aged; Multipotent Stem Cells - immunology; Multipotent Stem Cells - physiology; Other; Stromal Cells - immunology; Stromal Cells - physiology; Tissue Donors; Transplantation Conditioning - methods; Umbilical Cord - immunology; Umbilical Cord - physiology; umbilical graft-versus-host disease; Young Adult
• ISSN: 1465-3249; 1477-2566
• DOI: 10.1016/j.jcyt.2013.04.007

Abstract Background aims The purpose of this study was to observe the outcome of co-transfusion of umbilical cord multipotent stromal cells (UC-MSC) and allogeneic hematopoietic stem cells in the treatment of heavily-transfused patients with severe aplastic anemia. Methods Of the 22 patients, eight cases received haploidentical hematopoietic stem cells from granulocyte colony-stimulating factor–primed bone marrow and peripheral blood grafts; the other patients received granulocyte colony-stimulating factor–mobilized peripheral blood grafts from human leukocyte antigen–matched related (six cases) and unrelated donors (eight cases). MSCs were intravenously infused at a mean dose of 1.2 × 106 / kg (range, 0.27–2.5 × 106 /kg). Fludarabine-based conditioning was conducted, and graft-versus-host disease prophylaxis containing cyclosporine A, methotrexate and mycophenolate mofetil with or without addition of anti-CD25 monoclonal antibody was performed. Hematopoietic engraftment, the occurrence of graft-versus-host disease (GVHD) and infections and overall survival were documented. Results All patients had rapid engraftment; mean time for neutrophil and platelet recovery was 13.95 d and 20.27 d, respectively. No acute toxicity associated with UC-MSC transfusion was observed. Acute GVHD developed in seven cases (grade I–II), and none had development of chronic GVHD. Cytomegalovirus reactivation was observed in 11 cases. One patient died of pulmonary complication 6 months after transplantation. Twenty-one patients are currently alive, at a median follow-up of 15 months; they are transfusion-independent and reached full donor chimerism at the time of reporting. Conclusions UC-MSC infusion might be an alternative option to promote hematopoietic engraftment and reduce the occurrence of GHVD in hematopoietic stem cell transplantation in the treatment of heavily transfused patients with severe aplastic anemia.