title: Efficacy and safety of human umbilical cord derived mesenchymal stem cell therapy in children with severe aplastic anemia following allogeneic hematopoietic stem cell transplantation: a retrospective case series of 37 patients.
abstract: The treatment of pediatric severe aplastic anemia (SAA) with allogeneic hematopoietic stem cell transplantation (allo-HSCT), presents major challenges including the risks of graft failure, septic complications, and graft-versus-host disease (GVHD). Additive infusions of human umbilical cord derived mesenchymal stem cell (hUC-MSC) may be administered to improve patient survival. We retrospectively examined 37 pediatric patients with SAA who received allo-HSCT and subsequent infusions of hUC-MSC suspension at a dose of 1.0 × 10(6 )/kg. The times and doses of hUC-MSC infusions were increased in patients with severe GVHD. All patients received hUC-MSC infusions. The median time to post-transplantation neutrophil count of greater than 0.5 × 10(9 )/L was 14 days (range, 11-20 days) and time to post-transplantation platelet count of greater than 20 × 10(9 )/L was 19 days (14-29 days). The overall frequency of acute GVHD (aGVHD) was 45.9% (17/37). These aGVHD episodes occurred at a median time of post-transplantation 47 days (15-83 days). The frequency of chronic GVHD (cGVHD) was 18.9% (7/37); cGVHD developed from aGVHD in 10.8% (4/37) of patients. The GVHD-associated mortality rate was 18.9% (7/37) and aGVHD-specific mortality rate was 8.1% (3/37). The median overall survival time was 35 months (9-67 months) and the three-year overall survival rate was 74.2% (28/37). Seven patients died of GVHD, one patient died of a severe invasive fungal infection, and one patient died of renal failure. In conclusion, post-transplantation hUC-MSC infusions seemed to be safely infused in children with SAA who have previously received allo-HSCT.
authors.affiliation: 1Department of Children's Hematology and Oncology, Bayi Children's Hospital Affiliated to General Hospital of Beijing Military Command , Beijing , China.
authors.forename: Yingjian
authors.initials: Y
authors.lastname: Si
authors.forename: Kai
authors.initials: K
authors.lastname: Yang
authors.forename: Maoquan
authors.initials: M
authors.lastname: Qin
authors.forename: Chuancang
authors.initials: C
authors.lastname: Zhang
authors.forename: Zhenlan
authors.initials: Z
authors.lastname: Du
authors.forename: Xiaomei
authors.initials: X
authors.lastname: Zhang
authors.forename: Yuhuan
authors.initials: Y
authors.lastname: Liu
authors.forename: Yan
authors.initials: Y
authors.lastname: Yue
authors.forename: Zhichun
authors.initials: Z
authors.lastname: Feng
mesh_terms: D064591:Allografts; D000741:Anemia, Aplastic / Q000601:surgery*; D001772:Blood Cell Count; D016026:Bone Marrow Transplantation / Q000706:statistics & numerical data; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000706:statistics & numerical data; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000175:diagnosis / Q000453:epidemiology; D019650:Hematopoietic Stem Cell Mobilization; D018380:Hematopoietic Stem Cell Transplantation / Q000706:statistics & numerical data; D006801:Humans; D007223:Infant; D008297:Male; D045164:Mesenchymal Stem Cell Transplantation / Q000706:statistics & numerical data; D009181:Mycoses / Q000209:etiology / Q000401:mortality; D036102:Peripheral Blood Stem Cell Transplantation / Q000706:statistics & numerical data; D011183:Postoperative Complications / Q000453:epidemiology; D051437:Renal Insufficiency / Q000209:etiology / Q000401:mortality; D012189:Retrospective Studies; D018805:Sepsis / Q000209:etiology / Q000401:mortality; D015996:Survival Rate; D019172:Transplantation Conditioning; D016896:Treatment Outcome