title: Efficacy and safety of mesenchymal stromal cell treatment from related donors for patients with refractory aplastic anemia.
abstract: OBJECTIVE
  This study evaluated the feasibility, safety and immunological effects of the intravenous administration of mesenchymal stromal cells (MSCs) from a related donor in patients with refractory aplastic anemia (AA).
  
  
  METHODS
  A mean of 6 × 10(5)/kg (range, 5.0-7.1 × 10(5)) MSCs were injected intravenously to 18 patients, including 14 patients with nonsevere AA and four patients with severe AA who were refractory to prior immunosuppressive treatment. The outcomes of patients treated with MSCs were evaluated and compared with a historic control cohort, including 18 patients with refractory AA.
  
  
  RESULTS
  Two patients had injection-related adverse events, including transient fever and headache. No major adverse events were reported during the follow-up period. An immunological analysis revealed an increased proportion of CD4(+)CD25(+) FOXP3(+)regulatory T cells in peripheral mononuclear cells. Following up for 1 year, six of 18 patients (33.3%) achieved a complete response or a partial response to MSC treatment. In six patients, two achieved a complete response including a recovery of three hematopoietic cell lines after MSCs therapy at days 88 and 92, two patients achieved only a red cell recovery with hemoglobin levels >100 g/L at days 30 and 48 and two patients had only a platelet recovery with a platelet count of >60 × 10(9)/L at days 54 and 81. In the control cohort, only one patient (5.56%) achieved a partial response during the follow-up period.
  
  
  CONCLUSIONS
  The data from the present study suggest that treatment with MSCs from a related donor may be a promising therapeutic strategy for patients with refractory AA. The trial has been registered at ClinicalTrials.gov: identifier NCT01305694.
authors.affiliation: Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangzhou, Guangdong, PR China. jdxiao111@163.com
authors.forename: Yang
authors.initials: Y
authors.lastname: Xiao
authors.forename: Zu-Jun
authors.initials: ZJ
authors.lastname: Jiang
authors.forename: Yan
authors.initials: Y
authors.lastname: Pang
authors.forename: Li
authors.initials: L
authors.lastname: Li
authors.forename: Yang
authors.initials: Y
authors.lastname: Gao
authors.forename: Hao-Wen
authors.initials: HW
authors.lastname: Xiao
authors.forename: Yong-Hua
authors.initials: YH
authors.lastname: Li
authors.forename: Hang
authors.initials: H
authors.lastname: Zhang
authors.forename: Qifa
authors.initials: Q
authors.lastname: Liu
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000473:pathology / Q000628:therapy*; D000753:Anemia, Refractory / Q000473:pathology / Q000628:therapy*; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage; D007275:Injections, Intravenous; D008297:Male; D045164:Mesenchymal Stem Cell Transplantation / Q000009:adverse effects*; D008875:Middle Aged; D050378:T-Lymphocytes, Regulatory; D014019:Tissue Donors
chemical_list: D007166:Immunosuppressive Agents