Abstract

Objective To explore the efficacy and safety of umbilical cord mesenchymal stem cells (UC-MSC) in the treatment of aplastic anemia (AA).Methods Eighteen patients with AA whose median age was 28 (7~49) were enrolled,including 10 severe AA; 7 of whom received immunosuppression treatment,but didn' t got good effect.UC-MSC were isolated from the umbilical cords of healthy fetuses and then cultured.The third to fifth generation cells were intravenously administered to the patients 1 × 106/kg once or twice a week.Before and after the infusion,complete blood cell counting,bone marrow aspiration,bone marrow biopsy,flow cytometry analysis of lymphocyte subsets,and clinical symptoms were observed.And treatmentrelated adverse reactions were observed.Results After a median of 38-time (12~96 times) infusions of UC-MSC 8-month(3-16 months) treatment,and 31-month (6~36 months) follow-up,3 cases were almost completely cured,7 cases relieved,1 case improved,2 cases got better in anemia and bleeding and longer transfusion interval,5 didn' t responded to the treatment,with a total response rate of 72.2%.4 of 7 frontline immunosuppressive-resistant patients responded to the treatment.7 of 12 patients with inverted CD4+/CD8+ recovered back to normal.No patient occurred adverse reaction.Conslusions UC-MSC in the treatment of AA is effective and safe and has no short term adverse reactions.It is a good therapy for the patients not responding to immunosuppression treatment,not suitable for transplant,or not able to tolerate the adverse reactions. Key words: Umbilical cord; Mesenchymal stem cells (MSC); Aplastic anemia (AA)

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