A novel cell-based therapy for patients with aplastic anemia

作者:Chen, Jiayu; Liu, Weiwei; Yu, Wei; Chen, Lingzhen; Wu, Jinming; Zhan, Yu; Wu, Lupo; Yang, Demao*
来源:Cytotherapy, 2010, 12(5): 678-683.
DOI:10.3109/14653241003695000

摘要

Background aims. Aplastic anemia (AA) is a rare but potentially life-threatening disease. There is a need for the development of new, more effective and less toxic therapies for treating AA. The safety and efficacy of an immune cell-based therapy for AA was examined. Methods. Thirty-one patients with idiopathic AA received intravenous infusions of ex vivo-activated autologous and allogeneic immune cells at least once a week. Response to therapy was assessed by symptoms, transfusion dependency, blood counts, bone marrow biopsy and survival. Results. Of the 31 patients, 25 (81%) had either complete (11, 35%) or partial (14, 45%) responses, while six (19%) showed no response to the therapy. The overall survival rates at 3 years were 90%. Conclusions. The therapy described appears to be safe and effective. The data from this pilot study suggest that a larger, controlled study is warranted.