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a novel cell-based therapy for patients with aplastic anemia：再生障碍性贫血患者的一种新的基于细胞的疗法
Cytotherapy, 2010; 12: 678–683
A novel cell-based therapy for patients with aplastic anemia
1 1 1 1 1 1
JIAYU CHEN , WEIWEI LIU , WEI YU , LINGZHEN CHEN , JINMING WU ,YU ZHAN ,
LUPO WU2 DEMAO YANG2
1Department of Hematology, The 12th Municipal Hospital of Guangzhou, Guangzhou, China, and 2Zhongxing Yangfan
Biotech Co. Ltd, Shenzhen, China
Abstract
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 effi cacy 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 sug-
gest that a larger, controlled study is warranted.
Key Words: aplastic anemia, cell therapy, hematopoiesis
Introduction
AA is more common in Asia than Europe and
Acquired aplastic anemia (AA) in humans is a rare North America (10,11). Studies of immunosuppres-
but potentially life-threatening disease character- sive therapy and the large registry of BM transplanta-
ized by pancytopenia of the peripheral blood and tion data have revealed several differences in clin
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