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 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.
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Disclosure of interest: The authors report no potential conflicts of interest.