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Cyclophosphamide is a nitrogen mustard alkylating agent and potent immunosuppressant. In the context of severe aplastic anemia (SAA), it is used to eliminate autoreactive T cells that attack hematopoietic stem cells. This specific regimen, investigated by Dr. Xiaofan Zhu at the Chinese Academy of Medical Sciences, utilizes a moderate dose of cyclophosphamide (30 mg/kg/day for 4 days) in combination with cyclosporine A (a calcineurin inhibitor) and recombinant human granulocyte colony-stimulating factor (rhG-CSF). The study aims to evaluate the efficacy and safety of this moderate-dose approach in children with acquired SAA, potentially offering a less toxic alternative to the traditional high-dose (50 mg/kg/day) cyclophosphamide therapy.
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