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Cyclophosphamide is a small molecule alkylating agent of the nitrogen mustard class, used as both an antineoplastic (chemotherapy) and immunosuppressive drug. It is indicated for the treatment of various cancers, including Hodgkin's lymphoma, non-Hodgkin's lymphoma, multiple myeloma, leukemia (acute and chronic forms), mycosis fungoides, neuroblastoma, retinoblastoma, ovarian cancer, and breast cancer. Additionally, it is used to treat nephrotic syndrome in certain children and has applications in severe autoimmune diseases such as vasculitis and systemic lupus erythematosus. Cyclophosphamide is a prodrug that requires hepatic activation to form its active metabolite phosphoramide mustard. The main mechanism involves cross-linking DNA at guanine N7 positions—both interstrand and intrastrand—leading to inhibition of DNA replication and transcription which results in cell cycle arrest and apoptosis. Its immunosuppressive effects are due to suppression of rapidly dividing immune cells. Cyclophosphamide also induces beneficial immunomodulatory effects by eliminating regulatory T cells and enhancing adoptive T cell therapies.
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