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This cytotoxic combination therapy is a multi-drug immunosuppressive regimen used as a standard-of-care or control arm in clinical trials for Behcet's disease, particularly for resistant ocular manifestations such as retinal vasculitis and posterior uveitis. The regimen consists of three primary components: pulse cyclophosphamide (an alkylating agent), azathioprine (a purine synthesis inhibitor), and prednisolone (a corticosteroid). Cyclophosphamide works by cross-linking DNA to inhibit cell division, azathioprine acts as a prodrug that interferes with purine metabolism to suppress T and B cell proliferation, and prednisolone provides broad anti-inflammatory effects by activating glucocorticoid receptors. In a Phase 2 pilot study conducted by the Tehran University of Medical Sciences (NCT00700297), this combination was used as the comparator arm against rituximab in patients with ocular lesions resistant to conventional therapy.
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