Drug intelligence / Profile preview

high-dose immunoablative cyclophosphamide

Development stage
Unknown
Lead developer
National Institute of Arthritis and Musculoskeletal and Skin Diseases
Modality
Small Molecules
Administration
Intravenous
01

Overview

High-dose immunoablative cyclophosphamide is an intensive immunosuppressive regimen developed by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) for the treatment of moderate-to-severe systemic lupus erythematosus (SLE). The therapy involves the intravenous administration of cyclophosphamide at a dose of 50 mg/kg/day for four consecutive days (total dose 200 mg/kg) without hematopoietic stem cell rescue. As an alkylating agent, cyclophosphamide cross-links DNA, leading to the depletion of autoreactive B and T lymphocytes. This "immune reset" aims to achieve long-term remission by eliminating the cells responsible for the autoimmune pathology of lupus. This regimen was evaluated in a Phase 3 clinical trial (NCT00005778) comparing its efficacy and safety against the standard monthly pulse cyclophosphamide treatment.

Brand names
CytoxanNeosar
Other names
immunoablative therapyhigh-dose cyclophosphamideHi-Dose Cytoxan
02

Targets

DNA

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