Drug intelligence / Profile preview

antithymocyte globulin + cyclophosphamide + cyclosporine + cord blood

Development stage
Unknown
Lead developer
960th Hospital
Modality
Cell Therapies, Antibody-Based Therapeutics, Small Molecules
Administration
Intravenous, Oral
01

Overview

This intensive immunosuppressive therapy (IST) regimen was developed by the Jinan Military General Hospital (now known as the 960th Hospital of the PLA) for the treatment of severe and very severe aplastic anemia (SAA/VSAA). The protocol consists of a combination of rabbit antithymocyte globulin (ATG), low-dose cyclophosphamide (administered at 50 mg/kg/day for 2 days), cyclosporine A (CsA), and an infusion of umbilical cord blood. Cyclophosphamide serves as an alkylating agent that cross-links DNA to provide potent immunosuppression, while ATG and CsA further target T-cell mediated destruction of hematopoietic stem cells. The addition of cord blood is intended to provide hematopoietic support and accelerate recovery. A Phase 4 clinical trial (NCT02838992) demonstrated that this combination therapy achieved an 88% effectiveness rate with faster hematopoietic reconstruction and fewer complications compared to standard immunosuppressive therapy alone.

Other names
ATG + Cy + CsA + cord bloodJinan Military General Hospital SAA protocolIntensive immunosuppressive therapy regimen

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