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This investigational combination regimen consists of the alkylating agent cyclophosphamide and the thrombopoietin receptor agonist (TPO-RA) herombopag, administered in conjunction with standard immunosuppressive therapy (IST) comprising anti-lymphocyte globulin (ALG) and cyclosporine. Developed by the Institute of Hematology & Blood Diseases Hospital in China, the regimen is being evaluated as a first-line treatment for patients with severe aplastic anemia (SAA) who are not candidates for hematopoietic stem cell transplantation. Cyclophosphamide acts as a potent immunosuppressant by cross-linking DNA to deplete autoreactive T-cells, while herombopag stimulates the TPO receptor on hematopoietic stem cells and megakaryocytes to promote multilineage hematopoiesis and increase platelet production. The addition of moderate-dose cyclophosphamide to the standard ALG, cyclosporine, and herombopag backbone aims to improve early response rates and overall efficacy in treatment-naive SAA patients.
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