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ist + hetrombopag
- Development stage
- Phase 4
- Lead developer
- Hengrui Therapeutics
- Modality
- Small Molecules, Recombinant Proteins and Enzymes
- Administration
- Oral, Intravenous
01
Overview
**ist + hetrombopag** refers to the combination of **immunosuppressive therapy (IST)**, typically consisting of antithymocyte globulin (ATG) and cyclosporine, with **hetrombopag**, an oral, small molecule, synthetic, nonpeptide thrombopoietin receptor agonist (TPO-RA). This combination is primarily used as a first-line regimen or for refractory cases in patients with **severe aplastic anemia (SAA)** who are ineligible for hematopoietic stem cell transplantation (HSCT). Hetrombopag binds to the transmembrane domain of the thrombopoietin (TPO) receptor (encoded by the MPL gene), stimulating platelet production and hematopoiesis. Clinical studies indicate that adding hetrombopag to IST increases hematologic response rates, shortens time to response, and improves transfusion independence compared with IST alone, without significant new safety concerns. The combination is also under investigation for other thrombocytopenic disorders[1][3][6].
- Other names
- ist + hetrombopag
02
Targets
TPOR (Thrombopoietin receptor)CN (Calcineurin)
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