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avatrombopag + all-trans retinoic acid
- Development stage
- Unknown
- Lead developer
- Institute of Hematology and Blood Diseases Hospital
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
This combination therapy consists of avatrombopag, a small molecule thrombopoietin receptor agonist (TPO-RA), and all-trans retinoic acid (ATRA), a vitamin A derivative. Avatrombopag stimulates the proliferation and differentiation of megakaryocytes from hematopoietic stem cells by binding to the thrombopoietin receptor (MPL), leading to increased platelet production. ATRA is included for its immunomodulatory effects; in the context of immune thrombocytopenia (ITP), it is thought to restore immune tolerance by increasing the proportion of regulatory T cells (Tregs) and modulating the Th17/Treg balance. The combination is being investigated by the Institute of Hematology & Blood Diseases Hospital in China as a treatment for patients with first-line ineffective or recurrent primary ITP to improve response rates and durability compared to TPO-RA monotherapy.
- Other names
- avatrombopag + ATRAavatrombopag + tretinoin
02
Targets
TPOR (Thrombopoietin receptor)RARA (Retinoic acid receptor alpha)RARB (Retinoic acid receptor beta)RARG (Retinoic acid receptor gamma)
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