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all-trans retinoic acid + eltrombopag
- Development stage
- Unknown
- Lead developer
- Peking University
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
- Administration
- Oral
01
Overview
This is a combination therapy of all-trans retinoic acid (ATRA, also known as tretinoin) and eltrombopag. ATRA is a retinoid that modulates gene expression by binding to nuclear retinoic acid receptors (RARs), promoting cell differentiation and immune modulation. Eltrombopag is a small molecule thrombopoietin receptor agonist that stimulates the production of platelets by activating the thrombopoietin receptor (MPL). This combination has been investigated in clinical trials for the treatment of steroid-resistant or relapsed immune thrombocytopenia (ITP), an autoimmune disorder characterized by low platelet counts due to increased destruction and decreased production. The rationale for combining these agents is based on evidence that ATRA can improve response rates and quality of life in ITP patients, while eltrombopag directly increases platelet production[1][2][3]. Recent studies have used biomarker-guided approaches (e.g., MSC-C5b-9 status) to stratify patients who may benefit most from this combination[2][3].
- Other names
- ATRA + eltrombopagtretinoin + eltrombopag
02
Targets
RARA (Retinoic acid receptor alpha)TPOR (Thrombopoietin receptor)
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