Eltromin (Eltrombopag) – Mechanism of Action and Therapeutic Indications

Eltromin (Eltrombopag) – Mechanism of Action and Therapeutic Indications

Eltromin, whose generic name is eltrombopag, is an oral thrombopoietin‑receptor agonist (TPO‑RA) widely used in hematology. Below is a detailed overview of how it works and the conditions for which it is approved.

1、Mechanism of Action

• In normal physiology, thrombopoietin (TPO) binds to the TPO receptor (also known as MPL) on the surface of megakaryocyte progenitor cells, activating the JAK‑STAT pathway and promoting megakaryocyte proliferation and differentiation, ultimately generating platelets. 国家生物技术信息中心+2go.drugbank.com+2

• Eltrombopag binds uniquely to the trans‑membrane domain of the TPO receptor (rather than the extracellular domain where TPO binds) and thus stimulates the same downstream signaling pathways. go.drugbank.com+1

• Through activation of JAK2/STAT pathways and megakaryocyte maturation, eltrombopag enhances platelet production. Some studies suggest it may also improve hematopoiesis of multiple lineages in conditions like aplastic anemia. MDPI

• Regarding pharmacokinetics: Oral bioavailability is over 50 %; peak plasma concentrations occur about 2–6 hours after dosing; half‑life is approximately 21–35 hours. go.drugbank.com+1

2、Therapeutic Indications

Based on current regulatory status and clinical literature, the key indications for eltrombopag include:

• Chronic immune thrombocytopenia (ITP): Adults and some pediatric patients with persistent or chronic ITP who did not adequately respond to corticosteroids, immune globulin therapy or splenectomy. FDA Access Data+1

• Thrombocytopenia associated with chronic hepatitis C virus (HCV) infection: Used in patients whose low platelet count prevents initiation or maintenance of interferon‑based antiviral therapy. 国家生物技术信息中心+1

• Severe aplastic anemia (SAA): In combination with standard immunosuppressive therapy in patients aged 2 years or older, including those who have insufficient response to first‑line therapy. haematologica.org+1

3、Summary

In summary, Eltromin/eltrombopag is a potent oral TPO‑receptor agonist that enhances platelet production via direct receptor stimulation and downstream signaling. It fills a therapeutic gap in disorders characterized by insufficient platelet generation, such as ITP, HCV‑related thrombocytopenia and SAA. Because of its mechanism and indications, clinicians must monitor platelet counts, liver function, and thrombotic risks when using it.

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