eltrombopag
Eltrombopag is a TPO-receptor agonist that interacts with the transmembrane domain of the human TPO-receptor (also known as cMpl) and initiates signaling cascades that induce proliferation and differentiation of megakaryocytes leading to increased platelet production.
Immune Thrombocytopenia (ITP) in adults and children ≥6 years who did not respond to other treatments.
Thrombocytopenia associated with chronic hepatitis C, to enable initiation and continuation of interferon-based therapy.
Severe Aplastic Anemia (SAA) in adults with inadequate response to immunosuppressive therapy.
ITP: 36 mg once daily (adults/pediatric ≥6), reduced to 18 mg for hepatic impairment or East-/Southeast-Asian ancestry.
Hepatitis C: 18 mg once daily.
SAA: 36 mg once daily, reduced to 18 mg if liver-impaired or East-/Southeast-Asian.
Most Common (≥20% in any indication):
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Anemia, fatigue, headache, diarrhea, ALT increase, pyrexia, cough.
ITP-Specific Reactions:
- Adults: Nausea, vomiting, UTI, ALT/AST elevations, rash.
- Pediatrics: Upper respiratory tract infections, nasopharyngitis, diarrhea, ALT/AST elevations.
SAA-Specific:
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Nausea (33%), fatigue (28%), cough (23%), diarrhea (21%).
None
Hepatic risk: Elevated ALT/AST, bilirubin.
Progression of MDS to AML: Increased risk when combined with azacitidine.
Thromboembolic events: Especially portal vein thrombosis.
Cataracts: Observed in trials and animal studies—eye monitoring recommended.