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Eltrombopag is an oral thrombopoietin receptor agonist used to raise the platelet count in chronic immune thrombocytopenia and in severe aplastic anaemia.
- Eltrombopag (Promacta/Revolade) was FDA-approved in 2008 for chronic immune thrombocytopenia and later for severe aplastic anaemia and chronic hepatitis C-associated thrombocytopenia; it is an orally bioavailable non-peptide TPO-receptor agonist that binds the receptor's transmembrane domain rather than the TPO binding site.
- In the phase 2 dose-ranging trial, the platelet-count endpoint of 50,000/mm3 or more at day 43 was reached by 28%, 70% and 81% of patients at 30, 50 and 75 mg/day respectively versus 11% on placebo [1].
- In the RAISE phase 3 trial, only 18% of eltrombopag patients needed rescue treatment versus 40% on placebo (p=0.001), and serious bleeding occurred in under 1% versus 7% [2].
- In the randomized RACE trial, adding eltrombopag to standard immunosuppression in treatment-naive severe aplastic anaemia improved the rate, speed and depth of haematologic response, with median time to first response of 3.0 months versus 8.8 months, without additional toxicity [3].
- In the RAISE trial, 7% of eltrombopag patients had mild ALT rises and 4% had bilirubin increases, with three thromboembolic events versus none on placebo [2].
Mechanism
It binds the transmembrane domain of the thrombopoietin receptor at a site distinct from where thrombopoietin itself binds and triggers the same JAK and STAT signalling that drives megakaryocyte proliferation and platelet production. Because it chelates polyvalent cations it has to be separated from dairy, antacids and iron by several hours or absorption collapses.
receptor fingerprint
Thrombopoietin receptor (MPL / TPO-R), transmembrane domainSmall-molecule non-peptide agonist that binds the transmembrane domain at a site distinct from where endogenous TPO binds the extracellular domain, triggering JAK2/STAT5 signalling and megakaryopoiesis
Intracellular labile iron pool (eltrombopag is a potent iron chelator)Chelates intracellular iron, which is proposed to contribute to its trilineage haematopoietic effect in aplastic anaemia and to its effect on leukaemic blasts
Haematopoietic stem cell compartment (CD34+ progenitors)Expands early progenitors and increases marrow cellularity, not just megakaryocyte lineage
JAK2 / STAT5 signalling axis (downstream of MPL)Activated by receptor dimerisation, driving megakaryocyte proliferation and differentiation
Safetyrisks and cautions, not medical advice
a platelet raising agent titrated against serial platelet counts, with hepatotoxicity and thrombosis if the count overshoots; it cannot be dosed safely without repeated blood tests
Subjective profileweighing the evidence above
Refusing the sourcing question here is not a comment on the drug, which is a good one; it is that the amount is meaningless without the blood test. Serial platelet counts are what it gets titrated against, overshooting the count causes clots, and the drug can injure the liver on its own, so a supply without a haematologist and a regular blood draw is two hazards and no target. Immune thrombocytopenia also has to be the real diagnosis before any of this applies, and that is a marrow and antibody question rather than a low number on one result.
Resources
No suppliers are provided for compounds like this. This entry is here for reference.
Research
- 2007first citedEltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura
- 2016meta-analysisTolerability and Efficacy of Eltrombopag in Chronic Immune Thrombocytopenia: Meta-Analysis of R…
- 2022most recentEltrombopag Added to Immunosuppression in Severe Aplastic Anemia
- 1.Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura
- 2.Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomised, phase 3 study
- 3.Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia
- 4.Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia
- 5.Eltrombopag before procedures in patients with cirrhosis and thrombocytopenia
- 6.Tolerability and Efficacy of Eltrombopag in Chronic Immune Thrombocytopenia: Meta-Analysis of Randomized Controlled Trials
6 listed here; entry last updated August 2026
Reviews
My notesprivate to this device
Notes and cautions
- Promacta carries a boxed warning for risk of hepatic decompensation in patients with chronic hepatitis C when used with interferon and ribavirin, and for hepatotoxicity generally; liver enzymes and bilirubin must be measured before starting and monitored during treatment.
- Thromboembolic events occur even at normal platelet counts, and the drug was associated with portal vein thrombosis in the cirrhosis pre-procedure trial [5], which is why that indication was not pursued.
- In myelodysplastic syndrome there is a theoretical and partially observed risk of accelerating progression to AML.
- Eltrombopag chelates polyvalent cations, so it must be separated by at least two hours from antacids, dairy and mineral supplements or absorption collapses; East Asian patients require a reduced starting dose.