Eltrombopag

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Eltrombopag

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Medically reviewed

Marina Burgos

Last updated on 10/01/2026

This page provides general, reference-level information compiled from official medical sources. It is not a substitute for professional medical advice, diagnosis, or treatment. For decisions about your health, please consult a qualified healthcare professional.

Overview of Eltrombopag

Property Description
Active ingredient Eltrombopag (as the olamine salt)
Form Film-coated tablet, powder for oral suspension
Pharmacological class Thrombopoietin Receptor Agonist (TPO-RA)
General purpose To increase blood platelet counts in low platelet conditions
Origin Synthetic, small-molecule, non-peptide

What Type of Medicine is Eltrombopag?

Eltrombopag is a synthetic and prescription-only medication used in managing blood disorders. It is classified as a Thrombopoietin Receptor Agonist (TPO-RA). As a non-peptide, small-molecule compound, Eltrombopag is an orally active medication. Its primary purpose is to address thrombocytopenia, the deficiency of platelets, for patients experiencing abnormally low blood platelet counts.

Composition, Form, and Delivery

The core medicinal substance is the active ingredient, Eltrombopag, delivered primarily as the salt form, Eltrombopag olamine, which is used to optimize its stability for oral absorption. Eltrombopag is a single active ingredient product that is available for oral administration in two distinct forms: a solid film-coated tablet and a powder for oral suspension. The availability of both forms allows the medicine to be administered across different patient groups, including pediatric patients. This flexibility in delivery supports the therapeutic presence of the drug.

High-Level Action: How Eltrombopag Stimulates Platelets

Eltrombopag works by selectively activating the body’s TPO receptor (c-Mpl), which is the mechanism responsible for initiating platelet generation. This targeted action sends a signal to the bone marrow, the specialized site of blood cell production. This process results in the proliferation and maturation of precursor cells, leading to an increase in the circulating platelet count. This physiological process supports the body’s essential ability to form blood clots, a function for managing conditions where platelet counts are compromised.

Regulatory References

  1. U.S. Food and Drug Administration
  2. [(FDA Drug Label)]

What side effects are possible with Eltrombopag?

Eltrombopag's official safety profile, as documented by regulatory agencies, is structured by frequency and the organ systems affected. Adverse reactions are classified according to the likelihood of their occurrence in clinical studies.


Adverse Reaction Frequencies

Classification Representative Adverse Effects
Very Common (ge 1 in 10) Nausea, diarrhea, upper respiratory tract infection, transaminase elevations (liver enzymes), retinal hemorrhage.
Common (ge 1 in 100 to < 1 in 10) Thromboembolic events, cataract, alopecia, abdominal pain, vomiting, headache, myalgia.

Serious Adverse Reactions and Systemic Safety

The label officially documents a risk of Severe Hepatotoxicity and Thromboembolic Events (blood clots), which are classified as serious adverse reactions. Adverse effects are grouped into System-Organ Classes, including Hepatobiliary disorders, Vascular disorders, Eye disorders, and Gastrointestinal disorders.


Population-Specific and Time-Related Safety

Hepatotoxicity (liver enzyme elevations) is documented to carry the highest risk early in the course of treatment. Furthermore, the risk of thromboembolic events is associated with the achieved increase in platelet counts. Specific safety considerations are noted for patients with severe hepatic impairment (increased drug exposure) and older adults in certain therapeutic contexts, who have shown a higher incidence of thromboembolic events.

Safety limitations also include the documented risk for developing or worsening bone marrow reticulin formation and potential progression to fibrosis, a constraint related to the drug's pharmacological class. The overall regulatory structure emphasizes that the risk profile is dynamic, requiring awareness of time-related and population-specific differences.

Overdose and Emergency Response

Eltrombopag Overdose is characterized by the pharmacological risk of excessive platelet count elevation, resulting in thrombocytosis. The central regulatory concern is the potential for serious or life-threatening thrombotic and thromboembolic events (TEEs), which can involve both arterial and venous systems. Officially documented manifestations of overexposure include rash, slowed heartbeat, and excessive tiredness. Due to differing plasma concentrations, regulatory documents note that patients with hepatic impairment and individuals of East Asian ancestry may possess an increased susceptibility to the effects of overexposure.

When to Seek Immediate Help

Regulatory guidance mandates seeking immediate medical attention if an overdose is suspected. Individuals should contact the Poison Control Helpline immediately for reporting and guidance. Furthermore, emergency services must be called without delay if the person has collapsed, experienced a seizure, has trouble breathing, or is unable to be awakened, as these are defined as critical signs requiring urgent intervention.

Management and Treatment

Management is defined as symptomatic and supportive because there is no specific antidote for Eltrombopag listed in regulatory documents. Procedural management includes frequent monitoring of platelet counts to rapidly detect and manage severe thrombocytosis. The administration of a polyvalent cation-containing product, such as an aluminum or magnesium antacid, may be employed to limit systemic absorption.

Therapeutic Uses of Eltrombopag

What Eltrombopag Treats: Main Uses and Benefits

Eltrombopag is used for conditions characterized by abnormally low platelet counts, where the primary focus is managing bleeding risk. The medication plays a supportive role across several distinct therapeutic contexts.

The medication is used for managing conditions that require symptomatic support, particularly Chronic Immune Thrombocytopenia (ITP) and Severe Aplastic Anemia (SAA), and in specific cases of thrombocytopenia associated with chronic Hepatitis C virus (HCV) infection. In these scenarios, Eltrombopag is applied to help raise platelet counts, which may assist with reducing the risk of spontaneous bleeding. This supportive function supports the management of symptoms during periods of low platelet levels.

The primary goal of use is to provide supportive relief that helps address the overall symptom burden associated with a deficiency of platelets.


Quick Fact: Symptomatic Support for Hemorrhagic Manifestations

Eltrombopag is used for managing symptoms that interfere with daily comfort, such as frequent bruising and petechiae, and assists with maintaining functional stability.

Eligibility and Restrictions for Use

Eligibility for Eltrombopag: Official Regulatory Profile

Eltrombopag is approved for adults and pediatric patients starting at age one year for Chronic Immune Thrombocytopenia (ITP), and at age two years for Severe Aplastic Anemia (SAA). Use is not established in infants under one year of age for ITP or for thrombocytopenia associated with Chronic Hepatitis C in children.

Classification Population Restriction
Contraindicated Patients with known hypersensitivity to Eltrombopag or its components.
Absolute Exclusion Patients with Myelodysplastic Syndromes (MDS), due to risk of malignancy progression.
Prohibited State Women who are breastfeeding (must not be used).

Eligibility requires specific conditions based on individual patient characteristics. Patients with hepatic impairment (liver dysfunction) and individuals of East Asian ancestry must initiate treatment at a reduced starting dose, as clearance of the medicine is affected. Use is not recommended for women who are pregnant, and they are advised to use effective contraception during treatment. Furthermore, use requires caution in patients with known risk factors for thromboembolism or those with impaired renal function, as stated in regulatory documentation.

What should I know about interactions with other medicines?

Interactions with other medicines and products

Eltrombopag’s interaction profile is defined by pharmacokinetic and pharmacodynamic constraints documented in regulatory labeling.

Pharmacokinetic Interactions

Eltrombopag administration requires specific separation from products containing polyvalent cations (such as iron, calcium, aluminum, and zinc). This is because the cations bind to Eltrombopag in the digestive tract, a process known as chelation, which significantly reduces the absorption of the medicine. The official restriction is a mandatory 4-hour time separation between Eltrombopag and antacids, mineral supplements, or calcium-rich foods.

Eltrombopag also acts as an inhibitor of key drug transporters, specifically Organic Anion Transporting Polypeptide 1B1 (OATP1B1) and Breast Cancer Resistance Protein (BCRP). This inhibition can result in excessive exposure and reduced clearance of co-administered medicines that are substrates for these transporters. Regulatory documents cite rosuvastatin as an example of a medication affected by this interaction, which may necessitate a dose review for the co-administered substrate.

Pharmacodynamic and Population Constraints

A specific pharmacodynamic risk is documented concerning the use of Eltrombopag in patients with Chronic Hepatitis C when co-administered with Interferon and Ribavirin. This combination is associated with an increased risk of hepatic decompensation in this patient population. Furthermore, official labeling notes that hepatic impairment itself increases Eltrombopag exposure, which is a population-specific consideration that may alter the overall interaction risk profile.

Mechanism of Action

Eltrombopag is a synthetic small-molecule agent that selectively activates the Thrombopoietin Receptor ( TPO-R), or c-Mpl, which is expressed on megakaryocyte progenitor cells in the bone marrow. It acts as an agonist by binding to a distinct site on the receptor's transmembrane domain, rather than competing with the natural TPO hormone. This binding induces receptor dimerization, initiating the necessary intracellular signaling. The dimerization activates the JAK/ STAT cascade inside the precursor cells. This molecular signal accelerates the proliferation, differentiation, and maturation of megakaryocytes, the platelet-producing cells, through enhanced megakaryopoiesis. The physiological consequence is an increase in the release of new platelets into the peripheral circulation. This mechanism is dependent on the availability of responsive progenitor cells and does not modulate the rate of platelet destruction or clearance in the bloodstream.

Dosage and Administration Information

Administration Guidelines

Eltrombopag is administered once daily as an oral medication, available in film-coated tablets or as a reconstituted powder for oral suspension. The usage of this medication follows specific parameters regarding dosing, timing, and monitoring.

Dosing and Timing Constraints

Constraint Instructional Parameter
Route & Frequency Oral, once daily.
Administration Timing Must be taken on an empty stomach (1 hour before or 2 hours after a meal).
Cation Separation A 4-hour interval is required between the Eltrombopag dose and products containing polyvalent cations (e.g., calcium, iron, or aluminum-containing antacids or supplements).
Missed Dose If a dose is missed, the patient should skip that dose and take the next dose at the regular time; the dose should not be doubled.

Dosing Regimens and Adjustments

The initial daily dose is typically 50 mg for most adult patients with Immune Thrombocytopenia (ITP). For Severe Aplastic Anemia (SAA), the maximum daily dose is 150 mg, while for ITP, the maximum is 75 mg.

Dose titration involves adjustments typically made in 25 mg increments for ITP and in 50 mg increments for SAA, usually no more frequently than once every two weeks. Treatment is adjusted to maintain a platelet count ge 50 imes 10^9/L but is not used to normalize platelet counts.

Specific dose adjustments include a reduced initial dose of 25 mg once daily for patients of East Asian ancestry and for patients with hepatic impairment, regardless of the indication.

Recent Clinical Evidence

Eltrombopag: Recent Clinical Evidence

Eltrombopag is a small molecule, non-peptide thrombopoietin receptor (TPO-R) agonist. It is authorized for use in the treatment of persistent or chronic Immune Thrombocytopenia (ITP) and for Severe Aplasti c Anemia (SAA).


Clinical Efficacy in ITP

Studies in adult patients with ITP who have had an inadequate response to prior treatment have evaluated the use of eltrombopag. Randomized Controlled Trials (RCTs) reported that, compared to placebo, the initiation of eltrombopag was associated with a greater rate of achieving and maintaining platelet counts above 50,000 cells/muL without requiring rescue medication. This effect was maintained during the active treatment phase of the trials.

Role in Severe Aplastic Anemia (SAA)

Research has explored the use of eltrombopag in combination with standard immunosuppressive therapy (IST) for patients with treatment-naïve SAA. Studies reported that the addition of eltrombopag to standard IST regimens was associated with higher rates of overall hematologic response and complete response compared to IST alone. Further investigation continues regarding the long-term impact on overall survival and relapse rates in this patient population.


Safety Profile Highlights

Across clinical trials, the most commonly reported adverse events included nausea, diarrhea, upper respiratory tract infection, and fatigue. Specific attention in research is paid to potential risks of hepatotoxicity (liver enzyme elevations) and thrombotic events (blood clots). Current guidelines recommend monitoring liver function tests prior to and during treatment. Study populations generally excluded patients with existing severe hepatic impairment.

Key Studies & References Eltrombopag for treating chronic immune thrombocytopenia (NICE Technology Appraisal Guidance TA293)

Frequently Asked Questions (FAQ)

Common questions about Eltrombopag (FAQ)


Q: What is the difference between Eltrombopag and other platelet-raising medicines?

Eltrombopag is categorized as a Thrombopoietin Receptor Agonist (TPO-RA). The official information highlights that Eltrombopag is an oral medication, available in tablet or suspension form. This key difference in administration method is noted in the official product information when compared to other TPO-RAs, such as romiplostim.


Q: Does Eltrombopag affect pregnancy or fertility?

Regulatory studies in animals indicate no functional effect on male fertility. No negative effects on female fertility were seen up to the highest dose tested in the definitive study. The official guidance advises patients to use effective contraception during treatment and states that the medication is not recommended for use during pregnancy or while breastfeeding.


Q: What kind of monitoring or blood tests are needed while on Eltrombopag?

Official information emphasizes that regular monitoring is required throughout the course of treatment. Complete Blood Counts (CBCs), including platelet counts, are required to be assessed frequently until a stable count is achieved. Additionally, blood tests for liver chemistries (serum aminotransferase levels and bilirubin) must be measured before and regularly during treatment.


Q: Is there any research on long-term effects of Eltrombopag use?

Official documentation describes a potential risk for the development or progression of reticulin fiber deposition within the bone marrow with long-term use. For this reason, regulatory guidelines recommend monitoring of peripheral blood smears to check for signs of marrow fibrosis (scarring) while on therapy.


Q: Is Eltrombopag used as a first-line treatment for ITP?

According to the official product information, Eltrombopag is indicated for Chronic Immune Thrombocytopenia (ITP) in patients who have already had an insufficient response to prior treatments. These prior treatments typically include corticosteroids, immunoglobulins, or splenectomy.


Q: Are there any specific dietary restrictions when taking Eltrombopag?

Yes, there is a key timing restriction related to polyvalent cations. Official guidelines define that Eltrombopag must be administered at least 4 hours apart from medications, supplements, or foods (like dairy or calcium-fortified juices) that contain polyvalent cations such as iron, calcium, aluminum, magnesium, selenium, and zinc, as these can significantly reduce the drug's absorption.


Q: Is Eltrombopag a steroid or an immunosuppressant?

Eltrombopag is classified as a Thrombopoietin Receptor Agonist (TPO-RA). This is a small-molecule agent that works by stimulating the platelet-producing receptors in the bone marrow. It is not classified as a steroid or a classical immunosuppressant, though it may be used in combination with immunosuppressive therapy for some conditions.


Q: Are there specific symptoms that require immediate medical attention while taking Eltrombopag?

Official warnings describe that immediate medical attention is required if signs of a blood clot (like swelling, pain, or tenderness in one leg, or sudden shortness of breath) or signs of severe liver problems (such as yellowing of the skin/eyes, unusual darkening of the urine, or upper stomach pain) are noticed.


Q: How does Eltrombopag compare to Romiplostim regarding how they are taken?

Both are TPO-RAs used to treat low platelet counts. However, Eltrombopag is administered orally as a tablet or liquid suspension. In contrast, Romiplostim, another drug in the same class, is administered by subcutaneous injection.


Q: Is Eltrombopag considered a type of chemotherapy?

No, Eltrombopag is not classified as a cytotoxic agent or chemotherapy. It is officially categorized as a Thrombopoietin Receptor Agonist (TPO-RA). It works by stimulating the body's bone marrow to produce platelets, rather than killing cells.


Q: Do you have to take Eltrombopag for the rest of your life?

The required duration of therapy depends on the condition being treated. While treatment for Chronic ITP is adjusted and often continued as needed to maintain platelet counts, the recommended duration of therapy for first-line Severe Aplastic Anemia is specified as 6 months in the official information.


Q: How quickly can someone expect Eltrombopag to start working?

Studies and official information indicate that platelet counts generally begin to increase within 1 to 2 weeks after initiation of Eltrombopag treatment.


Q: Can Eltrombopag be taken with common pain relievers like Tylenol?

Official drug information indicates a potential pharmacokinetic interaction with acetaminophen (Tylenol), suggesting Eltrombopag may affect the levels of acetaminophen in the body by influencing its metabolism. This is noted in regulatory databases.


Q: If my blood counts improve, can I stop taking Eltrombopag?

Regulatory documents provide specific criteria for discontinuation. For example, treatment may be stopped if the platelet count remains above a very high threshold (e.g., 400 imes 10^9/ L) after two weeks at the lowest dose, or if no response is achieved within a certain timeframe.


Q: What happens if I accidentally take too much Eltrombopag?

Information on overdose is limited. The key concern documented in regulatory information is that a significantly elevated platelet count increases the risk of serious thrombotic or thromboembolic complications (blood clots).


Q: How is Eltrombopag different from a platelet transfusion?

The key difference is the mechanism of action. Eltrombopag is an oral medication that works by stimulating your body's own bone marrow to produce new platelets over time. A platelet transfusion is an external medical procedure that provides platelets immediately from a blood product.


Q: Are there any driving restrictions while using Eltrombopag?

Official warnings note that dizziness is a common side effect of Eltrombopag. Official warnings indicate that patients must be aware of this potential effect, as dizziness is documented as potentially impairing the ability to drive or operate machinery.


Q: What should I do if I feel dizzy after taking Eltrombopag?

Dizziness is noted as a common side effect in clinical studies and official information cautions that it may affect the ability to drive or operate machinery.


Q: Does Eltrombopag interact with alcohol?

While direct pharmacological interaction between Eltrombopag and alcohol is not fully established, some regulatory patient information notes that alcohol use should be limited because it can increase the risk of bleeding, which is a key concern with low platelet counts.


Q: What is the maximum duration for which Eltrombopag is approved for use?

For conditions like Chronic ITP and refractory SAA, there is no specified absolute maximum duration, as treatment is maintained to keep platelet counts in the target range. However, for first-line Severe Aplastic Anemia, the duration is specifically set at 6 months.


Q: What is the usual time it takes to stop treatment with Eltrombopag?

Regulatory studies show that if the medication is discontinued, platelet counts are expected to decrease, often falling back toward pre-treatment levels within 1 to 2 weeks. For this reason, platelet count monitoring is required following discontinuation.


Q: Can Eltrombopag be crushed or split?

Official administration instructions dictate that Eltrombopag tablets should not be split, chewed, or crushed and should not be mixed with food or liquids. The tablet must be swallowed whole.


Q: What is the risk of platelet levels dropping after stopping Eltrombopag?

The official product information indicates that if the medication is stopped, platelet counts are expected to drop, often returning to near pre-treatment levels within 1 to 2 weeks. For this reason, official guidance states that platelet count monitoring is required following discontinuation.


Q: What happens if I develop a fever while taking Eltrombopag?

Fever (pyrexia) is listed as a common adverse reaction in the official safety data, particularly noted in pediatric patients.

How should Eltrombopag be stored and disposed of?

How to Store and Dispose of Eltrombopag?

Eltrombopag, in both tablet and powder forms, must be stored at Controlled Room Temperature, defined as 20 C to 25 C (68 F to 77 F). The product must be kept in its original container, which should be tightly closed to protect it from moisture, and must not be refrigerated or frozen. The medicine must be stored out of the reach of children.

Stability and Handling

For the powder for oral suspension, use cold water only for mixing. The prepared suspension has a short stability period and must be used immediately or discarded if not administered within 30 minutes.

Official Disposal

Unused or expired Eltrombopag must be disposed of through an official drug take-back program or by following local regulations. The medicine should not be flushed down a toilet or poured into a drain unless explicitly stated on the label.

Attention! Always consult to a doctor or pharmacist before using pills or medicines.

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