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eltrombopag (Promacta)
Thrombopoietin Receptor Agonists

How it is administered

Eltrombopag is taken by mouth and is available in several forms, including tablets and oral suspension (a liquid). The tablets come in different strengths (such as 12.5 mg, 25 mg, 50 mg, and 75 mg), and the oral suspension is available in packets that are mixed with water before use.

It is important to take eltrombopag either on an empty stomach or with a meal that is low in calcium (less than 50 mg). You should take eltrombopag at least 2 hours before or 4 hours after any foods or supplements containing calcium, iron, magnesium, aluminum, selenium, or zinc, as these can reduce how much medication your body absorbs.

How it works

Eltrombopag is a thrombopoietin (TPO) receptor agonist. It works by binding to and activating the TPO receptor, which is found on the surface of certain cells in the bone marrow called megakaryocytes. These cells are responsible for producing platelets, which are important for blood clotting.

By stimulating the TPO receptor, eltrombopag encourages the growth and development of megakaryocytes, leading to increased production of platelets. This helps to raise platelet counts in people whose bone marrow is not making enough platelets, reducing the risk of bleeding. The effects of eltrombopag are usually seen within 1-2 weeks of starting treatment, and platelet counts typically return to baseline within 1-2 weeks after stopping the medication.

Eltrombopag is not a cure for the underlying disease but helps manage symptoms related to low platelet counts.

Common side effects

The most common side effects of eltrombopag (reported in clinical trials) include:

  • Nausea
  • Diarrhea
  • Headache
  • Fatigue
  • Cough
  • Fever (pyrexia)
  • Increased liver enzymes (alanine aminotransferase, aspartate aminotransferase)
  • Rash
  • Upper respiratory tract infection
  • Abdominal pain
  • Muscle aches (myalgia)
  • Cataracts (clouding of the lens in the eye)

Other possible side effects include increased risk of blood clots, liver problems, and skin discoloration. Regular monitoring of blood counts and liver function is recommended during treatment.

Who Should take it

Eltrombopag is used to treat thrombocytopenia (low platelet count) in adults and children 1 year and older with persistent or chronic immune thrombocytopenia (ITP) who have not responded well to other treatments such as corticosteroids, immunoglobulins, or splenectomy.

It is also approved for use in patients with chronic hepatitis C to allow the initiation and maintenance of interferon-based therapy when low platelet counts would otherwise prevent or interrupt this treatment. Additionally, eltrombopag is used in combination with standard immunosuppressive therapy for the first-line treatment of severe aplastic anemia in adults and children 2 years and older, and for those with severe aplastic anemia who have not responded to previous immunosuppressive therapy.

Important Note: Eltrombopag is NOT indicated for the treatment of patients with myelodysplastic syndromes (MDS), as studies have shown an increased risk of progression to acute myeloid leukemia (AML) in these patients.

Who should not take it

Eltrombopag should not be used in patients with myelodysplastic syndromes (MDS), as it has been shown to increase the risk of progression to acute myeloid leukemia (AML) and may increase the risk of death in these patients.

There are no absolute contraindications listed, but caution should be used in patients with liver problems, as eltrombopag can cause or worsen liver injury. Patients with a history of blood clots or risk factors for blood clots should also use eltrombopag with caution, as it can increase the risk of thrombotic events. Women who are pregnant or breastfeeding should discuss the risks and benefits with their healthcare provider, as there is limited information on use in these populations.

Always inform your doctor about all your medical conditions and medications before starting eltrombopag.

Commonly used with

Eltrombopag is sometimes used in combination with standard immunosuppressive therapy (such as antithymocyte globulin and cyclosporine) for the treatment of severe aplastic anemia.

In patients with chronic hepatitis C, eltrombopag may be used alongside antiviral medications like pegylated interferon and ribavirin to help maintain adequate platelet counts during treatment. It is not typically used with other platelet-boosting agents unless specifically directed by a healthcare provider.

Commonly tested with

Eltrombopag has been tested in combination with standard immunosuppressive therapies (such as antithymocyte globulin and cyclosporine) in patients with severe aplastic anemia.

In studies for hepatitis C, it has been tested with pegylated interferon and ribavirin. It has also been studied in combination with azacitidine in patients with myelodysplastic syndromes (MDS), but this combination is not recommended due to increased risks. Eltrombopag is not recommended for use in combination with direct-acting antiviral agents for hepatitis C unless interferon is also part of the regimen.

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