Eltrombopag

(Promacta®)

Eltrombopag

Drug updated on 5/17/2024

Dosage FormTablet (oral; 12.5 mg, 25 mg, 50 mg, and 75 mg); Oral suspension (oral; 12.5 mg, 25 mg)
Drug ClassThrombopoietin receptor agonists
Ongoing and
Completed Studies
ClinicalTrials.gov

Indication

  • Indicated for the treatment of thrombocytopenia in adult and pediatric patients 1 year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. PROMACTA should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.
  • Indicated for the treatment of thrombocytopenia in patients with chronic hepatitis C to allow the initiation and maintenance of interferon-based therapy. PROMACTA should be used only in patients with chronic hepatitis C whose degree of thrombocytopenia prevents the initiation of interferon-based therapy or limits the ability to maintain interferon-based therapy.
  • Indicated in combination with standard immunosuppressive therapy for the first-line treatment of adult and pediatric patients 2 years and older with severe aplastic anemia.
  • Indicated for the treatment of patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy.

Summary
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  • Eltrombopag (Promacta) is indicated for the treatment of thrombocytopenia in adult and pediatric patients with persistent or chronic immune thrombocytopenia (ITP), severe aplastic anemia, and those with chronic hepatitis C. It has shown effectiveness by increasing platelet production.
  • Thirteen studies were reviewed to gather information about eltrombopag's safety and efficacy compared to other treatments such as romiplostim, avatrombopag, hetrombopag, rituximab, and immunosuppressive therapy alone.
  • In treating ITP specifically, eltrombopag demonstrated a significant improvement in platelet response outcomes when compared to placebo or rituximab. This was observed both in adults with refractory ITP as well as children suffering from the condition.
  • Regarding ITP treatment scenarios, eltrombopag did not significantly increase the risk of critical adverse events like thrombotic occurrences when compared against controls or other agents like romiplostim or avatrombopag within six months of administration.
  • For severe aplastic anemia (SAA) cases, combining eltrombopag with standard immunosuppressive therapy showed higher overall response rates at three and six months than using immunosuppressive therapy alone. However, these benefits seem to plateau after twelve months of treatment regimen.
  • The addition of eltrombopag also improved earlier hematologic responses without affecting event-free survival rate nor increased incidence of clonal evolution during SAA treatments, indicating its good safety profile even when combined with immunosuppressive therapies.
  • In terms of population types/subgroup considerations, age does not influence treatment effects, suggesting broad applicability across different patient demographics including pediatrics where it protected against severe disease/death indicating a favorable risk-benefit profile. However, caution should be exercised when interpreting its efficacy and safety data, especially for pediatric SAA cases.
  • Compared to other Thrombopoietin Receptor Agonists (TPO-RAs) like romiplostim, avatrombopag, and hetrombopag, eltrombopag has shown significant effectiveness in increasing platelet counts with a comparable safety profile regarding serious adverse events.

Product Monograph / Prescribing Information

Document TitleYearSource
Promacta (eltrombopag) Prescribing Information.2023Novartis Pharmaceuticals Corporation, East Hanover, NJ

Systematic Reviews / Meta-Analyses

Document TitleYearSource
Thrombopoietin receptor agonists use and risk of thrombotic events in patients with immune thrombocytopenic purpura: a systematic review and meta analysis of randomized controlled trials.2024Biomedical Reports
Efficacy and safety of immunosuppressive therapy combined with eltrombopag for severe aplastic anemia: a systematic review and meta-analysis.2024Systematic Reviews
Thrombopoietin receptor agonists use and risk of thrombotic events in patients with immune thrombocytopenic purpura: a systematic review and meta analysis of randomized controlled trials.2024Biomedical Reports
Eltrombopag for adults and children with immune-refractory thrombocytopenic purpura: a systematic review.2023Journal of Clinical Medicine
The efficacy and the safety of eltrombopag in pediatric patients with severe aplastic anemia: a systematic review.2023Frontiers in Pediatrics
Safety of non peptide thrombopoietin receptor agonists in patients with immune thrombocytopenia: a systematic review and meta analysis of short term double blind randomized clinical trials.2023Experimental and Therapeutic Medicine
Risk of thrombotic events in immune thrombocytopenia patients treated with thrombopoietic agents: a systematic review and meta-analysis. 2023Thrombosis Journal
Safety of non‑peptide thrombopoietin receptor agonists in patients with immune thrombocytopenia: a systematic review and meta‑analysis of short‑term double‑blind randomized clinical trials.2023Experimental and Therapeutic Medicine
Systematic literature review and meta-analysis on use of Thrombopoietic agents for chemotherapy-induced thrombocytopenia.2022PloS one
Efficacy and safety of avatrombopag in patients with chronic immune thrombocytopenia: a systematic literature review and network meta-analysis. 2021Advances in Therapy
Eltrombopag effectiveness and tolerability in chronic immune thrombocytopenia: a meta-analysis.2021Clinical and Applied Thrombosis/Hemostasis
Treatment efficacy for adult persistent immune thrombocytopenia: a systematic review and network meta-analysis.2020British Journal of Haematology
Therapeutic options for adult patients with previously treated immune thrombocytopenia – a systematic review and network meta-analysis.2019Hematology

Clinical Practice Guidelines