avatrombopag in chronic immune thrombocytopenia

Last edited 12/2022 and last reviewed 02/2023

Avatrombopag in Thrombocytopaenia

  • is an orally administered second generation thrombopoietin receptor agonist (TPO-RA) approved for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory or have an unsatisfactory response to other treatments, as well as for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) scheduled to undergo an invasive procedure (1)
  • mimics the natural compound (thrombopoietin) responsible for stimulating the production of platelets, an essential component of the clotting process that prevents excessive bleeding

NICE state that:

  • Avatrombopag is recommended, within its marketing authorisation, as an option for treating primary chronic immune thrombocytopenia (ITP) refractory to other treatments (for example, corticosteroids, immunoglobulins) in adults. It is only recommended if the company provides it according to the commercial arrangement
  • the NICE committee note that
    • "Current treatment for newly diagnosed primary chronic ITP usually includes corticosteroids and immunoglobulins. This is followed by thrombopoietin receptor agonists (TPO-RAs). Avatrombopag is another TPO-RA...Clinical trial evidence shows that avatrombopag is more effective than placebo at increasing the number of platelets in the blood (cells that help the blood to clot) to a level that meaningfully reduces the risk of bleeding.."

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