Eltrombopag (Revolade) para anemia aplásica severa - segunda línea

Eltrombopag is intended to be used for the treatment of cytopenias in patients with severe aplastic anaemia who have had an insufficient response to immunosuppressive therapy. If licensed, eltrombopag will offer a treatment option for this patient group. Eltrombopag is a non-peptidyl thrombopoietin (TPO) receptor agonist. TPO is the principal cytokine involved in the regulation of megakaryopoiesis and platelet production and acts through the activation of the TPO receptor. Eltrombopag mimics the effect of TPO thereby stimulating platelet production. Eltrombopag is licensed in the EU for the treatment of chronic immune (idiopathic) thrombocytopenic purpura in adult splenectomised patients who are refractory to other treatments, second line treatment of thrombocytopenia in adult non-splenectomised patients where surgery is contraindicated, and for the treatment of thrombocytopenia in adult patients with chronic hepatitis C virus infection. Aplastic anaemia is a rare condition and its precise incidence is difficult to determine due to the imprecision in establishing a diagnosis. However, estimates indicate that annual incidence is around two cases per million population. The incidence of aplastic anaemia varies throughout the world and is 2-3 times more common in Southeast Asia. In England, there were 12,987 admissions due to forms of aplastic anaemia in 2012-13, resulting in 14,390 finished consultant episodes and 19,658 bed days. In 2012, forms of aplastic anaemia accounted for 216 deaths in England and Wales. Treatment for aplastic anaemia aims to correct the hypo-cellular bone marrow of the patient while providing supportive care where appropriate. The main two effective treatments for those with acquired severe aplastic anaemia are allogeneic bone marrow stem cell transplantation and immunosuppressive therapy. Supportive care can include platelet and red blood cell transfusions, growth factors and antibiotics. Eltrombopag is currently in uncontrolled phase II clinical trials to determine its effect on haematological response in patients with severe aplastic anaemia. These trials are expected to complete in December 2016.