Eltrombopag (Revolade) para niños con púrpura trombocitopénica inmune crónica

Eltrombopag is intended to be used for the treatment of chronic immune thrombocytopenic purpura (ITP) in children. If licensed, it will provide a treatment option for this patient group, for whom there are currently no licensed therapies available. 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 adult ITP and thrombocytopenia in adults with chronic hepatitis C virus infection. ITP is an acquired autoimmune disorder of adults and children characterised by increased platelet destruction, and in some cases, inadequate platelet production. The incidence of ITP in children is around 4 per 100,000 population, and it is more common in two groups: young children and young adults. Approximately 25% of children develop chronic disease, which is often a mild disorder that does not significantly interfere with daily life. However in some children, frequent and serious haemorrhages may occur, including intracranial haemorrhage, which is fatal in around 50%. In 2012-13, there were 1,094 finished consultant episodes due to ITP in children in England, and in 2012, one death was registered in England and Wales. Most children with ITP do not require specific treatment. Treatment is dependent on a range of factors including the presence and severity of bleeding, co-morbidities, specific instances such as surgery, and the side effects of treatment. Current options include intravenous immunoglobin, steroids, rituximab, thrombopoeitin receptor agonists, platelet transfusion or splenectomy. Eltrombopag is currently in a phase II clinical trial comparing its effect on platelet counts against treatment with placebo. The completion date of this trial has not reported.