Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomized, multicenter, placebo-controlled trial

Grainger JD (2015), Lancet (Link to Abstract)         

Eltrombopag is a thrombopoietin receptor agonist, which is used effectively in adults with chronic immune thrombocytopenia. This study involved a randomized, multicenter, placebo-controlled, double blinded study looking at the safety and efficacy of eltrombopag in pediatric patients with chronic ITP. 92 patients: 63 in the treatment arm and 29 in the placebo arm. 40% of patients achieved platelet counts of greater than or equal to 50 during the initial 12 weeks of the study, while only 3% of the placebo treated group achieved these counts. 80% of patients achieved a platelet count of greater than 50 in the 24 weeks following the randomized portion of the study, when all patients were treated with Eltrombopag. There was no significant difference in adverse events between the two groups.

* Eltrombopag, a thrombopoietin receptor agonist, is a suitable therapeutic option to consider in pediatric patients with chronic ITP.