GSK eltrombopag study shows statistically significant platelet response in children with ITP

GSK plc announced on June 13, 2014, results from its Phase III PETIT2 study that assessed the efficacy of eltrombopag versus placebo in pediatric patients with chronic immune (idiopathic) thrombocytopenic purpura (cITP). The study met its primary endpoint, demonstrating a statistically significant improvement.

Nearly 40 percent of patients treated with eltrombopag achieved a consistent platelet response for at least six out of eight weeks, compared to only 3.4 percent in the placebo group (p<0.001). These findings were presented at the European Hematology Association Annual Congress in Milan.

Eltrombopag, known as Promacta™ in the U.S. and Revolade™ in Europe and other countries, showed a safety profile consistent with previous findings, with no new safety concerns identified. Common adverse events in the eltrombopag arm included nasopharyngitis, rhinitis, cough, and respiratory tract infections. Serious adverse events occurred in 8 percent of eltrombopag-treated patients versus 14 percent in the placebo group.

GSK plans to pursue regulatory submissions for a pediatric indication in cITP later in 2014. cITP is a condition characterized by low platelet counts, affecting up to 5 in 100,000 children annually. Approximately 30 percent of pediatric ITP cases become persistent, posing a risk of severe bleeding.