About 93 subjects at 38 centres in 14 countries were enrolled in PETIT2, a 2-part, double-blind, randomised placebo-controlled and open-label trial, which was designed to investigate the efficacy, safety and tolerability of eltrombopag in these patients.
Eltrombopag is marketed as Promacta in the US and as Revolade in Europe and other countries worldwide.
In the trial, eltrombopag met its primary endpoint, achieving a statistically significant improvement in platelet counts with almost 40% of patients treated with eltrombopag attaining a consistent platelet response for 6 of 8 weeks compared to placebo.
GlaxoSmithKline head of Oncology R&D Rafael Amado said the PETIT2 study results presented at the European Haematology Association Annual Congress, 12-15 June in Milan, Italy, show an increase in platelet response rate with eltrombopag treatment — an important result given that these children had failed other standard therapies.
"We look forward to continuing to assess the potential of eltrombopag in these patients and to moving forward with planned regulatory submissions for a paediatric indication in cITP later this year," Amado said.
The company said that efficacy results for the trial were consistent across age cohorts, while the safety profile was consistent with the established profile for eltrombopag and no new safety concerns were observed.
Image: GlaxoSmithKline headquaters in London, UK. Photo: Courtesy of Maxwell Hamilton