ASH 2024: Eltrombopag shows promise in treating newly diagnosed pediatric ITP

December 11, 2024

Study design: The phase 3, randomized Pediatric ITP Newly diagnosed pts Epag vs Standard therapy (PINES) trial compared eltrombopag with standard first-line treatments (corticosteroids, IVIg, and anti-D globulin) in children with newly diagnosed immune thrombocytopenia (ITP). Participants were randomly assigned to receive either eltrombopag or standard treatment at specified doses, and the primary endpoint was the proportion of responders.

Results: The eltrombopag group had a significantly higher proportion of responders compared with the standard treatment group (p = 0.0023; z-score = 3.04), crossing the monitoring boundary for efficacy in the second planned interim analysis. The primary endpoint of platelet response was achieved by 65% patients in the eltrombopag arm, compared with 33% patients in the standard treatment arm (p = 0.0007).

Impact on clinical practice: These findings, presented at the American Society of Hematology’s annual meeting, suggest that eltrombopag could be a more effective first-line treatment for newly diagnosed pediatric ITP, potentially leading to changes in treatment protocols and improved patient outcomes.

Source:

Shimano, K et al. (2024, December 9). Efficacy Findings in a Phase 3, Randomized Trial of Eltrombopag Vs. Standard First-Line Treatment for Newly Diagnosed Immune Thrombocytopenia in Children. Abstract #709. Presented at American Society of Hematology Annual Meeting & Exposition. https://ash.confex.com/ash/2024/webprogram/Paper193644.html