Eltrombopag for Newly Diagnosed Pediatric Immune Thrombocytopenia Requiring Treatment: The PINES Randomized Clinical Trial
치료가 필요한 새로 진단된 소아 면역성 혈소판 감소증에 대한 엘트롬보파그 (Eltrombopag)의 효과 PINES 무작위 임상시험
JAMA 334(20):p 1816-1826, November 25, 2025. | DOI: 10.1001/jama.2025.18168
Eltrombopag, a thrombopoietin receptor agonist, is approved by the US Food and Drug Administration for children with chronic immune thrombocytopenia. The efficacy of eltrombopag during the newly diagnosed phase of pediatric immune thrombocytopenia is unknown. Source JAMA 11/25/2025
According to the 2025-07-29 following report
Study Overview
Brief Summary
This is an investigator-initiated, multicenter, open-label, randomized phase 3 study for subjects with newly diagnosed ITP from ages 1 to less than 18 years old.
Detailed Description
This is a prospective, open-label, randomized, two-arm, multi-center Phase 3 trial.
Patients with newly diagnosed ITP are randomized 2:1 to receive the experimental treatment, eltrombopag, or the investigator’s choice of 3 standard therapies. The primary objective is to determine whether the proportion of patients with platelet response is significantly higher in those treated with eltrombopag than in those treated with standard therapies.
12/1/2025