Eltrombopag Improves Sustained Platelet Response in Pediatric ITP: JAMA
USA: A new study has revealed that among children with newly diagnosed immune thrombocytopenia needing pharmacologic treatment, eltrombopag produced a higher rate of sustained platelet response compared with standard therapy. Therefore, it may be a useful option for pediatric patients with non-severe bleeding who require medical intervention.
Researchers conducted the phase 3 PINES randomized clinical trial to rigorously compare eltrombopag with commonly used first-line therapies in this population, aiming to clarify whether early use of this thrombopoietin receptor agonist could offer a more dependable improvement in platelet counts. Published in JAMA, the study sheds new light on treatment strategies for pediatric immune thrombocytopenia, a condition in which optimal therapy during the newly diagnosed phase remains an area of uncertainty.
The PINES randomized clinical trial enrolled 118 children aged 1 to under 18 years who had newly diagnosed primary ITP with platelet counts below 30 × 10⁹/L. All participants required pharmacologic treatment but did not have severe bleeding or an urgent need for rapid platelet elevation. Patients were recruited from 23 centers across North America between May 2019 and January 2024, with final follow-up in February 2025.
Participants were randomized to receive either eltrombopag according to a standard oral dosing regimen or standard therapy, which included investigator-selected treatments such as glucocorticoids, intravenous immunoglobulin (IVIG), or anti-D immunoglobulin. The primary endpoint was achieving a sustained platelet response—defined as three or more platelet counts above 50 × 10⁹/L during weeks 6 to 12 without requiring rescue medication.
Key Findings:
- Eltrombopag showed a clear advantage, with 65% of the 71 treated children achieving a sustained platelet response.
- Only 35% of the 37 children receiving standard therapy reached the same response threshold.
- The 30% absolute difference between the two groups met the predefined efficacy criteria, leading to early termination of trial enrollment.
- Adverse events were comparable in both groups, indicating that the higher response rate with eltrombopag did not result in increased toxicity.
- Around 63% of participants had previously shown inadequate response to observation or initial medical management before joining the study.
- The effective response seen with eltrombopag in this population highlights its potential value for children who do not respond sufficiently to first-line treatment options.
The authors did outline several limitations. Children with severe bleeding were excluded, meaning the findings cannot be generalized to high-risk patients needing rapid platelet improvement. The modest sample size also limited subgroup analyses, and the open-label trial design could introduce bias in rescue treatment decisions. Additionally, the study was US-based, and drug availability may differ internationally. It is also unknown whether these results can be applied to other thrombopoietin receptor agonists or to adults, though early studies in adult populations have shown potential.
Future research from this trial will explore the need for second-line therapy, remission rates, biologic correlates, platelet kinetics, cost considerations, and detailed patient-reported outcomes.
"Overall, the trial provides strong evidence supporting eltrombopag as an effective therapeutic option for children with newly diagnosed ITP who require pharmacologic intervention but do not present with severe bleeding," the authors concluded.
Reference:
Shimano KA, Grimes AB, Kaicker S, et al. Eltrombopag for Newly Diagnosed Pediatric Immune Thrombocytopenia Requiring Treatment: The PINES Randomized Clinical Trial. JAMA. 2025;334(20):1816–1826. doi:10.1001/jama.2025.18168
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