Eltrombopag Improves Sustained Platelet Response in Pediatric Immune Thrombocytopenia: JAMA

A new study published in the Journal of American Medical Association showed that in children with newly diagnosed immune thrombocytopenia, eltrombopag produced a greater sustained platelet response than conventional medication, indicating that it might be a useful therapeutic choice for patients with non-severe bleeding who need medical attention.

For children with persistent immunological thrombocytopenia, the US Food and Drug Administration has licensed eltrombopag, a thrombopoietin receptor agonist. Eltrombopag's effectiveness in the recently identified stage of juvenile immune thrombocytopenia is uncertain. Therefore, this research was set to ascertain if individuals with newly diagnosed immune thrombocytopenia treated with eltrombopag had a substantially higher proportion of platelet responses than patients treated with conventional therapy (first-line therapies).

Patients (aged 1–18 years) with newly diagnosed primary immune thrombocytopenia (platelet count <30 × 109/L) who needed pharmacological treatment but did not have severe bleeding or require a rapid increase in platelet count were recruited for this trial at 23 centers that are part of the Pediatric ITP Consortium of North America. The trial ran from May 7, 2019, to January 25, 2024. February 26, 2025, was the date of the last follow-up.

In contrast to usual treatment (investigator choice of intravenous immunoglobulin, glucocorticoids, or anti-D immunoglobulin) (n = 40), eltrombopag was given orally according to a regular dose regimen (n = 78). A sustained platelet response defined as 3 or more of 4 platelet counts over 50 × 109/L during weeks 6–12 without rescue treatment, was the main result. Serious adverse events, bleeding scores, and changes in health-related quality of life were among the secondary outcomes.

After observation or medicinal therapy, 63% of the 118 pediatric patients (median age, 8 years [IQR, 4-12 years]; 49% were male) had an initial treatment failure. After a planned interim analysis reached a predetermined effectiveness threshold, enrollment was terminated.

13 out of 37 patients (35% [95% CI, 20%-51%]) in the conventional treatment group (between-group difference, 30% [95% CI, 11%-49%]; P =.002) had a sustained platelet response, whereas 46 (65% [95% CI, 54%-76%]) of the 71 patients in the eltrombopag group had a sustained platelet response.

Overall, the quantity and kind of adverse events did not differ across groups. The incidence of sustained platelet response was greater with eltrombopag than with conventional therapy in pediatric patients with newly diagnosed immune thrombocytopenia who needed pharmacological treatment.

Source:

Shimano, K. A., Grimes, A. B., Kaicker, S., Shah, S. J., Gunn, E., Bhat, R. V., Kochhar, M., Rothman, J. A., Rose, M. J., Briones, M., Nakano, T. A., Lebensburger, J. D., Lambert, M. P., Fritch Lilla, S. A., Jesudas, R., Lee-Miller, C. A., Thompson, A. A., Rifkin-Zenenberg, S., Majumdar, S., … Grace, R. F. (2025). Eltrombopag for newly diagnosed pediatric immune thrombocytopenia requiring treatment: The PINES randomized clinical trial: The PINES randomized clinical trial. JAMA: The Journal of the American Medical Association. https://doi.org/10.1001/jama.2025.18168