Eltrombopag in pediatrics: revealing hidden signals of adverse drug events

doi:10.5246/jcps.2025.11.077

Abstract

Abstract:

This study sought to investigate adverse drug event (ADE) signals associated with eltrombopag use in pediatric patients aged 0–18 years, utilizing data from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS). By analyzing this extensive pharmacovigilance database, the study aimed to offer meaningful insights for improving the clinical safety of eltrombopag in children. Data covering eltrombopag-related ADEs from Q1 2004 to Q4 2023 were extracted from FAERS, and signal detection was conducted using both the reporting odds ratio (ROR) and proportional reporting ratio (PRR) methods. ADEs were categorized based on the System Organ Class (SOC) classification in MedDRA version 25.0. A total of 582 reports involving pediatric patients receiving eltrombopag were identified, encompassing 21 SOC categories. The analysis revealed that, in addition to the known ADEs listed in the drug label, clinicians should remain vigilant for potential off-label ADE signals. These included abnormal platelet counts, thrombocytosis, antiphospholipid syndrome, myelofibrosis, reduced serum iron levels, myelodysplastic syndrome, hepatic infections, and other related conditions. Given these findings, it is strongly recommended that serum iron and ferritin levels should be routinely monitored in pediatric patients undergoing eltrombopag therapy, particularly during long-term treatment. Such proactive surveillance may help prevent the onset of iron deficiency anemia and enhance overall treatment safety.

Key words: Children, Off-label medication, Eltrombopag, Signal mining, Adverse drug events

Supporting:

Yingqiu Tu, Tiantian Xu, Nan Zhong, Xin Lai. Eltrombopag in pediatrics: revealing hidden signals of adverse drug events[J]. Journal of Chinese Pharmaceutical Sciences, 2025, 34(11): 1033-1040.

Figures/Tables 8

Table 1. Proportional imbalance method table.

Table 2. Basic information of ADE reports for eltrombopag (n).

Table 3. The 30 most frequently reported ADEs for eltrombopag.

Table 4. Aggregate ADE reporting rates for investigation associated with eltrombopag use in pediatric patients aged 0–18 years.

Table 5. Aggregate ADE reporting rates for general disorders and administration site conditions associated with eltrombopag use in pediatric patients aged 0–18 years.

Table 6. Aggregate ADE reporting rates for gastrointestinal disorders with eltrombopag use in pediatric patients aged 0–18 years.

Table 7. Incidence of PT associated with eltrombopag in children aged 0–1 year.

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