Efficacy and safety comparison of hetrombopag and eltrombopag in children with cancer therapy-induced thrombocytopenia: a single-center retrospective study.

OBJECTIVE

To compare the efficacy and safety of hetrombopag and eltrombopag in children with cancer therapy-induced thrombocytopenia (CTIT), and to explore the clinical value of eltrombopag for this specific pediatric population given the current lack of dedicated literature.

METHODS

This was a single-center retrospective study. A total of 185 pediatric CTIT patients were enrolled, including 114 cases in the hetrombopag group and 71 cases in the eltrombopag group. Baseline characteristics, platelet (PLT) parameters, efficacy outcomes (platelet response rate, response duration, transfusion dependence), and adverse events (AEs) were compared between the two groups. Statistical analyses were performed using appropriate tests (t-test, Mann-Whitney U test, χ2 test, and proportional odds logistic regression), with p < 0.05 considered statistically significant.

RESULTS

Baseline characteristics were generally balanced between the two groups, with no significant differences in age, height, weight, primary diagnosis, or baseline PLT count (all p > 0.05), except for gender distribution (p = 0.013). Regarding efficacy: (1) Eltrombopag was associated with higher rates of partial or complete platelet response relative to hetrombopag in this retrospective cohort, with an odds ratio (OR) of 1.978 (95% confidence interval [95% CI]: 1.295 ~ 3.021, p = 0.001) after adjusting for confounding factors. (2) The rate of platelet transfusion dependence was significantly lower in the eltrombopag group (15.7% vs. 31.3%, p = 0.019). (3) No significant difference was observed in PLT count at drug withdrawal (p = 0.493) or response duration (39.42 ± 102.99 days vs. 63.75 ± 129.33 days, p = 0.192) between the two groups. No significant intergroup differences in response duration were found between hetrombopag and eltrombopag across the primary and three sensitivity analyses (all p > 0.05). For safety: (1) The hetrombopag group had higher incidences of Grade 1-2 leukopenia (41.2% vs. 23.9%, p = 0.016) and anemia (45.6% vs. 25.4%, p = 0.006). (2) The eltrombopag group had a higher incidence of elevated bilirubin levels (69.0% vs. 47.4%, p = 0.028). Thrombocytosis and thrombotic events were rare in both groups with no significant differences (p > 0.05).

CONCLUSION

In pediatric CTIT patients, eltrombopag was linked to improved platelet response and less transfusion dependence, accompanied by a tolerable safety profile. As the first study focusing on eltrombopag for pediatric CTIT, these findings provide critical evidence supporting its clinical application as a promising therapeutic option for this understudied population.