Efficacy and safety of immunosuppressive therapy with or without eltrombopag in pediatric patients with acquired aplastic anemia: A Chinese retrospective study

Abstract

To determine the efficacy and safety of eltrombopag (E-PAG) combined with intensive immunosuppressive therapy (IST) for the treatment of pediatric patients with severe aplastic anemia (SAA). A total of 57 pediatric patients with newly diagnosed severe aplastic anemia were enrolled in this study. Thirty nine patients were treated with IST alone, consisting of porcine anti-human thymocyte globulin (30 mg/kg/day × 5 days) and cyclosporine A (CsA) (treated for 2 years, with a trough concentration maintained at 200–250 ng/mL), and 18 patients were treated with IST + E-PAG (12.5–50 mg/day, maintained for 6 months). We found no statistical difference between the response rates at 3 months for the two groups (CR: 12.8% vs. 22.2% p > 0.05, ORR: 56.4% vs. 77.7% p > 0.05). However, we found a statistical difference between the response rates at 6 months for the two groups (CR: 17.9% vs. 50% p < 0.05, ORR: 69.2% vs. 94.4% p < 0.05). The main side-effect during treatment with E-PAG was having a slightly to moderately elevated bilirubin level, which was temporary and controllable, accounting for approximately 66.6% (12/18) of patients in the IST + E-PAG group vs. 20.5% (8/39) of those in the IST group (p < 0.05). IST + E-PAG therapy appears to be more effective than IST alone for the treatment of pediatric SAA, with good tolerability and compliance. This approach deserves further exploration.

Keywords:

• Children
• cyclosporine A
• eltrombopag
• porcine anti-human thymocyte globulin
• severe aplastic anemia

Acknowledgments

The authors would like to thank Computational Engineering and Networking (CEN) department for encouraging the research.

Disclosure statement

The authors declare no conflict of interest.

Data availability statement

The data that support the findings of this study are available on request from the corresponding author, Yongmin Tang. The data are not publicly available due to ethical restrictions.

Contributors’ statement

Meixin Fang conceptualized and designed the study, collected data, performed the data analysis and wrote the manuscript. Hua Song, Jingying Zhang, Sisi Li, Diying Shen: helped data analysis with constructive discussions. Yongmin Tang as the correspondence author takes the full responsibility of this manuscript with contribution for the conception of the study, data analysis, and manuscript amendment.

Additional information

Funding

This work was supported by a grant from the Fund of Zhejiang Provincial Scientific Technology Department No. 2019C03032.