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Eltrombopag combined with immunosuppressive therapy for pediatric severe aplastic anemia

Pediatric Research (2024)Cite this article

Abstract

Background

Severe aplastic anemia (SAA) is caused by immune-mediated destruction. Standard immunosuppressive therapy (IST) is effective but needs to be improved.

Methods

The data of patients with SAA and received IST were analyzed retrospectively to conducted this historical control study.

Results

A total of 115 SAA patients (60 males; median age of 5.77 years and median follow-up time of 45 months) were enrolled in this study. The complete response rates (CRR) of the eltrombopag group at 3 and 6 months were higher than the control group (30.3% vs.8.2% at 3 months; 50.0% vs. 10.2% at 6 months). The overall response rates (ORR) showed no differences. There were significant differences in the times from G-CSF, Red blood cell transfusion, and Platelet transfusion between the two groups. No difference in overall survival (OS), event-free survival (EFS), and relapse rate between two groups. There is no variable were associated with prognosis in both groups.

Conclusion

Addition of eltrombopag to IST confers faster hematological response and higher early hematological response in pediatric SAA patients.

Impact

  • Addition of eltrombopag to standard immunosuppressive therapy confers faster hematological response and higher early hematological response in pediatric severe aplastic anemia patients.

  • Eltrombopag showed reliable safety but had no impact on long-term response and prognosis. This article is a historical controlled study consisting of 115 pediatric severe aplastic anemia patients and makes up for the lack of clinical data deficient on pediatric severe aplastic anemia with TPO-RA combined with IST.

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Fig. 1: OS in both groups.
Fig. 2: EFS in both groups.

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Data availability

The data underlying this article are available in the article and in its online supplementary material.

References

  1. Young, N. S. Aplastic anemia. N. Engl. J. Med. 379, 1643–56 (2018).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  2. Montane, E. et al. Epidemiology of aplastic anemia: a prospective multicenter study. Haematologica 93, 518–23 (2008).

    Article  PubMed  Google Scholar 

  3. Hartung, H. D., Olson, T. S. & Bessler, M. Acquired aplastic anemia in children. Pediatr. Clin. North Am. 60, 1311–36 (2013).

    Article  PubMed  PubMed Central  Google Scholar 

  4. Peslak, S. A., Olson, T. & Babushok, D. V. Diagnosis and treatment of aplastic anemia[J]. Curr. Treat. Options Oncol. 18, 70 (2017).

    Article  PubMed  PubMed Central  Google Scholar 

  5. Scheinberg, P., Wu, C. O., Nunez, O. & Young, N. S. Long-term outcome of pediatric patients with severe aplastic anemia treated with antithymocyte globulin and cyclosporine. [J]. J. Pediatr. 153, 814–9 (2008).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  6. Rogers, Z. R. et al. Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study[J]. Haematologica 104, 1974–83 (2019).

  7. Yoshida, N. et al. First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy. [J]. Haematologica 99, 1784–91 (2014).

    Article  PubMed  PubMed Central  Google Scholar 

  8. Carlo et al. Outcome of aplastic anemia in adolescence: a survey of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation. [J]. Haematologica 99, 1574–81 (2014).

    Article  Google Scholar 

  9. Garanito, M. P., Carneiro, J. D. A., Filho, V.O. & Scheinberg, P. Outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine A, [J]. Jornal de pediatria 90, 523-7 (2014).

  10. Dufour, C. et al. Outcome of aplastic anaemia in children. A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant. [J]. Br. J. Haematol 169, 565–73 (2015).

  11. Townsley, D. M. et al. Eltrombopag added to standard immunosuppression for aplastic anemia[J]. N. Engl. J. Med. 376, 1540–50 (2017).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  12. Olnes, M. J. et al. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N. Engl. J. Med. 367, 11–9 (2012).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  13. Peffault de Latour, R. et al. Eltrombopag added to immunosuppression in severe aplastic anemia. N. Engl. J. Med. 386, 11–23 (2022).

    Article  CAS  PubMed  Google Scholar 

  14. Groarke, E. M. et al. Eltrombopag added to immunosuppression for children with treatment naive severe aplastic anaemia[J]. Br. J. Haematol. 192, 605–14 (2021).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  15. Harry, L. et al. Eltrombopag in children with severe aplastic anemia. Pediatr. Blood Cancer 68, e29066 (2021).

    Article  Google Scholar 

  16. Olga, G. et al. Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia. Blood advances 7, 953–62 (2023).

  17. Subspecialty Group of Hematology, Society of Pediatrics, Chinese Medical Association The Editorial Board, Chinese Journal of Pediatrics. Recommendations for diagnosis and treatment of acquired aplastic anemia in children. Zhonghua Er Ke Za Zhi. 52,103-106 (2014).

  18. Erickson-Miller, C. et al. Preclinical activity of eltrombopag (SB-497115), an oral, nonpeptide thrombopoietin receptor agonist[J]. Stem Cells 27, 424–30 (2009).

    Article  CAS  PubMed  Google Scholar 

  19. Filippidou, M. et al. Longitudinal evaluation of eltrombopag in paediatric acquired severe aplastic anaemia. Br. J. Haematol. 190, e157–e173 (2020).

    Article  PubMed  Google Scholar 

  20. Kim, T. O. et al. Eltrombopag for use in children with immunethrombocytopenia. Blood Adv. 2, 454–61 (2018).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

Download references

Funding

This work was supported in part by grants from the National Natural Science Foundation of China (No. 81970111), Beijing Natural Science Foundation of China (No. 7192064), the Pediatric Medical Coordinated Development Center of Beijing Municipal Administration of Hospitals (No. XTZD20180205), and National Science and Technology Key Projects (No.2017ZX09304029001).

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Authors and Affiliations

  1. Department of Hematology, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, China

    Bixi Yang, Lingling Fu, Hongmin Li, Rui Zhang, Jiafeng Yao, Liqiang Zhang, Runhui Wu & Jie Ma

  2. Hematologic Disease Laboratory, Hematology Center, Beijing, China

    Hui Chen

Authors
  1. Bixi Yang
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  2. Lingling Fu
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  3. Hongmin Li
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  7. Liqiang Zhang
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  8. Runhui Wu
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  9. Jie Ma
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Contributions

Bixi Yang and Lingling Fu designed the study and wrote the manuscript. Hongmin Li, Hui Chen, Rui Zhang, Jiafeng Yao, Liqiang Zhang collected the medical records and conducted data processing. Jie Ma and Runhui Wu reviewed and corrected this manuscript professionally. All authors reviewed the manuscript and approved the final submission of the manuscript.

Corresponding author

Correspondence to Jie Ma.

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Competing interests

The authors declare no competing interests.

Ethics approval

This study was approved by the Ethics Committee of Beijing Children’s Hospital affiliated to Capital Medical University (approval number: 2019-k-276), and all the guardians of the subjects were informed

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Yang, B., Fu, L., Li, H. et al. Eltrombopag combined with immunosuppressive therapy for pediatric severe aplastic anemia. Pediatr Res (2024). https://doi.org/10.1038/s41390-024-03253-w

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