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November 21, 2025Frontiers in MedicineOpen Access

Efficacy and influencing factors of immunosuppressive therapy combined with or without eltrombopag in children with severe aplastic anemia

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Authors

JGJu GaoHYHui YangMLMeiling Liao

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Overview

Retrospective study reports comparable remission rates across eltrombopag and immunosuppressive regimens in children with severe aplastic anemia, indicating early initiation improves response.

Key Points

  • To compare the efficacy, safety, and influencing clinical factors of eltrombopag combined with standard immunosuppressive regimens versus cyclosporine alone in pediatric severe aplastic anemia.
  • Retrospective study evaluating 41 pediatric patients divided into Group A (eltrombopag + rabbit antithymocyte globulin + cyclosporine, n = 12), Group B (eltrombopag + cyclosporine, n = 13), and Group C (cyclosporine alone, n = 16).
  • Overall remission rate was tracked at 1, 3, 6, and 12 months, and adverse events were evaluated alongside baseline hematological predictors of recovery.
  • Overall remission rates did not differ significantly among Groups A, B, and C at 1, 3, 6, and 12 months of follow-up (P > 0.05).
  • Adverse reaction rates at 6 months were 36.4% (4/11) in Group A, 58.3% (7/12) in Group B, and 69.2% (9/13) in Group C (P = 0.264), with zero cases of clonal evolution.
  • Hematopoietic response was more frequent in patients receiving eltrombopag within 60 days of diagnosis, and those with higher baseline platelets, white blood cells, lymphocytes, and CD4+/CD8+ ratios.

Cite This Study

Gao et al. (2025) studied this question.

synapsesocial.com/papers/6a7d90042a69415fbc1869fahttps://doi.org/10.3389/fmed.2025.1688771
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Efficacy and influencing factors of immunosuppressive therapy combined with or without eltrombopag in children with severe aplastic anemia2025
  2. 2A Multicenter, Retrospective Study Comparing Immunosuppressive Therapy Combined with Eltrombopag to Immunosuppressive Therapy Alone as Front-Line Treatment for Pediatric Severe Aplastic Anemia.2025 · 2 citations
  3. 3Long-term outcomes in patients with severe aplastic anemia treated with immunosuppression and eltrombopag: a phase 2 study2021 · 111 citations
  4. 4Outcomes of eltrombopag combined with immunosuppressive therapy in adults with severe aplastic anemia: A large, propensity-matched retrospective analysis2025
  5. 5Long-term outcomes of intensive immunosuppressive therapy combined with eltrombopag in patients with severe aplastic anemia2025