Rapid early hematological response in a newly diagnosed patient with very severe aplastic anemia: a case report of high-dose romiplostim, hetrombopag and IST combination therapy - Takeaways - MDSpire

Rapid early hematological response in a newly diagnosed patient with very severe aplastic anemia: a case report of high-dose romiplostim, hetrombopag and IST combination therapy

  • By

  • Liangkui Luo

  • Kaikai Huang

  • Yanbin Pang

  • May 25, 2026

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  • 1

    Very severe aplastic anemia (VSAA) is a critical condition requiring rapid treatment responses, especially for patients ineligible for HSCT.

  • 2

    A 16-year-old male with newly diagnosed VSAA achieved complete remission after treatment with high-dose romiplostim, hetrombopag, and immunosuppressive therapy.

  • 3

    The patient experienced a partial trilineage response by week 9 and complete remission by week 22, with sustained remission after 7 months.

  • 4

    The combination therapy resulted in transfusion independence and minimal adverse effects, indicating a promising treatment approach for VSAA.

  • 5

    This case suggests that dual TPO-RA therapy may be a viable first-line option for HSCT-ineligible patients with VSAA, warranting further investigation.

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