ArticleFrontiers in medicine2026
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.
Article in Frontiers in medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Background: Very severe aplastic anemia (VSAA) is a life-threatening bone marrow failure syndrome. For patients ineligible for allogeneic hematopoietic stem cell transplantation (HSCT), effective treatments that induce rapid responses remain limited. This case report describes a young VSAA patient who achieved an exceptionally rapid trilineage response to first-line therapy with high-dose romiplostim (ROMI), hetrombopag, and standard immunosuppressive therapy (IST). Case presentation: A 16-years-old male with newly diagnosed VSAA (neutrophils 0.13 × 10 Conclusion: The combination of high-dose ROMI, hetrombopag, and IST induced a rapid response in this young VSAA patient who refused HSCT. This dual TPO-RA regimen warrants further investigation as a potential first-line option for HSCT-ineligible VSAA patients.
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