ArticleAnnals of hematology2026
Haploidentical hematopoietic stem cell transplantation compared to immunosuppressive therapy plus eltrombopag as first-line treatment for severe aplastic anemia patients aged 40-65: a multicenter retrospective study.
Article in Annals of hematology, 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
Immunosuppressive therapy (IST) plus eltrombopag (EPAG) (IST+EPAG) is the preferred first-line treatment for severe aplastic anemia (SAA) patients over 40 years. However, direct comparison between IST+EPAG and upfront haploidentical hematopoietic stem cell transplantation (HID-HSCT) in this population remains limited. We retrospectively analyzed 123 newly diagnosed SAA patients aged 40-65 who received first-line HID-HSCT (n = 67) or IST+EPAG (n = 56) between 2016 and 2024. Hematological response, survival, and safety were compared. The HID-HSCT group demonstrated superior complete response (CR) rates at 3 months (17.9% vs. 5.4%), 6 months (44.8% vs. 17.9%), and 12 months (64.2% vs. 33.9%) (all P < 0.001). The 3-year overall survival (OS) was comparable between the HID-HSCT and IST+EPAG groups (78.0% vs. 80.3%, P = 0.662), and a landmark analysis conditional on survival to 6 months confirmed comparable subsequent 3-year OS (92.2% vs. 88.9%, P = 0.616). Meanwhile, the 3-year failure-free survival (FFS) was significantly higher in the HID-HSCT group among patients aged 40-49 (82.9% vs. 60.1%, P = 0.045), whereas no difference was observed in those aged 50-65 (P = 0.984). HID-HSCT may offer more durable benefits in selected SAA patients aged 40-49, while its advantage over IST+EPAG is not evident in patients aged 50-65.
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