Authors
Xiaohui Xiao, Guiping Chen, Cong Xu, Liangliang Wu, Xiaoyan Liu, Ming Zhou, Xiaowei Chen, Ruiqing Zhou, Wenjian Mo, Yumiao Li, Shilin Xu, Yuping Zhang, Dongjun Lin, Shunqing Wang
Published in
Annals of hematology. Volume 105. Issue 11. Sep 29, 2026. Epub Sep 29, 2026.
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.
PMID:
42900363
Bibliographic data and abstract were imported from PubMed on 21 Oct 2026.
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