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Peer-reviewedCC BYAnnals of hematology

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.

  • Xiaohui Xiao1,2
  • Guiping Chen2,3
  • Cong Xu1
  • Liangliang Wu2,3
  • Xiaoyan Liu4,5
  • Ming Zhou2,3
  • Xiaowei Chen2,3
  • Ruiqing Zhou2,3
  • Wenjian Mo2,3
  • Yumiao Li2,3
  • Shilin Xu2,3
  • Yuping Zhang6,7
2 more authors
  • Dongjun Lin8
  • Shunqing Wang9,10
10 affiliations
  1. 1Department of Hematology, The Seventh Affiliated Hospital, Sun Yat-Sen University, Shenzhen, China.
  2. 2Department of Hematology, Guangzhou First People's Hospital, Institute of Blood Transfusion and Hematology, Guangzhou Medical University, Guangzhou, China.
  3. 3Department of Hematology, Guangzhou First People's Hospital, South China University of Technology, Guangzhou, China.
  4. 4Department of Hematology, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, China.
  5. 5Guangdong Provincial Key Laboratory of Malignant Tumor Epigenetics and Gene Regulation, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, China.
  6. 6Department of Hematology, Guangzhou First People's Hospital, Institute of Blood Transfusion and Hematology, Guangzhou Medical University, Guangzhou, China. [email protected].
  7. 7Department of Hematology, Guangzhou First People's Hospital, South China University of Technology, Guangzhou, China. [email protected].
  8. 8Department of Hematology, The Seventh Affiliated Hospital, Sun Yat-Sen University, Shenzhen, China. [email protected].
  9. 9Department of Hematology, Guangzhou First People's Hospital, Institute of Blood Transfusion and Hematology, Guangzhou Medical University, Guangzhou, China. [email protected].
  10. 10Department of Hematology, Guangzhou First People's Hospital, South China University of Technology, Guangzhou, China. [email protected].
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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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Cite

BibTeXRIS
BibTeX
@article{xiao2026haploidentical,
  title = {{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.}},
  author = {Xiaohui Xiao and Guiping Chen and Cong Xu and Liangliang Wu and Xiaoyan Liu and Ming Zhou and Xiaowei Chen and Ruiqing Zhou and Wenjian Mo and Yumiao Li and Shilin Xu and Yuping Zhang and Dongjun Lin and Shunqing Wang},
  journal = {Annals of hematology},
  year = {2026},
  month = sep,
  doi = {10.1007/s00277-026-07289-2},
  url = {https://europepmc.org/article/MED/42900363},
  note = {CC BY},
}
RIS
TY  - JOUR
TI  - 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.
AU  - Xiaohui Xiao
AU  - Guiping Chen
AU  - Cong Xu
AU  - Liangliang Wu
AU  - Xiaoyan Liu
AU  - Ming Zhou
AU  - Xiaowei Chen
AU  - Ruiqing Zhou
AU  - Wenjian Mo
AU  - Yumiao Li
AU  - Shilin Xu
AU  - Yuping Zhang
AU  - Dongjun Lin
AU  - Shunqing Wang
PY  - 2026
DA  - 2026/09/01
JO  - Annals of hematology
DO  - 10.1007/s00277-026-07289-2
UR  - https://europepmc.org/article/MED/42900363
AB  - 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.
SN  - 0939-5555
SN  - 1432-0584
ER  -