Skip to content
DraconiticResearch
Search Sources API
190,869 studies31 sourcesUpdated 35 min ago
Peer-reviewedCC BY 4.0Frontiers in Immunology

Comparative prognostic analysis of allogeneic hematopoietic stem cell transplantation regimens in the treatment of severe aplastic anemia

  • Ting Lin1
  • Xianlin Chen1
  • Xiaohong Yuan1
  • Zhijuan Zhu1
  • Nainong Li1,2
  • Xiaofan Li1,2
  1. 1Fujian Institute of Hematology, Fujian Provincial Key Laboratory on Hematology,FUjian Medical University Union Hospital
  2. 2Translational Medicine Center on Hematology, Fujian Medical University
Read full textPDFDOICite

Abstract

Background: Severe aplastic anemia (SAA) is a fatal bone marrow failure disorder. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the definitive curative treatment for SAA. This study evaluated the prognosis of patients with severe aplastic anemia (SAA) receiving different allo-HSCT regimens.

Methods: A retrospective analysis was performed on 62 patients with aplastic anemia who underwent allo-HSCT between January 2015 and August 2024. The cohort comprised 32 males and 30 females, with a median age of 12 years (range, 1.5–44 years) at transplantation; 28 patients were ≥14 years old and 34 were <14 years. Forty-eight patients were diagnosed with severe AA (SAA) and 14 with very severe AA (VSAA). Twenty-two patients underwent HLA-matched sibling donor transplantation(MSD-HSCT), and 40 underwent HLA-haploidentical sibling donor transplantation(Haplo-HSCT).Three conditioning regimens were administered: cyclophosphamide plus anti-thymocyte globulin (CY+ATG, CA, n=11), fludarabine + cyclophosphamide + ATG (Flu+CY+ATG, FCA, n=26), and fludarabine + melphalan + ATG (Flu+Mel+ATG, FMA, n=25).

Results: The median follow-up duration of the 62 patients was 38 months (range: 0–136 months). Twelve deaths occurred during follow-up. Post-transplant complications included post-transplant lymphoproliferative disorder (PTLD) in 3 patients (4.84%), acute graft-versus-host disease (aGVHD) in 7 patients (11.29%), chronic GVHD (cGVHD) in 2 patients (3.23%), Epstein-Barr virus (EBV) viremia in 10 patients (16.13%), and invasive fungal disease in 1 patient (1.62%). The median time to neutrophil engraftment was 13 days (evaluable in 55 patients). Kaplan-Meier survival analysis revealed no statistically significant difference in 5-year overall survival (OS) between MSD-HSCT and Haplo-HSCT groups (P = 0.582). The 5-year OS rate was 76.8% for the MSD-HSCT (n=22) and 80.6% for the Haplo-HSCT group (n=40). No significant intergroup difference in 5-year OS was detected across the three conditioning regimens (P = 0.85); the 5-year OS rates of the CA, FCA, and FMA groups were 81.6%, 76.8%, and 84.0%, respectively.

Conclusions: (1)MSD-HSCT and Haplo-HSCT yield comparable 5-year OS. Haploidentical related donors serve as a reliable alternative when fully matched donors are unavailable. (2) No statistically significant difference in 5-year overall survival (OS) was observed among the CA, FCA, and FMA conditioning regimens. (3) the FMA regimen may be considered as a favorable alternative to avoid the cyclophosphamide related toxicities.

Related

  1. Longitudinal plasma proteome profiling after allogeneic hematopoietic stem cell transplantation: heterogeneous development and associations with acute GvHD
    Iris C. Kreft, Myrddin W. Verheij, Maartje van den Biggelaar +8
    Frontiers in immunologyPeer-reviewedCC BYFull textImmunologyEurope PMC
  2. Case Report: Fulminant aggressive NK-cell leukemia with hemophagocytic lymphohistiocytosis and multiple organ dysfunction successfully bridged to allogeneic hematopoietic stem cell transplantation
    Shutan Li, Chao Fang
    Frontiers in ImmunologyPeer-reviewedCC BY 4.0Full textImmunologyCrossref
  3. Allogeneic hematopoietic cell transplantation with reduced-toxicity conditioning for X-linked hyper-IgM syndrome
    Ryosuke Wakatsuki, Satoshi Miyamoto, Akihiro Hoshino +14
    International Journal of HematologyPeer-reviewedCC BY 4.0Full textImmunologyPMC
  4. Indications for chimerism analysis on sorted cell populations after allogeneic hematopoietic stem cell transplantation (SFGM-TC)
    Debora Jorge-Cordeiro, Marine Cargou, Roberto Crocchiolo +15
    Bulletin du CancerPeer-reviewedCC BYGeneral biologyGeneral medicineHAL
  5. T-cell polyfunctionality reconstitution after modified anti-thymocyte globulin plus post-transplant cyclophosphamide in allogeneic hematopoietic stem cell transplantation
    Cen Jiang, Luxiang Wang, Lilan Jin +4
    Frontiers in ImmunologyPeer-reviewedCC BY 4.0ImmunologyFrontiersCrossref

Cite

BibTeXRIS
BibTeX
@article{lin2026comparative,
  title = {{Comparative prognostic analysis of allogeneic hematopoietic stem cell transplantation regimens in the treatment of severe aplastic anemia}},
  author = {Ting Lin and Xianlin Chen and Xiaohong Yuan and Zhijuan Zhu and Nainong Li and Xiaofan Li},
  journal = {Frontiers in Immunology},
  year = {2026},
  month = oct,
  doi = {10.3389/fimmu.2026.1936533},
  url = {https://www.frontiersin.org/articles/10.3389/fimmu.2026.1936533/full},
  note = {CC BY 4.0},
}
RIS
TY  - JOUR
TI  - Comparative prognostic analysis of allogeneic hematopoietic stem cell transplantation regimens in the treatment of severe aplastic anemia
AU  - Ting Lin
AU  - Xianlin Chen
AU  - Xiaohong Yuan
AU  - Zhijuan Zhu
AU  - Nainong Li
AU  - Xiaofan Li
PY  - 2026
DA  - 2026/10/05
JO  - Frontiers in Immunology
DO  - 10.3389/fimmu.2026.1936533
UR  - https://www.frontiersin.org/articles/10.3389/fimmu.2026.1936533/full
AB  - Background: Severe aplastic anemia (SAA) is a fatal bone marrow failure disorder. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the definitive curative treatment for SAA. This study evaluated the prognosis of patients with severe aplastic anemia (SAA) receiving different allo-HSCT regimens.  Methods: A retrospective analysis was performed on 62 patients with aplastic anemia who underwent allo-HSCT between January 2015 and August 2024. The cohort comprised 32 males and 30 females, with a median age of 12 years (range, 1.5–44 years) at transplantation; 28 patients were ≥14 years old and 34 were <14 years. Forty-eight patients were diagnosed with severe AA (SAA) and 14 with very severe AA (VSAA). Twenty-two patients underwent HLA-matched sibling donor transplantation(MSD-HSCT), and 40 underwent HLA-haploidentical sibling donor transplantation(Haplo-HSCT).Three conditioning regimens were administered: cyclophosphamide plus anti-thymocyte globulin (CY+ATG, CA, n=11), fludarabine + cyclophosphamide + ATG (Flu+CY+ATG, FCA, n=26), and fludarabine + melphalan + ATG (Flu+Mel+ATG, FMA, n=25).  Results: The median follow-up duration of the 62 patients was 38 months (range: 0–136 months). Twelve deaths occurred during follow-up. Post-transplant complications included post-transplant lymphoproliferative disorder (PTLD) in 3 patients (4.84%), acute graft-versus-host disease (aGVHD) in 7 patients (11.29%), chronic GVHD (cGVHD) in 2 patients (3.23%), Epstein-Barr virus (EBV) viremia in 10 patients (16.13%), and invasive fungal disease in 1 patient (1.62%). The median time to neutrophil engraftment was 13 days (evaluable in 55 patients). Kaplan-Meier survival analysis revealed no statistically significant difference in 5-year overall survival (OS) between MSD-HSCT and Haplo-HSCT groups (P = 0.582). The 5-year OS rate was 76.8% for the MSD-HSCT (n=22) and 80.6% for the Haplo-HSCT group (n=40). No significant intergroup difference in 5-year OS was detected across the three conditioning regimens (P = 0.85); the 5-year OS rates of the CA, FCA, and FMA groups were 81.6%, 76.8%, and 84.0%, respectively.  Conclusions: (1)MSD-HSCT and Haplo-HSCT yield comparable 5-year OS. Haploidentical related donors serve as a reliable alternative when fully matched donors are unavailable. (2) No statistically significant difference in 5-year overall survival (OS) was observed among the CA, FCA, and FMA conditioning regimens. (3) the FMA regimen may be considered as a favorable alternative to avoid the cyclophosphamide related toxicities.
SN  - 1664-3224
ER  -