ArticleFrontiers in immunology2026
Comparative prognostic analysis of allogeneic hematopoietic stem cell transplantation regimens in the treatment of severe aplastic anemia.
Article in Frontiers in immunology, 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: 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.
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