High-dose immunosuppressive therapy combined with cord blood infusion and non-myeloablative peripheral blood stem cell transplantation for patients with severe aplastic anemia

Abstract

Objectives: The aim of this study was to compare the clinical effective rates of high-dose immunosuppressive therapy combined with cord blood infusion (IS + CBI) and non-myeloablative peripheral blood stem cell transplantation (NSCT) for severe aplastic anemia (SAA).

Patients and methods: Human leukocyte antigen (HLA)-mismatched patients received immunosuppressive therapy combined with IS + CBI, whereas those with HLA matches received NSCT. Clinical effective rates, hematopoietic recovery, and prevalence of complications were compared between the two groups.

Results: No significant difference in total effective rate or 2 years long-term survival was observed between the two groups. The total effective treatment in the NSCT, IS + CBI group was 80%, 68.75%, and the 2 years long-term survival rate in two groups was 2 years 76.66%, 68.75%, respectively. The median times of WBC > 1.0×109/L in the NSCT group was faster than that of IS + CBI group (13 vs 19 days) (p = 0.027). The median recovery times of PLT and Hb in the NSCT group was significantly faster than that of IS + CBI group (19 vs 50 days) (p = 0.00), (27 vs 57 days) (p = 0.001). The SAA group and the very SAA (VSAA) group did not show a significant difference in effective rate (76.74% vs 68.42%) (p = 0.490). In the NSCT group, two preparative regimens did not show a significant difference in effect (70.59% vs 92.31%) (p = 0.141).

Conclusions: IS + CBI is applicable to HLA-mismatched patients with SAA. NSCT is the treatment of choice for patients with HLA matching alleles. Both treatment methods are effective on VSAA.