Clinical analysis of haplotype allogeneic hematopoietic stem cell transplantation for children with severe aplastic anemia

Objective To explore the efficacy and safety of haplotype allogeneic hematopoietic stem cell transplantation (allo-HSCT) in treatment of childhood severe aplastic anemia(SAA). Methods From Jan.2010 to Jan.2013, 16 children with SAA who received haploidentical allo-HSCT were studied, including 10 male and 6 female, aged from 3 to 13 years old, and the mean age was 7.8 years.The median time from diagnosis to transplantation was 15.5 months (1 to 80 months). Before transplantation, all patients received Cyclosporin A(CSA) therapy, and 10 of them received Anti-Thymocyte Globulin(ATG) intensive immune therapy.Donors received granulocyte colony-stimulating factor (G-CSF) mobilization, and stem cell transplantation were collected from both peripheral blood and bone marrow.Cyclophosphamide(CTX)+ Fludarabine(FLU)+ ATG program was used as conditioning regimen, and combined immunosuppressive agents were used for graft-versus-host disease(GVHD)prophylaxis, including CSA, Amethopterin (MTX), tacrolimus (FK506), etc.Toxic and side effect, GVHD and disease-free survival of these children after transplantation were observed. Results Fifteen cases of children reached hematopoietic reconstitution, one patient had no evidence of engraftment, one showed rejection after implantation, for the 14 engrafted children with neutrophils≥0.5×109/L and platelets≥20×109/L, the average time was 18.5 days and 24.6 days, respectively.Implantation was confirmed by the evidence of 100% of donor hematopoiesis.With a median follow-up duration of 24.8 months (3-45 months), 6 cases developed acute GVHD, 3 cases showed chronic GVHD, 1 died of GVHD and 2 died of infection, the other 11 patients remained in disease-free survival, and the disease-free survival rate was 68.8% (11/16 cases). Conclusions Haplotype allo-HSCT were safe and very effective in treatment of childhood SAA, and can be widely carried out in clinical treatment. Key words: Haplotype; Allogeneic hematopoietic stem cell transplantation; Severe aplastic anemia; Graft-versus-host disease; Child