Objective: To evaluate the clinical efficacy and safety of human leukocyte antigen (HLA) haploidentical stem cell transplantation in nonmalignant hematologic diseases.
Method: To analyze the outcome of 13 patients with nonmalignant hematologic diseases who underwent HLA haploidentical stem cell transplantation from September 2001 to October 2013.
Results: Thirteen patients including 9 of severe aplastic anemia, 3 of severe β thalassemia, 1 of congenital pure red cell aplastic anemia underwent HLA haploidentical stem cell transplantation. Three HLA loci mismatched in 4 cases, two HLA loci mismatched in 8 cases and one HLA locus mismatched in 1 case. The conditioning regime consisted of Fludarabine (30 mg×m(-2)×d(-1)×5 d ), Busulfan(0.8 mg×kg(-1)×6h(-1)×4 d), Cyclophosphamide (60 mg×kg(-1)×d(-1)×2 d ), rabbit anti-human lymphocyte globulin ( 2.5 mg×kg(-1)×d(-1)×5 d ). To prevent from graft-versus-host disease (GVHD), cyclosporin A and short term methotrexate (MTX) were used. All patients were successfully engrafted. The incidence of grade 1-2 acute graft-versus-host disease (aGVHD) was 3/13, and that of grade 3-4 was 1/13. The cumulative incidence of total chronic GVHD (cGVHD) was 3/13. Eleven patients survived free of disease at a median follow-up period of 13 months (2-145).
Conclusion: HLA haploidentical stem cell transplantation is an effective and safe therapy for nonmalignant hematologic diseases.