title: [The outcome of thirteen patients with nonmalignant hematologic diseases treated with HLA haploidentical stem cell transplantation].
abstract: OBJECTIVE
  To evaluate the clinical efficacy and safety of human leukocyte antigen (HLA) haploidentical stem cell transplantation in nonmalignant hematologic diseases.
  
  
  METHODS
  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).
  
  
  CONCLUSIONS
  HLA haploidentical stem cell transplantation is an effective and safe therapy for nonmalignant hematologic diseases.
authors.affiliation: Department of Hematology, Zhujiang Hospital, Southern Medical University, Guangzhou 510280, China.
authors.forename: Yuan
authors.initials: Y
authors.lastname: Tao
authors.affiliation: Department of Hematology, Zhujiang Hospital, Southern Medical University, Guangzhou 510280, China. Email: wubingyi@aliyun.com.
authors.forename: Bingyi
authors.initials: B
authors.lastname: Wu
authors.affiliation: Department of Hematology, Zhujiang Hospital, Southern Medical University, Guangzhou 510280, China.
authors.forename: Qingfeng
authors.initials: Q
authors.lastname: Du
authors.affiliation: Department of Hematology, Zhujiang Hospital, Southern Medical University, Guangzhou 510280, China.
authors.forename: Can
authors.initials: C
authors.lastname: Sun
authors.affiliation: Department of Hematology, Zhujiang Hospital, Southern Medical University, Guangzhou 510280, China.
authors.forename: Xia
authors.initials: X
authors.lastname: Lin
authors.affiliation: Department of Hematology, Zhujiang Hospital, Southern Medical University, Guangzhou 510280, China.
authors.forename: Yuxian
authors.initials: Y
authors.lastname: Huang
authors.affiliation: Department of Hematology, Zhujiang Hospital, Southern Medical University, Guangzhou 510280, China.
authors.forename: Sanfang
authors.initials: S
authors.lastname: Tu
authors.affiliation: Department of Hematology, Zhujiang Hospital, Southern Medical University, Guangzhou 510280, China.
authors.forename: Chaoyang
authors.initials: C
authors.lastname: Song
authors.affiliation: Department of Hematology, Zhujiang Hospital, Southern Medical University, Guangzhou 510280, China.
authors.forename: Zhigang
authors.initials: Z
authors.lastname: Lu
authors.affiliation: Department of Hematology, Zhujiang Hospital, Southern Medical University, Guangzhou 510280, China.
authors.forename: Tuzhen
authors.initials: T
authors.lastname: Chen
authors.affiliation: Department of Hematology, Zhujiang Hospital, Southern Medical University, Guangzhou 510280, China.
authors.forename: Caixia
authors.initials: C
authors.lastname: Sun
mesh_terms: D000741:Anemia, Aplastic; D002066:Busulfan; D006086:Graft vs Host Disease; D006680:HLA Antigens; D006402:Hematologic Diseases / Q000628:therapy*; D006648:Histocompatibility; D006801:Humans; D015994:Incidence; D008727:Methotrexate; D033581:Stem Cell Transplantation; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D014740:Vidarabine / Q000031:analogs & derivatives
chemical_list: D006680:HLA Antigens; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine; D008727:Methotrexate