title: Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia.
abstract: Techniques for haploidentical hematopoietic stem cell transplantation (haplo-HSCT) to treat severe aplastic anemia (SAA) have recently improved, but no protocol has been evaluated in a large number of pediatric patients. Fifty-two children with SAA received haplo-HSCT in our center. The treatment protocol used G-CSF-primed bone marrow with G-CSF-mobilized PBSCs without in vitro T-cell depletion. The conditioning regimen included busulfan/cyclophosphamide and antithymocyte globulin. Fifty-one patients achieved primary engraftment; one child died of regimen-related toxicity on the day +1. Secondary graft failure occurred in three patients. The cumulative incidences of aGVHD grade II-IV and grade III-IV were 39.2±0.5 and 13.7±0.2%, respectively. The cumulative incidence of cGVHD was 34.2±0.5%. The 3-year overall and failure-free survival rates were 84.5±5.0 and 82.7±5.2%, respectively, with a median follow-up time of 744.5 days (100-3294) for surviving patients. The Eastern Cooperative Oncology Group score was the only predictor of overall and failure-free survival rates. Clinical outcomes were similar between the upfront and salvage group. This result suggests that both newly diagnosed and refractory pediatric SAA patients benefit from haplo-HSCT, especially when patients are in good general condition. Therefore, haplo-HSCT might be an alternative therapy for pediatric SAA patients without HLA-matched sibling donors.
authors.affiliation: Hematology Department, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.
authors.forename: L P
authors.initials: LP
authors.lastname: Xu
authors.affiliation: Hematology Department, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.
authors.forename: X H
authors.initials: XH
authors.lastname: Zhang
authors.affiliation: Hematology Department, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.
authors.forename: F R
authors.initials: FR
authors.lastname: Wang
authors.affiliation: Hematology Department, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.
authors.forename: X D
authors.initials: XD
authors.lastname: Mo
authors.affiliation: Hematology Department, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.
authors.forename: T T
authors.initials: TT
authors.lastname: Han
authors.affiliation: Hematology Department, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.
authors.forename: W
authors.initials: W
authors.lastname: Han
authors.affiliation: Hematology Department, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.
authors.forename: Y H
authors.initials: YH
authors.lastname: Chen
authors.affiliation: Hematology Department, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.
authors.forename: Y Y
authors.initials: YY
authors.lastname: Zhang
authors.affiliation: Hematology Department, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.
authors.forename: J Z
authors.initials: JZ
authors.lastname: Wang
authors.affiliation: Hematology Department, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.
authors.forename: C H
authors.initials: CH
authors.lastname: Yan
authors.affiliation: Hematology Department, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.
authors.forename: Y Q
authors.initials: YQ
authors.lastname: Sun
authors.affiliation: Hematology Department, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.
authors.forename: S N
authors.initials: SN
authors.lastname: Zuo
authors.affiliation: Hematology Department, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.|Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.|Peking-Tsinghua Center for Life Sciences, Beijing, China.
authors.forename: X J
authors.initials: XJ
authors.lastname: Huang
mesh_terms: D000293:Adolescent; D064591:Allografts; D000741:Anemia, Aplastic / Q000401:mortality* / Q000628:therapy*; D002066:Busulfan / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage; D018572:Disease-Free Survival; D005260:Female; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage; D019650:Hematopoietic Stem Cell Mobilization; D006801:Humans; D007223:Infant; D008297:Male; D036102:Peripheral Blood Stem Cell Transplantation; D015996:Survival Rate; D014019:Tissue Donors; D019172:Transplantation Conditioning
chemical_list: D016179:Granulocyte Colony-Stimulating Factor; D003520:Cyclophosphamide; D002066:Busulfan