title: Comparison of Haploidentical Hematopoietic Stem Cell Transplantation and Immunosuppressive Therapy for the Treatment of Acquired Severe Aplastic Anemia in Pediatric Patients.
abstract: This study aimed to compare the efficacy and safety between haploidentical hematopoietic stem cell transplantation (HHCT) and immunosuppressive therapy (IST) for the treatment of pediatric acquired severe aplastic anemia (SAA). The clinical data of 28 children with SAA treated from June 2010 to October 2014 at our hospital were retrospectively reviewed. Of these patients, 18 were treated with HHCT and 10 with IST. The median follow-up time was 23.5 months (range, 3-52 months). There was no significant difference in overall survival rate between the HHCT group and the IST group (66.7% vs. 70%, P > 0.05). Graft-versus-host disease occurred in 83.3% (15/18) of the HHCT group, including 5 cases with grade III or higher. In comparison with IST, HHCT has similar efficacy and safety profiles in the treatment of pediatric SAA.
authors.affiliation: Department of Hematology, Beijing Military General Hospital, Beijing, China.
authors.forename: Yuan
authors.initials: Y
authors.lastname: Zhang
authors.forename: Zhi
authors.initials: Z
authors.lastname: Guo
authors.forename: Xiao-Dong
authors.initials: XD
authors.lastname: Liu
authors.forename: Xue-Peng
authors.initials: XP
authors.lastname: He
authors.forename: Kai
authors.initials: K
authors.lastname: Yang
authors.forename: Peng
authors.initials: P
authors.lastname: Chen
authors.forename: Hui-Ren
authors.initials: HR
authors.lastname: Chen
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000401:mortality / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use; D016572:Cyclosporine / Q000627:therapeutic use*; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control*; D006239:Haplotypes; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007239:Infections / Q000453:epidemiology* / Q000401:mortality; D053208:Kaplan-Meier Estimate; D008297:Male; D008727:Methotrexate / Q000627:therapeutic use; D008775:Methylprednisolone / Q000627:therapeutic use*; D009173:Mycophenolic Acid / Q000627:therapeutic use; D019653:Myeloablative Agonists / Q000627:therapeutic use; D012720:Severity of Illness Index; D015996:Survival Rate; D019172:Transplantation Conditioning; D016896:Treatment Outcome; D014740:Vidarabine / Q000031:analogs & derivatives / Q000627:therapeutic use; D014916:Whole-Body Irradiation
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D019653:Myeloablative Agonists; D016572:Cyclosporine; D003520:Cyclophosphamide; D014740:Vidarabine; D009173:Mycophenolic Acid; C024352:fludarabine; D008775:Methylprednisolone; D008727:Methotrexate