title: [A comparative study of unrelated donor and matched-sibling donor allogeneic hematopoietic stem cell transplantation in children and adolescents with acquired severe aplastic anemia].
abstract: Objective:To evaluate the efficacy of unrelated donor allogeneic hematopoietic stem cell transplantation(URD allo-HSCT) for children and adolescents with severe aplastic anemia (SAA).Methods:Clinical data of 34 SAA children and adolescents undergoing allo-HSCT were retrospectively analyzed from October 2001 to October 2015. According to the source of donor, the patients were divided into matched sibling donor allo-HSCT group (MSD group) and unrelated donor group (URD group). The clinical outcome of SAA children and adolescents receiving URD allo-HSCT was assessed, and patients in MSD allo-HSCT group were enrolled as control at the same period.Results:The rate of hematopoietic reconstitution, the time of neutrophil and platelet engraftment, incidence of chimerism and graft rejection between two groups were not statistically different.The incidence of acute graft-versus-host disease (GVHD) in URD group was significantly higher than that in MSD group [42.9%(6/14) vs 10.5%(2/19),P=0.047]. The incidence of grade Ⅱ-Ⅳ acute GVHD and chronic GVHD in URD were higher than those in MSD group [21.4%(3/14) vs 5.3%(1/19),P=0.288; 35.7%(5/14) vs 5.3%(1/19),P=0.062, respectively], yet without significant difference between two groups. Other transplant-related complications including pulmonary complications, hemorrhagic cystitis, incidence of EBV and CMV reactivation and venous occlusive disease were comparable with two regimens. Estimated 5-years overall survival (OS) rate and disease free survival (DFS) rate were not statistically significant between URD group and MSD group [(84.4±6.6)% vs (89.4±7.1)%, (82.5±5.4)% vs (82.1±4.3)%;P=0.766,P=0.884, respectively].Conclusions:By multivariate analysis, the outcome of URD allo-HSCT in SAA children and adolescent is similar to MSD allo-HSCT. It could be an alternative option as the first-line treatment for SAA children and adolescents without HLA matched sibling donors.
authors.affiliation: Department of Hematology, the Affiliated Cancer Hospital of Zhengzhou University, Henan Cancer Hospital, Zhengzhou 450003, China.
authors.forename: J
authors.initials: J
authors.lastname: Zhou
authors.forename: Y W
authors.initials: YW
authors.lastname: Fu
authors.forename: L J
authors.initials: LJ
authors.lastname: Liang
authors.forename: Q
authors.initials: Q
authors.lastname: Wang
authors.forename: L J
authors.initials: LJ
authors.lastname: Han
authors.forename: Y L
authors.initials: YL
authors.lastname: Zu
authors.forename: Yanli
authors.initials: Y
authors.lastname: Zhang
authors.forename: X H
authors.initials: XH
authors.lastname: Zhu
authors.forename: F K
authors.initials: FK
authors.lastname: Yu
authors.forename: B J
authors.initials: BJ
authors.lastname: Fang
authors.forename: X D
authors.initials: XD
authors.lastname: Wei
authors.forename: Y P
authors.initials: YP
authors.lastname: Song
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D002648:Child; D046528:Chimerism; D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000453:epidemiology / Q000401:mortality; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects* / Q000401:mortality; D006650:Histocompatibility Testing; D006801:Humans; D015994:Incidence; D008297:Male; D012189:Retrospective Studies; D012720:Severity of Illness Index; D035781:Siblings; D014019:Tissue Donors; D014184:Transplantation, Homologous / Q000009:adverse effects; D016896:Treatment Outcome; D061349:Unrelated Donors