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Comparative Study
. 2018 Mar 14;39(3):184-189.
doi: 10.3760/cma.j.issn.0253-2727.2018.03.003.

[Comparison of different styles of allogeneic hematopoietic stem cell transplantation as first-line treatment treated with severe aplastic anemia in children and adolescents]

[Article in Chinese]
S W Yang  1 , R J Ma, J J Zhao, H F Zhong, X L Yuan, L Jiang, J Yang, P C Lei, Y Zhang, Y W Fu, D M Wan, Z M Zhu
Affiliations
Comparative Study

[Comparison of different styles of allogeneic hematopoietic stem cell transplantation as first-line treatment treated with severe aplastic anemia in children and adolescents]

[Article in Chinese]
S W Yang et al. Zhonghua Xue Ye Xue Za Zhi. .

Abstract
in English, Chinese

Objective: To evaluate the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) from different donors as first-line treatment for children and adolescents with severe aplastic anemia (SAA) . Methods: The clinical data of 79 children and adolescents with SAA diagnosed from January 2013 to December 2016 in Henan Province were retrospectively analyzed. There were 50 males and 29 females, with a median age of 14(4-18) years. 40 cases received matched sibling transplantation (MSD-HSCT), 17 with unrelated donor transplantation (UD-HSCT), and 22 with haploidentical transplantation (haplo-HSCT). Results: The comparison of MSD-HSCT, UD-HSCT, haplo-HSCT groups was conducted and the median times of neutrophils engraftment were statistically significant [12(9-25) d, 14(10-22) d, 16(11-26) d, respectively (χ2=13.302, P=0.001)], but no difference in+30 d engraftment rate [97.3%(36/37), 100%(15/15), 100%(20/20), χ2=0.959, P=0.619]. The median times of PLT engraftment were not statistically significant [14(6-34)d, 16(7-32)d, 19(10-34)d, respectively, χ2=5.892, P=0.053], and the +30 d engraftment rate had no difference [97.3%(36/37), 100%(15/15), 100%(20/20), χ2=0.959, P=0.619]. The post-transplant infection rate showed no statistically significance [35.0% (14/40), 29.4% (5/17), 45.5% (10/22), χ2=1.158, P=0.560], as well as the incidences of aGVHD, grade III/IV aGVHD and cGVHD(χ2=0.230, P=0.891; χ2=2.628, P=0.269; χ2=3.187, P=0.203). The two-years OS rate was not statistically significant respectively [(77.1±6.7)%, (70.6±11.1)%, (77.3±8.9)%, χ2=0.330, P=0.845]. Severe post-transplant infection (RR=4.617, P=0.009), grade Ⅲ/Ⅳ aGVHD (RR=2.707, P=0.048) were independent risk factors for OS. Conclusion: The overall efficacy of MSD-HSCT, UD-HSCT and haplo-HSCT as first-line therapy for children and adolescents with SAA/VSAA is comparable.

目的： 评估不同供者来源的异基因造血干细胞移植一线治疗儿童及青少年重型再生障碍性贫血（SAA）的疗效。 方法： 回顾性分析2013年1月至2016年12月河南地区79例儿童及青少年SAA患者的临床资料，其中男50例，女29例，中位年龄14（4~18）岁。同胞相合造血干细胞移植（MSD-HSCT）40例、无关供者造血干细胞移植（UD-HSCT）17例，单倍体相合造血干细胞移植（haplo-HSCT）22例。 结果： MSD-HSCT、UD-HSCT、haplo-HSCT三组比较：中性粒细胞植入中位时间差异有统计学意义[分别为12（9~25）、14（10~22）、16（11~26）d，χ2=13.302，P=0.001]；+30 d中性粒细胞植入率差异无统计学意义[分别为97.3%（36/37）、100%（15/15）、100%（20/20），χ2=0.959，P=0.619]；血小板中位植入时间差异无统计学意义[分别为14（6~34）、16（7~32）、19（10~34）d，χ2=5.892，P=0.053]，+30 d血小板植入率差异无统计学意义[分别为97.3%（36/37）、100%（15/15）、100%（20/20），χ2=0.959，P=0.619]；移植后感染发生率差异无统计学意义[分别为35.0%（14/40）、29.4%（5/17）、45.5%（10/22），χ2=1.158，P=0.560]；急性移植物抗宿主病（GVHD）、Ⅲ/Ⅳ度急性GVHD及慢性GVHD发生率差异均无统计学意义（χ2=0.230，P=0.891；χ2=2.628，P=0.269；χ2=3.187，P=0.203）；2年总生存率差异无统计学意义[分别为（77.1±6.7）%、（70.6±11.1）%、（77.3±8.9）%，χ2=0.330，P=0.845]。多因素分析显示移植后重度感染、Ⅲ/Ⅳ度急性GVHD是影响总生存率的独立危险因素（RR=4.617，P=0.009；RR=2.707，P=0.048）。 结论： MSD-HSCT、UD-HSCT、haplo-HSCT一线治疗儿童及青少年SAA的总体疗效相当。.

Keywords: Anemia, aplastic; Haploidentical transplantation; Survival analysis.

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