临床儿科杂志 ›› 2017, Vol. 35 ›› Issue (10): 769-.doi: 10.3969/j.issn.1000-3606.2017.10.013

• 综合报道 • 上一篇    下一篇

脐带血移植治疗儿童血液病疗效的回顾性分析

刘虎, 肖佩芳, 卢俊, 姚艳华, 李捷, 凌婧, 翟宗, 胡昳歆, 万琳, 卞馨妮, 胡绍燕   

  1. 苏州大学附属儿童医院血液科(江苏苏州 215025)
  • 收稿日期:2017-10-15 出版日期:2017-10-15 发布日期:2017-10-15
  • 通讯作者: 胡绍燕 E-mail:hsy139@126.com
  • 基金资助:
    江苏省自然基金课题(No.BL2013014、BL20140286);苏州市临床重点专病(No.LCZX201507) ;苏州市小儿内科临 床医学中心(No.SZZX201504);苏州市儿童白血病重点实验室(No.SZSL201615)

Retrospective analysis of curative effect of umbilical cord blood transplantation on children with hematologic diseases

 LIU Hu, XIAO Peifang, LU Jun, YAO Yanhua, LI Jie, LING Jing, ZHAI Zong, HU Yixin, WAN Lin, BIAN Xinni, HU Shaoyan   

  1. Department of Hematology, Children’s Hospital of Soochow University, Suzhou 215025, Jiangsu, China
  • Received:2017-10-15 Online:2017-10-15 Published:2017-10-15

摘要:  目的 观察脐带血治疗儿童血液病的治疗效果及预后。方法 回顾分析2011年1月至2016年6月期间51例 接受脐带血移植患儿的临床资料。结果 51例患儿中,男34例、女17例,中位年龄62个月;恶性血液病32例,非恶性血液 病19例。 2例在粒细胞未重建时死亡, 4例发生原发性植入失败,45例植入成功。粒细胞植入中位时间16天,血小板植入中 位时间23天。围植入综合征发生率46.94%,围植入综合征患儿移植后100 天内以及长期总生存率(OS)分别为(73.9±9.2) % 和(50.2±11.7) %,均低于无围植入综合征患儿的OS 100%,差异有统计学意义(P<0.01)。急性移植物抗宿主病(aGVHD) 发生率为55.10%,其中II~III度aGVHD占28.57%,IV度aGVHD 26.53%;IV度aGVHD患儿移植100天内OS为(61.5±13.5) %; III和IV度aGVHD患儿的长期OS分别为(75.0±21.7) %和(44.9±14.1) %,未发生aGVHD患儿的长期OS为(90.2±6.6) %, 差异有统计学意义(χ2=14.35, P=0.002)。慢性GVHD(cGVHD)发生率为28.57%;cGVHD患儿的长期OS为(72.7±13.4) %, 无cGVHD患儿100%存活。脐血移植后100天内OS(86.0±4.9)%;脐血移植长期OS(77.9±6.3)%,其中恶性血液病 为(76.6±7.8)%,非恶性血液病为(79.5±11.3)%。恶性血液病中急性淋巴细胞白血病(ALL)的OS为(87.5±11.7)%, 急性髓细胞白血病(AML)为(76.7±10.3)%,骨髓增生异常综合征(MDS)为(33.3±27.2)%。结论 脐带血移植是治 疗儿童血液病的有效手段,重视围植入综合征的处理,积极防治III/IV度aGVHD和cGVHD是提高脐带血移植疗效的重要 策略。

Abstract: Objective To explore the curative effect and prognosis of umbilical cord blood in the treatment of hematological diseases in children. Method The clinical data of 51 children who underwent umbilical cord blood transplantation from January 2011 to June 2016 were analyzed retrospectively. Results In 51 children (34 males and 17 females) with median age of 62 months, 32 children had malignant hematologic diseases and 19 children had nonmalignant hematologic diseases. Two children died before the granulocytes were reconstructed, 4 children had primary implantation failure, and 45 children had successfully implantation. The median time of implantation was 16 d, and the median time of platelet implantation was 23 d. The incidence of peri-implantation syndrome was 46.94%. The 100 day survival rate and long-term overall survival (OS) in children with peri-implantation syndrome were (73.9±9.2)% and (50.2±11.7)% respectively, which were significantly lower than the OS (100%) in children without peri-implantation syndrome (P<0.01). The incidence of acute graft versus host disease (aGVHD) was 55.10%, among which Ⅱ-Ⅲ degrees of aGVHD was 28.57% and Ⅳ degrees of aGVHD was 26.53%. The 100 day OS in children with Ⅳ degrees of aGVHD was (61.5±13.5)%, and The OS in children with Ⅲ and Ⅳ degrees of aGVHD were (75.0±21.7)% and (44.9±14.1)% respectively, and the OS in children without aGVHD was (90.2±6.6)%. The difference was statistically significant (χ2=14.35, P=0.002). The incidence of chronic GVHD (cGVHD) was 28.57%. The long-term OS in children with cGVHD was (72.7±13.4)%, while OS in children without cGVHD was 100%. The 100 days OS was (86.0±4.9)%. Long-term OS in cord blood transplantation was (77.9±6.3)%, among which OS for malignant hematological diseases was (76.6±7.8)% and OS for nonmalignant hematological diseases was (79.5±11.3)%. Among malignant hematological diseases, the OS in acute lymphoblastic leukemia (ALL) was (87.5±11.7)%, OS in acute myeloid lymphocytic leukemia (AML) was (76.7±10.3)%, and OS in myelodysplastic syndrome (MDS) was (33.3±27.2)%. Conclusions Umbilical cord blood transplantation is an effective treatment for hematologic diseases in children. It is important to treat the peri-implantation syndrome. Prevention and treatment Ⅲ/Ⅳ degree of aGVHD and cGVHD are important strategies to improve the efficacy of umbilical cord blood transplantation.