脐带血移植治疗儿童血液病疗效的回顾性分析

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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(c GVHD)发生率为28.57%;c GVHD患儿的长期OS为(72.7±13.4)%,无c GVHD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c GVHD是提高脐带血移植疗效的重要策略。 Objective To observe the curative effect and prognosis of umbilical cord blood in children’s hematological diseases. Methods The clinical data of 51 children undergoing cord blood transplantation from January 2011 to June 2016 were retrospectively analyzed. Results Of the 51 children, 34 were males and 17 were females, with a median age of 62 months. Thirty-two patients had hematologic malignancies and 19 had non-hematologic malignancies. Two patients died when granulocytes were not reconstructed, four had failed primary implantation, and 45 had successful implantation. The median time for granulocyte implantation was 16 days, and the median platelet engraftment time was 23 days. The incidence of periarterial implants syndrome was 46.94%. The perioperative survival rates and long-term overall survival (OS) of periarterial implanted syndrome were (73.9 ± 9.2)% and (50.2 ± 11.7)%, respectively, OS 100% implantation in children with syndrome, the difference was statistically significant (P <0.01). The incidence of acute graft-versus-host disease (aGVHD) was 55.10%, of which 28.57% were grade II-III aGVHD and 26.53% were grade IV aGVHD; OS was (61.5 ± 13.5)% within 100 days after transplantation for grade IV aGVHD; The long-term OS was (75.0 ± 21.7)% and (44.9 ± 14.1)% respectively in children with grade Ⅳ aGVHD and (90.2 ± 6.6)% in those without aGVHD, the difference was statistically significant (χ2 = 14.35, P = 0.002). The incidence of chronic GVHD (cGVHD) was 28.57%. The long-term OS of children with GVHD was (72.7 ± 13.4)%, and 100% of children without cGVHD survived. OS was 100.0 days after cord blood transplantation (86.0 ± 4.9)%; long-term OS was (77.9 ± 6.3)% with cord blood transplantation (76.6 ± 7.8%) and non-hematologic disease was (79.5 ± 11.3)%. The OS of acute lymphoblastic leukemia (ALL) was (87.5 ± 11.7)% in acute hematologic malignancies, (76.7 ± 10.3)% in acute myeloid leukemia (AML) and (33.3 ± 27.2) in myelodysplastic syndrome %. Conclusion Umbilical cord blood transplantation is an effective treatment for children with hematological diseases. Paying attention to the treatment of peri-implant syndrome, active prevention and treatment of grade Ⅲ / Ⅳ aGVHD and cGVHD are important strategies to improve the efficacy of cord blood transplantation.
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