免疫抑制联合艾曲泊帕治疗儿童重型再生障碍性贫血的疗效

The efficacy of immunosuppression combined with Eltrombopag in the treatment of severe aplastic anemia in children

目的探讨艾曲泊帕联合免疫抑制治疗(IST)儿童重型再生障碍性贫血(SAA)的疗效和临床意义。方法回顾性分析2017年5月至2019年6月在新乡市中心医院血液科初诊为SAA的63例患儿的临床资料,均为无同胞全合供者,按照治疗方案不同分为观察组和对照组,观察组31例接受IST联合艾曲泊帕治疗;对照组32例接受IST治疗。采用χ^(2)检验比较2组患儿感染、显著出血的发生率及治疗3个月、6个月、12个月的总缓解(OR)率、完全缓解(CR)率;采用t检验比较2组患儿粒细胞集落刺激因子应用量、红细胞平均输注量、血小板平均输注量;2组生存率和生存分析采用Kaplan-Meier法,应用Log-rank检验比较2组患儿2年总生存(OS)率及无失败生存(FFS)率。结果观察组3个月、6个月的OR率分别为61.29%(19/31例)、80.64%(25/31例),均高于对照组的37.50%(12/32例)、59.38%(19/32例),差异均有统计学意义(χ^(2)=45.27、43.81,均P<0.05);观察组3个月、6个月CR率分别为32.26%(10/31例)、45.16%(14/31例),均高于对照组的15.62%(5/32例)、28.13%(9/32例),差异均有统计学意义(χ^(2)=47.02、48.35,均P<0.05);观察组12个月的OR率为83.87%(26/31例)、CR率为64.52%(20/31例),与对照组12个月的OR率81.25%(26/32例)、CR率59.38%(19/32例)比较,差异均无统计学意义(均P>0.05)。观察组患儿治疗6个月内粒细胞集落刺激因子的应用总量、红细胞和血小板平均输注量均明显少于对照组[(13.58±4.28)支比(23.24±6.68)支、(5.48±1.67)U比(10.58±3.67)U、(4.15±2.47)袋比(9.15±3.87)袋],差异均有统计学意义(t=2.591、2.040、2.744,均P<0.05);观察组患儿感染率、显著出血率较对照组均明显减低(16.13%比43.75%、16.13%比37.50%),差异均有统计学意义(χ^(2)=47.12、44.52,均P<0.05);观察组与对照组的2年OS率分别为93.55%(29/31例)、87.50%(28/32),差异无统计学意义(P=0.4073);观察组与对照组2年FFS率分别为87.10%、78.13%,差异无统计学意义(P=0.3266)。结论对于无同胞全相合供者的SAA患儿,IST联合艾曲泊帕治疗可明显提高SAA患儿的早期治疗反应率,并能减少红细胞、血小板输注量,降低感染、出血的发生率。

Objective To explore the efficacy and clinical significance of Eltrombopag combined with immunosuppression therapy(IST)in the treatment of severe aplastic anemia(SAA)in children.Methods Clinical data of 63 children with initially diagnosed SAA in the Department of Hematology of Xinxiang Central Hospital from May 2017 to June 2019 were retrospectively analyzed.All of them were all donors without siblings and they were classified into observation group(31 cases)and control group(32 cases).Patients in the observation group received IST combined with Eltrombopag treatment,and those in the control group received IST treatment.The Chi-square test was used to compare the overall remission(OR)rate and complete remission(CR)rate at 3 months,6 months and 12 months,and incidence of infection and significant bleeding between groups.The t-test was used to compare the application of gra-nulocyte colony stimulating factor,the mean red blood cell transfusion,and the mean platelet infusion volume between groups.Kaplan-Meier method was adopted to analyze the 2-year overall survival(OS)rate and failure-free survival(FFS)rate,followed by the Log-rank test.Results The 3-month and 6-month OR rate of the observation group were 61.29%(19/31 cases)and 80.64%(25/31 cases),respectively,which was significantly higher than that of the control group[37.50%(12/32 cases)and 59.38%(19/32 cases),χ^(2)=45.27,43.81,respectively,all P<0.05].The 3-month and 6-month CR rate of the observation group were 32.26%(10/31 cases)and 45.16%(14/31 cases),respectively,which was significantly higher than that of the control group[15.62%(5/32 cases)and 28.13%(9/32 cases),χ^(2)=47.02,48.35,respectively,all P<0.05].The 12-month OR rate and CR rate in the observation group were 83.87%(26/31 cases),and 64.52%(20/31 cases),respectively,which were 81.25%(26/32 cases),and 59.38%(19/32 cases),respectively in the control group,and no significant differences in them were detected between the two groups(all P>0.05).The total amount of granulocyte colony stimulating factors[(13.58±4.28)doses vs.(23.24±6.68)doses,t=2.591],and the mean infusion volume of red blood cells[(5.48±1.67)U vs.(10.58±3.67)U,t=2.040]and platelets(4.15±2.47)bags vs.(9.15±3.87)bags,t=2.744)used in observation group within 6 months of treatment were significantly lower than those of the control group(all P<0.05).The rate of infection(16.13%vs.43.75%,χ^(2)=47.12)and significant bleeding(16.13%vs.37.50%,χ^(2)=44.52)in the observation group were significantly lower than those of the control group(all P<0.05).The 2-year OS rate of the observation group and control group were 93.55%(29/31 cases),and 87.50%(28/32 cases),respectively.No significant difference in the OS rate was found between groups(P=0.4073),nor as the 2-year FFS rate(87.10%vs.78.13%,P=0.3266).Conclusions IST combined with Eltrombopag can significantly improve the early treatment response rate of SAA children without a sibling identical donor,which can reduce red blood cell and platelet transfusion,and the incidence of infection and bleeding.