摘要
目的:比较一线免疫抑制治疗(immunosuppressive therapy,IST)及造血干细胞移植(hematopoietic stem cell transplantation,HSCT)治疗肝炎相关性再生障碍性贫血(hepatitis-associated aplastic anemia,HAAA)的疗效。方法:选择2013年1月1日至2022年8月31日诊断为重度或极重度HAAA的患者41例,进行一线IST(12例)或HSCT治疗(29例),其中同胞全相合供者异基因造血干细胞移植(matched-sibling donor hematopoietic stem cell transplantation,MSD-HSCT)13例,单倍体异基因造血干细胞移植(haplo-identical donor hematopoietic stem cell transplantation,HID-HSCT)16例。结果:除死亡事件外,所有患者移植后中位随访时间为67(16~129)个月。MSD-HSCT组和HID-HSCT组患者造血重建时间、感染发生率、Ⅱ~Ⅳ度急性移植物抗宿主病、慢性移植物抗宿主病发生率差异均无统计学意义。IST组治疗失败率明显高于HSCT组(41.7%vs 10.3%,P=0.034)。整个队列显示5年总生存率(overall survival,OS)和无失败生存率(failure-free survival,FFS)分别为85.4%和80.5%。MSD-HSCT组、HID-HSCT组、IST组5年OS率分别为92.3%、87.5%和75.0%,5年FFS率分别为92.3%、87.5%和58.3%。HSCT组和IST组5年OS率差异无统计学意义;MSD-HSCT组5年FFS率较IST组明显延长(P=0.039);MSD-HSCT组和HID-HSCT组5年OS率及FFS率差异均无统计学意义。结论:HSCT较IST更适合于极重型或爆发性HAAA患者。MSD-HSCT仍是HAAA治疗的一线选择,对于没有MSD供者的年轻患者来说,HID-HSCT可以替代IST作为一线选择。
Objective To compare the clinical efficacy between frontline immunosuppressive therapy(IST)and hematopoietic stem cell transplantation(HSCT)for patients with severe hepatitis-associated aplastic anemia(HAAA).Methods A total of 41 patients who received frontline IST(12 cases)or HSCT(29 cases)between January 1,2013 and August 31,2022 were retrospectively analyzed.Among 29 patients undergoing HSCT,there were 13 cases of matched-sibling donor hematopoietic stem cell transplantation(MSD-HSCT)and 16 cases of haplo-identical donor hematopoietic stem cell transplantation(HID-HSCT).Results Except for death events,the median follow-up time for all patients after transplantation was 67(16-129)months.There was no significant difference in hematopoietic reconstruction time,the infection rate,the incidences of gradeⅡ-Ⅳacute graft-versus-host disease and chronic graft-versus-host disease between the MSD-HSCT group and the HID-HSCT group.The incidence of treatment failure in the IST group was higher than that in the HSCT group(41.7%vs 10.3%,P=0.034).The estimated 5-year overall survival(OS)and failure-free survival(FFS)were 85.4%and 80.5%for all of the 41 patients.The estimated 5-year OS in the MSD-HSCT group,HID-HSCT group and IST group were 92.3%,87.5%and 75.0%,respectively.The estimated 5-year FFS in the MSD-HSCT group,HID-HSCT group and IST group were 92.3%,87.5%and 58.3%,respectively.There was no significant difference in the estimated 5-year OS between the HSCT group and the IST group,but the estimated 5-year FFS in the MSD-HSCT group was significantly higher than that in the IST group(P=0.039).There was no significant difference in the expected 5-year OS and FFS between the MSD-HSCT group and the HID-HSCT group.Conclusion HSCT is more suitable for patients with severe HAAA than IST.MSD-HSCT remains the first-line option for HAAA,and HID-HSCT can replace IST as the first-line option for younger patients without a MSD donor.The frontline haplo-HSCT was an effective and safe approach for the treatment of patients with SAA who lack a HLA-matched sibling donor.
作者
郭欢绪
范丹
张静宜
齐美颖
严学倩
肖方
GUO Huanxu;FAN Dan;ZHANG Jingyi;QI Meiying;YAN Xueqian;XIAO Fang(Department of Hematology,the Second Affiliated Hospital of Air Force Medical University,Xi'an,710038,China)
出处
《临床血液学杂志》
CAS
2024年第5期343-348,共6页
Journal of Clinical Hematology
关键词
肝炎相关再生障碍性贫血
异基因造血干细胞移植
免疫抑制治疗
hepatitis-associated aplastic anemia
allogeneic hematopoietic stem cell transplantation
immunosuppressive therapy