首页|异基因造血干细胞移植治疗重型再生障碍性贫血的临床研究

异基因造血干细胞移植治疗重型再生障碍性贫血的临床研究

扫码查看
目的 探讨异基因造血干细胞移植(allo-HSCT)治疗重型再生障碍性贫血(SAA)/极重型再生障碍性贫血(vSAA)的疗效,并比较接受单倍体相合造血干细胞移植(HID-HSCT)与同胞全相合造血干细胞移植(MSD-HSCT)患者的生存情况。方法 回顾性分析2013年1月至2022年12月于解放军联勤保障部队第九四〇医院采用allo-HSCT治疗的66例SAA/vSAA患者的临床资料,根据供者情况分为HID-HSCT组(40例)和MSD-HSCT组(26例)。分析66例患者的造血重建、移植物抗宿主病(GVHD)、移植后并发症发生情况并比较两组患者的生存情况。结果 66例SAA/vSAA患者中,63例患者达到造血重建,造血重建率为95。45%,3例未达到造血重建的患者死于脓毒血症及消化道出血。移植后中性粒细胞植入时间为11~21 d,中位植入时间15 d,血小板植入时间为11~28 d,中位植入时间16 d。31例发生急性移植物抗宿主病(aGVHD),累积发病率为47。0%,其中Ⅰ/Ⅱ度aGVHD 23例,Ⅲ/Ⅳ度aGVHD 8例。23例发生上呼吸道感染,34例患者发生不同程度的肺部感染,4例发生皮下软组织感染,7例发生泌尿系感染,7例发生肠道感染,6例发生颅内感染,14例发生脓毒血症,14例发生EB病毒血症,20例发生巨细胞病毒血症;3例患者移植后出现重度肠道GVHD合并消化道出血,1例移植后血象持续不恢复并发脓毒血症、消化道出血死亡,1例在造血重建后发生感染继发血小板减低出现颅内出血,2例移植后由于重度感染和aGVHD继发脑出血;9例移植后出现(迟发型)出血性膀胱炎,3例发生移植后EB病毒相关淋巴增殖性疾病,3例发生血栓性微血管病,1例发生血栓性微血管病肾损伤,6例发生继发性低免疫球蛋白血症。截至2023年7月1日,共存活48例,死亡18例,5年预计总生存率为76。1%,HID-HSCT组与MSD-HSCT组5年预计总生存率分别为73。5%和80。0%,经比较差异无统计学意义(P=0。59)。结论 Allo-HSCT是治愈SAA的有效方法,其中MSD-HSCT效果好,移植相关并发症少,可作为首选移植方案,无同胞全相合供者时优先考虑单倍体相合供者作为替代治疗。
A clinical study of allogeneic hematopoietic stem cell transplantation for the treatment of severe aplastic anemia
Objective To investigate the efficacy of allogeneic hematopoietic stem cell transplantation(allo-HSCT)in the treatment of SAA/vSAA,and to compare the survival of haploidentical donor-hematopoietic stem cell transplantation(HID-HSCT)with matched sibling donor-hematopoietic stem cell transplantation(MSD-HSCT).Method The clinical data of 66 SAA/vSAA patients treated with allo-HSCT in the 940th Hospital of Joint Logistics Support Force of Chinese People's Liberation Army from January 2013 to December 2022 were retrospectively analyzed,and they were divided into 40 cases in HID-HSCT group and 26 cases in MSD-HSCT group according to the donor situation.Hematopoietic reconstruction,graft versus host disease(GVHD)and postoperative complications of 66 patients were analyzed and the survival of the two groups were compared.Result Among 66 SAA/vSAA patients,63 patients achieved hematopoietic reconstruction,the rate of hematopoietic reconstruction was 95.45%,and 3 patients who did not achieve hematopoietic reconstruction died of sepsis and gastrointestinal hemorrhage.The median implantation time of neutrophil was 15 days and the median implantation time of platelet was 16 days and 11 to 28 days after transplantation.Acute graft versus host disease(aGVHD)occurred in 31 patients,with a cumulative incidence of 47.0%,including 23 cases with grade Ⅰ/Ⅱ aGVHD and 8 cases with grade Ⅲ/Ⅳ aGVHD.There were 23 patients with upper respiratory tract infection,34 patients with different degrees of lung infection,4 patients with subcutaneous soft tissue infection,7 patients with urinary tract infection,7 patients with intestinal infection,6 patients with intracranial infection,14 patients with sepsis,14 patients with Epstein-Barr viremia,and 20 patients with cytomegaloviremia.3 patients developed severe intestinal GVHD combined with gastrointestinal hemorrhage after transplantation,1 patient died of sepsis and gastrointestinal hemorrhage after continuous non-recovery of blood image after transplantation,1 patient suffered intracranial hemorrhage after infection secondary to thrombocytopenia after hematopoietic reconstruction,and 2 patients suffered secondary cerebral hemorrhage due to severe infection and aGVHD after transplantation.9 patients developed(delayed)hemorrhagic cystitis after transplantation,3 patients developed Epstein-Barr virus associated lymphoproliferative disease after transplantation,3 patients developed thrombotic microangiopathy,1 patient developed thrombotic microangiopathy renal injury,and 6 patients developed secondary hypoimmunoglobulinemia.As of July 1,2023,48 patients were alive and 18 died,and the 5-year estimated overall survival rate was 76.1%.The 5-year estimated overall survival rate of the HID-HSCT group and the MSD-HSCT group were 73.5%and 80.0%,respectively,with no statistically significant difference(P=0.59).Conclusion Allo-HSCT is the effective way to cure severe aplastic anemia,with good sib matching effect and few transplant-related complications.As the first choice,HID-HSCT is preferred as an alternative treatment when there is no MSD-HSCT.

Severe aplastic anemiaAllogeneic hematopoietic stem cell transplantationHaploid transplantationHematopoietic reconstructionGraft versus host disease

杨小兰、潘耀柱、葸瑞、吴涛、张茜、周进茂

展开 >

宁夏医科大学临床医学院,宁夏银川 750000

解放军联勤保障部队第九四〇医院血液科,甘肃兰州 730050

重型再生障碍性贫血 异基因造血干细胞移植 单倍体移植 造血重建 移植物抗宿主病

甘肃省卫生健康行业科研计划项目西北民族大学高校项目甘肃省创新基地与人才计划项目

GSWSKY-2019-633192023019521JR7RA015

2024

中国医刊
人民卫生出版社

中国医刊

CSTPCD
影响因子:1.14
ISSN:1008-1070
年,卷(期):2024.59(8)