外周血干细胞移植治疗重型地中海贫血的相关并发症及死亡原因分析  

Complications and death causes of peripheral blood stem cell transplantation in the treatment of thalassemia major

作　　者：黄楚雯 江华 李敏清 Huang Chuwen;Jiang Hua;Li Minqing(Department of Hematology and Oncology,Guangzhou Women and Children Medical Center,Guangzhou 510623,Guangdong Province,China;Department of Preventive Health of Ministry of Medical Affairs,Guangzhou Women and Children Medical Center,Guangzhou 510623,Guangdong Province,China)

机构地区：[1]广州市妇女儿童医疗中心血液肿瘤科,广东省广州市510623 [2]广州市妇女儿童医疗中心医务部预防保健科,广东省广州市510623

出　　处：《中国组织工程研究》2023年第1期42-48,共7页Chinese Journal of Tissue Engineering Research

摘　　要：背景:异基因造血干细胞移植是目前治疗重型地中海贫血的主要方法,但影响移植预后的危险因素尚不明确。目的:分析重型地中海贫血患儿异基因造血干细胞移植后常见并发症分布及死亡原因,探讨预后影响因素,为提高患儿生存率提供参考。方法:选择2013年9月至2019年9月在广州市妇女儿童医疗中心血液肿瘤科诊断为重型地中海贫血并同意接受异基因造血干细胞移植的257例患者,男172例,女85例,移植时中位年龄6岁,采用单因素比较存活患者组与死亡患者组接受移植时的基本资料及移植后并发症差异,总生存率采用Kaplan-Meier法分析,总生存率比较采用Log-rank检验。采用多因素Cox回归分析影响生存的因素。结果与结论:中位随访时间为29个月,无一例失访。①单因素分析结果显示,存活患者和死亡患者在危险度分级方面存在统计学差异(P=0.033),危险度分级高的患者移植后死亡风险增多;多因素分析结果显示,危险度分级和重症肺炎是重型地中海贫血患者造血干细胞移植后影响总生存率的独立危险因素(P<0.05)。②20例死亡患者中,重症肺炎伴呼吸衰竭13例,肠道Ⅳ度急性移植物抗宿主病2例,血小板低下致颅内出血1例,血小板低下伴急性肺出血1例,脓毒血症伴休克1例,重症肌无力1例。③17例死于移植后1年以内,3例患者因重症肺炎死于移植后1年以上。④移植后死亡患者闭塞性细支气管炎的发生率显著高于存活患者(P<0.0001)。⑤结果表明,感染及重度移植物抗宿主病是目前影响重型地中海贫血患者造血干细胞移植治疗后生存率的主要原因。移植后并发闭塞性细支气管炎的患者死亡风险增高。BACKGROUND:Allogeneic hematopoietic stem cell transplantation is the only effective method to cure thalassemia major.However,the risk factors influencing the prognosis of transplantation remain unclear.OBJECTIVE:To analyze the distribution of common complications and cause of death after allogeneic hematopoietic stem cell transplantation in children with thalassemia major,and to explore the prognostic factors so as to provide references for improving the survival rate of patients.METHODS:This retrospective cohort study included 257 patients withβ-thalassemia major who underwent allogeneic hematopoietic stem cell transplantation at Department of Hematology and Oncology,Guangzhou Women and Children Medical Center between September 2013 and September 2019.There were 172 males and 85 females,with a median age of 6 years at the time of transplantation.The basic clinical data before transplantation and complications after transplantation were compared between the surviving group and dead group using single factor.The overall survival rate was analyzed by the Kaplan-Meier method,and the overall survival rate was compared by the Log-rank test.Multivariate Cox regression was used to analyze factors affecting survival.RESULTS AND CONCLUSION:The median follow-up time was 29 months,and no cases were lost to follow-up.(1)Univariate analysis results showed that there were significant differences in the risk classification between surviving patients and dead patients(P=0.033).Patients with higher risk class had an increased risk of death after transplantation.Multivariate analysis showed that risk classification and severe pneumonia were independent risk factors for overall survival after thalassemia major hematopoietic stem cell transplantation(P<0.05).(2)Among 20 dead patients,there were 13 patients with severe pneumonia and respiratory failure,2 patients with grade IV intestinal graft-versus-host disease,1 patient with intracranial hemorrhage due to thrombocytopenia,1 patient with thrombocytopenia with acute pulmonary hemor

关 键 词：重型地中海贫血 造血干细胞移植 感染 移植物抗宿主病 生存率 

分 类 号：R459.9[医药卫生—治疗学] R394.2[医药卫生—临床医学]