骨髓增生异常综合征患者异基因造血干细胞移植前评估与管理

Evaluation and management before allogeneic hematopoietic stem cell transplantation in patients with myelodysplastic syndrome

骨髓增生异常综合征(MDS)是一种异质性克隆性造血干细胞疾病,其发病率随着患者年龄增长而增高,好发于年龄≥70岁人群。异基因造血干细胞移植(allo-HSCT)是目前唯一可治愈MDS的方法。由于MDS主要影响老年人,并且大多数患者由于存在合并症或机体功能状态低下,而不适宜进行allo-HSCT[1]。因此,准确评估MDS患者的肿瘤负荷,判断细胞遗传学异常及疾病分期,关注MDS疾病演变过程及有无转化为急性髓细胞白血病(AML)的风险,对降低移植风险至关重要。这需要对MDS患者做好移植前风险评估及管理,提高其移植后生活质量,延缓疾病进展,以获取最大限度生存获益。笔者基于疾病危险分层、骨髓原始细胞比例、细胞遗传学异常、基因突变、患者年龄,以及移植前合并症、减瘤治疗及袪铁治疗等可能影响移植结局的相关因素,对MDS患者移植前注意事项进行阐述,以完善移植前的评估与管理,提高allo-HSCT成功率。

Myelodysplastic syndrome(MDS)is a group of heterogeneous clonal hematopoietic stem cell diseases.The incidence of MDS increases with age,especially in people over 70 years old.Allogeneic hematopoietic stem cell transplantation(allo-HSCT)is currently the only possible cure for MDS.Because MDS mainly affects the elderly,and most of the patients do not suit for allo-HSCT due to frailty and comorbidity.The assessment of tumor load,cytogenetic abnormalities,the stage of disease,the evolution process,the risk of turning to acute myeloid leukemia(AML)is crucial to reduce the risk of trans plantation.The management of MDS patients requires careful risk assessment to improve the quality of life after allo-HSCT,delay the progression of the disease,and obtain the maximum survival benefit.This article was based on disease risk stratification,proportion of bone marrow blasts,cytogenetic abnormalities,gene mutations,patient′s age,comorbidities before transplantation,pre-transplant cytoreductive therapy and iron depletion therapy and other related factors that may affect the outcome of transplantation,attention should be paid to MDS patients before transplantation.The precautions are explained in order to do a good job in the evaluation and management before transplantation,so as to promote the success of allo-HSCT.