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[异基因造血干细胞移植治疗骨髓增生异常综合征]

[Allogeneic hematopoietic stem cell transplantation for myelodysplastic syndromes].

作者信息

Gournay Viviane, Robin Marie

机构信息

AP-HP, université de Paris Cité, hôpital Saint-Louis, Paris, France.

AP-HP, université de Paris Cité, hôpital Saint-Louis, Paris, France.

出版信息

Bull Cancer. 2023 Nov;110(11):1168-1175. doi: 10.1016/j.bulcan.2023.02.025. Epub 2023 Jul 27.

Abstract

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the treatment options for myelodysplastic syndromes (MDS). This treatment is indicated as first-line treatment for high-risk MDS according to the IPSS and R-IPSS classifications and improves overall survival and progression-free survival. However, allo-HSCT is not indicated in first intention for low-risk MDS. It can be discussed in case of cytopenias needing transfusions, poor evolution under other treatment, or in case of poor prognosis molecular anomaly. Allo-HSCT is a treatment that can be complicated by early or late toxicities (graft versus host disease, infections, chemotherapy toxicity…). The decision to do an allo-HSCT is based on the benefit/risk ratio between the risk of progression from MDS to myeloid leukemia and the risk of transplant related mortality, which increases with the patient's age and comorbidities. The indication of a cytoreductive treatment before allo-HSCT depends on the blasts count, and on the delay before the allograft. The use of reduced intensity conditioning regimen and alternative donors such as haploidentical donors, expanded the indications for allo-HSCT. Relapse remains one of the main causes of mortality after allo-HSCT. Some genetic mutations and karyotype anomalies increase the risk of post-transplant relapse. Preventive treatments for relapse are currently being studied. Treatments such as azacytidine, donor lymphocytes infusions or targeted therapies can be used, prophylactically or preemptively.

摘要

异基因造血干细胞移植(allo-HSCT)是骨髓增生异常综合征(MDS)的治疗选择之一。根据国际预后评分系统(IPSS)和修订的国际预后评分系统(R-IPSS)分类,该治疗被指定为高危MDS的一线治疗方法,可提高总生存率和无进展生存率。然而,低危MDS并非首选allo-HSCT。对于需要输血的血细胞减少症、其他治疗效果不佳或预后不良的分子异常情况,可以考虑采用allo-HSCT。allo-HSCT可能会出现早期或晚期毒性反应(移植物抗宿主病、感染、化疗毒性等)。决定是否进行allo-HSCT基于MDS进展为髓系白血病的风险与移植相关死亡率之间的获益/风险比,而移植相关死亡率会随着患者年龄和合并症的增加而升高。allo-HSCT前减瘤治疗的指征取决于原始细胞计数以及移植前的时间间隔。降低强度预处理方案以及单倍体相合供体等替代供体的使用扩大了allo-HSCT的适应证。复发仍然是allo-HSCT后主要的死亡原因之一。一些基因突变和核型异常会增加移植后复发的风险。目前正在研究复发的预防性治疗方法。阿扎胞苷、供体淋巴细胞输注或靶向治疗等可以预防性或抢先性地使用。

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