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骨髓增生异常综合征患者的移植策略。

Transplant strategies for patients with myelodysplastic syndromes.

作者信息

Deeg H Joachim

机构信息

Fred Hutchinson Cancer Research Center and the University of Washington, 1100 Fairview Avenue North, Seattle, WA 98109-1024, USA.

出版信息

Curr Opin Hematol. 2006 Mar;13(2):61-6. doi: 10.1097/01.moh.0000208466.63861.9f.

Abstract

PURPOSE OF REVIEW

Hematopoietic cell transplantation offers potentially curative therapy for patients with myelodysplastic syndrome. The median patient age at diagnosis, however, is about 70 years, which has limited the application of conventional hematopoietic cell transplantation. In addition, the optimum timing of transplantation remains controversial.

RECENT FINDINGS

The omission of high-dose total body irradiation, dose adjustments of busulfan to achieve defined target levels, the use of fludarabine instead of cyclophosphamide as an immune suppressive agent, the addition of melphalan, and the incorporation of antithymocyte globulin appear to have contributed to better tolerability of new transplant regimens. Reduced-intensity conditioning regimens are associated with a decrease in nonrelapse mortality and allow for successful hematopoietic cell transplantation even in patients 60-70 years of age. Among patients with less advanced/low-risk myelodysplastic syndrome (< 5% marrow myeloblasts), 3-year survivals of 65-75% are achievable with human leucocyte antigen-matched related and unrelated donors. Among patients with more advanced/high-risk disease, the probability of posttransplant relapse ranges from 10 to 40%, and, as a result, relapse-free survival is inferior. In addition to disease stage, comorbid conditions, pretransplant chemotherapy, conditioning regimen and source of stem cells, affect transplant outcome.

SUMMARY

Modifications of transplant conditioning regimens have reduced transplant-related mortality, allowing successful hematopoietic cell transplantation in increasingly older patients. Graft versus host disease, both acute and chronic, and posttransplant relapse remain challenging problems.

摘要

综述目的

造血细胞移植为骨髓增生异常综合征患者提供了潜在的治愈性治疗方法。然而,诊断时患者的中位年龄约为70岁,这限制了传统造血细胞移植的应用。此外,移植的最佳时机仍存在争议。

最新发现

省略高剂量全身照射、调整白消安剂量以达到确定的目标水平、使用氟达拉滨而非环磷酰胺作为免疫抑制剂、添加美法仑以及加入抗胸腺细胞球蛋白似乎有助于提高新移植方案的耐受性。减低强度预处理方案与非复发死亡率的降低相关,甚至在60 - 70岁的患者中也能成功进行造血细胞移植。在病情较轻/低危骨髓增生异常综合征(骨髓原始细胞<5%)的患者中,与人类白细胞抗原匹配的相关和无关供者移植后3年生存率可达65 - 75%。在病情较重/高危疾病的患者中,移植后复发概率为10%至40%,因此无复发生存率较低。除疾病阶段外,合并症、移植前化疗、预处理方案和干细胞来源也会影响移植结果。

总结

移植预处理方案的改进降低了移植相关死亡率,使越来越多的老年患者能够成功进行造血细胞移植。急性和慢性移植物抗宿主病以及移植后复发仍然是具有挑战性的问题。

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