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采用非清髓性预处理的异基因造血干细胞移植——一项本地经验。

Allogeneic haemopoietic stem cell transplantation using non-myeloablative conditioning--a local experience.

作者信息

Leong C F, Cheong S K, Fadilah S A, Ainoon O, Hamidah N H

机构信息

Clinical Haematology and Stem Cell Transplantation Services, MAKNA-HUKM Cancer Institute, Universiti Kebangsaan Malaysia, Kuala Lumpur.

出版信息

Med J Malaysia. 2003 Jun;58(2):229-35.

Abstract

Allogeneic haemopoietic stem cell transplantation was initially considered as a means of delivering supralethal doses of chemotherapy with or without total body irradiation for the treatment of malignancy. However, it has become clear that this mode of therapy does not eradicate the malignancy in many patients and its benefit is largely due to the immune mediated graft versus malignancy effect. This has led to development of alternative strategy to utilize a less intensive preparative regimen pre-transplantation that provides sufficient immunosuppression to achieve engraftment of an allogeneic stem cell graft, thus allowing the evolution of a graft versus malignancy effect post-transplantation. Since September 1999, we had carried out 10 cases of allogeneic peripheral blood stem cell transplantation: one case of aplastic anaemia, four cases of acute myeloid leukemia (AML) in first remission, and five cases of chronic myeloid leukemia (CML) in chronic phase. The preparative regimen was non-myeloablative comprising Fludarabine with Cyclophosphamide or Busulphan. Recovery from transplantation was rapid with no or brief period of neutropenia or thrombocytopenia. Engraftment was established by determining donor's short tandem repeats in the recipient's bone marrow at day 30, 60 and 100 post-transplantation. Seven cases (70%) show partial or complete donor's chimerism by day 30 indicating successful engraftment. No treatment mortality was noted at day 100. Graft versus host disease was generally limited. Up to the date of reporting, two patients with CML had graft failure, one was successfully re-transplanted later. Two patients with AML had since relapsed and passed away. The others remain alive and well. The cost of transplantation on average was estimated to be about a quarter of that using a myeloablative regimen. It appears that this treatment strategy is a promising approach for the management of blood disorders.

摘要

异基因造血干细胞移植最初被视为一种给予超致死剂量化疗(有或无全身照射)以治疗恶性肿瘤的方法。然而,现已明确这种治疗方式并不能根除许多患者体内的恶性肿瘤,其益处主要归因于免疫介导的移植物抗恶性肿瘤效应。这促使人们开发了另一种策略,即在移植前采用强度较低的预处理方案,该方案可提供足够的免疫抑制以实现异基因干细胞移植物的植入,从而使移植后产生移植物抗恶性肿瘤效应。自1999年9月以来,我们进行了10例异基因外周血干细胞移植:1例再生障碍性贫血,4例首次缓解期的急性髓系白血病(AML),以及5例慢性期的慢性髓系白血病(CML)。预处理方案为非清髓性,包括氟达拉滨联合环磷酰胺或白消安。移植后的恢复很快,中性粒细胞减少或血小板减少期无或短暂。通过在移植后第30、60和100天测定受者骨髓中供者的短串联重复序列来确定植入情况。7例(70%)在第30天时显示部分或完全供者嵌合,表明植入成功。在第100天时未观察到治疗相关死亡。移植物抗宿主病通常较局限。截至报告时,2例CML患者出现移植失败,其中1例后来成功再次移植。2例AML患者随后复发并死亡。其他患者仍存活且情况良好。据估计,移植的平均费用约为采用清髓性方案的四分之一。看来这种治疗策略是治疗血液疾病的一种有前景的方法。

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