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骨髓增生异常综合征中的干细胞移植

Stem cell transplantation in myelodysplastic syndromes.

作者信息

de Witte T

机构信息

Department of Haematology, University Hospital St. Radboud, Nijmegen, The Netherlands.

出版信息

Forum (Genova). 1999 Jan-Mar;9(1):75-81.

Abstract

The cornerstone of therapeutic management for most patients with myelodysplastic syndrome (MDS) is supportive care, mainly in view of the average advanced age in MDS and the poor response to therapy. Due to the lack of satisfactory therapies, allogeneic stem cell transplantation is today the treatment of choice in the majority of young patients with histocompatible siblings. Results of treatment with allogeneic stem cell transplantation varies considerably depending on the stage of disease at transplantation and various clinical factors, such as the presence of cytogenetic abnormalities, age, and the percentage of blasts in the bone marrow at transplantation. Most patients may benefit optimally from an allogeneic stem cell transplantation when the transplant is performed as soon as an HLA-identical family member has been identified. Progression to more advanced leukaemic conditions will be associated with a higher failure rate mainly due to an increased incidence of relapse after transplantation. Delay of the transplant may be justified in a minority of patients with refractory anaemia without cytopenias or complex cytogenetic abnormalities. Patients who lack an HLA-identical family donor may be transplanted with either autologous stem cells or alternative allogeneic donors. The results are less compared to those obtained with histocompatible sibling transplantation due to an increased risk of relapse after autologous stem cell transplantation or a higher treatment-related mortality after transplantation with genotypically non-identical donors.

摘要

对于大多数骨髓增生异常综合征(MDS)患者而言,治疗管理的基石是支持性治疗,这主要是鉴于MDS患者的平均年龄较大且对治疗反应不佳。由于缺乏令人满意的治疗方法,如今对于大多数有组织相容性同胞的年轻患者来说,异基因干细胞移植是首选治疗方法。异基因干细胞移植的治疗结果因移植时疾病的阶段以及各种临床因素(如细胞遗传学异常的存在、年龄和移植时骨髓中原始细胞的百分比)而有很大差异。一旦确定了HLA相同的家庭成员,大多数患者可能会从异基因干细胞移植中获得最佳益处。进展为更晚期的白血病状态将与更高的失败率相关,主要是由于移植后复发率增加。对于少数难治性贫血且无血细胞减少或复杂细胞遗传学异常的患者,延迟移植可能是合理的。缺乏HLA相同家族供体的患者可以接受自体干细胞移植或替代异基因供体移植。与组织相容性同胞移植相比,结果较差,这是因为自体干细胞移植后复发风险增加,或者与基因型不相同的供体移植后治疗相关死亡率较高。

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