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骨髓增生异常综合征患者骨髓原始细胞<10%且细胞遗传学非不良者的生存和向急性髓系白血病进展的临床预后因素。

Clinical prognostic factors for survival and risk of progression to acute myeloid leukemia in patients with myelodysplastic syndromes with < 10% marrow blasts and non-unfavorable cytogenetic categories.

机构信息

Department of Hematology, University Hospital Virgen del Rocío, Instituto de Biomedicina de Sevilla, IBIS/CSIC/Universidad de Sevilla, Sevilla, Spain.

出版信息

Clin Lymphoma Myeloma Leuk. 2013 Apr;13(2):144-52. doi: 10.1016/j.clml.2012.09.013. Epub 2012 Nov 6.

Abstract

UNLABELLED

Prognosis of myelodysplastic syndromes (MDS) is an area of ongoing interest. Identification of patients with poor outcome in the categories of lower risk disease is critical. In this study, we classify a cohort of 332 lower risk MDS into 3 groups with differences in survival and risk for leukemic progression that could drive treatment approaches to improve prognosis in a fraction of these patients.

BACKGROUND

Prognosis of MDS and particularly in patients categorized as lower risk (< 10% blasts or low and intermediate-1 International Prognostic Scoring System [IPSS]) is very heterogeneous and includes patients with very different outcomes with current scoring systems. Recently, a new cytogenetic classification has been proposed for the revised IPSS in predicting the outcome for MDS.

PATIENTS AND METHODS

To evaluate the prognostic significance of multiple variables for survival and risk of progression to acute myeloid leukemia, we analyzed baseline characteristics of 332 lower risk MDS patients within the lower risk cytogenetic categories by IPSS and the recent proposal for the new cytogenetic classification.

RESULTS

In multivariate analysis, severity of cytopenias, age > 60 years, bone marrow blasts (5%-9%) and transfusion dependency significantly influenced outcome. The combination of these variables allowed development of a model which categorizes patients in 3 different groups with median survival of 95, 44, and 13 months for groups 1, 2, and 3, respectively (P < .001). In addition, this score also stratified patients for their risk for leukemic progression, estimated at 2 years in 3.1%, 7.6%, and 21.3% for each group (P = .024).

CONCLUSION

Although karyotype remains the main prognostic factor in MDS, the current study identifies clinical parameters predicting outcome among patients with the better cytogenetic profile. Degree of cytopenias, blasts 5%-9% and transfusion dependence might identify a subset of patients within the nonadverse karyotype, in which early or more aggressive approaches could possibly be required to improve survival or prevent disease progression.

摘要

目的

骨髓增生异常综合征(MDS)的预后是一个研究热点。在低危疾病分类中识别预后不良的患者至关重要。本研究将 332 例低危 MDS 患者分为 3 组,这些患者在生存和白血病进展风险方面存在差异,这种差异可能会影响治疗方法,从而改善部分患者的预后。

背景

MDS 的预后,尤其是在被归类为低危(<10%原始细胞或低危和中危-1 国际预后积分系统[IPSS])的患者中,具有高度异质性,且目前的评分系统包含了预后非常不同的患者。最近,提出了一种新的细胞遗传学分类方法,用于预测修订后的 IPSS 中 MDS 的结果。

患者和方法

为了评估多个变量对生存和向急性髓系白血病进展风险的预后意义,我们分析了 332 例低危 MDS 患者的基线特征,这些患者的低危细胞遗传学分类为 IPSS 和最近提出的新细胞遗传学分类。

结果

多变量分析显示,细胞减少的严重程度、年龄>60 岁、骨髓原始细胞(5%-9%)和输血依赖显著影响结局。这些变量的组合可用于建立一个模型,该模型将患者分为 3 个不同的组,第 1、2 和 3 组的中位生存期分别为 95、44 和 13 个月(P<0.001)。此外,该评分还对患者的白血病进展风险进行分层,每个组的 2 年进展风险估计分别为 3.1%、7.6%和 21.3%(P=0.024)。

结论

尽管核型仍然是 MDS 的主要预后因素,但本研究确定了预测具有更好细胞遗传学特征的患者结局的临床参数。细胞减少程度、原始细胞 5%-9%和输血依赖可能在非不良核型中确定了一组患者,在这些患者中,可能需要早期或更积极的治疗方法来提高生存率或预防疾病进展。

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