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根据世界卫生组织标准分类的骨髓增生异常综合征的预后因素和预期寿命:临床决策的基础

Prognostic factors and life expectancy in myelodysplastic syndromes classified according to WHO criteria: a basis for clinical decision making.

作者信息

Malcovati Luca, Porta Matteo Giovanni Della, Pascutto Cristiana, Invernizzi Rosangela, Boni Marina, Travaglino Erica, Passamonti Francesco, Arcaini Luca, Maffioli Margherita, Bernasconi Paolo, Lazzarino Mario, Cazzola Mario

机构信息

Division of Hematology, IRCCS, Policlinico San Matteo, 27100 Pavia, Italy.

出版信息

J Clin Oncol. 2005 Oct 20;23(30):7594-603. doi: 10.1200/JCO.2005.01.7038. Epub 2005 Sep 26.

Abstract

PURPOSE

The aim of this study was to evaluate the prognostic value of the WHO proposal, to assess the role of the main prognostic factors in myelodysplastic syndromes (MDSs) classified into WHO subgroups, and to estimate mortality (standardized mortality ratio [SMR]) and life expectancy in these groups as a basis for clinical decision making.

PATIENTS AND METHODS

Four hundred sixty-seven patients who were diagnosed as having de novo MDS at the Division of Hematology, University of Pavia (Pavia, Italy), between 1992 and 2002, were evaluated retrospectively for clinical and hematologic features at diagnosis, overall survival (OS), and progression to leukemia (leukemia-free survival).

RESULTS

Significant differences in survival were noted between patients with refractory anemia (RA), refractory cytopenia with multilineage dysplasia, RA with excess blasts, type 1 (RAEB-1), and RAEB-2. The effect of demographic factors on OS was observed in MDS patients without excess blasts (age, P = .001; sex, P = .006), as in the general population. The mortality of RA patients 70 years or older did not differ significantly from that of the general population (SMR, 1.62; P = .06). Cytogenetics was the only International Prognostic Scoring System variable showing a prognostic value in MDS classified into WHO subgroups. Transfusion-dependent patients had a significantly shorter survival than patients who did not require transfusions (P < .001). Developing a secondary iron overload significantly affected the survival of transfusion-dependent patients (P = .003).

CONCLUSION

These data show that the WHO classification of MDSs has a relevant prognostic value. This classification, along with cytogenetics, might be useful in decisions regarding transplantation. MDS with isolated erythroid lineage dysplasia identifies a subset of truly low-risk patients, for whom a conservative approach is advisable.

摘要

目的

本研究旨在评估世界卫生组织(WHO)提议的预后价值,评估主要预后因素在WHO亚组分类的骨髓增生异常综合征(MDS)中的作用,并估计这些组别的死亡率(标准化死亡率[SMR])和预期寿命,作为临床决策的依据。

患者与方法

回顾性评估1992年至2002年间在意大利帕维亚大学血液学系被诊断为初发性MDS的467例患者的诊断时临床和血液学特征、总生存期(OS)以及进展为白血病(无白血病生存期)情况。

结果

难治性贫血(RA)、多系发育异常的难治性血细胞减少症、1型原始细胞增多的RA(RAEB-1)和RAEB-2患者之间的生存存在显著差异。与普通人群一样,在无原始细胞增多的MDS患者中观察到人口统计学因素对OS的影响(年龄,P = 0.001;性别,P = 0.006)。70岁及以上RA患者的死亡率与普通人群无显著差异(SMR,1.62;P = 0.06)。细胞遗传学是在WHO亚组分类的MDS中唯一显示出预后价值的国际预后评分系统变量。依赖输血的患者生存期明显短于无需输血的患者(P < 0.001)。发生继发性铁过载显著影响依赖输血患者的生存(P = 0.003)。

结论

这些数据表明WHO对MDS的分类具有重要的预后价值。该分类连同细胞遗传学可能有助于移植决策。单纯红系发育异常的MDS确定了一组真正低风险患者,对其采取保守方法是可取的。

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