Malcovati Luca, Germing Ulrich, Kuendgen Andrea, Della Porta Matteo G, Pascutto Cristiana, Invernizzi Rosangela, Giagounidis Aristoteles, Hildebrandt Barbara, Bernasconi Paolo, Knipp Sabine, Strupp Corinna, Lazzarino Mario, Aul Carlo, Cazzola Mario
Department of Hematology, University of Pavia, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, 27100 Pavia, Italy.
J Clin Oncol. 2007 Aug 10;25(23):3503-10. doi: 10.1200/JCO.2006.08.5696.
The aims of this study were to identify the most significant prognostic factors in myelodysplastic syndromes (MDS) taking into account both their values at clinical onset and their changes in time and to develop a dynamic model for predicting survival and leukemic evolution that can be applied at any time during the course of the disease.
We studied a learning cohort of 426 MDS patients diagnosed at the Department of Hematology, San Matteo Hospital, Pavia, Italy, between 1992 and 2004, and a validation cohort of 739 patients diagnosed at the Heinrich-Heine-University Hospital, Düsseldorf, Germany, between 1982 and 2003. All patients were reclassified according to WHO criteria. Univariable and multivariable analyses were performed using Cox models with time-dependent covariates.
The most important variables for the prognostic model were WHO subgroups, karyotype, and transfusion requirement. We defined a WHO classification-based prognostic scoring system (WPSS) that was able to classify patients into five risk groups showing different survivals (median survival from 12 to 103 months) and probabilities of leukemic evolution (P < .001). WPSS was shown to predict survival and leukemia progression at any time during follow-up (P < .001), and its prognostic value was confirmed in the validation cohort.
WPSS is a dynamic prognostic scoring system that provides an accurate prediction of survival and risk of leukemic evolution in MDS patients at any time during the course of their disease. This time-dependent system seems particularly useful in lower risk patients and may be used for implementing risk-adapted treatment strategies.
本研究的目的是确定骨髓增生异常综合征(MDS)中最重要的预后因素,同时考虑其临床发病时的值及其随时间的变化,并开发一种动态模型来预测生存和白血病演变,该模型可在疾病过程中的任何时间应用。
我们研究了1992年至2004年间在意大利帕维亚圣马泰奥医院血液科诊断的426例MDS患者的学习队列,以及1982年至2003年间在德国杜塞尔多夫海因里希 - 海涅大学医院诊断的739例患者的验证队列。所有患者均根据世界卫生组织(WHO)标准重新分类。使用具有时间依赖性协变量的Cox模型进行单变量和多变量分析。
预后模型中最重要的变量是WHO亚组、核型和输血需求。我们定义了一种基于WHO分类的预后评分系统(WPSS),该系统能够将患者分为五个风险组,这些组显示出不同的生存率(中位生存期从12个月到103个月)和白血病演变的概率(P <.001)。WPSS被证明可以在随访期间的任何时间预测生存和白血病进展(P <.001),并且其预后价值在验证队列中得到证实。
WPSS是一种动态预后评分系统,可在MDS患者疾病过程中的任何时间准确预测生存和白血病演变风险。这种时间依赖性系统在低风险患者中似乎特别有用,可用于实施风险适应性治疗策略。