Bejar Rafael
1Moores Cancer Center, Division of Hematology and Oncology, University of California, San Diego, La Jolla, CA.
Hematology Am Soc Hematol Educ Program. 2013;2013:504-10. doi: 10.1182/asheducation-2013.1.504.
Establishing the prognosis for patients with myelodysplastic syndromes (MDS) is a key element of their care. It helps patients understand the severity of their disease and set expectations for their future. For physicians, an accurate estimate of prognosis drives decisions about the timing and choice of therapeutic options to consider. The International Prognostic Scoring System (IPSS) has been the standard tool for MDS risk stratification since it was released in 1997. It has been used to describe patients in pivotal clinical trials and is a key element of practice guidelines. Subsequent changes to the classification scheme for MDS and an underestimation of risk in some patients from the low and intermediate-1 categories have led to the development of several newer prognostic models. The most recent is the revised IPSS (IPSS-R), which addresses several of the perceived deficiencies of its predecessor. Despite their utility, none of the available prognostic systems incorporates disease-related molecular abnormalities such as somatic mutations. These lesions are present in the nearly all cases and many have been shown to improve upon existing prognostic models. However, the interpretation of somatic mutations can be challenging and it is not yet clear how best to combine them with clinical predictors of outcome. Here I review several prognostic scoring systems developed after the IPSS and describe the emerging use of molecular markers to refine risk stratification in the MDS patient population.
确定骨髓增生异常综合征（MDS）患者的预后是其治疗的关键环节。这有助于患者了解自身疾病的严重程度，并对未来形成预期。对于医生而言，准确的预后评估能推动关于治疗方案时机和选择的决策。自1997年发布以来，国际预后评分系统（IPSS）一直是MDS风险分层的标准工具。它被用于关键临床试验中描述患者情况，也是实践指南的关键要素。MDS分类方案随后的变化以及低危和中危-1组部分患者风险的低估促使了几种更新的预后模型的开发。最新的是修订后的IPSS（IPSS-R），它解决了其前身存在的一些明显缺陷。尽管这些系统有用，但现有的预后系统均未纳入与疾病相关的分子异常，如体细胞突变。这些病变几乎在所有病例中都存在，并且许多已被证明能改进现有的预后模型。然而，体细胞突变的解读可能具有挑战性，目前尚不清楚如何最好地将它们与临床预后预测指标相结合。在此，我回顾了IPSS之后开发的几种预后评分系统，并描述了分子标志物在MDS患者群体中用于优化风险分层的新用途。
