Greenberg P, Cox C, LeBeau M M, Fenaux P, Morel P, Sanz G, Sanz M, Vallespi T, Hamblin T, Oscier D, Ohyashiki K, Toyama K, Aul C, Mufti G, Bennett J
Hematology Division, Stanford University Medical Center, CA 94305, USA.
Blood. 1997 Mar 15;89(6):2079-88.
Despite multiple disparate prognostic risk analysis systems for evaluating clinical outcome for patients with myelodysplastic syndrome (MDS), imprecision persists with such analyses. To attempt to improve on these systems, an International MDS Risk Analysis Workshop combined cytogenetic, morphological, and clinical data from seven large previously reported risk-based studies that had generated prognostic systems. A global analysis was performed on these patients, and critical prognostic variables were re-evaluated to generate a consensus prognostic system, particularly using a more refined bone marrow (BM) cytogenetic classification. Univariate analysis indicated that the major variables having an impact on disease outcome for evolution to acute myeloid leukemia were cytogenetic abnormalities, percentage of BM myeloblasts, and number of cytopenias; for survival, in addition to the above, variables also included age and gender. Cytogenetic subgroups of outcome were as follows: "good" outcomes were normal, -Y alone, del(5q) alone, del(20q) alone; "poor" outcomes were complex (ie, > or = 3 abnormalities) or chromosome 7 anomalies; and "intermediate" outcomes were other abnormalities. Multivariate analysis combined these cytogenetic subgroups with percentage of BM blasts and number of cytopenias to generate a prognostic model. Weighting these variables by their statistical power separated patients into distinctive subgroups of risk for 25% of patients to undergo evolution to acute myeloid leukemia, with: low (31% of patients), 9.4 years; intermediate-1 (INT-1; 39%), 3.3 years; INT-2 (22%), 1.1 years; and high (8%), 0.2 year. These features also separated patients into similar distinctive risk groups for median survival: low, 5.7 years; INT-1, 3.5 years; INT-2, 1.2 years; and high, 0.4 year. Stratification for age further improved analysis of survival. Compared with prior risk-based classifications, this International Prognostic Scoring System provides an improved method for evaluating prognosis in MDS. This classification system should prove useful for more precise design and analysis of therapeutic trials in this disease.
尽管有多种不同的预后风险分析系统用于评估骨髓增生异常综合征（MDS）患者的临床结局，但此类分析仍存在不精确性。为了改进这些系统，一个国际MDS风险分析研讨会整合了来自七项先前报道的基于风险的大型研究的细胞遗传学、形态学和临床数据，这些研究已生成了预后系统。对这些患者进行了全面分析，并重新评估了关键的预后变量，以生成一个共识预后系统，特别是使用了更精细的骨髓（BM）细胞遗传学分类。单变量分析表明，对疾病进展为急性髓系白血病的结局有影响的主要变量是细胞遗传学异常、BM原始细胞百分比和血细胞减少症数量；对于生存而言，除上述因素外，变量还包括年龄和性别。结局的细胞遗传学亚组如下：“良好”结局为正常、单纯-Y、单纯del(5q)、单纯del(20q)；“不良”结局为复杂（即≥3种异常）或染色体7异常；“中间”结局为其他异常。多变量分析将这些细胞遗传学亚组与BM原始细胞百分比和血细胞减少症数量相结合，以生成一个预后模型。根据这些变量的统计效力对其进行加权，将患者分为不同的风险亚组，25%的患者会进展为急性髓系白血病，具体情况如下：低风险（31%的患者），9.4年；中间-1（INT-1；39%），3.3年；INT-2（22%），1.1年；高风险（8%），0.2年。这些特征也将患者分为类似的不同风险组，用于中位生存分析：低风险，5.7年；INT-1，3.5年；INT-2，1.2年；高风险，0.4年。年龄分层进一步改善了生存分析。与先前基于风险的分类相比，这个国际预后评分系统为评估MDS的预后提供了一种改进的方法。这种分类系统对于更精确地设计和分析该疾病的治疗试验应该会很有用。
