Primary myelodysplastic syndromes: analysis of prognostic factors in 235 patients and proposals for an improved scoring system.
Primary myelodysplastic syndromes: analysis of prognostic factors in 235 patients and proposals for an improved scoring system.
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原发性骨髓增生异常综合征：235 名患者的预后因素分析以及改进评分系统的建议。
作者：
C. Aul;N. Gattermann;A. Heyll;U. Germing;G. Derigs;W. Schneider
在一项单中心回顾性研究中，对235例未经治疗的原发性骨髓增生异常综合征（MDS）患者进行了分析，以确定生存和白血病转化的预后因素。对于MDS的众所周知的FAB分类，添加了具有纯铁粒幼细胞性贫血（PSA）的患者的补充组，其特征在于不存在非红系细胞的发育不良特征。因此，形态学亚型为难治性贫血（RA），n = 55; PSA，n = 40; RA伴环形铁粒幼细胞（RARS），n = 33; RA伴原始细胞过多（RAEB），n = 53; RAEB转化（RAEB/T）n = 29;和慢性粒单核细胞白血病（CMML），n = 25。通过单变量分析筛选了28个临床、细胞学和实验室参数，多元回归分析确定了6个具有独立预后价值的变量：骨髓原始细胞百分比、血清LDH活性、PSA、血红蛋白浓度、年龄和血小板计数。如果排除PSA患者，则FAB分类不再提供独立的预后信息。根据多变量分析的结果，设计了一个简单的评分系统来预测MDS患者的生存率。对以下各项参数进行统一评分：骨髓原始细胞大于或等于5%，LDH大于200 U/I，血红蛋白小于或等于9 g/dl，血小板小于或等于100 x 10（9）/I。根据他们的总分，患者被分为三个风险组（A组，0分; B组，1-2分; C组，3-4分），这三个风险组在生存率和白血病转化率方面都有显著差异。诊断后2年累积生存率A组为91%，B组为52%，C组为9%（p <0.00005）。2年时转化为急性髓性白血病的精算风险分别为0、19和54%（p <0.05）。LDH酶水平的纳入使该评分系统能够准确评估CMML患者的预后，当用其他评分评分时，这些患者的预后被认为过于有利。此外，该评分能够识别那些患有RA和RARS的患者，这些患者没有显示出过量的骨髓原始细胞，预后不良。
In an attempt to identify prognostic factors for survival and leukemic transformation, 235 untreated patients with primary myelodysplastic syndromes (MDS) were analyzed in a single center retrospective study. To the well known FAB classification of MDS a supplementary group of patients with pure sideroblastic anemia (PSA) was added, characterized by the absence of dysplastic features of non-erythroid cells. Accordingly, the morphological subtypes were refractory anemia (RA), n = 55; PSA, n = 40; RA with ring sideroblasts (RARS), n = 33; RA with excess of blasts (RAEB), n = 53; RAEB in transformation (RAEB/T) n = 29; and chronic myelomonocytic leukemia (CMML), n = 25. Having screened 28 clinical, cytological, and laboratory parameters by univariate analysis, multiple regression analysis identified six variables with independent prognostic value: percentage of bone marrow blasts, serum LDH activity, PSA, hemoglobin concentration, age, and platelet count. If patients with PSA were excluded, the FAB classification no longer contributed independent prognostic information. Based on the results of this multivariate analysis, a simple scoring system was devised for predicting the survival of patients with MDS. A score of unity was allocated to each of the following parameters: bone marrow blasts greater than or equal to 5%, LDH greater than 200 U/I, hemoglobin less than or equal to 9 g/dl, and platelets less than or equal to 100 x 10(9)/I. As a function of their total score, patients were divided into three risk groups (group A, score 0; group B, score 1-2; group C, score 3-4), which differed significantly in both survival and rates of leukemic transformation. The cumulative survival 2 years after diagnosis was 91% in group A, 52% in group B, and 9% in group C (p less than 0.00005). The actuarial risk of transformation to acute myeloid leukemia at 2 years was 0, 19, and 54%, respectively (p less than 0.05). The inclusion of LDH enzyme levels qualified this scoring system for an accurate assessment of patients with CMML whose prognosis is viewed too favorably when rated by other scores. Furthermore, this score was able to identify those patients with RA and RARS who, without showing an excess of marrow blasts, have an unfavorable prognosis.