Aul C, Gattermann N, Heyll A, Germing U, Derigs G, Schneider W
Department of Internal Medicine, Heinrich Heine University, Düsseldorf, Germany.
Leukemia. 1992 Jan;6(1):52-9.
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.
为了确定生存和白血病转化的预后因素，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。在MDS的著名FAB分类中增加了一组纯铁粒幼细胞贫血（PSA）患者，其特征为非红系细胞无发育异常特征。因此，形态学亚型为难治性贫血（RA），n = 55；PSA，n = 40；环形铁粒幼细胞性难治性贫血（RARS），n = 33；原始细胞过多的难治性贫血（RAEB），n = 53；转化中的RAEB（RAEB/T），n = 29；以及慢性粒单核细胞白血病（CMML），n = 25。通过单因素分析筛选了28项临床、细胞学和实验室参数后，多因素回归分析确定了6个具有独立预后价值的变量：骨髓原始细胞百分比、血清乳酸脱氢酶（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，FAB分类不再提供独立的预后信息。基于该多因素分析结果，设计了一个简单的评分系统来预测MDS患者的生存情况。对以下每个参数赋予1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以及血小板小于或等于100×10⁹/I。根据总分，患者被分为三个风险组（A组，评分0；B组，评分1 - 2；C组，评分3 - 4），这三组在生存和白血病转化率方面均有显著差异。诊断后2年的累积生存率在A组为91%，B组为52%，C组为9%（p < 0.00005）。2年时转化为急性髓系白血病的精算风险分别为0、19和54%（p < 0.05）。纳入LDH酶水平使该评分系统能够准确评估CMML患者，而其他评分对CMML患者预后的评估过于乐观。此外，该评分能够识别那些骨髓原始细胞未增多但预后不良的RA和RARS患者。
