Hellström-Lindberg E, Robèrt K H, Gahrton G, Lindberg G, Forsblom A M, Kock Y, Ost A
Department of Medicine, Huddinge University Hospital, Sweden.
Br J Haematol. 1992 Aug;81(4):503-11. doi: 10.1111/j.1365-2141.1992.tb02982.x.
The response to treatment with low-dose ara-C was studied in 102 consecutive patients; 79 with myelodysplastic syndrome (MDS) and 23 with acute myelogenous leukaemia (AML) following MDS. The aim was to find variables that could predict the response to treatment. All patients had clinical symptoms related to cytopenia. Peripheral blood values, bone marrow morphology histology and chromosomes were analysed before the start of treatment. The median survival of the patients was 9 months and a poor survival was predicted by advanced age, low platelet counts, the presence of pseudo-Pelger morphology and > or = 2 chromosomal aberrations. Thirty patients (29%) responded with either a complete remission or a significant increase in haemoglobin level. For the remaining 71%, the treatment was ineffective and in some cases hazardous. The factors associated with a poor response to treatment could be divided into two groups: one included low platelet counts and the presence of chromosomal aberrations, both signs of progressive MDS with a short survival, and the other comprised morphological findings, indicating ineffective haemopoiesis. Patients with platelet counts > 150 x 10(9)/l had a response rate of 55% compared to 23.5% in patients with subnormal platelet counts. Logistic regression identified low bone marrow cellularity, absence of ring sideroblasts and < 2 chromosomal aberrations as predictors of a favourable response in patients with platelet counts < 150 x 10(9)/l. These factors and the platelet count were combined in a predictive model which can divide patients into three groups with different probabilities of response: a favourable group, 38.6% of the patients, with a response rate of > 50%, an intermediate group, 32.7% of the patients, with a response rate of 24%, and an unfavourable group, 28.7% of the patients, with only 3% responses. While low-dose ara-C is an effective treatment for some patients, it is ineffective and hazardous for others. We present a model that can facilitate therapeutic decision making in two-thirds of patients with MDS and MDS-AML by identifying patients who should not be treated with low-dose ara-C as well as patients with a relatively high probability of response.
对102例连续患者进行了小剂量阿糖胞苷治疗反应的研究；其中79例为骨髓增生异常综合征（MDS）患者，23例为MDS继发的急性髓系白血病（AML）患者。目的是寻找能够预测治疗反应的变量。所有患者均有与血细胞减少相关的临床症状。在治疗开始前分析外周血值、骨髓形态学、组织学和染色体情况。患者的中位生存期为9个月，高龄、低血小板计数、假性Pelger核形态的存在以及≥2个染色体畸变提示生存期较短。30例患者（29%）获得完全缓解或血红蛋白水平显著升高。其余71%的患者治疗无效，在某些情况下甚至有危险。与治疗反应不佳相关的因素可分为两组：一组包括低血小板计数和染色体畸变，这两者都是MDS进展且生存期短的征象；另一组包括形态学表现，提示造血无效。血小板计数>150×10⁹/L的患者反应率为55%，而血小板计数低于正常的患者反应率为23.5%。逻辑回归分析确定，骨髓细胞减少、无环形铁粒幼细胞以及染色体畸变<2个是血小板计数<150×10⁹/L患者治疗反应良好的预测指标。这些因素与血小板计数相结合，构建了一个预测模型，可将患者分为三组，反应概率不同：良好组，占患者的38.6%，反应率>50%；中间组，占患者的32.7%，反应率为24%；不良组，占患者的28.7%，反应率仅为3%。虽然小剂量阿糖胞苷对部分患者是有效的治疗方法，但对其他患者则无效且有危险。我们提出了一个模型，通过识别不应接受小剂量阿糖胞苷治疗的患者以及反应概率相对较高的患者，可为三分之二的MDS和MDS-AML患者的治疗决策提供便利。
