Oguma S, Yoshida Y, Uchino H, Okuma M, Maekawa T, Nomura T
Department of Internal Medicine, Faculty of Medicine, Kyoto University, Japan.
Int J Hematol. 1993 Jan;57(1):15-25.
Based on 473 primary cases of refractory anemia (RA), RA with ring sideroblasts (RARS), or with an excess of blasts (RAEB), new staging systems for overall survival, leukemic transformation, and nonleukemic death were developed. The reproducibility of these new staging systems, our former staging systems for leukemic transformation and nonleukemic death, and that for overall survival provided by Sanz et al. (Blood 1989;74:395-408), were estimated using a total of 132 patients with RA, RARS, or RAEB (test sample). The correlation of risk ratios of the test sample from our system and that of Sanz et al. was poor (tau = 0.24; Kendall rank correlation). Our risk ratio was a significant (p = 0.0012) continuous covariate of the Cox model life-table analysis in the test sample but that of Sanz et al. was not. The new and previous staging systems for leukemic transformation were highly similar (tau = 0.7) and risk ratios of both systems were significant in the test sample (p = 0.00046 and 0.0017, respectively). Based upon the new system, patients at high risk for leukemic transformation RA or RARS were identified as males, with bone marrow myeloblasts higher than 2.5%, abnormal granules in the granulocytes, and circulating erythroblasts. Their cumulative leukemic transformation rate exceeded 50% within 2 years from presentation. The new and previous systems for nonleukemic death were moderately correlated (tau = 0.47). The risk ratios of both systems were significant in the test sample (p = 0.00012 and 0.000024, respectively) but failed to stratify test sample patients into three distinct risk groups. In conclusion, our new staging systems for overall survival and leukemic transformation were reproducible in RA, RARS, or RAEB patients and will be useful especially in identifying high-risk patients with RA or RARS, who are considered to be at lower risk by the FAB classification.
基于473例难治性贫血（RA）、环形铁粒幼细胞性难治性贫血（RARS）或原始细胞增多的难治性贫血（RAEB）的原发病例，制定了总生存、白血病转化和非白血病死亡的新分期系统。使用总共132例RA、RARS或RAEB患者（测试样本）评估了这些新分期系统、我们之前的白血病转化和非白血病死亡分期系统以及Sanz等人（《血液》1989年；74：395 - 408）提供的总生存分期系统的可重复性。我们的系统与Sanz等人的测试样本风险比之间的相关性较差（tau = 0.24；肯德尔等级相关性）。在测试样本中，我们的风险比是Cox模型生命表分析的显著（p = 0.0012）连续协变量，而Sanz等人的风险比则不是。白血病转化的新分期系统和之前的分期系统高度相似（tau = 0.7），并且两个系统的风险比在测试样本中均具有显著性（分别为p = 0.00046和0.0017）。基于新系统，被确定为白血病转化高风险的RA或RARS患者为男性，骨髓原始粒细胞高于2.5%，粒细胞中有异常颗粒，且有循环幼红细胞。从就诊起2年内，他们的累积白血病转化率超过50%。新的和之前的非白血病死亡系统具有中度相关性（tau = 0.47）。两个系统的风险比在测试样本中均具有显著性（分别为p = 0.00012和0.000024），但未能将测试样本患者分为三个不同的风险组。总之，我们的总生存和白血病转化新分期系统在RA、RARS或RAEB患者中具有可重复性，尤其将有助于识别被FAB分类认为风险较低的RA或RARS高风险患者。
