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一种用于接受α干扰素治疗的慢性髓性白血病患者生存情况的新预后评分系统。慢性粒细胞白血病协作预后因素项目组写作委员会。

A new prognostic score for survival of patients with chronic myeloid leukemia treated with interferon alfa. Writing Committee for the Collaborative CML Prognostic Factors Project Group.

作者信息

Hasford J, Pfirrmann M, Hehlmann R, Allan N C, Baccarani M, Kluin-Nelemans J C, Alimena G, Steegmann J L, Ansari H

机构信息

Institute for Medical Informatics, Biometry, and Epidemiology, University of Munich, Germany.

出版信息

J Natl Cancer Inst. 1998 Jun 3;90(11):850-8. doi: 10.1093/jnci/90.11.850.

Abstract

BACKGROUND

Interferon alfa is a conservative and widely used alternative to bone marrow transplantation in treatment of patients with early chronic myeloid leukemia (CML). A meta-analysis was conducted to develop a reliable prognostic scoring system for estimation of survival of patients with CML treated with interferon alfa.

METHODS

Patients treated in prospective studies, including major randomized trials, were separated into learning and validation samples. Cox regression analysis and the minimum P-value approach were used to identify prognostic factors for patient survival and to discover groups in the learning sample with the greatest differences in survival. These findings were then validated by applying the new scoring system to patients in the validation sample.

RESULTS

We collected data on 1573 patients who were participants in 14 studies involving 12 institutions; 1303 patients (learning sample, n = 981; validation sample, n = 322) were eligible for inclusion in this analysis, and their median survival time was 69 months (range, 1-117 months). Because two previously described prognostic scoring systems failed to discriminate risk groups satisfactorily, we developed a new scoring system that utilizes the following covariates: age, spleen size, blast count, platelet count, eosinophil count, and basophil count. Among 908 patients with complete data in the learning sample, three distinct risk groups were identified (median survival times of 98 months [n = 369; 40.6%], 65 months [n = 406; 44.7%], or 42 months [n = 133;14.6%]; two-sided logrank test, P< or =.0001). The ability of the new scoring system to discriminate these risk groups was confirmed by analysis of 285 patients with complete data in the validation sample (two-sided logrank test, P = .0002).

CONCLUSIONS

A new prognostic scoring system for estimating survival of patients with CML treated with interferon alfa has been developed and validated through use of a large dataset.

摘要

背景

干扰素α是治疗早期慢性粒细胞白血病(CML)患者的一种保守且广泛应用的替代骨髓移植的方法。进行了一项荟萃分析,以开发一种可靠的预后评分系统,用于评估接受干扰素α治疗的CML患者的生存率。

方法

前瞻性研究(包括主要随机试验)中治疗的患者被分为学习样本和验证样本。采用Cox回归分析和最小P值法来确定患者生存的预后因素,并在学习样本中发现生存差异最大的组。然后通过将新的评分系统应用于验证样本中的患者来验证这些发现。

结果

我们收集了1573例患者的数据,这些患者参与了涉及12个机构的14项研究;1303例患者(学习样本,n = 981;验证样本,n = 322)符合纳入本分析的条件,他们的中位生存时间为69个月(范围,1 - 117个月)。由于之前描述的两种预后评分系统未能令人满意地区分风险组,我们开发了一种新的评分系统,该系统利用以下协变量:年龄、脾脏大小、原始细胞计数、血小板计数、嗜酸性粒细胞计数和嗜碱性粒细胞计数。在学习样本中有完整数据的908例患者中,确定了三个不同的风险组(中位生存时间分别为98个月[n = 369;40.6%]、65个月[n = 406;44.7%]或42个月[n = 133;14.6%];双侧对数秩检验,P≤0.0001)。通过对验证样本中285例有完整数据的患者进行分析,证实了新评分系统区分这些风险组的能力(双侧对数秩检验,P = 0.0002)。

结论

通过使用大型数据集,已经开发并验证了一种用于评估接受干扰素α治疗的CML患者生存率的新的预后评分系统。

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