Department of Oncology-Hematology, Pugliese-Ciaccio Hospital Center, Catanzaro, Italy.
Cancer. 2013 Mar 15;119(6):1177-85. doi: 10.1002/cncr.27900. Epub 2012 Dec 7.
A nomogram that incorporates traditional and newer prognostic factors to identify patients with chronic lymphocytic leukemia (CLL) who are at high risk of receiving therapy was developed by investigators at The University of Texas M. D. Anderson Cancer Center (MDACC). Because the model required validation before its extensive use could be recommended, the authors sought to externally validate the nomogram in an independent, community-based cohort of patients with CLL.
In total, 328 previously untreated patients with newly diagnosed, asymptomatic, Binet stage A CLL from different primary hematology centers who were registered on a prospective basis during 2006 to 2010 on an observational database of the Italian Lymphoma Study Group were considered suitable for external validation of the model.
A total point score was calculated for each patient using a formula proposed by MDACC investigators, and the median score was 19.9 (range, 0-69.5). Furthermore, when the score was evaluated as continuous variable (ie, by measuring the risk of each point increase), the total point score was associated with the time to first treatment (hazard ratio [HR], 1.04; 95% confidence interval [CI], 1.02-1.05; P < .0001). Receiver operating characteristic analysis identified a point score of 25 (area under curve; 0.64; sensitivity, 61.5; specificity, 72.1; P < .0001) as the best threshold capable of separating patients who needed therapy from patients who did not (HR, 3.27; 95% CI, 2,07-5.18; P < .0001). The prognostic index category also remained a predictor of the time to first treatment when the analysis was limited to patients with Rai stage 0 disease (HR, 4.05; 95% CI, 2.25-7.52; P < .0001). Finally, a goodness-of-fit test demonstrated that the nomogram model had a significantly good fit at 2 years (correlation coefficient [r(2) ] = 0.966; P = .002).
The current results confirmed the ability of a newly developed prognostic index to predict the time to first treatment among previously untreated patients with CLL who had early disease and extended the utility of the model to those with Rai stage 0 disease. In addition, the actual and predicted time to first treatment outcomes revealed good agreement, suggesting that, externally, the results provided by the model are well calibrated. Cancer 2013. © 2012 American Cancer Society.
德克萨斯大学 MD 安德森癌症中心（MDACC）的研究人员开发了一种列线图，该列线图将传统和较新的预后因素结合起来，以识别出患有慢性淋巴细胞白血病（CLL）且接受治疗风险较高的患者。由于在广泛推荐使用该模型之前需要对其进行验证，作者试图在一个独立的、基于社区的 CLL 患者队列中对该列线图进行外部验证。
共有 328 名新诊断、无症状、Binet 分期为 A 的初治 CLL 患者，来自不同的初级血液学中心，这些患者于 2006 年至 2010 年期间在意大利淋巴瘤研究组的一个观察数据库中进行前瞻性登记，适合用于验证模型。
根据 MDACC 研究人员提出的公式，为每位患者计算了总评分，中位评分为 19.9（范围，0-69.5）。此外，当评分作为连续变量进行评估时（即，通过测量每个评分增加的风险），总评分与首次治疗时间相关（风险比[HR]，1.04；95%置信区间[CI]，1.02-1.05；P<.0001）。接受者操作特征分析确定 25 分（曲线下面积；0.64；灵敏度，61.5；特异性，72.1；P<.0001）为最佳阈值，能够区分需要治疗的患者和无需治疗的患者（HR，3.27；95%CI，2.07-5.18；P<.0001）。当分析仅限于 Rai 分期为 0 期的患者时，预后指数类别仍然是首次治疗时间的预测因子（HR，4.05；95%CI，2.25-7.52；P<.0001）。最后，拟合优度检验表明，该列线图模型在 2 年内具有良好的拟合度（相关系数[r(2)]=0.966；P=.002）。
目前的结果证实了一种新开发的预后指数在预测早期疾病的未经治疗的 CLL 患者首次治疗时间方面的能力，并将模型的实用性扩展到 Rai 分期为 0 期的患者。此外，实际和预测的首次治疗时间结果显示出良好的一致性，表明模型提供的结果在外部验证中具有良好的校准度。癌症 2013。©2012 美国癌症协会。
