Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Leukemia. 2017 Jan;31(1):100-106. doi: 10.1038/leu.2016.168. Epub 2016 Jun 13.
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes) is a multisystem disorder with a good long-term prognosis. In its dozens of clinical features, those with independent prognostic value are still not well characterized. We retrospectively included 362 patients with newly diagnosed POEMS syndrome at our institute from 2000 to 2015. On the basis of a randomized sample splitting, we first identified four baseline clinical variables, including age >50 years (hazards ratio (HR) 4.07, 95% confidence interval (CI) 1.41-11.76, P=0.009), pulmonary hypertension (HR 3.99, 95% CI 1.44-11.04, P=0.008), pleural effusion (HR 3.81, 95% CI 1.23-11.79, P=0.02) and estimated glomerular filtration rate <30 ml/min/1.73 m (HR 8.25, 95% CI 2.18-31.25, P=0.002), associated with inferior overall survival in the derivation cohort, with the use of multivariate Cox regression model. These factors were incorporated together to develop a prognostic nomogram. Concordance index calculation (0.727, 95% CI 0.601-0.853, P=0.018) and calibration curve plotting demonstrated its significant predictive and discriminatory capacity in the validation cohort. This nomogram could be a useful and convenient tool in clinical practice to evaluate individualized prognosis in patients with newly diagnosed POEMS syndrome.
POEMS 综合征（多发性神经病、器官肿大、内分泌病、单克隆丙种球蛋白病和皮肤改变）是一种多系统疾病，具有良好的长期预后。在其数十种临床特征中，具有独立预后价值的特征仍未得到很好的描述。我们回顾性地纳入了 2000 年至 2015 年在我院新诊断为 POEMS 综合征的 362 例患者。基于随机样本拆分，我们首先确定了四个基线临床变量，包括年龄>50 岁（风险比（HR）4.07，95%置信区间（CI）1.41-11.76，P=0.009）、肺动脉高压（HR 3.99，95%CI 1.44-11.04，P=0.008）、胸腔积液（HR 3.81，95%CI 1.23-11.79，P=0.02）和估计肾小球滤过率<30 ml/min/1.73 m（HR 8.25，95%CI 2.18-31.25，P=0.002），与推导队列中的整体生存不良相关，使用多变量 Cox 回归模型。这些因素一起被纳入来开发预后列线图。一致性指数计算（0.727，95%CI 0.601-0.853，P=0.018）和校准曲线绘图表明，它在验证队列中具有显著的预测和区分能力。这个列线图可以成为临床实践中评估新诊断 POEMS 综合征患者个体化预后的有用且方便的工具。
