Uncovering symptom progression history from disease registry data with application to young cystic fibrosis patients.

Uncovering symptom progression history from disease registry data with application to young cystic fibrosis patients.
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DOI:
10.1111/j.1541-0420.2009.01288.x
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发表时间:
2010-06
期刊:
影响因子:
1.9
通讯作者:
Lai HJ
Lai HJ
中科院分区:
数学3区
文献类型:
--
作者:
Yan J;Cheng Y;Fine JP;Lai HJ

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各种疾病登记数据的日益可用性为流行病学家了解登记疾病的自然历史带来了宝贵的机会。它也提出了挑战,传统的数据分析技术,由于复杂的删失/截断方案和协变量影响的时间动态。在囊性纤维化基金会患者登记数据的案例研究中,我们建议使用时间过程回归分析进行性症状,作为常用比例风险模型的替代方案。考虑两个终点,即肺部铜绿假单胞菌(PA)感染的阳性率和阳性率,这两个终点反映了疾病过程的不同方面。通过时变系数模型对以往PA阳性的分析表明,在标准比例风险分析中缺乏拟合以及潜在的信息丢失。对目前PA阳性的分析产生了具有临床意义的结果,并且以前在囊性纤维化文献中没有报道过。我们的分析表明,与通过体征和症状进行的传统诊断相比,产前/新生儿筛查导致PA感染的患病率较低,但这种益处随着年龄的增长而减弱。诊断的历年也影响PA感染的风险;最近队列中诊断的患者显示出更高的PA阳性患病率,但目前PA阳性的患病率较低。
The growing availability of various disease registry data has brought precious opportunities to epidemiologists to understand the natural history of the registered diseases. It also presents challenges to the traditional data analysis techniques due to complicated censoring/truncation schemes and temporal dynamics of covariate influences. In a case study of the Cystic Fibrosis Foundation Patient Registry data, we propose analyses of progressive symptoms using temporal process regressions, as an alternative to the commonly employed proportional hazards models. Two end points are considered, the prevalence of ever positive and currently positive for Pseudomonas aeruginosa (PA) infection in the lungs, which capture different aspect of the disease process. The analysis of ever PA positive via a time-varying coefficient model demonstrates the lack of fit, as well as the potential loss of information, in the standard proportional hazards analysis. The analysis of currently PA positive yields results which are clinically meaningful and have not previously been reported in the cystic fibrosis literature. Our analyses demonstrate that prenatal/neonatal screening results in lower prevalence of PA infection compared to traditional diagnosis via signs and symptoms, but this benefit attenuates with age. Calendar years of diagnosis also affect the risk of PA infection; patients diagnosed in more recent cohort show higher prevalence of ever PA positive but lower prevalence of currently PA positive.
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