Predictive 5-year survivorship model of cystic fibrosis
Predictive 5-year survivorship model of cystic fibrosis
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DOI：
10.1093/aje/153.4.345
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发表时间：
2001-02-15
影响因子：
5
通讯作者：
Marshall, BC
中科院分区：
文献类型：
--
作者：
Liou, TG;Adler, FR;Marshall, BC
本研究的目的是建立一个5年生存率模型，以确定囊性纤维化的关键临床特征。这样的模型可以帮助研究人员和临床医生评估治疗方法，改进前瞻性研究的设计，监测实践模式，为个体患者提供咨询，并确定肺移植的最佳候选人。作者使用了囊性纤维化基金会患者登记处（CFFPR）的信息，该登记处收集了自1986年以来在美国诊断的约90%囊性纤维化患者的纵向数据。他们利用从1993年CFFPR的11,630名患者中随机选择的5,820名患者的数据建立了多变量logistic回归模型。对模型进行了拟合优度检验，并对1993年剩余的5 810名患者进行了验证。经验证的5年生存率模型包括年龄、1秒用力呼气量占预测正常值的百分比、性别、年龄别体重z评分、胰腺功能不全、糖尿病、金黄色葡萄球菌感染、洋葱伯克霍尔德菌感染和每年急性肺加重次数。该模型为囊性纤维化的复杂性质提供了见解，并为临床实践和研究提供了严格的工具。
The objective of this study was to create a 5-year survivorship model to identify key clinical features of cystic fibrosis. Such a model could help researchers and clinicians to evaluate therapies, improve the design of prospective studies, monitor practice patterns, counsel individual patients, and determine the best candidates for lung transplantation. The authors used information from the Cystic Fibrosis Foundation Patient Registry (CFFPR), which has collected longitudinal data on approximately 90% of cystic fibrosis patients diagnosed in the United States since 1986. They developed multivariate logistic regression models by using data on 5,820 patients randomly selected from 11,630 in the CFFPR in 1993. Models were tested for goodness of fit and were validated for the remaining 5,810 patients for 1993. The validated 5-year survivorship model included age, forced expiratory volume in 1 second as a percentage of predicted normal, gender, weight-for-age z score, pancreatic sufficiency, diabetes mellitus, Staphylococcus aureus infection, Burkerholderia cepacia infection, and annual number of acute pulmonary exacerbations. The model provides insights into the complex nature of cystic fibrosis and supplies a rigorous tool for clinical practice and research.