囊狀性纖維化是一種少見且具有地區性的遺傳疾病, 其中以肺部和消化系統所受的影響最為嚴重, 而在此篇論文主要是在討論肺部的囊狀纖維化。在1994年rhDNase(pulmozyme) 獲FDA 核准上市, 且列為囊狀纖維化患者的治療藥物。其中我們感興趣的是rhDNase 對於囊狀纖維化患者療效和肺活量對此疾病的影響, 本篇研究的資料來自於研究脫氧核醣核酸酶團隊(ThePulmozyme Study Group) , 分析方法主要使用了邊際模型(marginal model): AG model 、PWP model 、WLW model 和脆弱模型(frailty model) 。由邊際模型和脆弱模型可以得到相同的結論, rhDNase能降低囊狀纖維化患者的 復發風險和肺活量增加可以改善囊狀纖維化患者的復發風險。 Cystic fibrosis (CF) is a rare and regional genetic disease mainly developing in lungs and digestive system. In this study, we focused on lung cystic fibrosis. In 1994, rhDNase (pulmozyme) was listed and approved by FDA as a therapeutic drug for patients with cystic fibrosis. We are interested in the effect of rhDNase therapy and the impact of pulmonary forced expiratory volume (FEV). We applied various main stream statistical methods to analyze this recurrent event data obtained from pulmozyme study group. These statistical methods include marginal models(AG model,PWP model and WLW model) and frailty model. The results derived from different methods are consistent which suggest that rhDNase can reduce recurrent hazard for Cystic fibrosis patients and forced expiratory volume increasing could improve reccrrent hazard for Cystic fibrosis patients.
