Flume Patrick A, O'Sullivan Brian P, Robinson Karen A, Goss Christopher H, Mogayzel Peter J, Willey-Courand Donna Beth, Bujan Janet, Finder Jonathan, Lester Mary, Quittell Lynne, Rosenblatt Randall, Vender Robert L, Hazle Leslie, Sabadosa Kathy, Marshall Bruce
Department of Medicine, Medical University of South Carolina, Charleston, South Carolina 29425, USA.
Am J Respir Crit Care Med. 2007 Nov 15;176(10):957-69. doi: 10.1164/rccm.200705-664OC. Epub 2007 Aug 29.
Cystic fibrosis is a recessive genetic disease characterized by dehydration of the airway surface liquid and impaired mucociliary clearance. As a result, individuals with the disease have difficulty clearing pathogens from the lung and experience chronic pulmonary infections and inflammation. Death is usually a result of respiratory failure. Newly introduced therapies and aggressive management of the lung disease have resulted in great improvements in length and quality of life, with the result that the median expected survival age has reached 36 years. However, as the number of treatments expands, the medical regimen becomes increasingly burdensome in time, money, and health resources. Hence, it is important that treatments should be recommended on the basis of available evidence of efficacy and safety.
The Cystic Fibrosis Foundation therefore established a committee to examine the clinical evidence for each therapy and to provide guidance for the prescription of these therapies.
The committee members developed and refined a series of questions related to drug therapies used in the maintenance of pulmonary function. We addressed the questions in one of three ways, based on available evidence: (1) commissioned systematic review, (2) modified systematic review, or (3) summary of existing Cochrane reviews.
It is hoped that the guidelines provided in this article will facilitate the appropriate application of these treatments to improve and extend the lives of all individuals with cystic fibrosis.
囊性纤维化是一种隐性遗传病，其特征为气道表面液体脱水和黏液纤毛清除功能受损。因此，患有该疾病的个体难以清除肺部病原体，会经历慢性肺部感染和炎症。死亡通常是呼吸衰竭的结果。新引入的疗法以及对肺部疾病的积极管理已使患者的寿命长度和生活质量有了显著改善，中位预期生存年龄已达36岁。然而，随着治疗方法数量的增加，医疗方案在时间、金钱和健康资源方面的负担越来越重。因此，基于有效性和安全性的现有证据来推荐治疗方法非常重要。
囊性纤维化基金会因此成立了一个委员会，以审查每种疗法的临床证据，并为这些疗法的处方提供指导。
委员会成员制定并完善了一系列与用于维持肺功能的药物疗法相关的问题。我们根据现有证据，通过以下三种方式之一来解决这些问题：（1）委托进行系统评价，（2）修改系统评价，或（3）总结现有Cochrane系统评价。
希望本文提供的指南将有助于这些治疗方法的合理应用，以改善和延长所有囊性纤维化患者的生命。
