PDF(Pubmed)
Abstract：
Cystic fibrosis (CF) is a genetic disease that results in dysfunction of the CF transmembrane conductance regulator (CFTR) protein, which is a chloride and bicarbonate channel expressed in the apical portion of epithelial cells of various organs. Dysfunction of that protein results in diverse clinical manifestations, primarily involving the respiratory and gastrointestinal systems, impairing quality of life and reducing life expectancy. Although CF is still an incurable pathology, the therapeutic and prognostic perspectives are now totally different and much more favorable. The purpose of these guidelines is to define evidence-based recommendations regarding the use of pharmacological agents in the treatment of the pulmonary symptoms of CF in Brazil. Questions in the Patients of interest, Intervention to be studied, Comparison of interventions, and Outcome of interest (PICO) format were employed to address aspects related to the use of modulators of this protein (ivacaftor, lumacaftor+ivacaftor, and tezacaftor+ivacaftor), use of dornase alfa, eradication therapy and chronic suppression of Pseudomonas aeruginosa, and eradication of methicillin-resistant Staphylococcus aureus and Burkholderia cepacia complex. To formulate the PICO questions, a group of Brazilian specialists was assembled and a systematic review was carried out on the themes, with meta-analysis when applicable. The results obtained were analyzed in terms of the strength of the evidence compiled, the recommendations being devised by employing the GRADE approach. We believe that these guidelines represent a major advance to be incorporated into the approach to patients with CF, mainly aiming to favor the management of the disease, and could become an auxiliary tool in the definition of public policies related to CF.
摘要：
囊性纤维化（CF）是一种导致CF跨膜传导调节蛋白（CFTR）功能障碍的遗传性疾病，它是在各种器官的上皮细胞的顶端部分表达的氯化物和碳酸氢盐通道。该蛋白质的功能障碍导致不同的临床表现，主要涉及呼吸系统和胃肠道系统，影响生活质量和降低预期寿命。尽管CF仍然是一种无法治愈的病理，治疗和预后的观点现在完全不同，并且更加有利。本指南的目的是确定在巴西使用药物治疗CF肺部症状的循证建议。患者感兴趣的问题，要研究的干预措施，干预措施的比较，和感兴趣的结果（PICO）格式用于解决与使用该蛋白调节剂有关的方面（ivacaftor，lumacaftor+ivacaftor,和tezacaftor+ivacaftor)，使用dornasealfa，铜绿假单胞菌的根除治疗和慢性抑制，并根除耐甲氧西林金黄色葡萄球菌和洋葱伯克霍尔德菌。为了制定PICO问题，召集了一组巴西专家，并对这些主题进行了系统审查，适用时进行荟萃分析。所获得的结果根据所收集的证据的强度进行了分析，这些建议是通过采用等级方法设计的。我们认为，这些指南代表了将纳入CF患者治疗方法的重大进展，主要是为了有利于疾病的管理，并可能成为定义与CF相关的公共政策的辅助工具。
