Rodrigo Abensur Athanazio1, Suzana Erico Tanni8, Juliana Ferreira1, Paulo de Tarso Roth Dalcin3,4, Marcelo B de Fuccio5, Concetta Esposito6, Mariane Gonçalves Martynychen Canan7, Liana Sousa Coelho8, Mônica de Cássia Firmida9, Marina Buarque de Almeida2, Paulo José Cauduro Marostica10, Luciana de Freitas Velloso Monte11,12, Edna Lúcia Souza13, Leonardo Araujo Pinto14, Samia Zahi Rached1, Verônica Stasiak Bednarczuk de Oliveira7,15, Carlos Antonio Riedi7, Luiz Vicente Ribeiro Ferreira da Silva Filho2
J Bras Pneumol.2023;49(2):e20230040
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.
Keywords: Cystic fibrosis; GRADE approach; Cystic fibrosis/drug treatment; Clinical practice guide.