Treatment of Pulmonary Disease of Cystic Fibrosis: A Comprehensive Review

Abstract

Cystic fibrosis (CF) is a genetic disease that causes absence or dysfunction of a protein named transmembrane conductance regulatory protein (CFTR) that works as an anion channel. As a result, the secretions of the organs where CFTR is expressed are very viscous, so their functionality is altered. The main cause of morbidity is due to the involvement of the respiratory system as a result of recurrent respiratory infections by different pathogens. In recent decades, survival has been increasing, rising by around age 50. This is due to the monitoring of patients in multidisciplinary units, early diagnosis with neonatal screening, and advances in treatments. In this chapter, we will approach the different therapies used in CF for the treatment of symptoms, obstruction, inflammation, and infection. Moreover, we will discuss specific and personalized treatments to correct the defective gene and repair the altered protein CFTR. The obstacle for personalized CF treatment is to predict the drug response of patients due to genetic complexity and heterogeneity of uncommon mutations.

Cystic Fibrosis – results of CFTR modulators in Croatia

“…Režim liječenja drastično se promijenio od prvog opisa bolesti, od ere prije antibiotika, do danas (5,18). Do prije desetak godina tradicionalna je skrb za bole snike s CFom bila usmjerena na simptomatsko liječe nje zahvaćenih organskih sistema, ponajprije na pluć ne i gastrointestinalne posljedice bolesti.…”

Chasing a Breath of Fresh Air in Cystic Fibrosis (CF): Therapeutic Potential of Selective HDAC6 Inhibitors to Tackle Multiple Pathways in CF Pathophysiology

“…In fact, in patients with persistent PA infection, azithromycin strongly reduces neutrophil chemiotaxis in the lung and the release of elastases by neutrophils themselves. 28 …”

“…In addition to its effect on bacterial protein synthesis, azithromycin has important immunomodulatory and anti-inflammatory properties, which in part could explain the effectiveness of this class of antibiotics. In fact, in patients with persistent PA infection, azithromycin strongly reduces neutrophil chemiotaxis in the lung and the release of elastases by neutrophils themselves …”

The effectiveness of hyaluronic acid in reducing adverse effects associated with inhaled hypertonic saline therapy in patients with cystic fibrosis: A systematic review and meta-analysis

“…4 Trials examining the potential of hypertonic saline solution (HS) on accelerating mucociliary clearance have recently been proposed as a new option for increasing airway surface liquid hydration in CF patients. 5 There is enough evidence to recommend inhaled HS as an alternative mucolytic agent in CF to improve quality of life and reduce pulmonary exacerbations. 6 Although most patients tolerate HS treatment well, some adverse events such as coughing, narrowing of the airways, and an unpleasant salty taste have been reported, limiting its use despite pre-treatment with an inhaled bronchodilator.…”