EFFICACY AND EFFECTIVENESS OF TREATING CHILDREN WITH CYSTIC FIBROSIS WITH CFTR PROTEIN MODULATORS
Keywords:
cystic fibrosis, CFTR protein, chlorine channelAbstract
Cystic fibrosis (CF) therapy with CF protein transmembrane transport modulators (CFTRP) is innovative, effective, and efficient for patients who are homo- or hetero-zygotes F508del. The combination of correctors and potentiators in a certain proportion and in a certain daily regime enabled increased production and intracellular function of CFTRP, better clearance of the respiratory tree and lungs, better chlorine transport, and better metabolism at the level of respiratory and intestinal membranes primarily, enabled reduction of chlorine concentration in sweat, improvement of lung function, rare lung exacerbations caused by infection, reduction of flatulence and other digestive problems related to CF, increased production of pancreatic elastase, improved body weight, better control of CF-related diabetes mellitus, better patient adherence to CFTRP modulators, the better quality of life for CF patients, reduction of mental health problems and loss of indication for lung transplantation. CFTRP modulators (elexacaftor 100mg, tezacaftor 50mg, ivacaftor 75mg) and Ivacaftor 150mg are effective and efficient in treating pulmonary and extrapulmonary CF symptoms in children over 12 years of age, and have a less number of mild adverse effects ie. drugs are well tolerated, and safe. The clinical phenotype of CF was significantly altered after the application of the CFTRP modulator.
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