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A study was conducted to assess the radiological and long-term clinical outcomes of the triple combination of cystic fibrosis transmembrane conductance regulator (CFTR) modulators in individuals diagnosed with cystic fibrosis and advanced lung disease.
Elexacaftor/Tezacaftor/Ivacaftor improves radiological features, sputum cultures, exacerbation rate, quality of life, BMI, and lung function in cystic fibrosis patients with advanced lung illness.
A study was conducted to assess the radiological and long-term clinical outcomes of the triple combination of cystic fibrosis transmembrane conductance regulator (CFTR) modulators in individuals diagnosed with cystic fibrosis and advanced lung disease.
Individuals diagnosed with cystic fibrosis who initiated treatment with Elexacaftor/Tezacaftor/Ivacaftor (ELE/TEZ/IVA) were continuously monitored to evaluate various factors including sweat chloride concentration (SCC), Cystic Fibrosis Questionnaire-Revised (CFQ-R) scores, spirometry, chest computerized tomography (CT) scans, and body mass index (BMI) prior to treatment initiation and following three months. Additionally, follow-up assessments of BMI, sputum cultures, and spirometry were conducted at 1, 6, 12, 18, and 24 months.
In this evaluation, a total of 18 subjects participated, consisting of 9 individuals with the F508del/F508del genotype, 8 of whom were receiving dual CFTR modulators, and 9 volunteers with the F508del/minimal function mutation. After three months, there was a noteworthy average decrease in SCC by -44.9, accompanied by a substantial enhancement in CT results (Brody score change: -28.27) and improvements in CFQ-R outcomes, specifically in the respiratory domain (change: +18.8).
Over the course of twenty-four months, there was an alteration in the percent predicted forced expiratory volume in 1 second (ppFEV1) by +8.89, an increase in BMI by +1.53 kg/m2, and a reduction in exacerbation rate from 5.94 in the preceding 24 months to 1.17 in the subsequent 24 months.
Individuals diagnosed with cystic fibrosis and advanced lung disease showed significant clinical improvement after two years of treatment with ELE/TEZ/IVA. There were notable improvements in BMI, exacerbation rate, quality of life, and structural lung damage. However, the increase in ppFEV1 (a measure of lung function) was reduced contrasted to the results observed in phase III trials that incorporated younger subjects with moderate lung function impairment.
Pediatric Pulmonology
Radiological and long-term clinical response to elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with advanced lung disease
Bente L. Aalbers et al.
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