CFTR Modulator Therapy in the Treatment of Cystic Fibrosis as Compared to Placebo
Faculty Advisor Name
Abby Massey
Department
Department of Health Professions
Description
Objective: To assess the efficacy of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies including ivacaftor, tezacaftor/ivacaftor, and elexacaftor/tezacaftor/ivacaftor combinations in the improvement of lung function in patients aged 12 and older with Cystic Fibrosis. Design: Systematic Literature Review Methods: A search was performed in PubMed using the search terms “CFTR modulators in Cystic Fibrosis.” Further parameters set were: published in the last 10 years, Randomized Control Trials, and trials that include participants under 18. Articles were excluded if they did not compare to placebo or had a primary endpoint measurement of FEV₁. Results: The Taylor-Cousar et al study showed CFTR combination therapy had a greater absolute change from baseline in FEV₁ as compared to placebo. The Rowe et al study showed both the ivacaftor and tezacaftor-ivacaftor groups had significant benefits with improved FEV₁ compared to placebo. The Donaldson et al study displayed significant FEV₁ improvements noted with tezacaftor/ivacaftor combination therapy, particularly with higher doses of tezacaftor, indicating a possible dose-related response. The Middleton et al study's absolute change in the percentage of predicted FEV₁ have a statistically significant mean treatment difference of 14.3 points compared to placebo. Conclusion: The use of CFTR modulators increases lung function improving quality of life in those 12 years of age and older with Cystic Fibrosis.
CFTR Modulator Therapy in the Treatment of Cystic Fibrosis as Compared to Placebo
Objective: To assess the efficacy of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies including ivacaftor, tezacaftor/ivacaftor, and elexacaftor/tezacaftor/ivacaftor combinations in the improvement of lung function in patients aged 12 and older with Cystic Fibrosis. Design: Systematic Literature Review Methods: A search was performed in PubMed using the search terms “CFTR modulators in Cystic Fibrosis.” Further parameters set were: published in the last 10 years, Randomized Control Trials, and trials that include participants under 18. Articles were excluded if they did not compare to placebo or had a primary endpoint measurement of FEV₁. Results: The Taylor-Cousar et al study showed CFTR combination therapy had a greater absolute change from baseline in FEV₁ as compared to placebo. The Rowe et al study showed both the ivacaftor and tezacaftor-ivacaftor groups had significant benefits with improved FEV₁ compared to placebo. The Donaldson et al study displayed significant FEV₁ improvements noted with tezacaftor/ivacaftor combination therapy, particularly with higher doses of tezacaftor, indicating a possible dose-related response. The Middleton et al study's absolute change in the percentage of predicted FEV₁ have a statistically significant mean treatment difference of 14.3 points compared to placebo. Conclusion: The use of CFTR modulators increases lung function improving quality of life in those 12 years of age and older with Cystic Fibrosis.
