Current reviews in clinical and experimental pharmacology · 2023
Cystic Fibrosis Transmembrane Conductance Regulator Protein Modulators in Children and Adolescents with Different CF Genotypes - Systematic Review and Meta-Analysis.
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Yousif Hamdan Alaa Hassan, Zakaria Faiza, Lourdes Pormento Maria Kezia, Lawal Odunayo Susan, Opiegbe Adaugo, Zahid Samina, Guntipalli Prathima, Nasr Ujala, Hasan Rizvi Syed Asad
Аннотация
OBJECTIVE: To determine the efficacy of the first triple CFTR protein modulators in children and adolescents with cystic fibrosis.
METHODS: Systematic review and meta-analysis were conducted, following PRISMA guidelines. The following databases were searched extensively: PubMed/Medline, Clinical trials.gov, Google Scholar, Scopus, Embase, and Europe PMC using the keywords: "Ivacaftor", "Elexacaftor", "Tezacaftor", VX_661", VX_770", "VX_445", "cystic fibrosis". A total of ten randomized clinical trials were included in our analysis. Primary outcomes included: Absolute change in predicted FEV1 from baseline, Absolute change in sweat chloride test from baseline, Absolute change in BMI from baseline, Absolute change in CF-QR from baseline, and Adverse Events.
RESULTS: Among primary findings, significant absolute change in predictive FEV1 from baseline through 4 weeks favoured the triple CFTR protein modulators. (MD = 11.80, 95% CI = 8.47_15.12, p value = <0.00001); as well as CF_QR score (MD = 0.00, 95% CI = -2.50_2.50, p value= 1.00), and BMI kg/m² change (MD = 16.90, 95% CI = 12.73_21.06, p value= <0.00001). No significant change was noted for CFTR channels activity in the treatment group when compared to placebo or VX_770/VX_661 (MD = -12.57, 95% CI = -94.46_69.32, p value= 0.76).
CONCLUSION: In children aged ≥ 6 y old and adolescents with F508del_CFTR mutation, Elexacaftor- Tezacaftor-Ivacaftor tend to be more effective than first-generation therapy, demonstrating promising results by exhibiting significant improvement in lung function, body weight, and respiratory-related quality of life.
METHODS: Systematic review and meta-analysis were conducted, following PRISMA guidelines. The following databases were searched extensively: PubMed/Medline, Clinical trials.gov, Google Scholar, Scopus, Embase, and Europe PMC using the keywords: "Ivacaftor", "Elexacaftor", "Tezacaftor", VX_661", VX_770", "VX_445", "cystic fibrosis". A total of ten randomized clinical trials were included in our analysis. Primary outcomes included: Absolute change in predicted FEV1 from baseline, Absolute change in sweat chloride test from baseline, Absolute change in BMI from baseline, Absolute change in CF-QR from baseline, and Adverse Events.
RESULTS: Among primary findings, significant absolute change in predictive FEV1 from baseline through 4 weeks favoured the triple CFTR protein modulators. (MD = 11.80, 95% CI = 8.47_15.12, p value = <0.00001); as well as CF_QR score (MD = 0.00, 95% CI = -2.50_2.50, p value= 1.00), and BMI kg/m² change (MD = 16.90, 95% CI = 12.73_21.06, p value= <0.00001). No significant change was noted for CFTR channels activity in the treatment group when compared to placebo or VX_770/VX_661 (MD = -12.57, 95% CI = -94.46_69.32, p value= 0.76).
CONCLUSION: In children aged ≥ 6 y old and adolescents with F508del_CFTR mutation, Elexacaftor- Tezacaftor-Ivacaftor tend to be more effective than first-generation therapy, demonstrating promising results by exhibiting significant improvement in lung function, body weight, and respiratory-related quality of life.