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Volume 19, Issue 1
  • ISSN: 2772-4328
  • E-ISSN: 2772-4336

Abstract

To determine the efficacy of the first triple CFTR protein modulators in children and adolescents with cystic fibrosis.

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.

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, value = <0.00001); as well as CF_QR score (MD = 0.00, 95% CI = -2.50_2.50, value= 1.00), and BMI kg/m2 change (MD = 16.90, 95% CI = 12.73_21.06, 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, value 0.76).

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.

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2023-02-24
2024-10-16

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/content/journals/crcep/10.2174/2772432818666230201094115
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Cystic Fibrosis Transmembrane Conductance Regulator Protein Modulators in Children and Adolescents with Different CF Genotypes - Systematic Review and Meta-Analysis
Curr Rev Clin Exper Pharm 19, 93 (2024); https://doi.org/10.2174/2772432818666230201094115
/content/journals/crcep/10.2174/2772432818666230201094115
/content/journals/crcep/10.2174/2772432818666230201094115
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  • Article Type: Research Article
Keyword(s): CFTR protein modulators; conductance; Cystic fibrosis; elexacaftor; ivacaftor; meta-analysis; tezacaftor

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