Year in review 2023 - Back to the future

Area of Science:

• Medical research
• Pulmonology
• Genetics

Background:

• Cystic Fibrosis (CF) is a genetic disorder affecting multiple organs.
• CFTR modulators represent a significant advancement in CF treatment.
• The impact of elexacaftor-tezacaftor-ivacaftor (ETI) requires ongoing evaluation.

Purpose of the Study:

• To review 2023 literature on ETI's impact in cystic fibrosis care.
• To assess real-world benefits and challenges associated with ETI therapy.
• To identify emerging trends and unmet needs in CF management.

Main Methods:

• Systematic literature review of articles published in 2023.
• Analysis of real-world data on ETI efficacy and safety.
• Synthesis of research on complementary and novel CF therapies.

Main Results:

• Real-world evidence confirms sustained clinical benefits of ETI across diverse age groups.
• Neuropsychological side effects associated with ETI therapy remain a notable challenge.
• Research is expanding into telemedicine applications and innovative therapeutic strategies for CF.

Conclusions:

• ETI demonstrates significant positive impact on cystic fibrosis care.
• Addressing persistent side effects and ensuring equitable access are critical for optimizing CF outcomes.
• Continued research into novel therapies and care delivery models is essential for comprehensive CF management.