Cystic Fibrosis Year in Review 2024

Area of Science:

• Pulmonology and Genetics
• Pediatric Care

Background:

• Cystic Fibrosis (CF) management is evolving with new therapeutic options.
• The landscape of CF care delivery is shifting, particularly in the post-CFTR modulator era.

Purpose of the Study:

• To review significant 2024 advances in cystic fibrosis care.
• To provide high-level information for potential clinical practice changes.

Main Methods:

• Literature review of 2024 publications relevant to CF care.
• Synthesis of new guidelines, position papers, and research findings.

Main Results:

• Publication of crucial guidelines for newborn screening and management of CF transmembrane conductance regulator (CFTR)-Related Metabolic Syndrome/Cystic Fibrosis Screen Positive Inconclusive Diagnosis (CRMS/CFSPID).
• Development of new position papers addressing care delivery and team composition in the post-CFTR modulator era.
• Continued research on optimizing growth, nutrition, pulmonary exacerbation treatment, and understanding CFTR modulator side effects (mental health, pregnancy).

Conclusions:

• 2024 brought key updates in CF newborn screening and CRMS/CFSPID management.
• The post-CFTR modulator era necessitates updated approaches to care delivery and team structure.
• Ongoing research addresses treatment burden, side effects, and specific populations within CF care.