Improvements in symptomatic treatment strategies for cystic fibrosis: Delivering CF care in the 21st century

Charlotte Addy*, Damian G. Downey, J. Stuart Elborn

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

3 Citations (Scopus)


Introduction: Cystic Fibrosis (CF) is a life-limiting multisystem disease characterized by viscid secretions in multiple organ systems. Survival has increased significantly with average survival now over 40 years. CF care in the 21st century will face different challenges to the 20th century- balancing modulating the basic genetic defect with caring for a larger adult population living with complex multisystem disease into middle age.Areas covered: We review the latest advances in CF therapeutics and their place within CF care. Precision Medicine and the impact of CFTR modulators on costs of care are highlighted. We discuss the role of the lung microbiome and its impact on management of lung infection. Advances in inhaled antibiotics and the rise of dry powder inhalers are evaluated. Screening and management of complications and comorbidities will form a much larger part of CF care, especially CF-related Diabetes, Renal and Bone disease. Current evidence surrounding managing complications is appraised and future research avenues highlighted.Expert opinion: Therapeutic options across all areas of CF care are increasing. Comparative effectiveness research is needed to establish where new treatments fit into 21st century CF Care. Increasing personalization comes with rising costs of care which must be balanced against cost-effectiveness in overall delivery of care.

Original languageEnglish
Pages (from-to)5-19
Number of pages15
JournalExpert Opinion on Orphan Drugs
Issue number1
Early online date02 Nov 2015
Publication statusPublished - 02 Jan 2016


  • CF complication
  • CFTR
  • cystic fibrosis
  • delivery of care
  • inhaled treatments
  • precision medicine

ASJC Scopus subject areas

  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Health Policy
  • Pharmacology (medical)


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