Cystic Fibrosis

**tip: Find an extended version of this teaching on the pulmonology rotation page

Objectives:

1. Develop an understanding of the molecular pathogenesis and CFTR dysfunction along with the natural history of the disease
2. Appreciate the myriad of organ system involvement (upper/lower respiratory tract, GI/nutrition, endocrine, reproductive, electrolyte complications, etc.)
3. Recognize infectious complications of CF lung disease (e.g. Pseudomonas aeruginosa, MRSA, Burkholderia cepacia ABPA, nontuberculous mycobacterial infection)
4. Understand the inpatient presentation and the treatment of pulmonary exacerbations and the complications of CF involving other organ systems
5. Appreciate the importance of a multidisciplinary team approach

Articles:

• There have been great advances in CF management over the past few decades. Read this 2020 review article from the Lancet Respiratory Medicine Commission to appreciate how CF care has evolved over time and better understand the natural history of the disease through a patient’s lifetime.  In addition, the paper offers a unique global perspective on CF.  [Objectives 1-5]
• For a better understanding of the multiple organ systems impacted by CF, please see the following Pediatrics in Review review article on lung, gastrointestinal and pancreatic complications. [Objectives 1-4]
• This state-of-the-art review article discusses the most common lung infections affecting cystic fibrosis patients.  It provides a great review of the pathophysiology, presentation, diagnosis and treatment of these infections.  There is a great explanation of how CFTR dysfunction predisposes patients to colonization by Pseudomonas aeruginosa. [Objectives 1-4]
• These guidelines on the management of acute pulmonary exacerbations highlight the recommendedantibiotic regimens, including the dosing and duration antibiotic treatment with graded evidence.  For more information on the treatment of lung infections with a focus on Pseudomonas aeruginosa read this review article. [Objectives 2-4]
• CF requires multidisciplinary care that must be standardized across institutions. The Cystic fibrosis Care Center Network exemplifies the importance of a team-based approach to improve patient outcomes as evidenced by this 2013 BMJ article. [Objective 5]

Additional resources:

• For more guidelines on the care of cystic fibrosis patients, visit the website of the Cystic Fibrosis Foundation.
• Cystic Fibrosis My Way is an excellent video series from CHOP highlighting the proper use of cystic fibrosis therapies for patients and their families.
• Fantastic book for patients and their families on living with cystic fibrosis.

Self-Assessment: Coming soon!