Cystic fibrosis: Therapeutic updates and optimizing treatment
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Cystic Fibrosis (CF) is a genetic disease that affects nearly 70,000 people worldwide with more than 90% of patients diagnosed of Caucasian descent and a median lifetime survival remains a mere 43.6 years. CF is caused by an autosomal recessive mutation in the CF transmembrane regulator (CFTR) gene, which controls the other chloride and sodium channels at the cell surface and is found in the lungs, liver, pancreas, intestine, sweat duct, and epididymis. The primary organs in which the disease manifests clinically are the pancreas, leading to malabsorption of nutrients, and the lungs due to the accumulation of thick, sticky mucous that contributes to airway obstruction. CF causes several clinical complications, including recurrent pulmonary infections, nasal polyps, CF-related diabetes, fat-soluble vitamin deficiencies, acid reflux, and liver failure.
The following HCPs: pulmonologists, pediatricians, gastroenterologists and primary care physicians; physician assistants, nurse practitioners, nurses, and pharmacists who practice in the aforementioned areas of specialty; and any other healthcare professionals with an interest in or who clinically encounter patients with CF.
By the end of the session the participant will be able to:
- Describe the pathophysiology of CF such that it might inform treatment mechanisms.
- Identify the currently available and emerging pharmacotherapeutic treatments for the management of CF and apply them to patient cases using evidence-based medicine.
- Describe newly approved and investigational therapies in development for CF.
- Evaluate an ongoing treatment plan for a specific patient with CF to optimize safety and efficacy, suggesting modifications for improvement, including the management of complications.