Cystic Fibrosis Afshaun Haniff, PharmD Candidate Mercer College of Pharmacy Part 3 of 3 Quinton PM. Cystic fibrosis: Lessons from the sweat gland. Physiology 2007;22:212–225. "Woe to that child which when kissed on the forehead tastes salty. He is bewitched and soon must die." This European adage accurately describes the fate of an individual diagnosed with cystic fibrosis during ancient times.

Pancreatic and GI Treatments

Pancreatic Enzymes (Creon, Pancreaze, Pancrelipase, Zenpep) Pancreatic enzyme replacement Contains lipase, amylase, protease Aids in the digestion of fats, protein, and starch in the duodenum Titrated with meals to reduce fatty stools Dipiro JT. Pharmacotherapy, A Pathophysiological Approach. 7th ed. New York City, NY: McGraw Hill. Cystic Fibrosis section.

Dipiro JT. Pharmacotherapy, A Pathophysiological Approach. 7th ed Dipiro JT. Pharmacotherapy, A Pathophysiological Approach. 7th ed. New York City, NY: McGraw Hill.  Cystic Fibrosis section.

CF Related Diabetes (CFRD) Since insulin deficiency is the hallmark of CFRD, insulin is the recommended medical treatment. Oral antidiabetic agents have inconsistent results in literature, therefore, support for their use in therapy for CFRD patients is not recommended. Insulin regimens are individualized based on the patient's lifestyle and circumstances. Dipiro JT. Pharmacotherapy, A Pathophysiological Approach. 7th ed. New York City, NY: McGraw Hill.  Cystic Fibrosis section.

Gastrointestinal Stool softeners Acid suppression Fat soluble vitamin supplements Appetite stimulants High calorie diet Dipiro JT. Pharmacotherapy, A Pathophysiological Approach. 7th ed. New York City, NY: McGraw Hill.  Cystic Fibrosis section.

Pulmonary Exacerbations

IV antibiotics inpatient unless available in non-hospital setting CF Foundation and American Thoracic Society Guidelines for Pulmonary Exacerbations IV antibiotics inpatient unless available in non-hospital setting Continue chronic outpatient regimen Evidence is lacking to recommend for or against inhaled and IV antimicrobials Increase therapies that improve airway clearance Double cover for pseudomonas

High-dose once daily aminoglycosides CF Foundation and American Thoracic Society Guidelines for Pulmonary Exacerbation High-dose once daily aminoglycosides Minimal evidence for continuous infusion -lactams No established length of therapy (14-21 days) No data for steroid use

Antipseudomonal Coverage Image from “Cystic Fibrosis” PowerPoint presentation by Metzger NL. 11/28/2012

Reproduction Drug-drug interactions between oral contraceptive pills (OCPs) and antibiotics should be monitored. Studies have shown that OCP use in CF patients is safe and effective in comparison with other contraception methods. Patches may not reliably adhere to the skin as a result of increased sweat on the surface of the skin. Men should not assume that they are infertile Dipiro JT. Pharmacotherapy, A Pathophysiological Approach. 7th ed. New York City, NY: McGraw Hill.  Cystic Fibrosis section.

Bone Health Many studies have observed that 50% to 75% of CF adults have low bone density and increased rates of fractures. Special multivitamin formulations contain high amounts of fat-soluble vitamins designed to deliver the appropriate doses required. Adequate vitamin D levels may be difficult to maintain due to altered absorption, reduced fat mass, and minimal exposure to sunlight. From “Cystic Fibrosis: Nutritional Issues” Available on UpToDate.com: http://www.uptodate.com/contents/cystic-fibrosis-nutritional-issues?detectedLanguage=en&source=search_result&search=Cystic+fibrosis&selectedTitle=6~150&provider=noProvider#H31

New Therapies

Attempts to Restore Normal Airway Hydration Mannitol, which works by creating an osmotic gradient; this is being studied as a dried powder inhaler.  Amiloride inhibits ion transport, but its short half-life limits its usefulness. Compounds with similar structures are also being studied for potential use. Dipiro JT. Pharmacotherapy, A Pathophysiological Approach. 7th ed. New York City, NY: McGraw Hill. Cystic Fibrosis section.

New Antibiotoics Tobramycin inhaled powder (TIP) and inhaled aztreonam (AZLI) are both currently in phase 3 trials. TIP's advantage is that it is administered faster than TOBI®. AZLI uses a new nebulizer to also reduce treatment time. A new inhaled antibiotic, Arikace, is now in phase 2 trials. This liposomal amikacin penetrates into CF mucus and delivers high concentrations of the drug to the site of infection. Cayston® (aztreonam powder) has recently received approval from the European commission. It will use the Altera® nebulizer, which will decrease administration time from 30 minutes to 3 minutes. Dipiro JT. Pharmacotherapy, A Pathophysiological Approach. 7th ed. New York City, NY: McGraw Hill. Cystic Fibrosis section.

Improvements in QOL Perhaps the best evidence that many people with cystic fibrosis are living longer is the fact that more than 45 percent of the CF patient population is now age 18 or older.  Recently developed therapies and specialized care have enabled people with CF to gain greater control over their disease. So, children and adults are not only living longer, but also living a better quality of life. Statistics from Cystic Fibrosis Foundation: http://www.cff.org/AboutCF/