Cystic Fibrosis
Overview
What is cystic fibrosis?
Cystic fibrosis (CF) is a progressive, genetically-inherited disease that leads to a decrease in lung function and increased frequency of lung infections. CF also causes decreased pancreatic function, which can affect the patient’s ability to absorb food.
CF is caused by a gene mutation that increases the amount of mucus in the body, as well as increasing its viscosity (thickness) and stickiness. The mucus can obstruct organs like the lungs, pancreas, and liver. 1 In the lungs, it enables bacteria to stick and multiply more easily, increasing the risk of infection. It obstructs the lungs, making breathing more difficult. In the pancreas, the mucus prevents pancreatic enzymes from digesting food, leading to possible malabsorption and fatty stools. The mucus in the liver can block the bile duct, which can result in liver disease. Increased mucus in the reproductive system can lower fertility in women, and can cause infertility in men.2
Although there is no cure for CF, recent advances in research have greatly improved quality of life, and the average lifespan has been steadily increasing. Children with CF can now go to college, get married, and even have children.
Treatment of cystic fibrosis includes multiple inhaled and oral medications. Airway clearance, nutrition, and psychosocial support are all integral components of a patient’s care plan.
How common is cystic fibrosis?
CF is most common in Caucasian patients, with 1 out of every 3,000 births having CF. Latin Americans
have a rate of 1 out of 4,000 to 10,000, and African Americans have a rate of 1 out of 15,000 to
20,000. About 1,000 children are born with CF every year in the United States. 3
The Cystic Fibrosis Foundation has 29,497 people with CF in the United States in their registry as of
2016. The worldwide total of people with CF is estimated to be around 70,000 people. 3
Questions regarding your cystic fibrosis therapy?
You can reach the Accredo Cystic Fibrosis care team, anytime, day or night, seven days a week.
24-hour Customer Service Center
1 “Cystic Fibrosis.” Mayo Clinic, Mayo Foundation for Medical Education and Research, 13 Oct. 2016, www.mayoclinic.org/diseases-conditions/cystic-fibrosis/symptoms-causes/syc-20353700.
2 “Cystic Fibrosis (CF).” American Lung Association, 2018, www.lung.org/lung-health-and-diseases/lung-disease-lookup/cystic-fibrosis/.
3 Cystic Fibrosis Foundation Patient Registry. 2016 Annual Data Report. Bethesda, Maryland. ©2017 Cystic Fibrosis Foundation. Accessed February 19, 2018.
Symptoms
What are the symptoms of cystic fibrosis? 1 2
- In newborns, you may notice:
- Persistent coughing, at times with phlegm
- Salty-tasting skin
- Frequent respiratory infections
- Wheezing/shortness of breath
- Poor growth or limited weight gain, in spite of a good appetite
- Frequent greasy stools, or bowel movements that may be pale or clay-colored
- Obstruction in the intestines or difficulty with bowel movements
- Symptoms noticed later in life include:
- Clubbed fingers
- Male infertility
- Pancreatitis (inflammation of the pancreas)
- Wheezing/shortness of breath, frequent respiratory infections, persistent coughing
How is it diagnosed?1 2
All newborns are screened in the United States. There may also be prenatal screening available in your area.
Newborns are screened using a heel prick and testing for Immunoreactive Trypsinogen (IRT). A second, confirmatory IRT test is often required if the initial IRT is positive. Sweat testing is then performed, as well as confirmatory DNA testing.
Other possible tests include nasal potential difference (NPD) or intestinal current monitoring (ICM).
1 “Cystic Fibrosis Foundation.” CF Foundation, Cystic Fibrosis Foundation, 2017, www.cff.org/What-is-CF/About-Cystic-Fibrosis/.
2 “Cystic Fibrosis | CF | Cystic Fibrosis Symptoms | MedlinePlus.” MedlinePlus Trusted Health Information for You, U.S. National Library of Medicine, 31 Jan. 2018, medlineplus.gov/cysticfibrosis.html.
Medications
Cystic Fibrosis
The following specialty medications are available at Accredo, a specialty pharmacy for cystic fibrosis.| Drug | Manufacturer |
|---|---|
| Gilead | |
| Novartis | |
| PARI | |
| Chiesi | |
|
Tobramycin (generic) |
Various |
| Genentech | |
| Vertex | |
| Vertex |
Financing Your Care
Financial assistance may be available to help with your medication costs, including manufacturer and community programs. Accredo specialists are available to help find a program that may work for you.
Community Financial Resources
- Gilead Cayston® Copay Coupon Program | 877-722-9786
- Novartis PodCare+® Patient Support Copay Program | 877-999-8624
- PARI Kitabis Pak® Copay Assistance Program | 844-548-2247
- Chiesi CareDirect Bethkis® Copay Assistance Program | 888-865-1222
- Pulmozyme® Copay Card Program | 800-690-3023
- Kalydeco/Orkambi® Vertex Copay Card Program | 877-752-5933
Financial Guidance
Life-saving specialty medication can be expensive. Learn how the Accredo teams help individuals find ways to afford the medication they need to survive in this video.
Find Support
Cystic Fibrosis Organizations
There are many organizations that support research and advocacy for cystic fibrosis. See below for a few of those organizations.
Support organizations
Government Organizations
Meet the Team
Accredo’s Cystic Fibrosis care team is dedicated to serving you and we understand the complexity of your condition. Our expert clinicians are available 24 hours a day, seven days a week to answer any questions.
Why We Do It
Accredo supports patients with chronic and complex conditions and helps them live their best life. Watch our video to learn why we do what we do for our patients.