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 multiple organs such as the lungs, pancreas, and liver. In the lungs, it enables bacteria to adhere and multiply more easily, increasing the risk of infection. It also obstructs the lungs, making breathing more difficult. In the pancreas, the mucus prevents pancreatic enzymes secretions used to digest 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.
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. Due to these advancements, for the first time ever there are currently more adults living in the US today with CF than 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.1
How common is cystic fibrosis
CF is most common in Caucasian patients, affecting 1 out of every 3,000 births. Latin Americans have a rate of 1 out of 4,000 to 10,000 births, and African Americans have a rate of 1 out of 15,000 to 20,000 births. About 1,000 children are born with CF every year in the United States.2
The Cystic Fibrosis Foundation displays 29,887 people with CF in the United States in their registry as of 2017. The worldwide total of people with CF is estimated to be around 70,000 people.2
Questions about your cystic fibrosis therapy
You can reach the Accredo Cystic Fibrosis care team, anytime, day or night, seven days a week by phone or online.
24-hour Customer Service Center
(800) 803-2523
Send a Message
Note: Most messages received via the online contact form will be responded to within one business day of receipt, Monday - Friday (excluding holidays). For urgent matters, please call us for immediate support.
1. Cystic Fibrosis Foundation Patient Registry. 2016 Annual Data Report. Bethesda, Maryland. ©2017 Cystic Fibrosis Foundation. Accessed January 25, 2019.
2. “Cystic Fibrosis Foundation.” CF Foundation, Cystic Fibrosis Foundation, 2017, www.cff.org/What-is-CF/About-Cystic-Fibrosis/.
Symptoms
What are the symptoms of cystic fibrosis3,4
- 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
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).
3. “Cystic Fibrosis (CF).” American Lung Association, 2018, www.lung.org/lung-health-and-diseases/lung-disease-lookup/cystic-fibrosis/. Accessed May 2019.
4. “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. Accessed May 2019.
Medications
Cystic fibrosis
Accredo, a specialty pharmacy for cystic fibrosis, dispenses specialty and non-specialty cystic fibrosis medications including (but not limited to):
| Drug | Generic/Biosimilar | Manufacturer |
|---|---|---|
| Bethkis® (tobramycin inhalation solution) | Chiesi Catalent Pharm | |
| Cayston® (aztreonam inhalation solution) | Gilead Sciences, Inc | |
| Kalydeco™ (ivacaftor) | Vertex Pharmaceuticals | |
| Kitabis Pak™ (tobramycin inhalation solution) | Generic(s) Available | OSI Pharmaceuticals |
| Orkambi® (lumacaftor/ivacaftor) | Vertex Pharmaceuticals | |
| Pulmozyme® (dornase alfa) | Genentech, Inc | |
| Symdeko™ (texacaftor/ivacaftor) | Vertex Pharmaceuticals | |
| Tobi Podhaler™ (tobramycin inhalation powder) | Novartis Pharmaceuticals Corp | |
| Tobi® (tobramycin) | Generic(s) Available | Novartis Pharmaceuticals Corp |
| Trikafta™ (elexacaftor, ivacaftor and tezacaftor) | Vertex Pharmaceuticals |
Financing Your Care
Financial assistance coordination may be available to help with your medication costs, including manufacturer and community programs. Accredo representatives are available to help find a program that may work for you.
- 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/Symdeko® Vertex Copay Card Program | 877-752-5933
- Healthwell Foundation | 800-675-8416
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
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 has a dedicated team that supports Cystic Fibrosis. We understand the complexity of the disease and are here to care for patients throughout their journey. You can reach the CF dedicated team Monday – Friday 8am – 8pm at 855-315-3408.
24-hour Customer Service Center
(855) 315-3408
Send a Message
Note: Most messages received via the online contact form will be responded to within one business day of receipt, Monday - Friday (excluding holidays). For urgent matters, please call us for immediate support.