Cystic fibrosis

Overview

What is cystic fibrosis?1, 2

Cystic fibrosis (CF) is a progressive, genetically-inherited disease impacting the cells that produce mucus, sweat, and digestive juices. Rather than being thin and slippery lubricants, these secreted fluids become sticky and thick because of a defective CF gene. This causes the secretions to obstruct tubes, ducts, and passageways, especially in the lungs and pancreas.

In the lungs, it enables bacteria to stick and multiply more easily, increasing the risk of infection. The lungs become obstructed, making breathing more difficult. In the pancreas, scarring from blocked ducts reduces the absorption of fats and vitamins. Furthermore, blockages in the liver can result in liver disease, and increased mucus in the reproductive tract can cause decreased fertility or infertility in men and women.

How common is cystic fibrosis?1, 2, 3

CF is most common in Caucasian patients, but it is growing more diverse every year. In 2022, more than 15% of people with CF were identified as a non-Caucasian race. It is anticipated that this trend towards increased diversity will continue.

In the United States alone, there are about 30,000 people with cystic fibrosis, and there are an estimated 70,000 people with CF worldwide. Approximately 1 out of every 30 Americans is a CF carrier. Because of this, every state in the U.S. routinely screens newborns for CF, to allow for early diagnosis and treatment.


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

855-315-3408

  • 1a1b"Cystic Fibrosis." Mayo Clinic, Mayo Foundation for Medical Education and Research, 5 July 2023. .<a href="www.mayoclinic.org/diseases-%20conditions/cystic-fibrosis/symptoms-causes/syc-20353700" target="_blank">Mayo Clinic</a>
  • 2a2b"Cystic Fibrosis (CF)." American Lung Association, 5 July 2023. <a href="https://www.lung.org/lung-health-diseases/lung-disease-lookup/cystic-%20fibrosis/learn-about-cystic-fibrosis" target="_blank">American Lung Association</a>
  • 32022 Cystic Fibrosis Foundation Patient Registry Highlights. Bethesda, Maryland. &copy;2023 Cystic Fibrosis Foundation. Accessed July 5, 2023.

Symptoms

What are the symptoms of cystic fibrosis?1

  • Symptoms can vary in severity and based on age:
    • Persistent coughing, at times with phlegm
    • Salty-tasting skin
    • Frequent respiratory and/or sinus infections
    • Wheezing/shortness of breath
    • Poor growth or limited weight gain, in spite of a good appetite
    • Frequent greasy stools, or bowel movements
    • Nasal polyps
    • Clubbed fingers
    • Male infertility

How is it diagnosed?1

All newborns are screened for cystic fibrosis (CF) in the United States. This is done using a heel prick and testing for Immunoreactive Trypsinogen (IRT). IRT levels can be elevated due to CF, prematurity, stressful birth, and other reasons, so additional testing is performed to confirm CF. One confirmatory test is known as a sweat test, where the amount of salt in the baby’s sweat is measured.

In addition to newborn screening, prenatal genetic testing may be available prior to birth.

  • 1a1b"Newborn Screening for CF." CF Foundation, 5 July 2023. <a href="https://www.cff.org/intro-cf/newborn-screening-cf" target="_blank">Cystic Fibrosis Foundation</a>

Medications

Cystic Fibrosis

The following specialty medications are available at Accredo, a specialty pharmacy for cystic fibrosis.

Medication Manufacturer
Bethkis® (tobramycin inhalation solution) Chiesi Catalent Pharm
Bronchitol® (mannitol inhalation powder) Chiesi USA, Inc.
Cayston® (aztreonam inhalation solution) Gilead Sciences, Inc.
Kalydeco™ (ivacaftor) tablets for oral use, oral granules Vertex Pharmaceuticals, Inc.
Kitabis Pak™ (tobramycin inhalation solution) PARI Respiratory, Inc.
Pulmozyme® (dornase alfa inhalation solution) Genentech USA, Inc.
Symdeko® (tezacafter/ivacaftor) tablets for oral use Vertex Pharmaceuticals, Inc.
Tobi Podhaler™ (tobramycin inhalation powder) Novartis Pharmaceuticals Corp.
tobramycin (generic) Various
Trikafta™ (elexacaftor, ivacaftor and tezacaftor) tablets for oral use, oral granules Vertex Pharmaceuticals, Inc.

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


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.

Why We Do It video transcript

24-hour Customer Service Center

855-315-3408