Trikafta for Cystic Fibrosis
Cystic fibrosis is an inherited genetic disease caused by a defect in the protein channel found in many organs in the body. This results in much thicker secretions and electrolyte imbalances. This causes issues like difficulty breathing. It has traditionally been treated with anti-inflammatories, antibiotics, medications to thin the mucus and more. While those therapies improve the symptoms of cystic fibrosis, they do not slow down the disease overall.
Trikafta treats the mutation causing cystic fibrosis. It not only improves symptoms but slows the progression of the disease overall. It has been a breakthrough medication for people with cystic fibrosis, allowing them to plan on living longer and to participate in life more fully.
What is Trikafta?
Trikafta combines 3 medications – elexacaftor, ivacaftor and tezacaftor – to treat patients with the most common cystic fibrosis mutation. It targets the defect from specific genetic mutations that cause cystic fibrosis, slowing the progression of the disease.
Who can have Trikafta?
Patients at least 2 years old with at least 1 F508del mutation may be able to take Trikafta. This is estimated to be about 85-90% of the people living with cystic fibrosis. Talk with your doctor to see if your gene mutation qualifies.
Sarah Johnson began taking Trikafta in 2018 as part of a clinical trial at the health system. She describes how the medication has allowed her to accomplish things she didn’t think were possible with cystic fibrosis.
I was considering quitting my job because my health was declining.
Sarah Johnson has cystic fibrosis, an inherited life-threatening disorder that damages the lungs and digestive tract. In 2018, Sarah's condition made doing her job almost impossible. She's a special education teacher in Grain Valley. The thought of quitting was devastating. Then she got a call from The University of Kansas Health System about a new clinical trial for a drug called Trikafta.
As someone with a rare disease, it's our way to gain control in an uncontrollable situation.
Sarah started seeing results immediately.
I noticed that I could walk as fast as others. I noticed that I could pick up and move things at my work, keep up with my students at my job. I noticed an overall quality of life increase that is just remarkable.
Sarah said Trikafta gave her a life she never imagined possible for someone with cystic fibrosis.
I am now able to have a social life. I can go out at night. I don't get tired easily. I participated in a 5K since then, which is incredible, something I've never done before.
Sarah said participating in a clinical trial is how cures are found. Until then, Trikafta offers her a longer, more normal life.
I am so grateful to be a part of the research here at The University of Kansas Hospital. And more importantly, I am so thankful that my doctors and nurses are here at KU. We get the best care. We get the most immediate care, and there's passion in everything that they do.
How does Trikafta work?
Trikafta is taken orally. It contains compounds called cystic fibrosis transmembrane conductance regulator (CFTR) modulators, which directly modify the CFTR protein. These drugs bind to the CFTR proteins that aren’t working correctly and kick-start them to do what they’re supposed to. Learn more about how Trikafta works from the manufacturer.
Benefits and risks of Trikafta
The biggest benefit of taking Trikafta is a significant increase in lung function for most patients. Additionally, it has been shown to:
- Improve pulmonary function
- Reduce cystic fibrosis exacerbations
- Enhance quality of life
As with any medication, there is a risk of potential side effects. The most common ones with Trikafta are:
- Upper respiratory tract infection (common cold), including stuffy and runny nose
- Stomach (abdominal) pain
- Increase in liver enzymes
- Increase in a blood enzyme called creatine phosphokinase
- Flu (influenza)
- Inflamed sinuses
- Increase in blood bilirubin
Your doctor will request regular blood tests to check your liver function while taking Trikafta. Some patients may also need to get vision exams to check for cataracts while taking the medication.
What happens during Trikafta treatment?
Trikafta is taken at home, by mouth, as either granules or tablets. They must be taken twice a day – once in the morning and once in the evening. Granules are mixed with soft food or liquids, while tablets are swallowed whole.
Why choose us for cystic fibrosis treatment with Trikafta?
The University of Kansas Health System actively participates in clinical trials to ensure we are at the leading edge of care. This is evidenced by our participation as 1 of 115 sites for the Trikafta clinical trial. We will continue to pursue research opportunities that benefit our patients.
Additionally, we are a nationally accredited cystic fibrosis care center that cares for both adult and pediatric cystic fibrosis patients. The adult cystic fibrosis clinic was founded in 1993 and was accredited by the Cystic Fibrosis Foundation in 1996. We have grown to become one of the largest cystic fibrosis adult treatment centers in the Midwest region.
Our staff are well-versed in working with the specialty pharmacies that provide Trikafta. Additionally, because this medication can be quite expensive, our team has developed resources to work with third-party payers as well as to help patients access financial assistance or copay programs.
We are focused on patient care and welcome community-based collaboration, quickly and routinely providing telephone and email referring-provider consultations.
Leading research and clinical trials
As part of one of the nation's premier academic medical centers, our care providers are committed to research and scientific discovery through the University of Kansas Medical Center. We can often include our patients in potentially lifesaving clinical trials and treatment options not available anywhere else.