Treatment with Kaftrio, a triple combo of elexacaftor, tezacaftor, and ivacaftor sold as Trikafta in the U.S., may help reduce the number of medications people with cystic fibrosis (CF) take each day, a study found.

Patients may have to take multiple medications to manage all symptoms of CF, so being able to take fewer may offer a way to improve the lives of these patients by reducing treatment burden.

The study, “Effects of elexacaftor-tezacaftor-ivacaftor on daily treatment burden and airflow obstruction in adults with cystic fibrosis,” was published in Pulmonary Pharmacology and Therapeutics by researchers in France.

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CF happens because of mutations in a gene that provides instructions for producing CFTR, a channel protein that helps control the movement of salt and water molecules in and out of cells.

The mutations reduce the amount of CFTR available on the surface of cells or affects the way the protein works. This makes mucus in the body’s organs become too thick, causing problems such as infections and trouble digesting food.

Developed by Vertex Pharmaceuticals, Kaftrio helps put more of the protein on the surface of cells, while making it work more effectively. This makes mucus less sticky and thick, which helps ease the symptoms of CF.

In Europe, its use is indicated for patients ages 6 years and older who have at least one copy of the F508del mutation. In the U.S., its approval covers children as young as 2 and patients with other mutations responsive to treatment.

Study participants were in Nancy, France

To find out if Kaftrio may reduce daily treatment burden, the researchers looked at data from 65 adults with CF who started treatment with Kaftrio sometime between March 2020 and April 2022 in a CF center in Nancy, France. Their mean age was 29.4; of those, 43 (66%) were male.

Nearly half (48%) carried two copies of the F508del mutation and had received previous treatment with Orkambi (ivacaftor/lumacaftor), another CFTR modulator marketed by Vertex. The remaining 34 (52%) patients hadn’t received any treatment with a CFTR modulator.

The majority (97%) of patients had productive cough, or cough with sputum. In 58 (89%), sputum analysis came back positive for Pseudomonas aeruginosa, a type of bacteria that often causes lung infection in people with CF.

Most (95%) had pancreatic insufficiency, which occurs when the pancreas doesn’t release enough enzymes to break down food in the intestines, causing digestive symptoms. For example, about one-third (32%) had diarrhea.

Many (68%) had chronic rhinosinusitis, a long-lasting infection of the nose and the air-filled pockets in the face (sinuses) that usually causes facial pain and a stuffy or runny nose. Nearly half (46%) had allergic bronchopulmonary aspergillosis, which is an allergic reaction to a lung infection with a fungus.

At the beginning of the study, the median number of medications taken each day was 13. There were six patients who also were on long-term oxygen therapy and two on noninvasive ventilation to help with breathing.

Medications decreased after six months of Kaftrio

After six months of treatment with Kaftrio, the number of medications decreased to a median of nine “due to patient decision,” the scientists noted. The proportion of patients who took inhaled corticosteroids in combination with a long-acting beta-agonist was cut by half (62% vs. 31%).

Even greater reductions were observed for inhaled antibiotics (71% vs. 22%) and mucolytics, a type of medication that breaks up and thins mucus so it’s easier to clear from the lungs (23% vs. 2%).

Other significant reductions were observed for nasal corticosteroids, the mucolytic Pulmozyme (dornase alfa), oral antibiotics, medications to treat diarrhea, and proton pump inhibitors that ease acid reflux from the stomach.

The proportion of patients who required long-term oxygen therapy also decreased (9% vs. 2%), as did that of patients on non-invasive ventilation (3% vs. 2%).

Kaftrio also improved lung function

Treatment with Kaftrio also improved lung function. For example, the amount of air patients could exhale in one second (FEV1) improved by 18%, and the total amount of air they could exhale (FVC) improved by 14%.

Pulmonary exacerbations, which are episodes of worsening lung symptoms, were reduced from an average rate of 1.1 in the six months prior to entry to the study, to 0.2 in the six months after starting treatment with Kaftrio.

Taking Kaftrio “can reduce daily treatment burden in adult CF patients with at least one F508del mutation in real-life at 6 months of treatment while maintaining its effectiveness on respiratory symptoms and pulmonary exacerbations rate,” the researchers wrote.

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