Cystic fibrosis is a hereditary disease with thick, sticky mucus in the lungs, leading to a decline in lung function over time. Triple combination therapy targets the root causes of the disease and has been approved for children and adults.
A recent study by Charité — Universitätsmedizin Berlin reveals that this new treatment significantly improves the health of school-aged children with cystic fibrosis. The findings have been published in the European Respiratory Journal.
Individuals with cystic fibrosis experience lung congestion due to thick mucus, causing breathing difficulties, infections, and inflammation. Current treatment involves daily inhalation of mucus-thinning medications, antibiotics, and physical therapy.
In 2020, a new approach emerged: triple combination therapy with three drugs – elexacaftor, tezacaftor, and ivacaftor. This treatment, taken orally, was first approved for children aged six and older in 2022 and later for children as young as two in 2023.
Prof. Mirjam Stahl, head of the Division of Cystic Fibrosis at Charité, describes the therapy as a game-changer for children. The three drugs work together to enhance the function of a protein responsible for mucus production in the lungs, significantly improving lung health.
The research team, led by Stahl and Prof. Marcus Mall, conducted a study on 107 children aged six to eleven with cystic fibrosis. The study showed that triple combination therapy reduced lung disease severity, prevented progression, and even restored normal lung function in most children.
Ongoing research aims to evaluate the benefits of this therapy in younger children and explore the possibility of reducing symptom-based treatment during triple combination therapy, which could enhance patients’ quality of life.
Cystic fibrosis is a common fatal hereditary disease globally, affecting thousands in Germany alone. The imbalance of salt and water in the body leads to the production of harmful secretions that damage organs like the lungs and pancreas, affecting life expectancy.
The triple combination therapy of elexacaftor, tezacaftor, and ivacaftor, approved in 2020, is a promising option for nearly 90% of cystic fibrosis patients. Its approval for children as young as two marks a significant advancement in treating this challenging disease.
