Current opinion in pulmonary medicine · 2024
Epidemiology of advanced cystic fibrosis lung disease in the modulator era.
СтатьяОбзорПоддержка исследования: вне правительства СШАНазвание и аннотация: Перевод пока не подготовлен
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Westhoff Julia, Naehrlich Lutz
Аннотация
PURPOSE OF REVIEW: Advanced cystic fibrosis lung disease remains the main cause of death in people with cystic fibrosis (pwCF). Cystic fibrosis transmembrane regulator (CFTR) modulators have changed the disease burden for eligible pwCF with access to this therapy.
RECENT FINDINGS: Real-world data show that there are no safety concerns for patients with advanced cystic fibrosis lung disease treated with highly effective triple CFTR modulator therapy. The improvements are comparable to those in other people with cystic fibrosis and in part even better. Mortality and rates of lung transplantation have decreased since the approval of CFTR modulator therapy and, especially, highly effective triple CFTR modulator therapy. Nevertheless, at least 10% of people with cystic fibrosis are not eligible for highly effective CFTR modulator therapy, and the development of alternative treatments remains important.
SUMMARY: The approval of highly effective CFTR modulator therapies has been a breakthrough in treatment for most people with cystic fibrosis, especially those with advanced lung disease, improving survival and reducing the burden of the disease.
RECENT FINDINGS: Real-world data show that there are no safety concerns for patients with advanced cystic fibrosis lung disease treated with highly effective triple CFTR modulator therapy. The improvements are comparable to those in other people with cystic fibrosis and in part even better. Mortality and rates of lung transplantation have decreased since the approval of CFTR modulator therapy and, especially, highly effective triple CFTR modulator therapy. Nevertheless, at least 10% of people with cystic fibrosis are not eligible for highly effective CFTR modulator therapy, and the development of alternative treatments remains important.
SUMMARY: The approval of highly effective CFTR modulator therapies has been a breakthrough in treatment for most people with cystic fibrosis, especially those with advanced lung disease, improving survival and reducing the burden of the disease.