NEJM: "combination of three drugs" to improve lung function in CF patients
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Last Update: 2019-11-05
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Source: Internet
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Author: User
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Recently, a phase III clinical trial led by researchers from the Southwest Medical Center of the University of Texas found that the combination of three drugs can improve lung function and reduce symptoms in patients with cystic fibrosis (CF) caused by single gene mutation The FDA approved the treatment earlier this month based on the findings, which were recently published in the New England Journal of medicine A similar study, also published in the journal Lancet, reported the results of treatment for symptoms in patients with cystic fibrosis caused by other mutations Dr Raksha Jain, associate professor of internal medicine of Southwest Medical Center, is the corresponding author of NEJM article and also participated in the study of Lancet CF is a chronic, progressive and fatal genetic disease that affects the respiratory and digestive systems of children and young people Sweat glands and the reproductive system are also usually involved People with CF have a shorter life span than normal people "Despite thousands of different pathogenic mutations, nearly 90% of patients with cystic fibrosis have at least one of the most common mutations, a deletion mutation of phenylalanine 508 in the CFTR allele," said Dr Jain Dr Jain said an estimated 80000 people worldwide are affected by mutations in the CFTR protein "The combination therapy of these three drugs can improve physical health and relieve symptoms for patients with 508 deletion mutant of CFTR gene." The study was conducted in 115 locations in 13 countries from June 2018 to April 2019 Among them, 403 patients aged 12 and over were randomly treated with elexacaftor tezacaftor ivacaftor combined therapy or placebo The trial was co sponsored by vertex pharma Patients were scheduled for pulmonary function tests at weeks 4 and 24 Compared with patients receiving placebo, the pulmonary function of patients in the treatment group improved significantly at the 4th week, and the effect lasted until the 24th week The study participants also answered questionnaires about their quality of life and respiratory symptoms - respondents in the treatment group scored higher in these areas.
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