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Observational study shows significant decline in systemic inflammation levels after treatment with ETI medication.
Cystic fibrosis (CF) is the most common inherited metabolic disease in Europe. More than 8,000 people in Germany are affected. Every year, around 150 to 200 children are born with this incurable disease. It is caused by a defect in the gene for the CFTR transport channel. This disrupts the salt and water balance in the body and leads to thick mucus that can block various organs, including the lungs. This results in repeated infections and inflammation, and lung function gradually deteriorates. Until 2020, lung disease in CF was treated exclusively symptomatically with mucolytic inhalations and antibiotics. Since then, however, a drug has been available that combines the active ingredients elexacaftor, tezacaftor, and ivacaftor (ETI), known as CFTR modulators.
The triple therapy improves the function of the transport channel and helps to reduce the formation of thick mucus. Now, researchers led by Prof. Dr. Anna-Maria Dittrich, senior physician at the Department for Pediatric Pneumology, Allergology, and Neonatology at Hannover Medical School (MHH), in cooperation with the German Center for Lung Research (DZL) and Charité Universitätsmedizin Berlin, have investigated the long-term effects of ETI treatment. The multicenter observational study MODULATE-CF shows that inflammation levels in those treated fell to around 40 to 80 percent of baseline values after just three months. At the same time, lung function values improved significantly and sustainably. The results have been published in the journal European Respiratory Journal.
Study under real-world conditions
Well over 2,000 mutations of the CFTR gene are known. However, 85 to 90 percent of all patients with cystic fibrosis in Germany have the same mutation. This is where the triple drug comes in. Previous studies have already shown that ETI improves lung function and alleviates symptoms. However, until now, there has been a lack of data on how inflammatory activity develops over longer periods of time. The current study is based on biomaterials and clinical data from a CF cohort of the German Center for Lung Research, ‘MODULATE-CF’, with a total of 198 participants aged six and older, which provides a particularly robust data basis. After just three months, a significant decrease in central inflammatory markers in the blood was already evident. This effect persisted in many participants over a period of two years. ‘It is particularly noteworthy that the study was conducted in the context of real-world medical care,’ emphasizes Dr. Olga Halle, human biologist at the Children's Hospital and scientist at the DZL site BREATH Hannover. ‘It thus confirms that the positive effects of triple therapy do not only occur under the previously selected, controlled conditions of clinical trials, but can also be seen in the everyday lives of patients,’ says the co-first author.
Better assessment of disease progression
Previous reports have already shown that triple therapy is superior to older dual modulator combinations with the active ingredients lumacaftor and ivacaftor, for example in terms of improved lung function. The data now available allow the effect of ETI to be described much more precisely: it not only improves the removal of mucus from the lungs, but also appears to influence fundamental biological processes of the disease – in particular chronic inflammation, which plays a central role in long-term tissue damage. ‘With this study, we are showing for the first time in everyday care that triple therapy not only relieves the lungs, but also reduces systemic inflammation in the long term – an important step in understanding the biological benefits of this treatment,’ says Professor Dittrich.
The results also provide a solid scientific basis for assessing how sustainably ETI can influence the course of the disease. This is particularly important for young patients, whose long-term prognosis depends heavily on controlling inflammation at an early stage. Nevertheless, despite significant improvements, a certain amount of residual inflammation remained in many of the patients in the study. The work therefore underscores not only the importance of precise long-term observation, but also the need to further develop future treatment strategies. ‘The data suggest that complementary anti-inflammatory or anti-infective approaches could be valuable in further reducing residual inflammatory activity,’ notes the pediatric pulmonologist.
SERVICE:
Further information is available from Prof. Dr. Anna-Maria Dittrich, dittrich.anna-maria@mh-hannover.de.
The original paper, ‘Longitudinal real-world effects of elexacaftor/tezacaftor/ivacaftor on systemic inflammation in cystic fibrosis,’ can be found here: https://publications.ersnet.org/content/erj/66/6/2500150
In cystic fibrosis, a genetic defect causes lung function to gradually deteriorate. Triple therapy h ...
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