Mukoviszidose – eine Erfolgsgeschichte der Kinderheilkunde

Few diseases in pediatrics have changed as much as cystic fibrosis. Approximately 50 years ago most patients were diagnosed in the first years of life. With a life expectancy of < 18 years most affected children had less than 15 years before they died. In 2020 the life expectancy is > 50 years, so that patients, who are diagnosed predominantly in the first weeks of life, can often live longer than 50 years. In addition, the morbidity has been greatly reduced especially in childhood and adolescence, so that many more years of life are experienced with a good quality of life. This article provides an overview of the current state of knowledge regarding the disease of the lungs in cystic fibrosis, including the changes that can be achieved by early diagnosis, comprehensive care and novel forms of treatment. It describes the examinations necessary in order to address the course of the lung disease as comprehensively as possible and possible intersections between care in specialized cystic fibrosis centers and care by a general pediatrician. The interaction of the highly efficient diagnostic algorithms presented, comprehensive care structures and multimodal therapeutics has led to the fact that the majority of affected patients can nowadays lead a productive adult life. Furthermore, the newest therapeutic developments in the form of cystic fibrosis transmembrane regulator (CFTR) modulators already enable individualized reduction of the very time-consuming complex respiratory treatment, particularly for pediatric patients and in the medium-term promise the achievement of an almost normal life expectancy. A special focus is therefore on information about CFTR modulators, with which the success story of the treatment of cystic fibrosis in pediatric medicine will be continued unrestrained.