Therapie der Mukoviszidose – neue Medikamente geben Hoffnung

 

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Therapieansatz Bis vor kurzem bestand die medikamentöse Therapie der Mukoviszidose nur aus einer rein symptomatischen Therapie, aber der Defekt des Chloridkanals konnte nicht beeinflusst werden. Mit der CFTR-Modulatortherapie besteht nun erstmals eine ursächliche und effiziente Therapie, die eine Verbesserung der Lungenfunktionswerte erzielt wie keine der bisherigen Therapien.

Abstract

For decades there were only symptom-oriented therapies for the monogenetic disease cystic fibrosis. With the new modulator therapies there are new hopes to better influence the course of the disease, which has its manifestation in several organs such as the lungs, the liver or pancreas.

In addition to ivacaftor, lumacaftor/ivacaftor and tezacaftor/ivacaftor, a triple combination with elexacaftor/tezacaftor/ivacaftor was developed. Studies in F508del homozygous and heterozygous patients with cystic fibrosis have resulted in significant clinical improvement and have therefore been approved by the FDA for the first time in the USA.

The results are very promising as already after 4 weeks a significant improvement of FEV1 and a significant decrease of the sweat chloride content could be detected. In the USA all patients with at least one F508del mutation can be prescribed the drug. This corresponds to approximately 90 % of all patients with cystic fibrosis and has the potential to change the landscape of cystic fibrosis.

Publication History

Article published online:
06 October 2020

© 2020. Thieme. All rights reserved.

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