Treatment burden in patients with at least one class IV or V CFTR mutation

Last updated on 23-3-2022 by Wesley Van Dessel

Peer reviewed scientific article



Article written during project(s) : 


CFTR mutations are grouped according to disease-causing mechanism. Several studies demonstrated that patients having at least one mutation of class IV/V, present with a milder phenotype, but little is known about their relative treatment burden. We compared treatment burden between patients with two class I, II, or III mutations and patients with at least one mutation of class IV/V in the 2010 database of the Belgian CF Registry. We calculated a “Treatment Burden Index” (TBI) by assigning long term therapies to categories low, medium and high intensity, for differential weighing in the tota…

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