Savi D; Schiavetto S; Simmonds NJ; Righelli D; Palange P.

Journal of Cystic Fibrosis. 18(3):420-424, 2019 05.

The combination of the corrector lumacaftor with the potentiator ivacaftor
has been approved for treatment of cystic fibrosis (CF) patients
homozygous for the Phe508del CFTR mutation. There are no reports detailing
the effect of lumacaftor-ivacaftor on physical activity (PA) and exercise
tolerance. We performed incremental cardiopulmonary exercise testing
(CPET) and we assessed PA pre- and post 2years initiation of
lumacaftor-ivacaftor in three CF adults. PA of mild intensity improved by
+13% in patient 1, + 84% in patients 2 and+89% in patient 3. Oxygen uptake
increased both at anaerobic threshold and at peak exercise (patient 1+33%,
patient 2+42% and patient 3+20%). Daily physical activities and exercise
tolerance improved after two years of lumacaftor-ivacaftor therapy.