Ivacaftor in adults with cystic fibrosis: a ten-year follow-pu of lung function and nutritional sttus
Abstract
Introduction: Causal therapy of cystic fibrosis (CF) became a clinical reality in 2012. Long-term results of ivacaftor treatment are the subject of current clinical research.
Methods: Participants in the STRIVE and PERSIST studies were included in the study. Demographic and clinical data were obtained from medical records covering the entire period of ivacaftor treatment from 9 to 10 years.
Changes in lung function were compared to the period before treatment. Results: Four adults with CF heterozygous for the G551D mutation were included in the study.
Stabilization of lung function (median decrease in FEV1 0.6% of baseline / year) and nutritional status (median increase in BMI 0.01 kg / m2 / year) was observed. Compared with the pre-treatment period, the change in FEV1 decrease (4.6% of baseline / year before treatment) tended to be statistically significant (p = 0.068).
Conclusion: Treatment with ivacaftor resulted in stabilization of lung function and nutritional status for ten years.