Ivacaftor modifies cystic fibrosis neutrophil phenotype in subjects with R117H residual function CFTR mutations

Gareth R Hardisty,Edward F Mckone,Pradeep K Singh,Robert D Gray,Sheonagh M Law,Brenda Grogan,Suzanne Carter

doi:10.1183/13993003.02161-2020

Abstract

CFTR modulation leads to changes in neutrophil phenotype even in patients with residual function CFTR mutations https://bit.ly/2EUk7xH

Highlights

The St Vincent’s Healthcare Ethics and Medical Research Committee, Dublin, Ireland, approved the study. 10 clinically stable cystic fibrosis (CF) subjects with one copy of the R117H allele and a second disease causing mutation were recruited
And even before treatment, R117H neutrophils had similar survival rates to healthy controls at 24 h. This is in contrast to neutrophils from class I–III mutation subjects with CF, where 40% were viable at 24 h
@ERSpublications CFTR modulation leads to changes in neutrophil phenotype even in patients with residual function CFTR mutations https://bit.ly/2EUk7xH

Summary

Introduction

The St Vincent’s Healthcare Ethics and Medical Research Committee, Dublin, Ireland, approved the study. 10 clinically stable CF subjects with one copy of the R117H allele and a second disease causing mutation were recruited. Treatment with ivacaftor had no significant impact on total isolated granulocyte number (figure 1a) or frequency of circulating neutrophils in peripheral blood (figure 1b). @ERSpublications CFTR modulation leads to changes in neutrophil phenotype even in patients with residual function CFTR mutations https://bit.ly/2EUk7xH

Results

Conclusion