Heterogeneity of CFTR modulator-induced sweat chloride concentrations in people with cystic fibrosis.

ABSTRACT:

Background

Sweat chloride (SC) concentrations in people with cystic fibrosis (PwCF) reflect relative CF transmembrane conductance regulator (CFTR) protein function, the primary CF defect. Populations with greater SC concentrations tend to have lesser CFTR function and more severe disease courses. CFTR modulator treatment can improve CFTR function within specific CF genotypes and is commonly associated with reduced SC concentration. However, SC concentrations do not necessarily fall to concentrations seen in the unaffected population, suggesting potential for better CFTR treatment outcomes. We characterized post-modulator SC concentration variability among CHEC-SC study participants by genotype and modulator.

Methods

PwCF receiving commercially approved modulators for ≥90 day

SUBMITTER: Zemanick ET 

PROVIDER: S-EPMC11322419 | biostudies-literature | 2024 Jul

REPOSITORIES: biostudies-literature