Females with cystic fibrosis have a larger decrease in sweat chloride in response to lumacaftor/ivacaftor compared to
B L Aalbers1, R W Hofland1, I Bronsveld1
1Department of Pulmonology, University Medical Center Utrecht, the Netherlands.
Summary
Female cystic fibrosis patients show greater sweat chloride response to CFTR modulators like lumacaftor/ivacaftor. This response did not correlate with lung function (ppFEV1) or BMI changes.
Area of Science:
- Medical research
- Clinical pharmacology
- Genetics
Background:
- Cystic Fibrosis (CF) is a genetic disorder affecting multiple organs.
- CFTR modulators are a new class of drugs targeting the underlying cause of CF.
- Understanding patient response variability is crucial for optimizing CFTR modulator therapy.
Purpose of the Study:
- To identify patient-related factors influencing sweat test response to CFTR modulators.
- To examine the correlation between sweat chloride response and changes in ppFEV1 or BMI.
- To analyze real-world clinical data for insights into treatment efficacy.
Main Methods:
- Retrospective analysis of 160 CF patients treated with lumacaftor/ivacaftor for at least six months.
- Collection of data on age, sweat chloride levels, ppFEV1, weight, and BMI at baseline and after six months.
- Application of Pearson and Spearman correlation tests to assess relationships between variables.
Main Results:
- Females exhibited a significantly larger sweat chloride response (mean difference 10.6 mmol/l) compared to males.
- A modest correlation was observed between patient weight and sweat chloride response, which diminished upon adjustment.
- Sweat chloride response did not correlate with changes in ppFEV1 or BMI after six months of treatment.
Conclusions:
- Female CF patients demonstrate a greater sweat chloride response to lumacaftor/ivacaftor.
- Sex is a significant factor influencing sweat chloride response, potentially explaining weight-related correlations.
- Sweat chloride response to this CFTR modulator does not correlate with ppFEV1 or BMI improvements, suggesting distinct response mechanisms.
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