Association between CFTR modulators and changes in iron deficiency markers in cystic fibrosis
Document Type
Article
Publication Date
9-2024
Institution/Department
Internal Medicine
Journal Title
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
MeSH Headings
Humans; Cystic Fibrosis (drug therapy, blood, epidemiology); Male; Female; Retrospective Studies; Cystic Fibrosis Transmembrane Conductance Regulator (genetics); Biomarkers (blood); Adult; Child; Adolescent; United States (epidemiology); Anemia, Iron-Deficiency (epidemiology, drug therapy, blood, diagnosis, etiology); Chloride Channel Agonists (therapeutic use); Iron Deficiencies; Quinolones (therapeutic use); Iron (blood, metabolism)
Abstract
BACKGROUND: Iron deficiency (ID) is a common extrapulmonary manifestation in cystic fibrosis (CF). CF transmembrane conductance regulator (CFTR) modulator therapies, particularly highly-effective modulator therapy (HEMT), have drastically improved health status in a majority of people with CF. We hypothesize that CFTR modulator use is associated with improved markers of ID. METHODS: In a multicenter retrospective cohort study across 4 United States CF centers 2012-2022, the association between modulator therapies and ID laboratory outcomes was estimated using multivariable linear mixed effects models overall and by key subgroups. Summary statistics describe the prevalence and trends of ID, defined a priori as transferrin saturation (TSAT) < 20 % or serum iron < 60 μg/dL (< 10.7 μmol/L). RESULTS: A total of 568 patients with 2571 person-years of follow-up were included in analyses. Compared to off modulator therapy, HEMT was associated with +8.4 % TSAT (95 % confidence interval [CI], +6.3-10.6 %; p < 0.0001) and +34.4 μg/dL serum iron (95 % CI, +26.7-42.1 μg/dL; p < 0.0001) overall; +5.4 % TSAT (95 % CI, +2.8-8.0 %; p = 0.0001) and +22.1 μg/dL serum iron (95 % CI, +13.5-30.8 μg/dL; p < 0.0001) in females; and +11.4 % TSAT (95 % CI, +7.9-14.8 %; p < 0.0001) and +46.0 μg/dL serum iron (95 % CI, +33.3-58.8 μg/dL; p < 0.0001) in males. Ferritin was not different in those taking modulator therapy relative to off modulator therapy. Hemoglobin was overall higher with use of modulator therapy. The prevalence of ID was high throughout the study period (32.8 % in those treated with HEMT). CONCLUSIONS: ID remains a prevalent comorbidity in CF, despite availability of HEMT. Modulator use, particularly of HEMT, is associated with improved markers for ID (TSAT, serum iron) and anemia (hemoglobin).
First Page
878
Last Page
884
Recommended Citation
Jia, Shijing; Wang, Yizhuo; Ross, Melissa H.; Zuckerman, Jonathan B.; Murray, Susan; Han, MeiLan K.; Cahalan, Shannon E.; Lenhan, Blair E.; and Best, Ryan N., "Association between CFTR modulators and changes in iron deficiency markers in cystic fibrosis" (2024). MaineHealth Maine Medical Center. 4047.
https://knowledgeconnection.mainehealth.org/mmc/4047