Changes in fecal elastase-1 following initiation of CFTR modulator therapy in pediatric patients with cystic fibrosis.
Document Type
Article
Publication Date
11-2023
Identifier
DOI: 10.1016/j.jcf.2023.09.005
Abstract
BACKGROUND: Improvement in exocrine pancreatic function in persons with CF (pwCF) on cystic fibrosis transmembrane conductance regulator (CFTR) modulators has been documented in clinical trials using fecal pancreatic elastase-1 (FE-1). Our group endeavored to evaluate real-world data on FE-1 in children on CFTR modulator therapy at three pediatric cystic fibrosis (CF) centers.
METHODS: Pediatric pwCF were offered FE-1 testing if they were on pancreatic enzyme replacement therapy (PERT) and on CFTR modulator therapy according to their center's guideline. FE-1 data were collected retrospectively. The primary outcome was absolute change in FE-1.
RESULTS: 70 pwCF were included for analysis. 53 had baseline and post-modulator FE-1 values. There was a significant increase in FE-1 from median 25 mcg/g (IQR 25-60) at baseline to 57 mcg/g (IQR 20-228) post-modulator (p < 0.001 by Wilcoxon matched pairs), with an absolute change in FE-1 of median 28 mcg/g (IQR -5-161) and mean 93.5 ± 146.8 mcg/g. Age was negatively correlated with change in FE-1 (Spearman r=-0.48, p<0.001). 15 pwCF (21%) had post-modulator FE-1 values ≥200 mcg/g, consistent with pancreatic sufficiency (PS). The PS group was significant for younger age at initiation of first CFTR modulator and a higher baseline FE-1.
CONCLUSIONS: Most pwCF experienced an increase in FE-1 while receiving CFTR modulator treatment and a small percentage demonstrated values reflective of PS. These data suggest that PS may be attained in those that initiated modulator therapy at a younger age or had a higher baseline FE-1. FE-1 testing is suggested for children on any CFTR modulator therapy.
Journal Title
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
Volume
22
Issue
6
First Page
996
Last Page
1001
MeSH Keywords
Humans; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Retrospective Studies; Pancreas; Pancreatic Elastase; Mutation
Keywords
CFTR modulators; Exocrine pancreatic function; Fecal elastase; Pancreatic insufficiency; Pancreatic sufficiency
Recommended Citation
Stephenson KG, Lingle AJ, Baumberger KA, et al. Changes in fecal elastase-1 following initiation of CFTR modulator therapy in pediatric patients with cystic fibrosis. J Cyst Fibros. 2023;22(6):996-1001. doi:10.1016/j.jcf.2023.09.005
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