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.
Subject(s)
Cystic Fibrosis , Child , Humans , Cystic Fibrosis/drug therapy , Cystic Fibrosis Transmembrane Conductance Regulator/metabolism , Mutation , Pancreas , Pancreatic Elastase/metabolism , Retrospective StudiesABSTRACT
PURPOSE: The purpose of this study was to develop and validate a novel medication regimen complexity-intensive care unit (MRC-ICU) scoring tool in critically ill patients and to correlate MRC with illness severity and patient outcomes. METHODS: This study was a single-center, retrospective observational chart review of adults admitted to the medical ICU (MICU) between November 2016 and June 2017. The primary aim was the development and internal validation of the MRC-ICU scoring tool. Secondary aims included external validation of the MRC-ICU and exploration of relationships between medication regimen complexity and patient outcomes. Exclusion criteria included a length of stay of less than 24 hours in the MICU, active transfer, or hospice orders at 24 hours. A total of 130 patient medication regimens were used to test, modify, and validate the MRC-ICU tool. RESULTS: The 39-line item medication regimen complexity scoring tool was validated both internally and externally. Convergent validity was confirmed with total medications (p < 0.0001). Score discriminant validity was confirmed by lack of association with age (p = 0.1039) or sex (p = 0.7829). The MRC-ICU score was significantly associated with ICU length of stay (p = 0.0166), ICU mortality (p = 0.0193), and patient acuity (p < 0.0001). CONCLUSION: The MRC-ICU scoring tool was validated and found to correlate with length of stay, inpatient mortality, and patient acuity.