Cystic fibrosis transmembrane conductance regulator modulators and the exocrine pancreas: A scoping review

Mitchell L. Ramsey, Susan S. Li, Luis F. Lara, Yevgeniya Gokun, Venkata S. Akshintala, Darwin L. Conwell, John Heintz, Stephen E. Kirkby, Karen S. McCoy, Georgios I. Papachristou, Alpa Patel, Vikesh K. Singh, Phil A. Hart

Research output: Contribution to journalReview articlepeer-review


Background: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators improve pulmonary outcomes in subjects with cystic fibrosis (CF); however, the effects on pancreatic manifestations are not well characterized. We hypothesized that CFTR modulators would improve measures of exocrine pancreatic function and outcomes. Methods: We performed a systematic search to identify studies reporting measures of the exocrine pancreas in humans treated with CFTR modulators. Only studies reporting baseline and on-treatment assessments were included. Results: Of 630 identified studies, 41 met inclusion criteria. CFTR modulators reduced acute pancreatitis events by 85% overall (rate ratio 0.15, 95% confidence interval (CI) 0.04, 0.52), with a greater effect seen in the subgroup with pancreas sufficient CF (PS-CF) (rate ratio 0.13 (95% CI 0.03, 0.53). Among 293 subjects with baseline and on-treatment evaluation of pancreas sufficiency, 253 were pancreas insufficient at baseline and 54 (21.3%) converted to pancreas sufficiency. Of 32 subjects with baseline FE-1 values <200 mcg/g, 16 (50%) increased to ≥200 mcg/g. Serum trypsin decreased by a mean of 565.9 ng/mL (standard deviation (SD) 311.8), amylase decreased by 38.2 U/L (SD 57.6), and lipase decreased by 232.3 U/L (SD 247.7). Conclusions: CFTR modulator use reduces acute pancreatitis frequency and improves indirect measures of exocrine pancreas function. Future interventional studies that evaluate the mechanism and impact of CFTR modulators on acute pancreatitis and pancreas sufficiency in patients with CFTR dysfunction are warranted.

Original languageEnglish (US)
Pages (from-to)193-200
Number of pages8
JournalJournal of Cystic Fibrosis
Issue number2
StatePublished - Mar 2023


  • Acute pancreatitis
  • Exocrine pancreatic insufficiency
  • Fecal elastase
  • Lipase
  • Trypsin

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine
  • Pediatrics, Perinatology, and Child Health


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