Cystic Fibrosis Transmembrane Conductance Regulator Modulator Use Is Associated with Reduced Pancreatitis Hospitalizations in Patients with Cystic Fibrosis

Mitchell L. Ramsey, Yevgeniya Gokun, Lindsay A. Sobotka, Michael R. Wellner, Kyle Porter, Stephen E. Kirkby, Susan S. Li, Georgios I. Papachristou, Somashekar G. Krishna, Peter P. Stanich, Phil A. Hart, Darwin L. Conwell, Luis F. Lara

Research output: Contribution to journalArticlepeer-review

7 Scopus citations

Abstract

INTRODUCTION:Acute pancreatitis (AP) occurs among patients with pancreas-sufficient cystic fibrosis (PS-CF) but is reportedly less common among patients with pancreas-insufficient cystic fibrosis (PI-CF). The incidence of AP may be influenced by cystic fibrosis transmembrane conductance regulator (CFTR) modulator use. We hypothesized that CFTR modulators would reduce AP hospitalizations, with the greatest benefit in PS-CF.Methods:MarketScan (2012-2018) was queried for AP hospitalizations and CFTR modulator use among patients with CF. Multivariable Poisson models that enabled crossover between CFTR modulator treatment groups were used to analyze the rate of AP hospitalizations on and off therapy. Pancreas insufficiency was defined by the use of pancreas enzyme replacement therapy.Results:A total of 10,417 patients with CF were identified, including 1,795 who received a CFTR modulator. AP was more common in PS-CF than PI-CF (2.9% vs 0.9%, P = 0.007). Overall, the observed rate ratio of AP during CFTR modulator use was 0.33 (95% confidence interval [CI] 0.10, 1.11, P = 0.07) for PS-CF and 0.38 (95% CI 0.16, 0.89, P = 0.03) for PI-CF, indicating a 67% and 62% relative reduction in AP hospitalizations, respectively. In a subset analysis of 1,795 patients who all had some CFTR modulator use, the rate ratio of AP during CFTR modulator use was 0.36 (95% CI 0.13, 1.01, P = 0.05) for PS-CF and 0.53 (95% CI 0.18, 1.58, P = 0.26) for PI-CF.DISCUSSION:CFTR modulator use is associated with a reduction in AP hospitalizations among patients with CF. These observational data support the prospective study of CFTR modulators to reduce AP hospitalizations among patients with CF.

Original languageEnglish
Pages (from-to)2446-2454
Number of pages9
JournalAmerican Journal of Gastroenterology
Volume116
Issue number12
DOIs
StatePublished - Dec 1 2021

Bibliographical note

Funding Information:
Financial support : The project described was supported by Award Number UL1TR002733 from the National Center for Advancing Translational Sciences. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Center for Advancing Translational Sciences or the National Institutes of Health.

Publisher Copyright:
© 2021 Wolters Kluwer Health. All rights reserved.

ASJC Scopus subject areas

  • Hepatology
  • Gastroenterology

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