Need to study simplification of gastrointestinal medication regimen in cystic fibrosis in the era of highly effective modulators

Meghana Sathe, Baha Moshiree, Enid Aliaj, Min Jae Lee, Jessica Hudson, Alex Gifford, Susan Attel, Breck Gamel, Steven D. Freedman, Sarah Jane Schwarzenberg, A. Jay Freeman

Research output: Contribution to journalArticlepeer-review

3 Scopus citations

Abstract

Introduction: The success of highly effective modulator therapy (HEMT) has led to consideration of simpler regimens for people with CF (PwCF) with opportunities to modify burdensome regimens. Despite the intuitive appeal of discontinuing chronic therapies no longer necessary, this process should be pursued systematically to ensure safety, adherence, and validate patient-centered preferences. We designed a questionnaire to determine the state of use of acid-suppressive medications (ASM) and pancreatic enzyme therapy (PERT), current self-withdrawal and provider-directed withdrawal practices, and interest in a standardized withdrawal study. Methods: In collaboration with CF Foundation (CFF), a questionnaire was developed and distributed to members of Community Voice (CV, comprised of PwCF and their loved ones), and CF providers regarding the need to study simplifying the gastrointestinal (GI) regimen for PwCF on HEMT. Results: Approximately 20-40% of CV or CF providers have decreased or stopped ASM for those on HEMT. For PERT, CV and CF providers have decreased dose (34%–48% and approximately 25%, respectively) more often than having stopped it altogether (13%–24% and 3%–12%, respectively). Cumulatively, there is interest in pursuing research in this area (86% CV and 89% CF providers) and willingness to enroll in such a study (80% CV and 89% CF providers). Conclusion: Systematically studying the withdrawal of common GI medications, ASM and PERT, is important to CV and CF providers. Decreases in dosing and withdrawal are already taking place without evidence to support this practice. This questionnaire is the first step in designing a GI medication simplification study in PwCF on HEMT.

Original languageEnglish (US)
Pages (from-to)811-818
Number of pages8
JournalPediatric pulmonology
Volume58
Issue number3
DOIs
StatePublished - Mar 2023

Bibliographical note

Funding Information:
We would like to thank all Community Voice members and CF providers who agreed to participate in this important questionnaire to allow us as a research team to determine the need to study the simplification of gastrointestinal treatments for people with CF on HEMT. This work was supported by the Cystic Fibrosis Foundation, SATHE20A0.

Publisher Copyright:
© 2022 Wiley Periodicals LLC.

Keywords

  • CFTR modulators
  • acid suppressive medications
  • cystic fibrosis
  • pancreatic enzyme replacement therapy
  • proton pump inhibitors

PubMed: MeSH publication types

  • Journal Article
  • Research Support, Non-U.S. Gov't

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