Qualitative understanding of experiences of people with cystic fibrosis in a treatment discontinuation trial: The QUEST study

Jill Maggs¹, Gregory S Sawicki², Callie Bacon², Emma McWilliams², Dana Yablon³, Benjamin Ertman², Liam Sweeney³, Jennifer L Butcher⁴, Robin S Everhart⁵, Michelle Prickett⁶, Christopher Siracusa⁷, Alex H Gifford⁸, Nicole Mayer-Hamblett⁹, David P Nichols¹⁰, Andrea Goodman³, Tia Woo³, Kristin A Riekert¹¹

Affiliations

¹ Community Member, former STRC Steering Committee Member, c/o Cystic Fibrosis Foundation, 4550 Montgomery Avenue, Suite 1100 N, Bethesda, MD, USA.
² Division of Pulmonary Medicine, Boston Children's Hospital, 333 Longwood Avenue, Boston, MA, USA.
³ Department of Medicine, Division of Pulmonary and Critical Care Medicine, Johns Hopkins University, 5501 Hopkins Bayview Circle, Baltimore, MD, USA.
⁴ Department of Pediatrics, Division of Pediatric Psychology, Mott Children's Hospital, University of Michigan Health, Ann Arbor, MI, USA.
⁵ Department of Psychology, Virginia Commonwealth University, Richmond, VA, USA.
⁶ Division of Pulmonary and Critical Care, Department of Medicine, Feinberg School of Medicine, Northwestern University, Chicago, IL, USA.
⁷ Division of Pulmonary Medicine, Cincinnati Children's Medical Center, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
⁸ Pulmonary, Critical Care, and Sleep Medicine, University Hospitals Cleveland Medical Center, Cleveland, OH, USA; Rainbow Babies and Children's Hospital, Cleveland, OH, USA.
⁹ Seattle Children's Research Institute, Seattle, WA, USA; Department of Pediatrics, University of Washington, Seattle, WA, USA; Department of Biostatistics, University of Washington, Seattle, WA, USA.
¹⁰ Cystic Fibrosis Foundation, Bethesda, MD, USA.
¹¹ Department of Medicine, Division of Pulmonary and Critical Care Medicine, Johns Hopkins University, 5501 Hopkins Bayview Circle, Baltimore, MD, USA.. Electronic address: kriekert@jhmi.edu.

PMID: 39557157
DOI: 10.1016/j.cct.2024.107752

Randomized Controlled Trial

Qualitative understanding of experiences of people with cystic fibrosis in a treatment discontinuation trial: The QUEST study

Jill Maggs et al. Contemp Clin Trials. 2025 Jan.

. 2025 Jan:148:107752.

doi: 10.1016/j.cct.2024.107752. Epub 2024 Nov 17.

Authors

Affiliations

¹ Community Member, former STRC Steering Committee Member, c/o Cystic Fibrosis Foundation, 4550 Montgomery Avenue, Suite 1100 N, Bethesda, MD, USA.
² Division of Pulmonary Medicine, Boston Children's Hospital, 333 Longwood Avenue, Boston, MA, USA.
³ Department of Medicine, Division of Pulmonary and Critical Care Medicine, Johns Hopkins University, 5501 Hopkins Bayview Circle, Baltimore, MD, USA.
⁴ Department of Pediatrics, Division of Pediatric Psychology, Mott Children's Hospital, University of Michigan Health, Ann Arbor, MI, USA.
⁵ Department of Psychology, Virginia Commonwealth University, Richmond, VA, USA.
⁶ Division of Pulmonary and Critical Care, Department of Medicine, Feinberg School of Medicine, Northwestern University, Chicago, IL, USA.
⁷ Division of Pulmonary Medicine, Cincinnati Children's Medical Center, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
⁸ Pulmonary, Critical Care, and Sleep Medicine, University Hospitals Cleveland Medical Center, Cleveland, OH, USA; Rainbow Babies and Children's Hospital, Cleveland, OH, USA.
⁹ Seattle Children's Research Institute, Seattle, WA, USA; Department of Pediatrics, University of Washington, Seattle, WA, USA; Department of Biostatistics, University of Washington, Seattle, WA, USA.
¹⁰ Cystic Fibrosis Foundation, Bethesda, MD, USA.
¹¹ Department of Medicine, Division of Pulmonary and Critical Care Medicine, Johns Hopkins University, 5501 Hopkins Bayview Circle, Baltimore, MD, USA.. Electronic address: kriekert@jhmi.edu.

PMID: 39557157
DOI: 10.1016/j.cct.2024.107752

Abstract

Background: As people with cystic fibrosis (PWCF) live longer due to the breakthrough drug elexacaftor-tezacaftor-ivacaftor (ETI), they have questioned whether other CF therapies could be safely discontinued. SIMPLIFY was the first prospective, randomized trial to evaluate non-inferiority of discontinuing versus continuing two therapies. The QUEST (Qualitative Understanding of Experiences in the SIMPLIFY Trial) study was conducted to understand experiences of PWCF enrolled in SIMPLIFY, including why they joined, perceptions of randomization, decision-making around study withdrawal, and considerations for future discontinuation studies.

Methods: QUEST enrolled SIMPLIFY participants 14 years or older stable on ETI and caregivers of the 14-17 year-olds. Interviews were audio-recorded, transcribed, and coded. A phenomenological approach was used to inductively develop codes with no a priori hypotheses; identified themes were then organized around current research and recruitment literature.

Results: 114 interviews were completed (68 adults, 23 teenagers, and 23 caregivers). Among PWCF, median age was 27.8 years, 49 % were female and 80 % had participated in research before SIMPLIFY. Five themes were identified: (1) Experience with SIMPLIFY randomization, [2] Trust, [3] Altruism, [4] Perceived personal benefits, and [5]) Perceived risks and protocol burden.

Conclusion: QUEST findings highlight how a long-standing culture of research and thoughtful protocol design contributed to SIMPLIFY's successful recruitment and retention. This included understanding the importance of remaining in the trial despite not being randomized to their preferred treatment assignment. Using patient-centered approaches to select research questions, design a protocol to minimize participant barriers, and frame recruitment materials messaging contribute to successful research participation.

Keywords: Cystic fibrosis; Discontinuation trial; Research participation.

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Conflict of interest statement

Declaration of competing interest The Authors declare that there is no conflict of interest with respect to the research, authorship, and/or publication of this article.

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Qualitative understanding of experiences of people with cystic fibrosis in a treatment discontinuation trial: The QUEST study

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Qualitative understanding of experiences of people with cystic fibrosis in a treatment discontinuation trial: The QUEST study

Authors

Affiliations

Abstract

Conflict of interest statement

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LinkOut - more resources

Full Text Sources

Medical

Research Materials