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Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
Published on: February 11, 2017
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Exploring perceptions of and decision-making about CFTR modulators
Lee Landess1, Mary G Prieur2, Ashley R Brown2
1University of North Carolina School of Medicine, Chapel Hill, North Carolina, USA.
Pediatric Pulmonology
|March 8, 2024
Summary
Cystic fibrosis transmembrane regulator (CFTR) modulator initiation requires thoughtful discussion, addressing patient emotions and knowledge gaps. Care teams should support all patients, including those ineligible for modulators.
Area of Science:
- Pulmonology
- Medical Genetics
- Patient Care
Background:
- Cystic fibrosis (CF) treatment increasingly utilizes highly effective modulators.
- Guidance for CF care teams on educating patients about modulator initiation is limited.
- This study explored patient, caregiver, and clinician perspectives on CFTR modulators.
Purpose of the Study:
- To explore patient, caregiver, and clinician perceptions of CFTR modulators.
- To identify influences on decisions regarding starting CFTR modulator therapy.
- To inform best practices for CF care teams in discussing modulator initiation.
Main Methods:
- Conducted semistructured interviews with CF clinicians, adults with CF, and caregivers of children with CF.
- Analyzed interview audio recordings by coding responses to identify central themes.
- Included 8 CF clinicians, 9 adults with CF, and 11 caregivers in the study.
Main Results:
- Key themes included emotional responses, decision-making influences, side effect concerns, and future planning related to modulators.
- Clinicians noted shifts in prescribing conversations post-elexacaftor/tezacaftor/ivacaftor approval.
- The multidisciplinary CF care team plays a crucial role in supporting treatment decisions.
- Unique needs of patients ineligible for modulators were also identified.
Conclusions:
- Clinicians should engage in thorough and thoughtful conversations about CFTR modulators.
- Utilize the multidisciplinary care model to address patient/caregiver emotions and knowledge gaps.
- Discuss treatment goals beyond clinical benefits and provide compassionate support for ineligible patients.
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