Journal article
Impact of Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapies on Liver Transplant Outcomes
Gastro hep advances, Vol.5(2), 100810
2026
DOI: 10.1016/j.gastha.2025.100810
PMCID: PMC12681715
PMID: 41362828
Abstract
Background and Aims
Up to 40% of patients with cystic fibrosis (CF) develop CF-related liver disease (CFrLD), which can progress to the point of requiring liver transplantation (LT). Advances in CF transmembrane conductance regulator (CFTR) modulator therapies, especially triple therapy modulators, have significantly improved pulmonary outcomes, but their impact on LT for CFrLD remains unclear.
Methods
Using data from the Scientific Registry of Transplant Recipients in 2000-2023, we analyzed trends in LT waitlisting for CFrLD pre- and post- U.S. Food and Drug Administration (FDA) approval of CFTR modulators: Ivacaftor (01/31/2012; single therapy), Ivacaftor-Lumacaftor (07/02/2015; dual therapy), and Ivacaftor-Tezacaftor-Elexacaftor (10/21/2019; triple therapy). We compared the waitlist characteristics and post-LT outcomes of pre- and post-FDA approval eras.
Results
Of 258,090 patients waitlisted for LT, 551 (0.2%) had CFrLD. The proportion of CFrLD patients on the LT waitlist decreased after FDA approval of triple CFTR modulators (0.23% to 0.14%, p<0.0004). Patients waitlisted after FDA approval of single and dual CFTR modulators were, on average, older (17.4 vs. 20.6 years, p<0.001 and 18.0 vs 20.8 years, p=0.004). Median MELD-Na scores were higher among individuals waitlisted following the approval of dual (9 [6–14] vs 10 [8–15], p<0.013) and triple (9 [6–14] vs 12.5 [8–17], p<0.003) CFTR modulators. There were no significant differences in post-LT survival pre- and post-FDA approval of single, dual, or triple CFTR therapy.
Conclusion
These findings suggest that CFTR modulators may mitigate CFrLD complications and delay the need for waitlisting as physicians await the patient’s response to therapy and reassess the need for LT.
Details
- Title: Subtitle
- Impact of Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapies on Liver Transplant Outcomes
- Creators
- Sara NaimimohassesAnkit RayEunice TanAsher WigginsBima J. HasjimShiyi ChenMamatha Bhat
- Resource Type
- Journal article
- Publication Details
- Gastro hep advances, Vol.5(2), 100810
- DOI
- 10.1016/j.gastha.2025.100810
- PMID
- 41362828
- PMCID
- PMC12681715
- NLM abbreviation
- Gastro Hep Adv
- ISSN
- 2772-5723
- eISSN
- 2772-5723
- Publisher
- ELSEVIER
- Language
- English
- Electronic publication date
- 09/14/2025
- Date published
- 2026
- Academic Unit
- Gastroenterology and Hepatology; Internal Medicine
- Record Identifier
- 9984963629402771
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