Gastro hep advances · 2025
Impact of Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapies on Liver Transplant Outcomes.
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Naimimohasses Sara, Ray Ankit, Tan Eunice, Wiggins Asher, Hasjim Bima J, Chen Shiyi, Bhat Mamatha
Аннотация
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 (January 31, 2012; single therapy), ivacaftor-lumacaftor (July 2, 2015; dual therapy), and ivacaftor-tezacaftor-elexacaftor (October 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 < .0004). Patients waitlisted after FDA approval of single and dual CFTR modulators were, on average, older (17.4 vs 20.6 years; P < .001 and 18.0 vs 20.8 years; P = .004). Median model for end-stage liver disease-sodium scores were higher among individuals waitlisted following the approval of dual (9 [6-14] vs 10 [8-15], P < .013) and triple (9 [6-14] vs 12.5 [8-17], P < .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.
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 (January 31, 2012; single therapy), ivacaftor-lumacaftor (July 2, 2015; dual therapy), and ivacaftor-tezacaftor-elexacaftor (October 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 < .0004). Patients waitlisted after FDA approval of single and dual CFTR modulators were, on average, older (17.4 vs 20.6 years; P < .001 and 18.0 vs 20.8 years; P = .004). Median model for end-stage liver disease-sodium scores were higher among individuals waitlisted following the approval of dual (9 [6-14] vs 10 [8-15], P < .013) and triple (9 [6-14] vs 12.5 [8-17], P < .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.