The use of elexacaftor/tezacaftor/ivacaftor in patients with cystic fibrosis postliver transplant: A case series. Issue 2 (12th December 2021)

• Record Type: Journal Article
• Authors: Ragan, Hunter; Autry, Elizabeth; Bomersback, Taryn; Hewlett, Jennifer; Kormelink, Lauren; Safirstein, Julie; Shanley, Laura; Lubsch, Lisa
• Is Part Of: Pediatric pulmonology. Volume 57:Issue 2(2022)
• Page Start: 411
• Page End: 417
• Publication Date: 2021-12-12
• Subjects: biliary atresia -- cystic fibrosis -- cystic fibrosis‐related liver disease -- elexacaftor/tezacaftor/ivacaftor (Trikafta) -- liver transplant; Pediatric respiratory diseases -- Periodicals; Pediatrics -- Periodicals; 618.922
• DOI: 10.1002/ppul.25779
• Languages: English
• ISSNs: 8755-6863

Abstract

Abstract: Introduction: Cystic fibrosis (CF)‐related liver disease (CFLD) manifests as a wide spectrum of hepatobiliary disease and can progress to need liver transplantation. Elexacaftor/tezacaftor/ivacaftor (elx/tez/iva) is a cystic fibrosis transmembrane conductance regulator modulator that has superior efficacy compared to previously approved modulators. Use of elx/tez/iva, should be approached with caution in individuals with CFLD or following liver transplantation due to possible increases in liver function tests (LFTs) and drug–drug interactions with several immunosuppressant medications. Objective: The purpose of this case series is to explore if the use of elx/tez/iva is safe and tolerable in patients with CF postliver transplantation. Methods: A retrospective case series including patients prescribed elx/tez/iva following liver transplantation and an immunosuppressive regimen consisting of drug therapy metabolized by P‐glycoprotein was completed. Results: Ten patients at six CF centers with a median age of 22.1 years (range 14–43.4 years) and the median time from the transplant of 6.9 years (range 0.6–22 years) were included. Most patients (8, 80%) received a reduced or full dose of elx/tez/iva for a mean duration of 10.4 months (range 7–12 months). Fluctuations in LFTs occurred in all patients (10, 100%) and led to therapy discontinuation in two patients (20%). Elx/tez/iva initiation resulted in elevations in tacrolimus trough concentration in seven patients (70%). Most patients who tolerated elx/tez/iva had symptomatic and quality of life improvement, increased body mass index, and maintained or improved lung function. Conclusion: Initiation of elx/tez/iva in patients with CF who received liver transplantation may be safe with clinical benefits.