Initial Clinical Response and Safety of Elafibranor
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Authors
Jones A.
Greener K.
Cripps S.
Rees F.
Shah S.
Crook J.
Boyle, A.
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2025
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Article
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Primary biliary cholangitis (PBC) is an immune-mediated chronic cholestatic liver disease. Until recently, there were only two licensed treatment options for PBC, ursodeoxycholic acid (UDCA) and obeticholic acid (OCA), along with off-label use of fibrates. Elafibranor, a dual peroxisome proliferator-activated receptor (PPAR) a/d agonist, is now licensed for treatment of PBC in patients unresponsive to or intolerant of UDCA monotherapy. This pharmacist-led study aims to describe initial clinical response and safety profile of elafibranor in real-world practice in the UK. Data was collected retrospectively across five UK centres by pharmacists on all patients treated with elafibranor since it became licensed. Patient records were used to review clinical characteristics including liver stiffness measurements (LSM), liver blood tests, and previous PBC therapies. The initial clinical response to elafibranor was also reported, alongside safety and tolerability. 27 patients were included, with 21 patients having AMA positive PBC (77.8%). 26 (96.2%) were female, with mean age 59 years (+/- 10). 6 (22%) patients had cirrhosis, 4 Child Pugh A (15.4%), 2 Child Pugh B (7.4%). Mean LSM prior to elafibranor initiation was 10.1 kPa (+/-7.6). 23 (85.1%) patients were on UDCA prior to elafibranor initiation and continued dual therapy, with mean dose of 14.3 mg/kg (+/- 1.8). 22 (81.5%) patients had trialled second-line therapies for PBC previously. 16 (59.3%) patients had trialled obeticholic acid (OCA) prior to elafibranor initiation. 2 (7.4%) patients continued OCA alongside elafibranor. 19 (70.4%) patients had trialled bezafibrate prior to elafibranor initiation. 13 (48.2%) patients had trialled both OCA and bezafibrate. Table 1 summarises the mean changes in liver blood test results after mean 6.1 weeks (+/-2.5) of elafibranor therapy. 1 (2.7%) patient reported improvement in itch and 1 (2.7%) patient reported improvement in fatigue. 14 (51.9%) patients reported adverse effects following elafibranor initiation, and 10 (37%) discontinued elafibranor due to adverse effects. Gastrointestinal effects were most common (n=10), followed by headache (n=4), muscle pain (n=3), rash (n=1). Pruritus worsened for 1 patient and 1 patient (baseline Child Pugh score A) developed decompensated cirrhosis with ascites, which may not have been related to elafibranor initiation. We report initial experience of elafibranor in patients who have not tolerated or not responded to other second-line PBC therapies. Approximately half of patients reported adverse effects leading to treatment discontinuation in over a third of patients. Biochemical response to therapy showed positive signs but further data is required to establish long-term efficacy in this cohort.
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Gut