Acalabrutinib monotherapy as a first-line treatment for CLL: 3 years follow-up of the real-world EPIC study.

Clinical trials indicate that acalabrutinib, a second-generation Bruton tyrosine kinase inhibitor, is safe and effective for treating patients with chronic lymphocytic leukemia (CLL), but real-world evidence on its clinical effectiveness is limited. This ongoing multi-center retrospective chart review included UK adults with previously untreated CLL who received acalabrutinib monotherapy as part of an UK-wide early access program. These patients received their first dose of acalabrutinib between 1st April 2020-1st April 2021. Data from 282 patients (55% male, n=156) collected from 29 sites across the UK observed a median age of 73.9 years (interquartile range [IQR]: 68.6-79.4) at acalabrutinib initiation (index date) and a median time of 3.0 years (IQR: 1.2-5.7 [n=281]) from CLL diagnosis to treatment. Median follow-up was 48.9 months (IQR: 45.0-52.3). At the 3-year landmark, real-world progression free survival was 82.5% (95% confidence interval (CI): 78.2-87.1), and real-world overall survival was 84.3% (95% CI 80.2-88.7%). Additionally, 72.3% (95% CI 67.3-77.8) of patients remained on acalabrutinib treatment. The most common reasons for acalabrutinib discontinuation were adverse events (AE), (36%, n=31/87), death (18%, n=16/87), and disease progression (16%, n=14/87). Of the 270 patients with recorded information, 99 patients (37%) experienced one or more pre-specified AE of which the most frequent were upper respiratory tract infection (21 patients [8%]), rash (13 patients [5%]) and neutropenia (12 patients [4%]). This study adds to a body of clinical trial and further post-marketing-evidence demonstrating the effectiveness and safety of acalabrutinib within routine UK clinical practice.