ORCID

Abstract

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 multicenter retrospective chart review included UK adults with previously untreated CLL who received acalabrutinib monotherapy as part of a UK-wide early access program. These patients received their first dose of acalabrutinib between 1 April 2020 and 1 April 2021. Data from 282 patients (male patients, n = 156 [55%]) collected from 29 sites across the United Kingdom revealed 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. The 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% of patients (95% CI, 67.3-77.8) remained on acalabrutinib treatment. The most common reasons for acalabrutinib discontinuation were adverse events (AEs; 31/87 [36%]), death (16/87 [18%]), and disease progression (14/87 [16%]). Of the 270 patients with recorded information, 99 patients (37%) experienced ≥1 prespecified AE, of which the most frequent were upper respiratory tract infection (n = 21 patients [8%]), rash (n = 13 [5%]), and neutropenia (n = 12 [4%]). This study adds to a body of clinical trial and further postmarketing evidence demonstrating the effectiveness and safety of acalabrutinib within routine UK clinical practice.

Publication Date

2026-07-28

Publication Title

Blood advances

Volume

10

Issue

14

ISSN

2473-9529

Acceptance Date

2026-04-07

Deposit Date

2026-07-14

Funding

The authors thank the patients and their families, UK CLL Forum, investigators, coinvestigators, and the study teams at each of the participating sites. They acknowledge Charlotte Richardson from AstraZeneca for her scientific support during the development of the manuscript, and OPEN Health, a health care consultancy company, for their help and involvement in the study design, study implementation, data collection, data analysis, interpretation, and reporting of results (funded by AstraZeneca). Medical writing support was provided by Fatemeh Saberi Hosnijeh from OPEN Health, funded by AstraZeneca. This research was funded by AstraZeneca. Contribution: T.A.E. N.M.-C. J.H. S.H. B.T.B. and R.W. contributed to study conception and design; J.H. performed the analysis; T.A.E. N.M.-C. S.P. M.N. D.T. H. Walter, A.B. M.K. N.P. P.E.M.P. N.S. M.R. S.O. T.T. F.F. H. Wardle, T.G. C.G. L.C. D.A. A.M. C.K. J.D. J.G. H.M. C.H. S.W. and R.W. contributed to data collection; and all authors contributed to interpretation of the data, reviewed the article, and approved the final version of the article to be published.

Keywords

Aged, Benzamides/therapeutic use, Female, Follow-Up Studies, Humans, Leukemia, Lymphocytic, Chronic, B-Cell/drug therapy, Male, Pyrazines/therapeutic use, Retrospective Studies, Treatment Outcome

First Page

4873

Last Page

4883

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