A 10-year phase 2 study published in the journal Blood found that ibrutinib alone can provide long-lasting benefits for patients with chronic lymphocytic leukemia (CLL), including those with high-risk disease features. The study included 84 patients who were either newly diagnosed or had relapsed disease, many of whom had TP53 abnormalities or unmutated IGHV, both linked to poorer outcomes.
Patients took daily oral ibrutinib until the cancer progressed or side effects became too severe. After 10 years, the median overall survival had still not been reached, and nearly 60% of patients were alive. Median progression-free survival was 7.2 years, although patients treated earlier in their disease course did much better than those with relapsed disease.
Some patients achieved undetectable minimal residual disease (uMRD), meaning no leukemia could be detected with sensitive testing. Most of these patients remained free of progression, even after stopping treatment in some cases.
Side effects were a major reason for discontinuation. Cardiac problems such as atrial fibrillation were the most common serious adverse events, followed by secondary cancers and infections. By July 2024, only about 11% of patients were still taking the drug.