The efficacy and safety of asciminib treatment in patients with chronic myeloid leukemia as a second-line or beyond second-line treatment: a systematic review and meta-analysis.

Background: Patients with chronic myeloid leukemia (CML) are currently experiencing intolerance or lack of efficacy with previous tyrosine kinase inhibitors (TKIs) and benefit from asciminib as a novel TKI.

Objectives: The purpose of this meta-analysis was to evaluate the efficacy and safety of asciminib as a second-line or beyond second-line treatment for patients with CML.

Design: A systematic review and meta-analysis.

Data sources and methods: We searched four databases (PubMed, Cochrane, Web of Science, and EMBASE) for relevant literature from the inception of the databases to February 4, 2024. Two authors independently performed data extraction to assess the efficacy of asciminib using metrics such as the rate of major molecular response (MMR) and the safety of asciminib using the rate of adverse event (AE). We also performed a subgroup analysis based on the reason for starting asciminib. Data were analyzed using either a fixed-effects model or a random-effects model to calculate the rate of MMR and the rate of AEs. We also assessed the quality of the studies by selecting appropriate tools according to the type of included studies.

Results: We included 8 studies involving a total of 691 patients. The overall MMR rate for patients with CML treated with asciminib was 46.9% (95% CI: 39.8-54.0, p < 0.05). AEs were reported in five studies, with a combined rate of 79.2% (95% CI: 46.6-98.7, p < 0.05) for all grades and 39.5% (95% CI: 17.6-61.3, p < 0.05) for grade ⩾3 AEs. Thrombocytopenia was the most common AE, with a combined rate of 22.5% (95% CI: 18.8-26.3, p < 0.05) for all grades.

Conclusion: Asciminib is effective in the treatment of patients with CML, and the most common AE during treatment is thrombocytopenia.