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The use of venetoclax in the treatment of acute lymphoblastic leukemia—a systematic review

Adrian Kowalczyk,Julia Zarychta,Joanna Zawitkowska,M. Lejman

Published 2026 in Therapeutic Advances in Medical Oncology

ABSTRACT

Background: The 5-year overall survival rates in acute lymphoblastic leukemia (ALL) vary depending on the patient’s age group (from 93% to under 30%). However, in the case of relapsed/refractory ALL (R/R ALL), the complete remission (CR) rate in the pediatric patient group is 44% and in the adult population—18%. Therefore, further research on new therapeutic compounds is necessary. Objectives: This systematic review aims to analyze the results of clinical studies concerning the use of venetoclax alone or in combination with other drugs in the treatment of ALL. Design: The systematic review was conducted in accordance with the guidelines of Preferred Reporting Items for Systematic Reviews and Meta-Analysis. The Quality Assessment Tool for Before-After (Pre-Post) Studies With No Control Group by the National Heart, Lung, and Blood Institute was used to assess the quality of included studies. Data sources and methods: PubMed and Web of Science were used for the literature review. All clinical studies reporting outcomes in patients with ALL treated with venetoclax met the inclusion criteria. As a result of our search process for articles, a total of 989 records were obtained. After excluding records that did not meet the inclusion criteria or met the exclusion criteria, seven articles were finally obtained. Results: In seven identified clinical trials, CR rate ranged from 22% to 59.6% in patients with R/R ALL, while in the group of patients with newly diagnosed ALL, it ranged from 90.9% to 96%. The most common adverse events of grade 3 or higher were hematological complications, including neutropenia (71 episodes among 110 patients), anemia (70/141), and thrombocytopenia (70/141). Conclusion: Based on the results of phase I clinical trials, further clinical trials should be conducted to assess the therapeutic potential of venetoclax in the treatment of ALL.

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