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Published on: February 17, 2022
Venetoclax plus azacitidine in relapsed or refractory T-cell acute lymphoblastic leukaemia: a multicentre,
Han-Yu Cao1, Hao Zhang2, Yang Zhang3
1Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, Jiangsu, China; Department of Blood and Marrow Transplantation, Soochow University, Suzhou, Jiangsu, China; The Leukemia Working Group of National Clinical Research Center for Hematologic Diseases, Suzhou, China.
Background:
Patients with relapsed or refractory T-cell acute lymphoblastic leukaemia have limited responses to conventional chemotherapy and poor prognoses. T-cell precursors exhibit high expression of BCL-2 and are sensitive to BCL-2 inhibitors. Retrospective case series have reported the successful use of venetoclax combined with chemotherapy or hypomethylating agents when treating relapsed or refractory T-cell acute lymphoblastic leukaemia. The study aimed to evaluate the activity and safety of the venetoclax plus azacitidine regimen in patients with relapsed or refractory T-cell acute lymphoblastic leukaemia.
Methods:
In this single-arm, phase 2, multicentre trial, patients aged 15-70 years with relapsed or refractory T-cell acute lymphoblastic leukaemia and an Eastern Cooperative Oncology Group performance status of 0-3 were eligible. The venetoclax plus azacitidine regimen consisted of venetoclax (100 mg on day 1, 200 mg on day 2, and 400 mg on days 3-21, orally) and azacitidine (75 mg/m2 per day on days 1-7, subcutaneously). The primary endpoint was overall response rate. Activity analyses were performed in the full analysis set that included all enrolled patients. This trial is registered with ClinicalTrials.gov (NCT05149378) and is complete.
Findings:
Between Nov 28, 2021, and Dec 31, 2024, 25 patients were enrolled. The median age at enrolment was 39·0 years (IQR 27·5-56·5); 18 patients (72%) were male and seven (28%) were female; and all patients were Asian. The overall response rate was 76% (19 of 25; 36% [nine of 25] for complete remission, 16% [four of 25] for complete remission with partial haematological recovery, 16% [four of 25] for complete remission with incomplete haematological recovery, and 8% [two of 25] for morphological leukaemia-free state). As of the data cutoff (April 7, 2025), median follow-up was 31·8 months (IQR 16·2-39·3). The most common grade 3 or worse haematological adverse events were neutropenia (21 [84%] of 25), anaemia (11 [44%] of 25), febrile neutropenia (ten [40%] of 25), and thrombocytopenia (five [20%] of 25), followed by infections (three [12%] of 25). No treatment-related serious adverse events were observed and no treatment-related deaths occurred.
Interpretation:
The venetoclax plus azacitidine regimen showed a manageable safety profile and promising activity as salvage therapy for relapsed or refractory T-cell acute lymphoblastic leukaemia. Given the limited sample size and single-arm design nature, further confirmatory trials are warranted to validate its efficacy as a potential salvage therapy for relapsed or refractory T-cell acute lymphoblastic leukaemia.
Funding:
National Natural Science Foundation of China.
Translation:
For the Chinese translation of the abstract see Supplementary Materials section.
