Outpatient Maintenance Therapy of Venetoclax Combined with Azacitidine in Patients with Acute Myeloid Leukemia– a Real-World Retrospective Study

Objective: With the increasing use of venetoclax in patients with acute myeloid leukemia (AML), several social issues have also emerged. Hence, we have proactively explored home treatment and outpatient follow-up management for AML patients undergoing venetoclax-based therapy. This approach aims to maintain treatment efficacy while facilitating patients' reintegration into society and reducing the burden of frequent hospitalizations.

Methods: A retrospective analysis was conducted on clinical data from AML patients who received venetoclax combined with azacitidine maintenance therapy at the outpatient department of Hebei Medical University Second Hospital from July 2021 to May 2024.We retrospectively studied the effectiveness and safety of this regimen.

Results: A total of 31 patients were included, with 17 males (54.8%) and 14 females (45.2%), and a median age of 61 years (range 21-85). Among the patients receiving outpatient VA maintenance therapy, the median follow-up was 11.9 months (range 2-35.1 ), and the median treatment period was 6 (range 2-24) cycles. The best overall response rate (ORR) was 92.6% (25/27). Of the 25 patients achieving the best response of CR/CRi, 3 patients experienced progression during outpatient VA treatment, and 5 patients had relapse, resulting in an overall relapse rate of 25.9% (7/27). A total of 26 patients were tested for minimal residual disease (MRD) during treatment, and the optimal MRD negative rate was 65.4% (17/26). The median relapse-free survival (RFS) for MRD-positive patients was 8.7 months, while it was not reached for MRD-negative patients. The most common adverse events (AE) was hematologic, and the most common non-hematologic AE was pulmonary infection. Among the 31 patients, 245 cycles of chemotherapy were administered, with grade 4 thrombocytopenia occurring in 4/19 patients (21%), neutropenia in 11/13 patients (85%), and anemia in 9/19 patients (47%). Compared to inpatient treatment, where thrombocytopenia (85%), neutropenia (100%), and anemia (100%) were more common, these rates were significantly reduced during outpatient treatment. The infection rate during inpatient treatment was 70%, while the average infection rate during outpatient VA maintenance therapy was 7%, a statistically significant difference (P < 0.05). The need for inpatient care due to infection was only 11 times.

Conclusions: Real-world data from our center indicate that venetoclax combined with azactidine is feasible for outpatient maintenance therapy. Patients who achieve MRD negativity can attain longer RFS and overall survival (OS), and both hematological and non-hematological toxicities are significantly reduced compared to inpatient treatment. Importantly, outpatient treatment of AML is more convenient and practical, allowing for broader implementation across various centers.

No relevant conflicts of interest to declare.