Purpose:Treatment options for myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) patients remain limited, underscoring the need for novel, more effective therapeutic strategies. This prospective, open-label study evaluated the efficacy and safety of a triple combination regimen comprising chidamide, azacitidine, and venetoclax (VAC regimen) in treatment-naïve patients with these hematologic malignancies.

Patients and Methods: Sixteen newly diagnosed patients (6 MDS, 10 AML) with an average age of 57.9 years were treated with the VAC regimen consisting of chidamide, azacitidine, and venetoclax. The primary endpoint was overall response rate (ORR). Secondary endpoints included complete remission (CR) rate, event-free survival (EFS), overall survival (OS), and safety/tolerability.

Results: The ORR was 93.8%, with a remarkable CR rate of 87.5% (CR in 12 patients, CRi in 2 patient). Rapid hematologic recovery was observed, with a median time to tri-lineage recovery of 15 days. The 6-month EFS and OS rates were 80.8% and 81.8%, respectively, demonstrating promising survival outcomes. Three MDS patients underwent allogeneic stem cell transplantation following remission.All participants experienced AEs, predominantly non-hematological in nature. The frequent non-hematological AEs included infections (notably pneumonia), nausea, fatigue, vomiting, hypokalemia, hypoalbuminemia, and febrile neutropenia, with most of these being mild or moderate (grade 1/2).Given the adverse event (AE) data, the VAC regimen demonstrated an acceptable safety profile.

Conclusions: The VAC regimen demonstrated exceptional efficacy and an encouraging safety signal in treatment-naïve MDS and AML patients. These promising results highlight the potential of this novel triple combination as a frontline therapeutic option warranting further investigation in larger randomized trials.

Disclosures

No relevant conflicts of interest to declare.

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2024
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