Phase 1 study of chidamide in combination with venetoclax, azacitidine, aclarubicin, cytarabine and G-CSF for refractory/relapsed acute myeloid leukemia: clinical safety, efficacy, and correlative analysis.

ABSTRACT:

Background

Relapsed or refractory AML (R/R-AML) remains a particularly adverse population necessitating improved therapeutic strategies. In this phase 1 study, we evaluated the efficacy and safety of chidamide, azacitidine, cytarabine, aclarubicin and granulocyte colony-stimulating factor (CACAG) in combination with the B cell lymphoma-2 inhibitor venetoclax (VEN) in R/R-AML.

Methods

We conducted a phase 1 trial to assess the safety and efficacy of CACAG-VEN regimen as a salvage induction regimen for patients with R/R AML. The primary endpoint was the treatment-related adverse events and overall response rate (ORR) following one cycle of the CACAG-VEN regimen. We also performed single-cell RNA sequencing on eight samples of bone marrow from four patients before and after CAC

SUBMITTER: Wen Y 

PROVIDER: S-EPMC12738368 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature