Venetoclax + 10-day decitabine in older/unfit patients with ND or R/R AML or HR-MDS

Swaminathan presented a poster during the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting, reporting long-term findings of a phase II study (NCT03404193) of venetoclax in combination with 10-day decitabine in acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). 

Among patients with newly diagnosed (ND) AML (n = 88), untreated secondary AML (sAML; n = 17), treated sAML (n = 30), relapsed/refractory (R/R) AML (n = 72), and high-risk MDS (n = 21):

• Overall response rates were 88%, 77%, 67%, 58%, and 52%, respectively.
• Complete response (CR) + CR with incomplete count recovery (CRi) rates were 81%, 65%, 47%, 40%, and 33%, respectively.
• • Durations of CR/CRi response were 23.6, 17.8, 8.4, 15.7, and 10.1 months, respectively.

Among patients with ND AML, untreated sAML, treated sAML, and R/R AML:

• Median overall survivals were 15.7, 11.1, 8.5, and 7.6 months respectively.
• Median relapse-free survivals were 10.0, 5.1, 7.8, and 7.5 months, respectively.

Patients receiving stem cell transplantation (SCT) had improved survival outcomes compared with those not receiving SCT.

Safety findings were consistent with the known safety profile of venetoclax + hypomethylating agents. Most common treatment-emergent adverse events were infection with absolute neutrophil count <1.0 × 10⁹/L (42%) and >1.0 × 10⁹/L (38%), febrile neutropenia (32%), thrombocytopenia (16%), neutropenia (13%), and oral mucositis (13%).

Venetoclax in combination with 10-day decitabine was highly effective and well tolerated in older/unfit patients with ND and R/R AML; however, more clinical trials are needed to validate the findings.