ASH 2023: Venetoclax Combined With Azacitidine For High-Risk MDS

Treating High-Risk MDS

At ASH 2023, Dr. Guillermo Garcia-Manero from MD Anderson Cancer Center presented the updated clinical outcomes of higher-risk MDS patients who received a stem cell transplant following a chemotherapy treatment combination of venetoclax and azacitidine. Previous reports showed that the venetoclax and azacitidine combination was safe and well tolerated. 

Clinical Trial For Venetoclax And Azacitidine

Researchers presented a global, open-label phase 1b clinical trial (NCT02942290) that magnified the use of the ven/aza treatment combination in “treatment-naïve higher-risk MDS comprising a dose-escalation portion and a safety expansion portion.” This study also reports on the outcomes of these patients who received a stem cell transplant. Recommended phase 2 doses were also determined.

Patient Demographics

All enrolled patients were older than 18 (median age was 64) and had less than 20% blasts in their bone marrow. The medications were administered in the following manner:

• Azacitidine- 75mg intravenously on Days 1–7 or Days 1–5, 8, and 9 of each 28-day cycle.
• Venetoclax- 100mg once daily for 14 days of a 28-day cycle, then escalated to 200 and 400 mg in separate cohorts.

The average number of cycles for the ven/aza treatment was 3. The median time from the first medication dose to the stem cell transplant was 5.6 months. 

Statistics

Before a stem cell transplant, patients experienced the following:

• 41.2% achieved complete remission.
• 45.1% achieved bone marrow complete remission.
• The composite response rate was 30.4%.
• The average time to best overall response (time from first dose of study drug to best response of complete response, partial response, or stable disease) was two months.
• Among patients who underwent transplants, 64.7% remained alive at the data cut-off date.

Conclusion

Positive, early responses from the ven/aza combination treatment provided an effective avenue for patients to receive a stem cell transplant. “These results suggest that Ven-based regimens before SCT may provide a path to curative therapy.”

More Information

Read about a similar study conducted and presented at the 2022 ASH HERE.

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