Objective:

To evaluate the clinical value of venetoclax combined with azacitidine in higher-risk myelodysplastic syndromes (HR-MDS) and propose a treatment-purpose framework.

Approach:

• Treatment Framework: The framework distinguishes between treatment aimed at bridging to allogeneic hematopoietic stem cell transplantation and treatment for non-transplant disease control.

Key Findings:

• Venetoclax combined with azacitidine shows high rates of complete remission, marrow complete remission, and overall response in treatment-naïve HR-MDS.
• Increased response depth does not necessarily correlate with improved overall survival.
• The clinical value of the treatment varies based on the intent of therapy and patient context.

Interpretation:

The findings indicate that venetoclax plus azacitidine should not be regarded merely as a universal substitute for azacitidine; its value instead varies according to treatment purpose and patient context.

Limitations:

• The study did not provide detailed reasons for patients not undergoing transplantation.
• Patient-level explanations for post-transplant relapse and non-relapse mortality were not included.

Conclusion:

Venetoclax plus azacitidine should be considered as a potential bridge to transplantation rather than a definitive means to enhance transplantation rates or post-transplant survival.

Sources:

• Garcia et al. Study