---

Clinical Report: Evaluating Treatment Outcomes of Venetoclax Combined with Azacitidine

Overview

Venetoclax combined with azacitidine demonstrates substantial clinical activity in treatment-naïve higher-risk myelodysplastic syndromes (HR-MDS), with notable rates of complete remission and overall response. However, increased response depth does not correlate with improved overall survival.

Background

Higher-risk myelodysplastic syndromes (HR-MDS) present significant therapeutic challenges due to poor survival rates and limited treatment options. The combination of venetoclax and azacitidine has emerged as a promising approach, yet recent findings indicate that achieving higher response rates does not necessarily translate to better overall survival outcomes.

Data Highlights

In a clinical study, venetoclax plus azacitidine resulted in a complete remission rate of 29.9% and a marrow complete remission rate of 50.5% among treatment-naïve HR-MDS patients. The overall response rate was reported at 80.4%, with a median overall survival of 26.0 months. Additionally, 39.3% of patients proceeded to allogeneic hematopoietic stem cell transplantation following treatment.

Key Findings

• Venetoclax plus azacitidine achieved complete remission in 29.9% and marrow complete remission in 50.5% of treatment-naïve HR-MDS patients.

• The overall response rate was reported at 80.4%, with a median overall survival of 26.0 months.

• 39.3% of patients in the study proceeded to allogeneic hematopoietic stem cell transplantation after treatment.

• Higher response rates do not guarantee improved overall survival.

• Clinical evaluation should consider molecular and cytogenetic factors, alongside treatment intent and patient context.

Clinical Implications

Clinicians should assess the intent of treatment when considering venetoclax plus azacitidine for HR-MDS, distinguishing between bridging to transplantation and disease control without transplantation.

Conclusion

The combination of venetoclax and azacitidine shows clinical activity in HR-MDS, but its value must be interpreted within a framework that considers treatment intent and individual patient factors.

Related Resources & Content

1. Garcia et al., ASH Annual Meeting, 2023 -- Venetoclax Plus Azacitidine Evaluated in High-Risk Myelodysplastic Syndromes

2. The ASCO Post, 2025 -- Doublet Therapy Yields Higher Response Rates and Fewer Complications Than Standard Induction in AML

3. Blood Cancer Journal, 2025 -- Efficacy of Venetoclax and Azacitidine in Newly Diagnosed Patients with Therapy-Related Acute Myeloid Leukemia

4. Nature Reviews Clinical Oncology, 2026 -- A 2026 update on myelodysplastic neoplasms: current state, challenges and future directions

5. AbbVie, 2025 -- AbbVie Provides Update on VERONA Trial for Newly Diagnosed Higher-Risk Myelodysplastic Syndromes

6. Blood Cancer Journal — Combination of Venetoclax with Low-Dose Cytarabine Achieves Significant Remission in Acute Myeloid Leukemia by Targeting Leukemic Stem Cells and Altering Clonal Hematopoiesis

7. A 2026 update on myelodysplastic neoplasms: current state, challenges and future directions | Nature Reviews Clinical Oncology

8. AbbVie Provides Update on VERONA Trial for Newly Diagnosed Higher-Risk Myelodysplastic Syndromes - Jun 16, 2025

9. Outcomes of patients with higher-risk myelodysplastic syndromes/neoplasms treated with hypomethylating agents + venetoclax—an analysis from the International Consortium for MDS (icMDS) VALIDATE database | Blood Cancer Journal

---