Clinical Study of Venetoclax Combined With Azacitidine as Bridging Therapy Prior to Hematopoietic Stem Cell Transplantation in Patients With Higher-Risk Myelodysplastic Syndromes

Launched by YEHUI TAN · Jun 24, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment approach for people with a serious blood condition called high-risk myelodysplastic syndromes (MDS), which affects how blood cells are made in the bone marrow. The study will test a combination of two medicines, venetoclax and azacitidine, given before patients undergo a special type of bone marrow transplant called allogeneic hematopoietic stem cell transplantation (allo-HSCT). The goal is to see if this combination can help control the disease better while patients wait for their transplant.

To take part in this study, patients need to be adults diagnosed recently with high-risk MDS, specifically those whose bone marrow has a higher number of immature blood cells (called blasts). Participants must be healthy enough for the transplant and have good heart, liver, and kidney function. People with other serious health problems, infections like HIV or active tuberculosis, or who are pregnant cannot join. If accepted, patients will receive the study drugs and then proceed to their planned transplant. This trial is not yet open for enrollment, but it aims to improve treatment options for patients facing this challenging condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Newly diagnosed MDS confirmed by morphological and immunophenotypic analysis of bone marrow；
• • 2. Age ≥18 years, any gender；
• • 3. Bone marrow blasts ≥10%；
• • 4. IPSS-R score \>4.5；
• • 5. ECOG performance status 0-2；
• • 6. Scheduled for allogeneic hematopoietic stem cell transplantation (allo-HSCT)；
• • 7. Adequate major organ function：
• • Cardiac: LVEF ≥50%
• • Hepatic: Bilirubin ≤1.5×ULN
• • AST/ALT ≤2.5×ULN
• • Renal: Creatinine clearance ≥60 mL/min；
• • 8. Written informed consent provided by the patient or legally authorized representative.
• Exclusion Criteria:
• • 1. Extramedullary disease involvement；
• • 2. Hypersensitivity to any study drugs；
• • 3. Clinically significant hepatic/renal dysfunction exceeding inclusion thresholds；
• • 4. Severe cardiac disease, including congestive heart failure, myocardial infarction, and cardiac insufficiency;
• • 5. Concurrent malignant tumors of other organs, which can be enrolled if previously cured;
• • 6. Active tuberculosis or HIV infection;
• • 7. Concomitant hematologic disorders;
• • 8. Pregnancy or lactation;
• • 9. Inability to comply with protocol requirements;
• • 10. Concurrently participating in other clinical studies.