Clinical Study of Venetoclax Combined With Azacitidine as Bridging Therapy Prior to Hematopoietic Stem Cell Transplantation in Patients With Higher-Risk Myelodysplastic Syndromes
VEN (Venetoclax): Enrolled patients will receive: Venetoclax: 100 mg on Day 1, 200 mg on Day 2, and 400 mg on Days 3-14. Azacitidine: 75 mg/m² on Days 1-7. Cycle duration: 28 days Total cycles: 1 to 2 cycles
Study summary
A Single-Arm, Prospective Clinical Study of Venetoclax Combined with Azacitidine Followed by Bridging Transplantation in Patients with High-Risk Myelodysplastic Neoplasms with Increased Blasts 2 (MDS-IB2)
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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.
Primary outcome measure(s)
2-Year Relapse-Free Survival(2-RFS) — From date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 24 months. Defined as the time from enrollment in this study to the occurrence of relapse. For patients without relapse, the time is calculated until death from any cause or the time of the last follow-up
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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