Overview
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)
Description
This is a single-center, single-arm, prospective study investigating pre-transplant bridging therapy in patients aged ≥18 years with higher-risk MDS-IB2. The study plans to enroll 46 eligible patients. Participants will receive Venetoclax combined with Azacitidine, administered in 28-day cycles for 1 to 2 cycles. Treatment response will be assessed according to the IWG 2023 HR-MDS response criteria.Patients achieving modified Composite Complete Remission (mCRc: CR or CR-equivalent + CRL + CRh) after Cycle 1 will proceed directly to transplantation. Patients not achieving mCRc will receive a second cycle of therapy. All patients will undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT) within 3 months after completing Cycle 2, regardless of mCRc status. Patients unable to proceed to transplantation will receive standard institutional care and undergo follow-up.
Eligibility
Inclusion Criteria:
- Newly diagnosed MDS confirmed by morphological and immunophenotypic analysis of bone marrow；
- Age ≥18 years, any gender；
- Bone marrow blasts ≥10%；
- IPSS-R score >4.5；
- ECOG performance status 0-2；
- Scheduled for allogeneic hematopoietic stem cell transplantation (allo-HSCT)；
- Adequate major organ function：
- Cardiac: LVEF ≥50%
- Hepatic: Bilirubin ≤1.5×ULN
- AST/ALT ≤2.5×ULN
- Renal: Creatinine clearance ≥60 mL/min；
- Written informed consent provided by the patient or legally authorized
representative.
Exclusion Criteria:
- Extramedullary disease involvement；
- Hypersensitivity to any study drugs；
- Clinically significant hepatic/renal dysfunction exceeding inclusion thresholds；
- Severe cardiac disease, including congestive heart failure, myocardial infarction, and cardiac insufficiency;
- Concurrent malignant tumors of other organs, which can be enrolled if previously cured;
- Active tuberculosis or HIV infection;
- Concomitant hematologic disorders;
- Pregnancy or lactation;
- Inability to comply with protocol requirements;
- Concurrently participating in other clinical studies.