Venetoclax plus azacitidine as a bridge to stem-cell transplant for higher-risk MDS-IB2

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)

This will test whether 1–2 cycles of venetoclax with azacitidine can lower bone marrow blasts and help adults with higher‑risk MDS‑IB2 reach allogeneic stem-cell transplantation.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 46 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: The First Hospital of Jilin University (Academic / other)
• Locations: 1 site (Changchun, Jilin)
• Trial ID: NCT07047183 on ClinicalTrials.gov

What this trial studies

This is a single-center, single-arm Phase 2 study enrolling about 46 adults with higher‑risk MDS‑IB2 and marrow blasts ≥10% who are planned for allogeneic hematopoietic stem-cell transplantation. Participants receive venetoclax combined with azacitidine in 28‑day cycles for one or two cycles, with response measured by the IWG 2023 HR‑MDS criteria. Patients who achieve a modified composite complete remission (mCRc) after Cycle 1 proceed directly to transplant, while others receive a second cycle and all are intended to undergo transplant within three months after completing Cycle 2. Patients unable to proceed to transplant will receive standard institutional care and continue follow‑up.

Who should consider this trial

Good fit: Adults (≥18 years) with newly diagnosed higher‑risk MDS‑IB2 (bone marrow blasts ≥10%, IPSS‑R >4.5), ECOG 0–2, adequate organ function, and a plan for allogeneic HSCT are the intended candidates.

Not a fit: Patients with extramedullary disease, significant organ dysfunction beyond entry thresholds, severe cardiac disease, active uncontrolled infections (e.g., TB, HIV), or those who cannot proceed to transplant are less likely to benefit.

Why it matters

Potential benefit: If successful, this approach could increase the number of higher‑risk MDS patients who reach transplant by reducing pre‑transplant disease burden.

How similar studies have performed: Venetoclax plus hypomethylating agents has shown promising activity in AML and some higher‑risk MDS case series, but prospective data specifically as a bridging regimen to transplant remain limited.

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.

Where this trial is running

Changchun, Jilin

• The First Hospital of Jilin University — Changchun, Jilin, China (Recruiting)

Study contacts

• Study coordinator: Yehui Tan

Conditions Myelodysplastic Neoplasms; Transplantation