Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN
Key claim: A phase I/II trial is evaluating the safety of venetoclax added to fludarabine/busulfan conditioning and to post-transplant maintenance (with azacitidine or oral decitabine/cedazuridine) in patients with AML, MDS, CMML, or MDS/MPN-unclassifiable undergoing allogeneic stem cell transplant.
Abstract
This clinical trial involves individuals who have been diagnosed with Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), Chronic Myelomonocytic Leukemia (CMML), or MDS/myeloproliferative neoplasm-unclassifiable (MDS/MPN-unclassifiable) and are planning to have an allogeneic hematopoietic stem cell transplant (“bone marrow transplant”). The goal of this research study is to (1) test the safety of adding the study drug, Venetoclax, to a standard of care conditioning regimen for bone marrow transplantation as a possible means of eliminating residual (left-over) disease prior to transplant, (2) to test the safety of combination Venetoclax and azacitidine as “maintenance therapy” after transplant to possibly prevent disease recurrence and (3) to test the safety of combination Venetoclax and oral decitabine/cedazuridine as “maintenance therapy” after transplant to possibly prevent disease recurrence.
- The name of the study drug involved in this study is Venetoclax.
- It is expected that about 102 people will take part in this research study.
Why this matters
Most transplant-intensification strategies act on only one phase (conditioning OR maintenance); this trial tests venetoclax across both, betting that continuous BCL2 inhibition before and after transplant can reduce relapse in myeloid neoplasms including MDS/MPN-unclassifiable and CMML. For the decision layer it doesn’t shift who gets transplanted, but if safe it could expand the operational toolkit for high-relapse-risk patients — and it sets up sequencing questions when HMAs and venetoclax are used both peri-conditioning and as maintenance.