Understanding How Azacitidine Works and Why It May Not Work in High-Risk Myelodysplastic Syndromes and Acute Myeloid Leukemia with Multilineage Dysplasia

What this Trial Is About

This research focuses on high-risk myelodysplastic syndromes MDS and acute myeloid leukemia with multilineage dysplasia AML-MD, conditions common in older adults with a median age of 71 years. The study aims to understand how the drug Azacitidine works and why resistance to it develops, particularly looking at its effects on cell death processes like apoptosis and autophagy. The investigation also explores new drugs that might reverse resistance to Azacitidine by targeting these cell death pathways. Participants in this observational study are adults aged 18 years or older with high-risk or intermediate 2 MDS or AML-MD who have undergone treatment with at least three to six cycles of Azacitidine. The study does not involve additional treatment but collects data to characterize molecular mechanisms of action and resistance. Researchers focus on hematological responses at three and six months and overall survival from the start of treatment and at death. During the study, participants clinical and molecular data related to their response to Azacitidine are observed and analyzed. The main outcomes include blood response measurements and survival tracking. The study is sponsored by the Centre Hospitalier Universitaire de Nice and began in 2010, with planned follow-up through September 2025. Participation involves regular monitoring without additional interventions, allowing researchers to evaluate treatment impact and resistance mechanisms over time.

Characterization of the Mechanisms of Resistance to Azacitidine