Expert Highlights Safety Profile of Oral Azacitidine in Lower-Risk MDS


Adverse effects associated with oral azacitidine in low- or intermediate-risk MDS are typically transient, according to Mikkael A. Sekeres, MD, MS.

Mikkael A. Sekeres, MD, MS, spoke with CancerNetwork® at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting about monitoring and managing toxicity in patients who receive oral azacitidine (Onureg) for low- or intermediate-risk myelodysplastic syndromes (MDS). He discussed the safety profile of the agent in the context of findings from the phase 2/3 ASTREON trial (NCT05469737), which assessed oral azacitidine plus best supportive care in patients with lower-risk MDS.

According to Sekeres, a professor of Medicine and chief of the Division of Hematology at Sylvester Comprehensive Cancer Center of the University of Miami Health System, most toxicity associated with oral azacitidine in the trial, including neutropenia and thrombocytopenia, were transient, as patient blood levels typically stabilized following these adverse effects. Additionally, any instances of nausea during treatment were mitigated with anti-nausea drugs.

Sekeres also described the potential next steps for researching orally available azacitidine in this patient population. Future research aims to replicate the responses reported in the initial ASTREON trial cohort across an expanded population of those with lower-risk MDS.


The next step in looking at oral azacitidine is to use it in an expanded patient population of those with lower-risk myelodysplastic syndromes and figure out if those response rates that we saw in this initial cohort of patients are replicated in a larger population. [We will] also look for any unusual [adverse] effects.

One of the most common adverse effects associated with any hypomethylating agent is that these drugs can make the blood counts worse before they make them better. Patients may develop neutropenia while taking these drugs, develop thrombocytopenia, and their anemia may not correct itself. Usually, these [adverse] effects are transient. Particularly, in those patients who respond to the drugs, they recover their blood counts. These drugs can also be associated with nausea. This has largely been entirely abrogated with the use of very effective anti-nausea medicines.


Garcia-Manero G, Yee KWL, Hernandez F, et al. Preliminary safety and efficacy of oral azacitidine (Oral-AZA) in patients (pts) with low-/Intermediate (Int)-risk myelodysplastic syndromes (MDS): phase 2 results from the ASTREON trial. J Clin Oncol. 2024;42(suppl 16):6509. doi:10.1200/JCO.2024.42.16_suppl.6509

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