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Challenges and New Horizons in the Treatment of Patients With AML

During the virtual 2020 Great Debates & Updates in Hematologic Malignancies, Gail Roboz, MD, Professor of Medicine and Director of the Clinical and Translational Leukemia programs at Weill Cornell Medicine and the NewYork-Presbyterian Hospital, discussed how clinicians can lead patients with acute myeloid leukemia (AML) down the correct treatment path, and highlighted a promising new treatment being actively investigated in patients with AML.

CC-486 (an investigational azacytidine) represents a new, promising therapeutic standard for patients aged ≥55 years with AML in first remission.

According to Dr Roboz, CC-486 is the first maintenance therapy that led to statistically significant and clinically meaningful improvements in overall survival (OS) and recurrence-free survival (RFS) in a broad range of patients with AML that was in remission after intensive chemotherapy, with or without consolidation.

In the double-blind QUAZAR AML-001 study by Wei et al, maintenance CC-486 was administered to patients with de novo or secondary AML and an ECOG performance status score 0-3. Across a median follow-up of 41.2 months, the median OS and RFS persisted through 9.9 months and 5.3 months, respectively,

In addition, OS and RFS benefits with CC-486 were observed across key patient subgroups.

The safety and tolerability of CC-486 was deemed controllable, with no unanticipated opposing events. Furthermore, CC-486 preserved overall health-related quality of life compared with placebo.

“For the last 20 to 25 years, we have been complaining that our options in AML are too limited, too few, and not good. But now I think I can show you that we have presented some much better options than what we have had in the past,” Dr Roboz concluded.Alexis Hyams

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