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Observational Study
. 2025 Apr 15;131(8):e35845.
doi: 10.1002/cncr.35845.

Real-world treatment patterns and outcomes with oral azacitidine maintenance therapy in patients with acute myeloid leukemia

Brian Leber  1 , Marcia T Ruiz  2 , Hany Elgendy  2 , Filippa Pettersson  2 , Thomas Prebet  2 , Carlos E Vigil  2 , Rohan C Parikh  3 , Siddhi Korgaonkar  3 , Fareedat Bello  3 , Keith L Davis  3 , Lona Gaugler  2 , Maria Strocchia  2 , Jan Sieluk  2 , Yeran Li  2 , Andre C Schuh  4
Affiliations
Observational Study

Real-world treatment patterns and outcomes with oral azacitidine maintenance therapy in patients with acute myeloid leukemia

Brian Leber et al. Cancer. .

Abstract

Introduction: This study describes baseline and clinical characteristics, treatment patterns, survival, and safety outcomes of patients with acute myeloid leukemia (AML) who received oral azacitidine (oral-AZA) maintenance therapy in Canada following its approval in 2021.

Methods: A retrospective, observational medical record review was conducted of patients with AML in remission after induction therapy and who initiated treatment with oral-AZA between March 2021 and July 2023 in Canada. Real-world relapse-free survival and overall survival outcomes were estimated using Kaplan-Meier methodology.

Results: Data from 119 patients were analyzed. The median age at oral-AZA initiation was 62.5 years. Most patients had favorable (39.5%) or intermediate (39.5%) genetic risk per the 2017/2022 European LeukemiaNet classification. Nearly all patients (99.2%) received cytarabine-based induction regimens. A total of 55.5% of patients received consolidation therapy, with a median of two cycles. After a median follow-up of 9.4 months, 68.1% of all patients were still receiving oral-AZA at last follow-up. After oral-AZA treatment, 21.0% of patients relapsed. Rates of real-world relapse-free survival and overall survival at 12 months from oral-AZA initiation were 66.9% and 74.5%, respectively. During oral-AZA treatment, 67.2% of patients experienced ≥1 adverse event. Concomitant antiemetic treatment was received by 78.2% of patients.

Conclusion: These findings provide real-world evidence further supporting the use of oral-AZA as a standard-of-care maintenance therapy in current routine clinical practice for patients with AML in remission who do not receive hematopoietic stem cell transplantation. These results may inform a broader clinical audience because of the inclusion of patients with diverse demographic and clinical characteristics.

Keywords: acute myeloid leukemia; maintenance therapy; oral‐AZA; real‐world evidence.

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References

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