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Published on: June 6, 2025
Azacitidine results in comparable outcome in newly diagnosed AML patients with more or less than 30% bone marrow
L H van der Helm1, N J G M Veeger, M van Marwijk Kooy
1Department of Hematology, University Medical Center Groningen, Groningen, The Netherlands.
Abstract:
The efficacy of azacitidine has been demonstrated in acute myeloid leukemia (AML) patients with 20-30% bone marrow (BM) blasts, but limited data is available on patients with ≥30% blasts. We analyzed 55 newly diagnosed AML patients, treated with azacitidine. The overall response rate was 42%. Median overall survival (OS) was 12.3 months. We confirmed poor-risk cytogenetics, therapy-related AML, performance score ≥2, and white blood cell count ≥15×10(9)/L as independent adverse predictors for OS. The BM blast percentage, however, had no impact on OS (P=0.55). In conclusion, administration of azacitidine is effective in AML patients with 20-30% and >30% BM blasts.
Insights
Azacitidine is effective for acute myeloid leukemia (AML) patients, regardless of bone marrow blast percentage. This study shows similar outcomes for AML patients with 20-30% and over 30% blasts.
Area of Science:
- Hematology
- Oncology
- Clinical Pharmacology
Background:
- Azacitidine is approved for acute myeloid leukemia (AML) with 20-30% bone marrow (BM) blasts.
- Limited data exists on azacitidine efficacy in AML patients with higher BM blast percentages (≥30%).
Purpose of the Study:
- To evaluate the efficacy of azacitidine in newly diagnosed AML patients with varying BM blast percentages.
- To identify independent prognostic factors for overall survival (OS) in AML patients treated with azacitidine.
Main Methods:
- Retrospective analysis of 55 newly diagnosed AML patients treated with azacitidine.
- Assessment of overall response rate (ORR) and OS.
- Multivariate analysis to identify predictors of OS.
Main Results:
- The overall response rate was 42% with a median OS of 12.3 months.
- Poor-risk cytogenetics, therapy-related AML, performance score ≥2, and WBC count ≥15×10(9)/L were independent adverse predictors for OS.
- BM blast percentage did not significantly impact OS (P=0.55).
Conclusions:
- Azacitidine demonstrates efficacy in AML patients with both 20-30% and >30% BM blasts.
- Treatment decisions for AML should consider factors beyond BM blast percentage, such as cytogenetics and performance status.
