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Peer-reviewedCC BYHematology, Transfusion and Cell Therapy

COMPARISON OF EFFICACY THROUGH RETROSPECTIVE ANALYSIS OF SURVIVAL RATE BETWEEN AZACITIDINE AND LOW DOSE CYTARABINE IN ADULT PATIENTS WITH ACUTE MYELOID LEUKEMIA

  1. 1Faculda Evangélica Mackenzie do Paraná, Curitiba, PR, Brazil

Abstract

Introduction: Acute Myeloid Leukemia (AML) is a malignancy with poor overall survival. Standard intensive chemotherapy remains the primary treatment, yet many patients are unfit due to age and comorbidities. Azacitidine (Aza) and low dose cytarabine (LDAC) are used in these cases, presenting different toxicity profiles, financial access and survival benefits. Objective: To compare survival outcomes and clinical efficacy between Aza and LDAC in adults unfit for intensive chemotherapy. Method: A retrospective, observational, analytical cohort study evaluating demographics, clinical and laboratory parameters, and survival outcomes via descriptive statistics. Conducted in a single tertiary care center in Brazil, between 2019 and 2024. The analysis included 22 adult AML patients unfit for intensive chemotherapy (age > 65 or significant comorbidities), treated with Aza or LDAC. AML inclusion required > 20% blasts in bone marrow or peripheral blood; > 10% blasts with complex cytogenetics; or > 20% immature monocytic cells with blasts. Percentages were primarily evaluated via bone marrow immunophenotyping. Patients receiving Venetoclax (Veneto) via private resources or donations combined with Aza were analyzed as a third group to prevent biases. Results: Of 22 patients, 9 received Aza only; 5 received Aza+Veneto; and 8 LDAC. Clinical remission was achieved in 5 patients: 2 with Aza only, 2 with Aza+Veneto, and 1 LDAC. One Aza+Veneto patient achieved negative minimal residual disease (MRD), and another achieved MRD with LDAC. Dose reduction due to toxicity occurred in 3 Aza only, 1 Aza+Veneto, and 1 LDAC patient. 8 patients were refractory to frontline treatment: 4 in the Aza cohort; 3 in Aza+Veneto; and 1 in LDAC. Furthermore, 3 patients experienced disease relapse after achieving clinical remission (1 per group). Second-line therapy was administered to 5 patients. Of 3 progressing from frontline Aza+Veneto, 1 received Gilteritinib and 2 Veneto (since they began Veneto several cycles after the first cycle of Aza). As second-line therapy, 1 patient from frontline Aza received Veneto, and 1 from LDAC received Aza. Ultimately, all 5 proved refractory to second-line treatments and transitioned to palliative care. In total, 9 patients required palliative management following failure of first- or second-line regimens (4 from initial Aza, 3 Aza+Veneto, 2 LDAC). Palliative interventions for the Aza only cohort consisted of hydroxyurea (n = 2) and LDAC (n = 2); the Aza+Veneto cohort received hydroxyurea (n = 1) and LDAC (n = 2); and the LDAC cohort received hydroxyurea (n = 1) and best supportive care (n = 1). The median overall survival (OS) was 13.4 months for Aza+Veneto; 4.03 months for Aza alone; and 3.3 months for LDAC. Conclusion: In this real-world cohort, frontline Aza+Veneto demonstrated an improved prognostic trend over Aza only or LDAC, having the other two groups similar outcomes. Second-line treatments showed limited efficacy across all groups. These findings underscore that Aza or LDAC have similar efficacy for treatment of unfit adult patients and should be chosen depending on availability and other clinical factors. The Aza + Veneto combination provides far better outcomes for unfit AML patients. This survival disparity highlights the critical necessity to update Brazil’s public health system (SUS) protocols to facilitate equitable access to Veneto, reflecting the complex realities of managing high-cost malignancies in developing nations.

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BibTeX
@article{biasi2026comparison,
  title = {{COMPARISON OF EFFICACY THROUGH RETROSPECTIVE ANALYSIS OF SURVIVAL RATE BETWEEN AZACITIDINE AND LOW DOSE CYTARABINE IN ADULT PATIENTS WITH ACUTE MYELOID LEUKEMIA}},
  author = {João Pedro Greca de Macedo BIASI and Júlia Riedel DERETI and Inácio Skraba SILVA and Maurício LITTIERI and Fabíola Pabst BREMER},
  journal = {Hematology, Transfusion and Cell Therapy},
  year = {2026},
  month = oct,
  doi = {10.1016/j.htct.2026.108459},
  url = {https://doi.org/10.1016/j.htct.2026.108459},
  note = {CC BY},
}
RIS
TY  - JOUR
TI  - COMPARISON OF EFFICACY THROUGH RETROSPECTIVE ANALYSIS OF SURVIVAL RATE BETWEEN AZACITIDINE AND LOW DOSE CYTARABINE IN ADULT PATIENTS WITH ACUTE MYELOID LEUKEMIA
AU  - João Pedro Greca de Macedo BIASI
AU  - Júlia Riedel DERETI
AU  - Inácio Skraba SILVA
AU  - Maurício LITTIERI
AU  - Fabíola Pabst BREMER
PY  - 2026
DA  - 2026/10/23
JO  - Hematology, Transfusion and Cell Therapy
DO  - 10.1016/j.htct.2026.108459
UR  - https://doi.org/10.1016/j.htct.2026.108459
AB  - Introduction: Acute Myeloid Leukemia (AML) is a malignancy with poor overall survival. Standard intensive chemotherapy remains the primary treatment, yet many patients are unfit due to age and comorbidities. Azacitidine (Aza) and low dose cytarabine (LDAC) are used in these cases, presenting different toxicity profiles, financial access and survival benefits. Objective: To compare survival outcomes and clinical efficacy between Aza and LDAC in adults unfit for intensive chemotherapy. Method: A retrospective, observational, analytical cohort study evaluating demographics, clinical and laboratory parameters, and survival outcomes via descriptive statistics. Conducted in a single tertiary care center in Brazil, between 2019 and 2024. The analysis included 22 adult AML patients unfit for intensive chemotherapy (age > 65 or significant comorbidities), treated with Aza or LDAC. AML inclusion required > 20% blasts in bone marrow or peripheral blood; > 10% blasts with complex cytogenetics; or > 20% immature monocytic cells with blasts. Percentages were primarily evaluated via bone marrow immunophenotyping. Patients receiving Venetoclax (Veneto) via private resources or donations combined with Aza were analyzed as a third group to prevent biases. Results: Of 22 patients, 9 received Aza only; 5 received Aza+Veneto; and 8 LDAC. Clinical remission was achieved in 5 patients: 2 with Aza only, 2 with Aza+Veneto, and 1 LDAC. One Aza+Veneto patient achieved negative minimal residual disease (MRD), and another achieved MRD with LDAC. Dose reduction due to toxicity occurred in 3 Aza only, 1 Aza+Veneto, and 1 LDAC patient. 8 patients were refractory to frontline treatment: 4 in the Aza cohort; 3 in Aza+Veneto; and 1 in LDAC. Furthermore, 3 patients experienced disease relapse after achieving clinical remission (1 per group). Second-line therapy was administered to 5 patients. Of 3 progressing from frontline Aza+Veneto, 1 received Gilteritinib and 2 Veneto (since they began Veneto several cycles after the first cycle of Aza). As second-line therapy, 1 patient from frontline Aza received Veneto, and 1 from LDAC received Aza. Ultimately, all 5 proved refractory to second-line treatments and transitioned to palliative care. In total, 9 patients required palliative management following failure of first- or second-line regimens (4 from initial Aza, 3 Aza+Veneto, 2 LDAC). Palliative interventions for the Aza only cohort consisted of hydroxyurea (n = 2) and LDAC (n = 2); the Aza+Veneto cohort received hydroxyurea (n = 1) and LDAC (n = 2); and the LDAC cohort received hydroxyurea (n = 1) and best supportive care (n = 1). The median overall survival (OS) was 13.4 months for Aza+Veneto; 4.03 months for Aza alone; and 3.3 months for LDAC. Conclusion: In this real-world cohort, frontline Aza+Veneto demonstrated an improved prognostic trend over Aza only or LDAC, having the other two groups similar outcomes. Second-line treatments showed limited efficacy across all groups. These findings underscore that Aza or LDAC have similar efficacy for treatment of unfit adult patients and should be chosen depending on availability and other clinical factors. The Aza + Veneto combination provides far better outcomes for unfit AML patients. This survival disparity highlights the critical necessity to update Brazil’s public health system (SUS) protocols to facilitate equitable access to Veneto, reflecting the complex realities of managing high-cost malignancies in developing nations.
SN  - 2531-1379
SN  - 2531-1387
ER  -