EPIDEMIOLOGICAL PROFILE AND OUTCOMES OF PATIENTS WITH ACUTE MYELOID LEUKEMIA TREATED WITH VENETOCLAX AND AZACITIDINE IN THE BRAZILIAN PUBLIC HEALTH SYSTEM (SUS): PRELIMINARY ANALYSIS OF A REAL-WORLD PILOT PROJECT
- 1Hospital Santa Izabel, Oncoclínicas, Salvador Bahia, Salvador, BA, Brazil
Abstract
Background: Venetoclax plus azacitidine (Ven-Aza) marked a milestone in the treatment of acute myeloid leukemia (AML) in patients ineligible for intensive chemotherapy, following the VIALE-A trial, which demonstrated a significant survival benefit. In 2024, a pilot project was implemented to expand access to Ven-Aza within the Brazilian Public Health System (SUS). Real-world data from this population may help evaluate treatment effectiveness and support policies for equitable access to novel technologies. Aim: To present a preliminary analysis of the epidemiological and clinical profile and outcomes of SUS patients with AML, ineligible for intensive chemotherapy, treated with venetoclax and azacitidine. Materials and methods: Retrospective study based on review of electronic medical records of AML patients followed between May 2024 and August 2026. Data collected included age, sex, AML classification (primary/secondary), prior azacitidine monotherapy, number of cycles, treatment duration, survival, and mortality. Overall survival (OS) was estimated using the Kaplan-Meier method, from treatment initiation to last assessment or death. Statistical analysis was performed in SPSS v25. Results: Fifteen patients were included, with a median age of 62 years (range, 48–84) and 73% female. Secondary AML was present in 53% of cases, and 47% had received prior azacitidine monotherapy. Median number of cycles was 2. Febrile neutropenia occurred in 80% of patients. Median OS was 6.8 months (95% CI, 1.3–18.2). Overall mortality was 60%, with early mortality of 6.7% (D30) and 29.5% (D60). Discussion: This analysis represents an initial real-world case series of AML patients treated with Ven-Aza within the SUS. Compared with the VIALE-A pivotal trial, 30-day mortality was similar (∼7%), but OS was markedly shorter (6.8 vs. 14.7 months). A UK NHS real-world cohort reported longer OS (13.6 months) and lower D30/D60 mortality (5%/8%), suggesting that population- and access-related differences may contribute to the outcomes observed. Febrile neutropenia was also more frequent than reported in VIALE-A (∼42%). Expanding this cohort and comparing findings with supplementary (private) health data may clarify these differences. Real-world evidence from SUS patients, particularly in the Northeast, is essential to evaluate treatment effectiveness under routine care, inform cost-effectiveness analyses, and support equitable AML treatment access in the public health system.
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BibTeX
@article{portugal2026epidemiological,
title = {{EPIDEMIOLOGICAL PROFILE AND OUTCOMES OF PATIENTS WITH ACUTE MYELOID LEUKEMIA TREATED WITH VENETOCLAX AND AZACITIDINE IN THE BRAZILIAN PUBLIC HEALTH SYSTEM (SUS): PRELIMINARY ANALYSIS OF A REAL-WORLD PILOT PROJECT}},
author = {Beatriz Moura PORTUGAL and Valney Oliveira Santos JUNIOR and Maura Alice Santos ROMEO and Luiz Henrique dos Santos de ASSIS and Giulia Beatriz Correia MATOS and Lorhane Nunes dos ANJOS},
journal = {Hematology, Transfusion and Cell Therapy},
year = {2026},
month = oct,
doi = {10.1016/j.htct.2026.107584},
url = {https://doi.org/10.1016/j.htct.2026.107584},
note = {CC BY},
}
RIS
TY - JOUR
TI - EPIDEMIOLOGICAL PROFILE AND OUTCOMES OF PATIENTS WITH ACUTE MYELOID LEUKEMIA TREATED WITH VENETOCLAX AND AZACITIDINE IN THE BRAZILIAN PUBLIC HEALTH SYSTEM (SUS): PRELIMINARY ANALYSIS OF A REAL-WORLD PILOT PROJECT
AU - Beatriz Moura PORTUGAL
AU - Valney Oliveira Santos JUNIOR
AU - Maura Alice Santos ROMEO
AU - Luiz Henrique dos Santos de ASSIS
AU - Giulia Beatriz Correia MATOS
AU - Lorhane Nunes dos ANJOS
PY - 2026
DA - 2026/10/23
JO - Hematology, Transfusion and Cell Therapy
DO - 10.1016/j.htct.2026.107584
UR - https://doi.org/10.1016/j.htct.2026.107584
AB - Background: Venetoclax plus azacitidine (Ven-Aza) marked a milestone in the treatment of acute myeloid leukemia (AML) in patients ineligible for intensive chemotherapy, following the VIALE-A trial, which demonstrated a significant survival benefit. In 2024, a pilot project was implemented to expand access to Ven-Aza within the Brazilian Public Health System (SUS). Real-world data from this population may help evaluate treatment effectiveness and support policies for equitable access to novel technologies. Aim: To present a preliminary analysis of the epidemiological and clinical profile and outcomes of SUS patients with AML, ineligible for intensive chemotherapy, treated with venetoclax and azacitidine. Materials and methods: Retrospective study based on review of electronic medical records of AML patients followed between May 2024 and August 2026. Data collected included age, sex, AML classification (primary/secondary), prior azacitidine monotherapy, number of cycles, treatment duration, survival, and mortality. Overall survival (OS) was estimated using the Kaplan-Meier method, from treatment initiation to last assessment or death. Statistical analysis was performed in SPSS v25. Results: Fifteen patients were included, with a median age of 62 years (range, 48–84) and 73% female. Secondary AML was present in 53% of cases, and 47% had received prior azacitidine monotherapy. Median number of cycles was 2. Febrile neutropenia occurred in 80% of patients. Median OS was 6.8 months (95% CI, 1.3–18.2). Overall mortality was 60%, with early mortality of 6.7% (D30) and 29.5% (D60). Discussion: This analysis represents an initial real-world case series of AML patients treated with Ven-Aza within the SUS. Compared with the VIALE-A pivotal trial, 30-day mortality was similar (∼7%), but OS was markedly shorter (6.8 vs. 14.7 months). A UK NHS real-world cohort reported longer OS (13.6 months) and lower D30/D60 mortality (5%/8%), suggesting that population- and access-related differences may contribute to the outcomes observed. Febrile neutropenia was also more frequent than reported in VIALE-A (∼42%). Expanding this cohort and comparing findings with supplementary (private) health data may clarify these differences. Real-world evidence from SUS patients, particularly in the Northeast, is essential to evaluate treatment effectiveness under routine care, inform cost-effectiveness analyses, and support equitable AML treatment access in the public health system.
SN - 2531-1379
SN - 2531-1387
ER -