Real-world Treatment Patterns of Acute Myeloid Leukemia (aml) in the Brazilian Public Healthcare System: a Retrospective Cohort Study Using Nexus Database, 2019–2023
Year: 2026
Type: article
Study design: Observational Study
Abstract: Background Acute myeloid leukemia (AML) is an aggressive hematologic malignancy associated with high mortality. However, population-level real-world evidence describing AML treatment patterns in the Brazilian public healthcare system remains limited. We sought to characterize treatment patterns in patients with newly diagnosed (ND) AML, including first-line regimen intensity (intensive, non-intensive, or mixed). Methods A retrospective cohort study was conducted using the NEXUS database. It links Brazil's national ambulatory and hospital claims systems (SIA/SIH DATASUS) and is mapped to the OMOP Common Data Model. Adults (≥18 years) with a first AML treatment claim between January 2019 and December 2023 were identified. Inclusion required ≥1 AML treatment claims and no prior AML treatment during a ≥12-month washout period, when available. Patients with treatment claims for acute promyelocytic leukemia (APL), non-AML acute leukemias and other cancers were excluded. Non-mandatory follow-up was extended to a minimum of 24 months per patient. Lines of therapy (LoT) were defined using a probabilistic algorithm applied to treatment claims. First-line regimens were classified as intensive, non-intensive, or mixed based on treatments received within 30 days of initiation. Outcomes included patient characteristics, LoT progression, treatment duration, medication use, and hematopoietic stem cell transplantation (HSCT) rates. Results A total of 2,006 AML patients met all eligibility criteria (mean age 42.7 ± 15.5 years; 53.8% male; mean follow-up 3.8 ± 4.3 months). The number of cases varied from 426 in 2019 to 339 in 2023. Most patients (88.6%) had no major baseline comorbidities. HSCT was performed in 5.8% of patients (n=116), and only two patients (0.1%) had documented palliative care. Among 1,912 patients evaluable for LoT analysis, 13.6% (n=273) advanced to second-line (2L), 2.4% (n=48) to third-line (3L), and 0.5% (n=9) to fourth-line or more (4L+) therapy; 85.7% of patients ended after first-line treatment. Cytarabine was the most commonly used agent in all lines. In 1L, cytarabine (n=577) predominated, followed by cytarabine combined with daunorubicin (n=534). Azacitidine was the most frequent hypomethylating agent, observed from 1L through 3L. Classification of first-line regimen intensity showed that 26.3% (n=503) of patients received intensive regimens, 5.5% (n=105) non-intensive regimens, and 68.2% (n=1,304) mixed regimens. Novel targeted therapies (e.g., FLT3 or IDH inhibitors and venetoclax-based combinations) were not identified in this public-sector cohort. Conclusions This study provides the first large-scale, population-level characterization of AML treatment patterns in the Brazilian public healthcare system. Conventional cytarabine-based regimens were the most frequent treatments across all lines of therapy, whereas novel targeted therapies were not identified. Most patients did not progress beyond first-line therapy and HSCT presented a low use. These findings suggest limited adoption of newer AML therapies and reinforce the value of real-world evidence to support healthcare planning, technology assessment, and future strategies to improve treatment access for patients with AML in Brazil.
Institutions: Universidade de São Paulo, Johnson & Johnson (Brazil)
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: diamond
OpenAlex ID: W7223138953