Real-world acute myeloid leukemia outcomes during a decade of molecular diagnostic expansion and azacitidine plus venetoclax adoption in Thailand: A single-center implementation cohort
Thanawat Rattanathammethee, Noppamas Panyasit, Piangrawee Niprapan, Sirichai Srichairatanakool, Teerachat Punnachet, Nonthakorn Hantrakun, Pokpong Piriyakhuntorn, Sasinee Hantrakool, Chatree Chai-Adisaksopha, Adisak Tantiworawit, Lalita Norasetthada, Ekarat RattarittamrongBackground
Expanded molecular diagnostics and introduction of azacitidine plus venetoclax have reshaped acute myeloid leukemia (AML) management. However, real-world data on these impacts in Southeast Asia remain limited.
Objectives
To evaluate clinical outcomes across two implementation eras defined by expanded molecular testing and adopting of azacitidine plus venetoclax.
Design
Retrospective single-center cohort study.
Methods
We included adults with newly diagnosed AML, comparing early (2016-2020) and later (2021-2025) eras. The primary endpoint was the adjusted between-era difference in 24-month restricted mean survival time (RMST) for overall survival (OS). Secondary approaches included propensity score matching (PSM) to adjust for baseline imbalances and a 6-month landmark analysis to evaluate allogeneic stem cell transplantation (alloSCT) outcomes without immortal time bias.
Results
Among 285 patients, 228 received AML-directed therapy (early n=107; later n=121). Later-era patients were significantly older and more frequently classified as ELN adverse risk, coinciding next-generation sequencing implementation (22.3%). Adjusted 24-month RMST for OS did not differ between eras (difference -0.06 months; 95% CI -2.41 to 2.27; p = 0.956). To control for referral bias, a 1:1 PSM yielded 84 well-balanced pairs (n=168). Within this matched cohort, median OS (15.4 vs. 14.7 months; HR 1.02; p = 0.908) and relapse-free survival remained comparable between eras. In the 6-month landmark analysis of the PSM subset, alloSCT demonstrated a clinical trend toward reduced mortality (adjusted HR 0.69, 95% CI 0.34 to 1.38).
Conclusion
Despite major shifts in treatment patterns, no improvement in adjusted short-term survival was observed at the population level. Notably, the higher proportion of adverse risk patients in the later era likely reflects both enhanced molecular ascertainment and changes in referral patterns, rather than a pure biological shift alone. Expanding transplant access and comprehensive molecularly guided therapy remain key priorities.