Novel therapies in myeloid neoplasms show limited benefit and increased costs over 15 years of follow-up in Southern Finland

Over the past two decades, targeted therapies have been adopted in the treatment of myeloid neoplasms. However, their impact on overall survival (OS) and healthcare spending remains uncertain. We performed a comprehensive retrospective observational study on patients with acute myeloid leukemia (AML, n = 684), myelodysplastic syndrome (MDS, n = 899) and myelofibrosis (MF, n = 276) diagnosed between 2009 and 2023 in a large hospital district in Southern Finland. During this 15-year period, the median age increased across all cohorts. OS remained unchanged after adjusting for age, comorbidity index and risk score in AML (HR 0.77, 95% CI 0.59–1.02), MDS (HR 1.03, 95% CI 0.81–1.31), and MF (HR 0.70, 95% CI 0.45–1.08). However, progression-free survival (PFS) improved among AML patients. For subgroup analyses, OS and PFS improved in AML receiving high-dose chemotherapy, mostly due to better stratification to fit and unfit treatment regimens. In MDS and MF cohorts, no OS benefit was observed in any subgroup. Hospitalization and drug expenses were the primary components in all cohorts and increased notably in AML and MF patients due to novel treatments. Advances in clinical care and adoption of novel agents show limited benefit but have gradually increased their financial burden. Future research should prioritize less toxic, outpatient-based treatments to enhance outcome and cost-effectiveness.