Incidence and Burden of Lower-Risk Myelodysplastic Syndrome: A Nationwide Population Study.

Abstract

Background: Myelodysplastic syndromes (MDS) are hematological malignancies that primarily affect older individuals, often leading to anemia, which significantly impacts quality of life. Until now, the management of lower-risk MDS (LR-MDS) typically includes erythropoiesis-stimulating agents (ESAs) as first-line treatment, with transfusions becoming necessary in cases of ESA resistance. This study aimed to assess the incidence, prevalence, and clinical outcomes of LR-MDS patients in France, using the French National Health Data System (SNDS).

Materials and methods: A retrospective cohort of 822 LR-MDS patients treated between 2018 and 2022 was analyzed, with patients classified based on transfusion dependency.

Results: Results showed a median LR-MDS extrapolated incidence of 5,850 patients per year in France (between 6.9 to 9.3 cases per 100,000 persons). Transfusion-dependent (TD) patients represented 32.5% of the cohort. TD patients had a significantly lower 2-year overall survival rate of 53% compared to 70% in nontransfusion-dependent (NTD) patients. Over the study period, 41.9% (N = 112) died out of 267 newly treated LR-MDS patients. Also, there were 88 events of death (with no progression), 26 estimated progression to HR-MDS and 11 progression to acute myeloid leukemia.

Conclusion: These findings highlight the substantial burden of transfusion dependency in LR-MDS patients, emphasizing the need for improved therapeutic strategies.