Impact of Transfusion Dependence on Clinical and Economic Burden in Patients with Lower-Risk Myelodysplastic...

Myelodysplastic syndromes comprise a diverse group of hematopoietic stem-cell malignancies characterized by ineffective hematopoiesis, cytopenia, and increased risk of acute myeloid leukemia. Anemia is a common manifestation of lower-risk myelodysplastic syndrome (LR-MDS) and often necessitates red blood cell (RBC) transfusions, although reliance on transfusions is associated with adverse outcomes and substantial burden. This 28-year, single-center, retrospective study aimed to assess the burden of RBC transfusion-dependence (RBC-TD) in terms of healthcare resource utilization (HCRU), medical costs, and clinical outcomes among patients with LR-MDS in South Korea.

Adult patients with LR-MDS initiating treatment between 1994 and 2022 at the Samsung Medical Center were included. HCRU, medical costs, and clinical outcomes were analyzed based on RBC-TD status.

Among 349 patients with LR-MDS, 103 (29.5%) were RBC-TD. RBC-TD was associated with increased HCRU and medical costs compared with non-transfusion-dependent (NTD) patients. Baseline erythropoietin (EPO) levels before erythropoiesis-stimulating agent (ESA) treatment were available for 76 patients with RBC-TD. Among 51 TD patients with baseline EPO levels ≤500U/L, 20 initiated ESA treatment after RBC-TD developed, to observe only limited effectiveness. Moreover, RBC-TD patients exhibited significantly shorter overall survival, leukemia-free survival and progression-free survival compared with NTD patients.

RBC-TD in LR-MDS imposes significant clinical and economic burdens, including poor outcomes, heightened HCRU, elevated risks of disease progression, and premature mortality. These trends were particularly pronounced in patients with LR-MDS compared with the overall MDS population, underscoring that "lower-risk" does not necessarily equate to "lower-burden".