OBJECTIVES:

Myelodysplastic syndromes (MDS) are a group of hematological malignancies characterized by cytopenias and an increased risk of progression to acute myeloid leukemia (AML). Red blood cell transfusions constitute the main supportive care for patients with MDS after failure of Erythropoiesis-stimulating agents. The aim was to assess the disease burden in low-risk MDS (LR-MDS) patients from the Spanish National Health System (NHS) perspective.

METHODS:

An incidence-based Markov model was developed to simulate the evolution of LR-MDS over a 5-year time horizon (one-month cycles). Health states modeled were: MDS transfusion-dependent (TD), MDS transfusion-independent (TI), AML, and death. A cohort of newly diagnosed LR-MDS patients based on the characteristics of the ERASME study (Spanish real-world data) entered the model according to transfusion dependence (TD or TI). Transition probabilities were based on ERASME study and a literature review. Utility values were obtained from a systematic literature review. Pharmacological, monitoring, transfusions, and complications management costs were included. A panel of experts validated the model and inputs. Results were expressed in cumulative (5-years) life years (LY), quality-adjusted life-years (QALYs), and costs (€, 2022). Outcomes and costs were discounted at 3%. Deterministic (DSA) and probabilistic sensitivity analyses (PSA) were conducted.

RESULTS:

Based on an incident population of 1,458 patients with LR-MDS, 5,299 LY and 4,286 QALYs over 5 years were calculated, implying a 27.3% reduction in LY and a 41.2% reduction in QALYs compared to a healthy population. TD patients accumulated 36.4% fewer QALYs than TI population. The 5-year cost was €105,582,606. The annual cost per patient with TD was estimated at €43,066 compared to €12,558/patient for TI. In TD patients, the transfusions represented 16.9% of the annual cost. DSA and PSA confirmed robustness of the results.

CONCLUSIONS:

The clinical and economic burden associated with LR-MDS is substantial in Spain, especially in patients with transfusion dependence.