Lenalidomide has been approved for the treatment of transfusion-dependent low- or intermediate-1-risk
myelodysplastic syndromes (MDS) associated with a chromosome 5q deletion with or without additional
cytogenetic abnormalities. We evaluated the cost effectiveness of
lenalidomide versus best supportive care (BSC) in these patients. We developed a decision analytic model to compare costs and outcomes of
lenalidomide with BSC without recombinant
erythropoietin (EPO) versus BSC with EPO over 1 year. Outcome measures were transfusion independence and quality-adjusted life years (QALYs) gained. The model incorporated costs of medications, transfusions, chelation, laboratory tests, office visits, and other resources associated with each
therapy.
Lenalidomide therapy was associated with an estimated incremental 0.53 transfusion-free and 0.25 QALY gain compared to BSC at 1 year. The costs of
lenalidomide therapy were substantially offset by reduced
blood transfusion and EPO costs. One-year total treatment costs were estimated at $63,385 for
lenalidomide and $54,940 for BSC. The incremental cost-effectiveness ratio for
lenalidomide vs BSC was estimated at $16,066 per transfusion-free year and $35,050 per QALY gained, values within the acceptable cost-effectiveness ranges for a new
therapy. Results suggest that oral
lenalidomide is cost effective in the United States in the treatment of transfusion-dependent, low- or intermediate-1-risk MDS associated with a deletion 5q
cytogenetic abnormality. Confirmation of these findings awaits results of an ongoing randomized phase III trial (MDS-004 study).