OBJECTIVES: In the era of novel agents, patients are confronted with various treatment combinations of autologous stem cell transplantation (ASCT) and maintenance therapy. This study aimed to assess the cost-effectiveness of different strategies involving ASCT and maintenance therapy for transplant-eligible patients with newly diagnosed multiple myeloma (NDMM) from a Chinese health care system perspective.

METHODS: We employed a partitioned survival model to determine the mean costs and quality-adjusted life-years (QALYs) of different treatment combinations of ASCT and maintenance therapy over a 5-year time horizon. A network meta-analysis (NMA) was conducted to provide clinical input for constructing the model. The utility was derived from published literature. Health care costs were informed from a single-center retrospective analysis and nation-level drug bidding and tendering database.

RESULTS: There were 9 combination strategies identified in the NMA：ASCT + carfilzomib, lenalidomide, and dexamethasone，ASCT +lenalidomide，ASCT + one-year lenalidomide，ASCT + two-years daratumumab，ASCT + two-year ixazomib，ASCT + two-year bortezomib，ASCT alone，no ASCT + lenalidomide and no ASCT + one-year lenalidomide. Of these strategies, the strategy involving ASCT followed by lenalidomide maintenance obtained the most undiscounted life years (4.44); the strategy involving ASCT followed by maintenance therapy with carfilzomib, lenalidomide, and dexamethasone gained the most discounted QALYs (3.33); and the strategy involving ASCT followed by two-year daratumumab maintenance incurred the least discounted costs (599,408 CNY), which was the most cost-effective option under the WHO-recommended WTP threshold of 257,094 CNY per QALY.

CONCLUSIONS: Considering its cost-effectiveness profile, two-year daratumumab maintenance therapy following ASCT is considered highly worthwhile for transplant-eligible patients with NDMM in clinical practice in China. Additionally, ASCT retains its value in the first-line setting of clinical practice due to its capacity to deliver superior gains in QALYs and cost reductions compared to the strategy without ASCT.