Integrating clinical and economic outcomes in the elderly myeloma management: a comparative cost-effectiveness...

Multiple myeloma (MM) is a hematologic malignancy that predominantly affects older adults and poses substantial therapeutic challenges due to the high prevalence of comorbidities and limited treatment tolerability. This study evaluates the cost-effectiveness of four first-line treatment regimens for elderly MM patients, ineligible for autologous stem cell transplantation (ASCT): bortezomib, melphalan, and prednisone (VMP); daratumumab in combination with VMP (D-VMP); lenalidomide and dexamethasone (Rd); and daratumumab combined with Rd (DARA-Rd).

A state-transition Markov model was developed, with transition probabilities parameterized using data derived from the pivotal ALCYONE and MAIA clinical trials. Cost-effectiveness was assessed from the perspective of the Italian National Health Service using a state-transition model comparing VMP, D-VMP, Rd and DARA-Rd over a 5.25-year horizon. Probabilistic sensitivity analyses (PSA) were conducted to account for parameter uncertainty and summarized through cost-effectiveness acceptability curves (CEACs).

Rd generated 1.53 QALYs for €60,483, whereas D-VMP generated 1.70 QALYs at €88,331, resulting in an ICER of €160,804/QALY. DARA-Rd and VMP were dominated.

The findings indicate that Rd is the strategy with the highest probability of being cost-effective at willingness-to-pay (WTP) thresholds below €250,000 per quality-adjusted life year (QALY) gained. Conversely, for WTP thresholds exceeding €250,000 per QALY gained, D-VMP emerges as the preferred option, offering the most favorable balance between additional costs and health benefits.