Cost-Effectiveness of Autologous Stem Cell Transplantation in Multiple Myeloma Patients Aged 65 to 69 Years...

Autologous stem cell transplantation (ASCT) remains a cornerstone therapy for eligible patients with multiple myeloma (MM), but its use in older adults is debated because of concerns about toxicity and cost-effectiveness (CE). This study aimed to evaluate the CE of upfront ASCT compared with nontransplant therapy in newly diagnosed patients with MM aged 65 to 69 years within a single-payer healthcare system.

Using nationwide claims data from the Korean Health Insurance Review and Assessment Service (HIRA database, 2009-2024; study enrollment period, 2017-2024), we conducted a CE analysis of 735 patients, of whom 458 received bortezomib-thalidomide-dexamethasone induction followed by ASCT and 277 received nontransplant therapy. A state-transition semi-Markov model-estimated life-years, quality-adjusted life-years (QALYs), and direct medical costs from a healthcare system perspective. Inverse probability of treatment weighting reduced confounding bias, and deterministic and probabilistic sensitivity analyses assessed parameter uncertainty.

ASCT yielded superior overall survival (4-year: 67.8% vs. 55.0%; HR 0.62, P = .001) and longer time to next treatment (29.8 vs 19.7 months, P < .001), with 0.71 additional QALYs at an incremental cost of $21 378. The incremental CE ratio was $30 251 per QALY, remaining below Korea's willingness-to-pay threshold of $37 453. Probabilistic sensitivity analysis demonstrated a 70.3% likelihood of CE at this threshold.

Upfront ASCT is a cost-effective consolidation strategy for appropriately selected patients with MM aged 65 to 69 years within Korea's single-payer healthcare system, supporting fitness-based rather than age-based transplant eligibility criteria.