从毒性到免疫：通过质量调整生命年 (QALY) 结局评估多发性骨髓瘤中的 CAR-T 与常规治疗

Multiple myeloma (MM) remains characterized by recurrent relapse and cumulative treatment burden despite major therapeutic advances.

This study compared conventional stepwise regimens with CAR-T cell therapy in terms of QoL, toxicity, cost, and ethical considerations within a model-based comparative framework. This comparative pharmacoeconomic modeling analysis was based on published clinical trial data. Kaplan-Meier survival curves were digitized to estimate mean overall survival (OS) and progression-free survival (PFS) using area-under-the-curve integration. Treatment costs were calculated based on published pricing and trial-derived treatment durations. A simple and transparent ECOG-based utility model was developed to enable clinicians, researchers, and health policy authorities to estimate quality-adjusted life years (QALY) and incremental cost-effectiveness ratios (ICER) across regimens spanning heterogeneous treatment lines. The present analysis synthesizes outcome data from 19 pivotal trials and real-world cohorts (N = 7,793 patients). Among heavily pretreated patients, CAR-T therapy approximately doubled OS and PFS compared with other late-line regimens and yielded higher QALY estimates with lower cumulative treatment burden.

Daratumumab-based combinations improved outcomes but reached very high costs ( USD 1 million/patient), while carfilzomib-based regimens remained costly but clinically important for high-risk disease. VMP represented a practical lower-cost option for transplant-ineligible or resource-limited patients. In treatment-line-stratified analysis, later-line/RRMM regimens had higher median ICER-equivalent values than early-line/induction regimens (USD 739,050/QALY vs USD 218,687/QALY; 3.

4-fold higher), whereas CAR-T regimens showed a median ICER-equivalent estimate of USD 299,723/QALY, approximately 2. 5-fold lower than later-line/RRMM conventional regimens. Within this model-based comparative framework, CAR-T provided substantial survival and quality-adjusted outcome gains after three or more prior therapy lines, with promising potential for earlier use. Despite its upfront single-payment structure, CAR-T did not show a disproportionate ICER-equivalent burden compared with later-line conventional regimens.

However, limited global availability raises ethical concerns regarding access, infrastructure, reimbursement, and equity.

Further studies incorporating longer follow-up and patient-level QoL data are needed to refine its role in multiple myeloma care. Multiple myeloma is a cancer of plasma cells that remains difficult to cure despite many new treatments. Most patients eventually relapse and need several lines of therapy.

This study compared traditional drug-based treatments with a new approach called CAR-T cell therapy.

We analyzed survival times, treatment costs, and patients quality of life, which reflects their physical comfort, emotional well-being, and ability to perform daily activities. Results showed that CAR-T therapy helped patients live longer and improved their daily well-being, even among those who had received many previous treatments.

It also reduced the need for repeated hospital visits and showed better long-term outcomes compared with older therapies. Although the initial price of CAR-T therapy seems high, it may actually cost less overall because it can replace many rounds of other expensive drugs.

Our findings suggest that CAR-T therapy could become a more accessible and sustainable treatment in the future.

However, more studies are needed to make this therapy widely available and to ensure fair access for all patients who may benefit from it.