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◆ Therapeutic advances in hematology2026-01-01

From toxicity to immunity: Evaluating CAR-T and conventional therapies in multiple myeloma through quality-adjusted life year (QALY) outcomes.

Guluzar Gulnur Itez, Asuman Sunguroğlu

原始摘要(英文原文)· Original abstract
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.
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From toxicity to immunity: Evaluating CAR-T and conventional therapies in multiple myeloma through quality-adjusted life year (QALY) outcomes. — 科研速览 Science Skim