接受商业化 CAR-T 细胞疗法治疗的复发/难治性多发性骨髓瘤患者的真实世界治疗模式与结局

To characterize physician-reported prescribing patterns and referral pathways for chimeric antigen receptor T-cell (CAR-T) therapy in patients with relapsed/refractory multiple myeloma (RRMM), and to describe treatment patterns and clinical outcomes among patients receiving commercial ciltacabtagene autoleucel (cilta-cel).

This study comprised a descriptive physician survey and retrospective medical chart review. Seventeen physicians representing geographically diverse private community practices (82.4%) and academic centers (17.6%) completed a standardized questionnaire addressing CAR-T referral patterns and perceived barriers. Medical records of 72 adult patients with RRMM who received cilta-cel between February 28, 2022, and June 30, 2024, following US Food and Drug Administration approval, were abstracted to evaluate treatment pathways and clinical outcomes; safety data were not captured.

Physicians reported treating 289 patients who were eligible for CAR-T therapy in the past year; 52.9% indicated that 31%-50% of eligible patients were not referred. For referred patients, efficacy of therapy (76.5%) and good performance status/low frailty (64.7%) were top reasons for referral, whereas patient choice (52.9%), poor performance status/high frailty (47.1%), comorbidity burden (41.2%), and older age (41.2%) were primary reasons for nonreferral. Among the 72 patients who received cilta-cel, approximately half (55.6%) traveled >30 miles to treatment centers, and 27.8% relocated for treatment. In those with a response assessment, the overall response rate was 95.2% and 60.3% achieved at least a complete response. At the end of follow-up (median: 5.6 months), 86.1% were alive, of whom 43.5% achieved remission and were not receiving active treatment.

This real-world study adds to the body of evidence on decision-making for use of commercial CAR-T therapies and supports the development of clinical best practices and guidelines, payer decisions, and health system planning for CAR-T treatment in RRMM.