Investigating CAR-T Treatment Access for Multiple Myeloma Patients Using Real-World Evidence

利用真实世界证据调查多发性骨髓瘤患者接受 CAR-T 疗法的途径

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Abstract

Importance: Multiple myeloma (MM) is the second most common hematologic malignancy in the U.S., with a higher incidence among Black patients than White patients. Chimeric antigen receptor T-cell (CAR-T) therapies show clinical promise, but their limited availability raises concerns about access. Objective: To examine associations between disease characteristics, treatment location, and patient demographics with receipt of CAR-T therapy among patients with MM. Design: Retrospective cohort study using electronic health record data from the University of California Health Data Warehouse (UCHDW) between January 2021 and January 2025. Setting: Six academic health centers and twelve affiliated hospitals within the UCHDW. Participants: A population-based cohort of 12,360 adult patients diagnosed with MM and treated at a University of California facility offering CAR-T administration. Analyses were conducted from February 2025 to March 2025. Exposures: Receipt of multiple cancer therapies following MM diagnosis. Main Outcomes and Measures: Logistic regression was used to estimate odds ratios (ORs) and 95% confidence intervals (CIs) for associations between disease characteristics, treatment locations, and patient demographics with receipt of CAR-T therapy. A zero-shot GPT-4 inference model was applied to UCSF clinical notes to assess whether CAR-T therapy was discussed, determine documented eligibility, and classify rationale for eligibility determinations. Results: Among 12,360 patients with MM (mean age, 68.5 years; 51.6% male), 320 (2.6%) received CAR-T therapy. Disease characteristics at diagnosis, measured by the International Staging System (ISS), was distributed as follows: Stage I (65.3%), Stage II (24.4%), Stage III (2.8%), and Unknown (7.5%). Patients treated at UC-1 (49.3%), and UC-2 (50.0%) were more frequently diagnosed with ISS Stage II, whereas patients treated at UC-3 (55.5%) were more frequently diagnosed with ISS Stage I. Our model showed that patients identifying as Black or African American had lower odds of receiving CAR-T therapy compared with White patients (OR, 0.33; [95% CI, 0.17-0.62]). Patients treated at UC-3 also had lower odds of receiving CAR-T therapy compared with UC-1 (OR, 0.42; [95% CI, 0.30-0.59]). Among 270 UCSF patients assessed for CAR-T eligibility using clinical notes, the proportion of patients deemed eligible without documented CAR-T discussions was highest among those identifying as Other Pacific Islander (50%), followed by Black or African American (4.2%), Asian (3.2%), and White patients (0.6%). Conclusions and Relevance: Within a large academic health system, receipt of CAR-T therapy varied by treatment location and patient-reported race. A subset of patients with documented eligibility lacked recorded discussions of CAR-T therapy, suggesting potential differences in referral, documentation, or care pathways influencing observed treatment patterns.

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