利用真实世界证据探讨多发性骨髓瘤患者的 CAR-T 治疗可及性

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.