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2025 ISPE Annual Meeting Main banner

Health Economics / Outcomes Research

4D-05 - Patient-Reported Outcomes Among Patients with Triple-Class Refractory Multiple Myeloma in Real-World Clinical Practice: A Prospective, Multi-Site Observational Study – Interim Results

Monday, August 25, 2025
5:15 PM - 5:30 PM ET
Background: Patients with triple-class refractory (TCR) multiple myeloma (MM) experience a significant decline in health-related quality of life (QOL) and poor clinical outcomes.

Objectives: To describe longitudinal changes in QOL and other patient-reported outcomes (PROs) among TCR MM patients who initiated a new line of therapy in real-world clinical practice.

Methods: A prospective, multi-site observational study was conducted within the Mayo Clinic system. Patients initiating a new line of therapy (index date) were followed for 12 months. For CAR-T patients, PRO surveys were collected at apheresis, lymphodepletion, and monthly through Month 6. Non-CAR-T patients were surveyed pre-index and monthly through Month 6. PRO measures included the EORTC Quality of Life Questionnaire (QLQ-C30; including the Global Health Score [GHS]), EORTC Multiple Myeloma Questionnaire (QLQ-MY20; including the Disease Symptoms [DS] score), EuroQoL-5 Dimensions (EQ-5D), the Patient Global Impression of Severity (PGIS) and Change (PGIC). This analysis reflects patient data as of May 2024.

Results: The interim analysis included 77 patients (58% male, 95% White, mean age 66 years). The median time from becoming TCR to index therapy was 5.6 months (IQR: 1.8, 18.2). Most patients had ECOG 0-1 (48% and 45%) and R-ISS I-II (27% and 36%); 42% had high-risk cytogenetics, and 14% had extramedullary disease. Patients had a median of 4 prior treatment lines. Among 42 CAR-T patients (ide-cel: 12, cilta-cel: 30), QOL remained stable from apheresis (GHS: 62.3, EQ-5D: 66.4) through Month 6 (64.2, 68.9). Disease symptoms improved (DS: 25.7 at apheresis, 20.1 at Month 6), but pain plateaued/worsened (pain scores: 24.5 at apheresis, 32.6 at Month 6). By Month 1, 76% of CAR-T patients reported feeling “a little better” or “much better” (75% at Month 6) per PGIC. Among 35 non-CAR-T patients, common regimens included pomalidomide + elotuzumab + corticosteroids (26%), carfilzomib + cyclophosphamide + corticosteroids (20%), and pomalidomide + carfilzomib + corticosteroids (17%). QOL remained stable (GHS: 62.9 at baseline, 64.9 at Month 6), while disease symptoms improved (DS: 28.0 at baseline, 22.8 at Month 6). Pain also decreased (40.8 at baseline, 28.1 at Month 6). PGIC scores improved over time, with 30.7% of patients reporting feeling “a little/much better” at Month 1, increasing to 52.7% by Month 6.

Conclusions: This interim analysis suggests a substantial QOL burden among TCR MM patients at baseline. QOL remained stable across both CAR-T and non-CAR-T cohorts post-index, with select improvements in disease symptoms. However, patients—particularly those receiving CAR-T—reported notable holistic improvements as early as Month 1 based on PGIC assessment.