接受标准治疗 Idecabtagene Vicleucel 的多发性骨髓瘤患者的患者报告结局

Idecabtagene vicleucel (ide-cel) was the first FDA-approved chimeric antigen receptor T-cell therapy for relapsed/refractory multiple myeloma (RRMM) patients. This was the first study to evaluate patient-reported outcomes (PROs) among RRMM patients receiving ide-cel in standard of care (SOC).

We prospectively assessed health-related quality of life (HRQOL) and symptoms from pre-infusion (baseline) through day (D)90 post-infusion. Baseline PRO associations with patient characteristics, mean PRO changes, and time to stable change were evaluated with t -tests, linear mixed-effects models, and Kaplan-Meier analyses, respectively. Within-person change scores and minimally important difference thresholds determined clinical and meaningful significance.

Participants ( n = 42) were a median of 66 years old (range: 43-81). At baseline, extramedullary disease was associated with worse physical well-being ( p = 0. 008), global pain ( p < 0. 001), performance status ( p = 0. 002), and overall symptom burden ( p < 0. 001). Fatigue ( p < 0. 001) and functional well-being ( p = 0. 003) worsened by D7 before returning to baseline levels.

Overall HRQOL ( p = 0. 008) and physical well-being ( p < 0. 001) improved by D60. Most participants reported PRO improvement (10-57%) or maintenance (23-69%) by D90. The median time it took to stabile deterioration in functional well-being was 14 days. The median time it took to stabile improvement in physical and emotional well-being was 60 days.

Overall, RRMM patients reported improvements or maintenance of HRQOL and symptom burden after SOC ide-cel.