Oswald Laura B, Gudenkauf Lisa M, Li Xiaoyin, De Avila Gabriel, Peres Lauren C, Kirtane Kedar, Gonzalez Brian D, Hoogland Aasha I, Nguyen Oanh, Rodriguez Yvelise, Baz Rachid C, Shain Kenneth H, Alsina Melissa, Locke Frederick L, Freeman Ciara, Castaneda Puglianini Omar, Nishihori Taiga, Liu Hien, Blue Brandon, Grajales-Cruz Ariel, Jim Heather S L, Hansen Doris K
Department of Health Outcomes and Behavior, Moffitt Cancer Center, 12902 USF Magnolia Dr., Tampa, FL 33216, USA.
Department of Blood and Marrow Transplant and Cellular Immunotherapy, Moffitt Cancer Center, 12902 USF Magnolia Dr., Tampa, FL 33216, USA.
Cancers (Basel). 2023 Sep 25;15(19):4711. doi: 10.3390/cancers15194711.
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 -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 ( = 42) were a median of 66 years old (range: 43-81). At baseline, extramedullary disease was associated with worse physical well-being ( = 0.008), global pain ( < 0.001), performance status ( = 0.002), and overall symptom burden ( < 0.001). Fatigue ( < 0.001) and functional well-being ( = 0.003) worsened by D7 before returning to baseline levels. Overall HRQOL ( = 0.008) and physical well-being ( < 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.
伊德凯布他赛(ide-cel)是首个获美国食品药品监督管理局(FDA)批准用于复发/难治性多发性骨髓瘤(RRMM)患者的嵌合抗原受体T细胞疗法。这是第一项评估接受伊德凯布他赛治疗的RRMM患者在标准治疗(SOC)中的患者报告结局(PRO)的研究。我们前瞻性地评估了从输注前(基线)到输注后第90天的健康相关生活质量(HRQOL)和症状。分别使用t检验、线性混合效应模型和Kaplan-Meier分析评估基线PRO与患者特征、PRO平均变化以及稳定变化时间之间的关联。个体内变化分数和最小重要差异阈值确定了临床和有意义的显著性。参与者(n = 42)的年龄中位数为66岁(范围:43 - 81岁)。在基线时,髓外疾病与较差的身体状况(P = 0.008)、全身疼痛(P < 0.001)、体能状态(P = 0.002)和总体症状负担(P < 0.001)相关。疲劳(P < 0.001)和功能状况(P = 0.003)在第7天恶化,之后恢复到基线水平。到第60天时,总体HRQOL(P = 0.008)和身体状况(P < 0.001)有所改善。到第90天时,大多数参与者报告PRO有所改善(10 - 57%)或维持不变(23 - 69%)。功能状况稳定恶化的中位时间为14天。身体和情绪状况稳定改善的中位时间为60天。总体而言,RRMM患者在接受SOC伊德凯布他赛治疗后报告HRQOL和症状负担有所改善或维持不变。