文章：

接受标准治疗伊德卡塔吉恩维克利尤治疗的多发性骨髓瘤患者报告结局分析

Patient-Reported Outcomes among Multiple Myeloma Patients Treated with Standard of Care Idecabtagene Vicleucel

原文发布日期：25 September 2023

DOI: 10.3390/cancers15194711

类型: Article

开放获取: 是

英文摘要：

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 witht-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.

摘要翻译： 

伊达妥昔单抗（ide-cel）是美国食品药品监督管理局批准的首个用于复发/难治性多发性骨髓瘤（RRMM）患者的嵌合抗原受体T细胞疗法。本研究首次在标准治疗（SOC）条件下评估接受ide-cel治疗的RRMM患者报告结局（PROs）。我们前瞻性评估了从输注前（基线）至输注后第90天（D90）期间健康相关生活质量（HRQOL）及症状变化。通过t检验、线性混合效应模型和Kaplan-Meier分析分别评估了基线PRO与患者特征的关联性、PRO平均变化及达到稳定变化的时间。个体内变化评分与最小重要差异阈值用于判定临床及有意义的显著性。42名参与者中位年龄为66岁（范围：43-81岁）。基线时，髓外病变与较差的生理健康（p=0.008）、整体疼痛（p<0.001）、体能状态（p=0.002）及总体症状负担（p<0.001）显著相关。疲劳（p<0.001）与功能健康（p=0.003）在D7时出现恶化，随后恢复至基线水平。至D60时总体HRQOL（p=0.008）与生理健康（p<0.001）均得到改善。截至D90，多数参与者报告PRO改善（10-57%）或维持稳定（23-69%）。功能健康达到稳定恶化的中位时间为14天，而生理与情绪健康达到稳定改善的中位时间均为60天。总体而言，RRMM患者在SOC ide-cel治疗后报告了HRQOL与症状负担的改善或维持。

原文链接：

Patient-Reported Outcomes among Multiple Myeloma Patients Treated with Standard of Care Idecabtagene Vicleucel