tisagenlecleucel 对比常规护理治疗复发/难治性滤泡淋巴瘤患者的疗效比较。
Efficacy comparison of tisagenlecleucel vs usual care in patients with relapsed or refractory follicular lymphoma.
机构信息
Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY.
Lymphoma Program, Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA.
出版信息
Blood Adv. 2022 Nov 22;6(22):5835-5843. doi: 10.1182/bloodadvances.2022008150.
The ELARA trial indicates tisagenlecleucel (tisa-cel) is an effective anti-CD19 chimeric antigen receptor T-cell therapy for relapsed or refractory follicular lymphoma (r/r FL). As ELARA is a single-arm trial, this study compares tisa-cel outcomes from the ELARA trial with usual care from a real-world cohort. ELARA enrolled 98 patients as of 29 March 2021 (median follow-up: 15 months from enrollment). Usual care data were obtained from ReCORD-FL, a global retrospective study of patients with r/r FL, who met similar eligibility criteria to ELARA. With a data cutoff date of 31 December 2020, 187 patients with ≥2 preceding treatment lines were included in the ReCORD-FL (median follow-up: 57 months from third-line) study. An indirect treatment comparison was performed for 97 patients from the ELARA trial and 143 patients from the ReCORD-FL study with no missing data on baseline factors. The line of therapy for which outcomes were assessed was selected or matched between cohorts using propensity score modeling. After baseline factor adjustment via weighting by odds, complete response rate (CRR; 95% confidence interval) was 69.1% (59.8%-78.3%) for tisa-cel vs. 37.3% (26.4%-48.3%) for usual care; overall response rate was 85.6% (78.7%-92.5%) vs. 63.6% (52.5%-74.7%). Kaplan-Meier probability of being progression/event-free at 12 months was 70.5% (61.4%-79.7%) for tisa-cel vs. 51.9% (40.6%-63.3%) for usual care, with hazard ratio (HR)=0.60 (0.34-0.86); 12-month overall survival was 96.6% (92.9%-100%) vs. 71.7% (61.2%-82.2%), with HR=0.2 (0.02-0.38). In conclusion, tisa-cel was associated with a 1.9-fold higher complete response rate and a 1.4-fold higher rate of being progression or event free at 12 months vs usual care, as well as a death risk reduction of 80%. The findings provide additional evidence on the benefit of tisa-cel in patients with r/r FL after ≥2 treatment lines. This trial was registered at www.clinicaltrials.gov as NCT03568461.
ELARA 试验表明，tisagenlecleucel（tisa-cel）是一种有效的抗 CD19 嵌合抗原受体 T 细胞疗法，用于治疗复发或难治性滤泡淋巴瘤（r/r FL）。由于 ELARA 是一项单臂试验，本研究将 tisa-cel 的结果与真实世界队列中的常规护理进行了比较。ELARA 截至 2021 年 3 月 29 日共纳入 98 例患者（中位随访：从入组起 15 个月）。常规护理数据来自 ReCORD-FL，这是一项全球回顾性研究，纳入了 r/r FL 患者，他们符合与 ELARA 类似的入选标准。截至 2020 年 12 月 31 日，187 例接受过≥2 线治疗的患者纳入了 ReCORD-FL（从三线治疗开始的中位随访：57 个月）研究。在无基线因素缺失数据的情况下，对来自 ELARA 试验的 97 例患者和来自 ReCORD-FL 研究的 143 例患者进行了间接治疗比较。使用倾向评分模型选择或匹配了评估疗效的治疗线。在通过优势比加权进行基线因素调整后，tisa-cel 的完全缓解率（CRR；95%置信区间）为 69.1%（59.8%-78.3%），常规护理为 37.3%（26.4%-48.3%）；总缓解率为 85.6%（78.7%-92.5%），常规护理为 63.6%（52.5%-74.7%）。tisa-cel 治疗 12 个月时无进展/事件的 Kaplan-Meier 概率为 70.5%（61.4%-79.7%），常规护理为 51.9%（40.6%-63.3%），风险比（HR）为 0.60（0.34-0.86）；12 个月总生存率为 96.6%（92.9%-100%），常规护理为 71.7%（61.2%-82.2%），HR 为 0.2（0.02-0.38）。综上所述，与常规护理相比，tisa-cel 治疗 r/r FL 患者的完全缓解率提高了 1.9 倍，12 个月时无进展/事件的比例提高了 1.4 倍，死亡风险降低了 80%。这些发现为 tisa-cel 在接受过≥2 线治疗的 r/r FL 患者中的获益提供了更多证据。该试验在 www.clinicaltrials.gov 上注册为 NCT03568461。
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