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替沙仑赛与博纳吐单抗在儿童复发/难治性急性淋巴细胞白血病中的间接比较

英文原题:Indirect comparison of tisagenlecleucel and blinatumomab in pediatric relapsed/refractory acute lymphoblastic leukemia.

查看英文原题

Indirect comparison of tisagenlecleucel and blinatumomab in pediatric relapsed/refractory acute lymphoblastic leukemia.

PubMed 2021/12/14(内容时间) Blood Adv Q1 · IF 7.7(JCR 2025)

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中文摘要

在缺乏头对头试验的情况下,间接治疗比较可以估计tisagenlecleucel与blinatumomab相比,在复发或原发性难治性(R/R)急性淋巴细胞白血病(ALL)患者中实现完全缓解(CR)率和总生存期(OS)方面的治疗效果。来自两项关键试验ELIANA(tisagenlecleucel;n = 79)和MT103-205(blinatumomab;n = 70)的患者水平数据被用于比较CR和OS,并控制了可用患者特征方面的跨试验差异。实施了五种不同的调整方法:稳定化逆概率治疗加权(sIPTW);修剪后的sIPTW;按倾向评分五分位数分层;针对预后因素进行调整;以及同时针对预后因素和倾向评分进行调整。

比较分析表明,与blinatumomab治疗相比,tisagenlecleucel治疗与实现CR的统计学显著更高可能性以及更低的死亡风险相关。无论采用何种调整方法,tisagenlecleucel组在每项分析中均表现出比blinatumomab组更高的CR可能性(比值比:6.71-9.76)。在每项分析中,tisagenlecleucel也与比blinatumomab更低的死亡风险相关,使用多种调整方法确定,其死亡风险比blinatumomab低68%至74%(风险比:0.26-0.32)。这些发现支持了越来越多的临床试验和真实世界证据,表明tisagenlecleucel是R/R ALL儿童和年轻成人的重要治疗选择。

展开英文摘要原文

In the absence of head-to-head trials, an indirect-treatment comparison can estimate the treatment effect of tisagenlecleucel in comparison with blinatumomab on rates of complete remission (CR) and overall survival (OS) in patients with relapsed or primary refractory (R/R) acute lymphoblastic leukemia (ALL). Patient-level data from two pivotal trials, ELIANA (tisagenlecleucel; n = 79) and MT103-205 (blinatumomab; n = 70), were used in comparisons of CR and OS, controlling for cross-trial difference in available patient characteristics. Five different adjustment approaches were implemented: stabilized inverse probability of treatment weight (sIPTW); trimmed sIPTW; stratification by propensity score quintiles; adjustment for prognostic factors; and adjustment for both prognostic factors and propensity score.

Comparative analyses indicate that treatment with tisagenlecleucel was associated with a statistically significant higher likelihood of achieving CR and lower hazard of death than treatment with blinatumomab. The tisagenlecleucel group exhibited a higher likelihood of CR than the blinatumomab group in every analysis regardless of adjustment approach (odds ratios: 6.

71-9. 76). Tisagenlecleucel was also associated with a lower hazard of death than blinatumomab in every analysis, ranging from 68% to 74% lower hazard of death than with blinatumomab, determined using multiple adjustment approaches (hazard ratios: 0. 26-0. 32).

These findings support the growing body of clinical trials and real-world evidence demonstrating that tisagenlecleucel is an important treatment option for children and young adults with R/R ALL.

论文信息

作者
Verneris MR、Ma Q、Zhang J、Keating A、Tiwari R、Li J、Yang H、Agarwal A
第一作者单位
Department of Pediatrics, University of Colorado Anschutz Medical Campus (CU Anschutz), Denver, CO.United States
通讯作者单位
Novartis Pharmaceuticals, East Hanover, NJ.United States
文献类型
临床试验 · 非美国政府资助研究
期刊
Blood advances2021 Dec 14
原文标识
PubMed 34597381 · DOI 10.1182/bloodadvances.2020004045