Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
Evidera, San Francisco, CA, USA.
J Comp Eff Res. 2020 Aug;9(12):849-860. doi: 10.2217/cer-2020-0069. Epub 2020 Jun 30.
We compared outcomes from a single-arm study of tisagenlecleucel with standard of care (SOC) regimens in pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia (ALL). The analysis included one tisagenlecleucel study, one blinatumomab study, one clofarabine monotherapy study, three studies of clofarabine combination regimens and two studies of other salvage chemotherapy. Matching-adjusted indirect comparison analyses were conducted. After adjusting for baseline characteristics, tisagenlecleucel was associated with significantly prolonged overall survival compared with blinatumomab (hazard ratio [95% CI], 0.32 [0.16-0.64]); clofarabine monotherapy (0.24 [0.13-0.42]); clofarabine combination regimens (0.26 [0.15-0.45]); two salvage therapies (0.15 [0.09-0.25] and 0.27 [0.15-0.49]). The analysis demonstrated tisagenlecleucel was associated with substantially greater survival benefit versus all SOC regimens.
我们比较了靶向 CD19 嵌合抗原受体 T 细胞（tisagenlecleucel）单臂研究与标准治疗（SOC）方案在儿科和年轻成人复发/难治性急性淋巴细胞白血病（ALL）患者中的疗效。该分析纳入了一项 tisagenlecleucel 研究、一项blinatumomab 研究、一项氯法拉滨单药治疗研究、三项氯法拉滨联合方案研究和两项其他挽救性化疗研究。采用匹配调整间接比较分析。在调整基线特征后，tisagenlecleucel 与 blinatumomab（风险比 [95%CI]，0.32 [0.16-0.64]）、氯法拉滨单药治疗（0.24 [0.13-0.42]）、氯法拉滨联合方案（0.26 [0.15-0.45]）、两种挽救性化疗方案（0.15 [0.09-0.25] 和 0.27 [0.15-0.49]）相比，总生存显著延长。该分析表明，与所有 SOC 方案相比，tisagenlecleucel 具有更大的生存获益。
