曲妥珠单抗乳酸甲氧基乙酯（T-DM1）已经证明在患有乳腺癌脑转移的患者中能够改善生存和神经症状。这项实际研究调查了T-DM1与拉帕替尼加卡培他滨（LC）在乳腺癌脑转移患者中的有效性。此项回顾性观察研究使用真实世界数据库评估了HER2阳性乳腺癌脑转移患者。符合条件的患者在之前被诊断出脑转移之前，已经接受了T-DM1或LC，并且接受过至少一次转移性乳腺癌治疗。主要终点是总生存率（OS）；次要终点是下一个相关治疗或死亡的时间（TTNT）和真实世界进展无瘤生存期（rwPFS）。使用逆概率处理权重（IPTW）方法以考虑治疗组之间的潜在基线特征差异。结果使用Kaplan-Meier方法描述，并使用加权Cox比例风险模型和风险比（HR）估计开始使用T-DM1与LC的平均治疗效果。共有214名患者可供分析（T-DM1，n = 161；LC，n = 53）。经过加权处理，治疗组之间的人口统计学和基线特征基本平衡。加权处理后，中位OS为17.7（T-DM1）与9.6（LC）个月（HR，0.55 [95% CI，0.34-0.89]；P = 0.013）。中位TTNT为9.0（T-DM1）与6.0（LC）个月（HR，0.55 [95% CI，0.36-0.85]；P = 0.005）。加权处理后，中位rwPFS为6.0（T-DM1）与4.0（LC）个月（HR，0.50 [95% CI，0.36-0.69]；P < 0.001）。这些结果支持T-DM1与LC在真实世界中在HER2阳性乳腺癌脑转移患者中的明显有效性和临床相关性。版权所有 © 2023作者。由爱思唯尔出版社发表。保留所有权利。

Trastuzumab emtansine (T-DM1) has demonstrated improvements in survival and neurological symptoms in patients with breast cancer with brain metastases (BCBM). This real-world study investigated the effectiveness of T-DM1 versus lapatinib plus capecitabine (LC) in patients with BCBM.This retrospective, observational study evaluated patients with HER2-positive BCBM using a real-world database. Eligible patients had initiated T-DM1 or LC with a prior diagnosis of brain metastasis and ≥1 prior metastatic breast cancer treatment. The primary endpoint was overall survival (OS); secondary endpoints were time to next relevant treatment or death (TTNT) and real-world progression-free survival (rwPFS). An inverse probability of treatment weighting (IPTW) approach was used to account for differences in potential baseline characteristics between treatment groups. Outcomes were described using the Kaplan-Meier method, and the average treatment effect of initiating T-DM1 versus LC was estimated using weighted Cox proportional hazard models and hazard ratio (HR).A total of 214 patients were available for analysis (T-DM1, n = 161; LC, n = 53). Demographics and baseline characteristics were generally well-balanced between treatment groups after weighting. After weighting, median OS was 17.7 (T-DM1) versus 9.6 (LC) months (HR, 0.55 [95% CI, 0.34-0.89]; P=0.013). Median TTNT was 9.0 (T-DM1) versus 6.0 (LC) months (HR, 0.55 [95% CI, 0.36-0.85]; P = 0.005). After weighting, median rwPFS was 6.0 (T-DM1) versus 4.0 (LC) months (HR, 0.50 [95% CI, 0.36-0.69]; P < 0.001).These results support the superior effectiveness and clinical relevance of T-DM1 versus LC in patients with HER2-positive BCBM in the real world.Copyright © 2023 The Authors. Published by Elsevier Ltd.. All rights reserved.