Bergen Elisabeth Sophie, Binter Amelie, Starzer Angelika Martina, Heller Gerwin, Kiesel Barbara, Tendl-Schulz Kristina, Bago-Horvath Zsuzsanna, Furtner Julia, Leitner Johannes, Exner Ruth, Fitzal Florian, Dieckmann Karin, Widhalm Georg, Preusser Matthias, Berghoff Anna Sophie, Bartsch Rupert
Division of Oncology, Department of Medicine 1, Medical University of Vienna, Austria.
Department of Neurosurgery, Medical University of Vienna, Austria.
Ther Adv Med Oncol. 2021 Apr 22;13:17588359211009002. doi: 10.1177/17588359211009002. eCollection 2021.
Dual human epidermal growth factor receptor 2 (HER2) blockade with trastuzumab and pertuzumab (TP) is a standard therapy of metastatic and localized HER2-positive breast cancer (BC), but its activity in breast cancer brain metastases (BCBM) is unknown.
Patients with HER2-positive BCBM were identified from the Vienna Brain Metastasis Registry and clinical data including patient characteristics, therapies and overall survival (OS) were obtained. Patients were grouped into 'TP', 'other-HER2-targeted therapy' and 'no-HER2-targeted therapy' according to received first-line systemic therapy after diagnosis of BCBM. Radiological re-assessment of intracranial lesions was performed in patients treated with TP as systemic first-line therapy according to RANO response criteria for brain metastases (BM).
A total of 252 HER2-positive BC patients with BM were available for this analysis. Patients treated with TP as systemic first-line therapy after diagnosis of BM had a significantly longer OS compared with treatment with other-HER2-targeted therapy and no-HER2-targeted therapy (44 17 3 months, < 0.001; log-rank test). Among radiologically re-assessed patients treated with TP as systemic first-line therapy after diagnosis of BM, 5/14 patients (35.7%) had complete intracranial remission (CR), 8/14 patients (57.1%) partial intracranial remission (PR), 1/14 patients (7.1%) stable intracranial disease (SD) and 0/14 patients (0.0%) progressive intracranial disease (PD) as best response resulting in an intracranial objective response rate (iORR) of 92.9% and an intracranial clinical benefit rate (iCBR) of 100.0%.
First-line therapy with dual HER2-inhibition of TP after BM diagnosis was associated with the longest median OS times in patients with BCBM.
曲妥珠单抗和帕妥珠单抗（TP）双重阻断人表皮生长因子受体2（HER2）是转移性和局限性HER2阳性乳腺癌（BC）的标准治疗方法，但其在乳腺癌脑转移（BCBM）中的活性尚不清楚。
从维也纳脑转移瘤登记处识别出HER2阳性BCBM患者，并获取包括患者特征、治疗方法和总生存期（OS）在内的临床数据。根据BCBM诊断后接受的一线全身治疗，将患者分为“TP组”、“其他HER2靶向治疗组”和“非HER2靶向治疗组”。对于接受TP作为全身一线治疗的患者，根据脑转移瘤（BM）的RANO反应标准对颅内病变进行放射学重新评估。
共有252例HER2阳性BCBM患者可用于该分析。与接受其他HER2靶向治疗和非HER2靶向治疗相比，BM诊断后接受TP作为全身一线治疗的患者OS明显更长（44±17±3个月，P<0.001；对数秩检验）。在BM诊断后接受TP作为全身一线治疗且经放射学重新评估的患者中，5/14例患者（35.7%）达到颅内完全缓解（CR），8/14例患者（57.1%）达到颅内部分缓解（PR），1/14例患者（7.1%）颅内疾病稳定（SD），0/14例患者（0.0%）颅内疾病进展（PD），最佳反应导致颅内客观缓解率（iORR）为92.9%，颅内临床获益率（iCBR）为100.0%。
BM诊断后采用TP双重HER2抑制进行一线治疗与BCBM患者最长的中位OS时间相关。
