Background and Method: The overexpression of the human epidermal growth factor receptor 2 (HER2) in breast cancer is correlated with accelerated tumor progression and an unfavorable clinical outcome. Since the introduction of trastuzumab in 2002, the treatment of HER2-positive breast cancer has been revolutionized, leading to significant improvements in survival. This retrospective, multicenter study aimed to describe the characteristics of patients with HER2-positive metastatic breast cancer (MBC) who maintained disease control for a minimum of three years after first-line therapy with trastuzumab and/or pertuzumab combined with chemotherapy.Results: Among 280 eligible patients, 48 (17.5%) were classified as long-term responders. The study population primarily consisted of women with a median age of 56.7 years at diagnosis; de novo metastatic presentation was observed in approximately 70% of cases. An objective response rate of nearly 90% was observed, with a median duration of response of 5.8 years. Median progression-free survival was 11.0 years [95% CI: 6.6—not reached], and median overall survival was not reached [95% CI: 10.9—not reached]. Furthermore, about 15% of patients were able to discontinue systemic therapy without immediate disease progression.Discussion and Conclusions: These findings indicate the potential of achieving prolonged disease control in a subset of patients with HER2-positive MBC, raising questions about therapeutic intensification and potential treatment discontinuation strategies. This study underscores the need for future research to identify predictive factors of durable response and assess the feasibility of adaptive treatment strategies, including planned treatment discontinuation.
背景与方法：人表皮生长因子受体2（HER2）在乳腺癌中的过度表达与肿瘤进展加速及不良临床结局相关。自2002年曲妥珠单抗问世以来，HER2阳性乳腺癌的治疗模式发生革命性变化，患者生存期显著改善。本项回顾性多中心研究旨在描述接受曲妥珠单抗和/或帕妥珠单抗联合化疗一线治疗后疾病控制至少维持三年的HER2阳性转移性乳腺癌（MBC）患者的临床特征。 结果：在280例符合条件患者中，48例（17.5%）被归类为长期应答者。研究人群以女性为主，诊断时中位年龄56.7岁；约70%病例为初诊转移性乳腺癌。观察到近90%的客观缓解率，中位缓解持续时间为5.8年。中位无进展生存期为11.0年[95% CI：6.6—未达到]，中位总生存期未达到[95% CI：10.9—未达到]。此外，约15%患者能够在不出现即刻疾病进展的情况下停止全身治疗。 讨论与结论：这些发现表明HER2阳性MBC患者亚群存在实现长期疾病控制的潜力，引发关于治疗强化策略及潜在治疗终止方案的思考。本研究强调未来需开展研究以确定持久应答的预测因素，并评估适应性治疗策略（包括计划性治疗终止）的可行性。
肿瘤(癌症)患者之家