Teodoro Maria Inês, Mayer Alexandra, da Costa Miranda Ana, Nunes Hugo, da Costa Filipa Alves, Lourenço António
Unidade de Investigação em Epidemiologia, Instituto Português de Oncologia de Lisboa Francisco Gentil, E.P.E., Lisbon, Portugal.
Research Institute for Medicines (iMED), Faculty of Pharmacy, University of Lisbon, Lisbon, Portugal.
J Pharm Policy Pract. 2024 Jan 17;17(1):2296551. doi: 10.1080/20523211.2023.2296551. eCollection 2024.
Monotherapy with aromatase inhibitors and fulvestrant were the standard-of-care for hormone receptor-positive (HR+)/human epidermal growth factor receptor-type2 negative (HER2-) advanced breast cancer, before integration of cyclin-dependent kinase 4/6 inhibitors. Effectiveness data is essential for regulatory action, but little is known about real-world use of aromatase inhibitors and fulvestrant.
A retrospective cohort study was conducted resorting to data from a cancer registry to identify adult women with HR+/HER- advanced breast cancer exposed to aromatase inhibitors or fulvestrant (31 May 2017-31 March 2019) at the main oncology hospital in Portugal. Cases were updated with follow-up until death or cut-off (31 March 2021) and pseudoanonymized data extracted. Primary outcome was overall survival (OS) and secondary time to treatment failure (TTF), estimated using survival analysis and compared with published trials.
192 patients were distributed by subgroups according to the medicine. Letrozole: OS 30.8 (95% confidence interval (CI) 20.6-41.4); TTF 11.2 (95%CI 8.7-13.7). Exemestane: OS 22.1 (95%CI 9.7-34.6); TTF 6.0 (95%CI 4.1-7.8). Fulvestrant: OS 21.6 (95%CI 16.5-26.7); TTF 5.6 (95%CI 4.5-6.6).
Estimated effectiveness (OS) of letrozole and fulvestrant was, respectively, 3.2-3.5 months lower than reported. The clinical meaning seems uncertain and may be explained a higher proportion of worse prognostic characteristics in patients treated in the real-world.
在细胞周期蛋白依赖性激酶4/6抑制剂纳入治疗之前，芳香化酶抑制剂和氟维司群单药治疗是激素受体阳性（HR+）/人表皮生长因子受体2型阴性（HER2-）晚期乳腺癌的标准治疗方案。有效性数据对于监管行动至关重要，但关于芳香化酶抑制剂和氟维司群的实际应用情况知之甚少。
采用一项回顾性队列研究，借助癌症登记处的数据，识别在葡萄牙主要肿瘤医院接受芳香化酶抑制剂或氟维司群治疗（2017年5月31日至2019年3月31日）的HR+/HER-晚期乳腺癌成年女性。对病例进行随访更新直至死亡或截止日期（2021年3月31日），并提取伪匿名数据。主要结局是总生存期（OS），次要结局是治疗失败时间（TTF），采用生存分析进行估计，并与已发表的试验进行比较。
192例患者根据所用药物分为亚组。来曲唑：OS为30.8（95%置信区间（CI）20.6 - 41.4）；TTF为ll.2（95%CI 8.7 - 13.7）。依西美坦：OS为22.1（95%CI 9.7 - 34.6）；TTF为6.0（95%CI 4.1 - 7.8）。氟维司群：OS为21.6（95%CI 16.5 - 26.7）；TTF为5.6（95%CI 4.5 - 6.6）。
来曲唑和氟维司群的估计有效性（OS）分别比报告的低3.2 - 3.5个月。其临床意义似乎不确定，可能是由于现实世界中接受治疗的患者中预后较差特征的比例较高所致。
