两万多例乳腺癌女性靶向治疗真实数据
近年来，随着乳腺癌治疗方法不断进步、乳腺癌治疗指南不断更新，人们迫切需要乳腺癌治疗用药的真实数据，尤其关于乳腺癌治疗指南推荐意见依从性的评定数据。
2020年11月8日，欧洲癌症治疗研究组织《欧洲癌症杂志》在线发表法国居里研究院、乔治斯-弗朗索瓦·勒克莱尔综合癌症中心、莱昂·贝拉德综合癌症中心、克莱蒙费朗大学、勒內·杜博斯医院中心、南阿尔萨斯医院集团、拉姆齐医疗集团、德罗姆·阿德什私立医院、罗氏、保罗·斯特拉斯综合癌症中心的大数据分析报告，根据2011～2018年法国个体化医疗保险报销模型数据库，对2万多例HER2阳性乳腺癌女性HER2靶向治疗策略变化的真实数据进行了分析。
该研究于2018年9月从法国120家随机入组医院的药房电子记录系统提取2万6350例患者数据，根据乳腺癌分期和治疗方案，分析2011年以来治疗方法以及联合治疗方案的变化。
结果，其中完成曲妥珠单抗、帕妥珠单抗或恩美曲妥珠单抗（T-DM1）治疗且数据完整的HER2阳性乳腺癌女性共计2万1119例，包括早期治疗1万6398例、晚期治疗6030例。
对于早期乳腺癌，1万4634例（89.2%）患者接受曲妥珠单抗＋紫杉类，其中1万0262例（62.6%）患者接受曲妥珠单抗＋紫杉类＋蒽环类。
对于晚期乳腺癌，一线治疗4841例、二线治疗2471例、至少三线治疗1707例，分别占80.3%、40.1%、28.3%。
2014年，帕妥珠单抗获得欧盟批准用于早期一线治疗之后，用药比例由5.8%增加至67.4%，而曲妥珠单抗＋紫杉类由47.2%减少至9.2%。
2014年，恩美曲妥珠单抗获得欧盟批准用于晚期二线治疗之后，用药比例由6.2%增加至53.8%。而曲妥珠单抗＋长春瑞滨或紫杉类分别由29.8%和29.1%减少至1.5%和6.7%。
因此，该大数据分析为治疗策略真实变化提供了可靠证据，根据HER2阳性乳腺癌患者可用治疗方法的最新变化，该研究结果表明HER2靶向治疗用药仍有较大的优化空间，此类数据将有助于为将来根据价值决定价格的解决方案提供药品相关指标。
Eur J Cancer. 2020 Nov 8;141:209-217.
Evolution in the real-world therapeutic strategies in more than 20,000 women with breast cancer having received human epidermal growth factor receptor 2-targeted treatments: Results from the french personalized reimbursement model database (2011-2018).
Paul Cottu, Bruno Coudert, David Perol, Anne Doly, Julien Manson, Olivier Aujoulat, Hugues Barletta, Nassera Chalabi, Laurence Samelson, Xavier Pivot.
Institut Curie, Paris, France; Georges Francois Leclerc Comprehensive Cancer Center, Dijon, France; Centre Léon Bérard Comprehensive Cancer Center, Lyon, France; Auvergne University, Clermont-Ferrand, France; Centre Hospitalier René-Dubos, Cergy Pontoise, France; South Alsace Hospital Group, Mulhouse, France; Ramsay Générale de Santé, Hopital Privé Drome Ardèche, Valence, France; Roche SAS, Boulogne-Billancourt, France; Paul Strauss Comprehensive Cancer Center, Strasbourg, France.
HIGHLIGHTS
There is a growing need for real-world data on how cancer treatments are used.
Use of therapies for early or advanced human epidermal growth factor receptor 2-positive (HER2+) breast cancer has changed over time.
We provide real-world data on 2011-2018 evolution in injectable HER2 treatments.
Results suggest that improvement is necessary for an optimal use.
Drug-related indicators will be further built for value-based pricingsolutions.
BACKGROUND: There is a growing need for real-world data on cancer treatments usage, especially to assess compliance with recommendations. We developed a French project using hospital data to analyse evolution in the therapeutic strategies implemented in patients with human epidermal growth factor receptor 2 (HER2)-overexpressed (HER2+) breast cancer (BC) and exposed to injectable HER2-targeted therapies, i.e. trastuzumab, pertuzumab or trastuzumab emtansine (T-DM1).
PATIENTS AND METHODS: Data from 26,350 women with BC were extracted in September 2018 from the Electronic Pharmacy Record systems of 120 French randomly recruited hospitals. Evolution in the treatments used, and combination regimens were described from 2011, in accordance with the BC stage and treatment line.
RESULTS: Overall, 21,119 patients treated since 2011 were analysed: 16,398 patients with early BC (eBC) and 6030 patients with metastatic BC (mBC) including patients treated at both stages. In eBC, 89.2% of patients received trastuzumab combined with at least taxanes (trastuzumab-taxane-anthracycline: 62.6%). Patients with mBC were treated in the first line (80.3%) and/or the second line (40.1%) and/or ≥ the third line (28.3%). After its approval in 2014, pertuzumab was first used in first-line therapy combinations in 67.4% of the total cases, while trastuzumab-taxane decreased from 47.2% to 9.2%. Similarly, T-DM1 was used as the second-line treatment in 53.8% of cases.
CONCLUSIONS: Given recent changes in available treatments for patients with HER2+ BC, this large French project provides robust information on real-world evolution in therapeutic strategies. Our data suggest there is room for significant improvement in optimal drug utilisation. Such data will be useful to build drug-related indicators for future value-based pricing solutions.
KEYWORDS: Breast cancer; HER2; Retrospective real-world data study; Pertuzumab; Trastuzumab; Trastuzumab emtansine
DOI: 10.1016/j.ejca.2020.10.012