中国医师对乳腺癌研究终点的看法
循证医学是现代医学区别于传统医学的重要特征，但是临床研究终点的设立其实是一把双刃剑，统计学意义和临床意义并不总能同时尽如人意。
2021年8月20日，瑞士巴塞尔《Breast Care》在线发表中国北京大学人民医院张易、刘淼、杨后圃、王殊等学者的研究报告，调查了中国乳腺外科医师对乳腺癌临床研究主要终点相关证据的看法。
该研究于2018年5月第十一届中国医师协会外科医师年会乳腺外科医师专场发放在线问卷，调查乳腺外科医师对临床研究终点、随访期、临床可接受获益的看法。
结果，278份问卷有效，大多数问题的未取得一致答案。
关于早期乳腺癌局部治疗最有意义的终点：
局部区域复发：30.6%
无病生存：28.8%
总生存：28.4%
关于早期乳腺癌局部治疗可改变临床实践的随访期：
5年：47.5%
>10年：34.5%
关于早期乳腺癌辅助治疗最有意义的终点：
无病生存：45.7%
总生存：38.5%
局部区域复发：12.9%
关于早期乳腺癌辅助治疗可改变临床实践的随访期：
>10年：52.5%
5年：37.4%
关于晚期乳腺癌最有意义的终点：
无进展生存：49.6%
生活质量：24.1%
总生存：23.7%
关于晚期乳腺癌最有意义的随访期：
1年：39.6%
3年：28.8%
关于主要终点最可接受的绝对获益：
总生存率提高5～10个百分点：33.8%
无病生存率提高5～10个百分点：40.6%
局部区域复发率降低5～10个百分点：36.7%
无进展生存时间延长6～12个月：38.5%
因此，该研究结果表明，虽然中国乳腺外科医师的看法极不一致，但是期盼的有临床意义研究终点似乎比大多数现有研究终点更严苛。虽然大多数中国乳腺外科医师主张某些情况可以采用替代终点，但是乳腺癌研究最可靠的终点仍然是总生存。医师对随访时间和获益程度的看法各不相同，反映关于支持癌症治疗新方法的许多问题亟待解决，故有必要取得共识，例如认可率非常高的替代终点、有足够临床意义的治疗获益。
Breast Care (Basel). 2021 Aug 20. Online ahead of print.
Physicians' Perception of the Evidence in Relation to Primary Endpoints of Clinical Trials on Breast Cancer.
Zhang Y, Liu M, Yang H, Wang S.
Peking University People's Hospital, Beijing, China.
OBJECTIVE: To investigate physicians' perception of the evidence of clinical trials on breast cancer.
METHODS: A survey was conducted by the Chinese Society of Breast Surgeons. We investigated the physicians' perception of meaningful endpoints, appropriate follow-up duration, and clinically acceptable benefit through online questionnaires.
RESULTS: Among 278 validated questionnaires, the majority of the questions had no consistent answer. For local treatment, 30.6, 28.8, and 28.4% of participants regarded locoregional recurrence (LRR), disease-free survival (DFS), and overall survival (OS) as the most meaningful endpoint, respectively, 47.5% believed that 5-year follow-up can alter clinical practice, and 34.5% thought it should be >10 years. In the adjuvant setting, 45.7, 38.5, and 12.9% regarded DFS, OS, and LRR as the most meaningful endpoint, respectively, 52.5% thought that 10-year follow-up was solid, while 37.4% thought that 5-year follow-up was enough. In the advanced setting, 49.6, 24.1, and 23.7% considered progression-free survival, quality of life, and OS the most meaningful endpoint, respectively, and 39.6 and 28.8% considered that a follow-up of 1 year and 3 years, respectively, was meaningful. Similarly, the clinically acceptable absolute difference was inconsistent.
CONCLUSION: Most Chinese oncologists advocated that surrogate endpoints could be used in certain circumstances, though OS was the most reliable one in breast cancer studies. Doctors' perceptions of follow-up time and magnitude of benefit vary widely, reflecting the fact that there are many unanswered questions about supporting the use of new cancer treatments; a common understanding needs to be reached, such as a very consensual surrogate endpoint and a meaningful sufficiently large therapeutic benefit.
KEYWORDS: Breast cancer; Primary endpoint; Clinical trial
DOI: 10.1159/000518260