Division of Hematology and Oncology, Department of Medicine, David Geffen School of Medicine, University of California at Los Angeles, Los Angeles, CA, USA.
Department of Gynecology and Obstetrics, University Hospital Erlangen, Comprehensive Cancer Center Erlangen-EMN, Friedrich-Alexander-University Erlangen-EMN, Erlangen, Germany.
Breast Cancer Res Treat. 2018 Oct;171(3):747-758. doi: 10.1007/s10549-018-4829-4. Epub 2018 Jun 27.
Clinical trials can offer novel and more advanced and/or novel treatments to cancer patients in advance of them being approved and available for all patients. While several studies have examined the effect of clinical trial participation on prognosis, there has been no clear conclusion from these studies. Therefore, we chose to test the influence of trial participation on pathological complete response (pCR) and mastectomy rates after neoadjuvant chemotherapy.
In this retrospective study, all patients treated with neoadjuvant chemotherapy from 2001 to 2014 were selected. A total of 1038 patients with complete treatment, patient, and tumor characteristics were included. A total of 260 of those were treated in clinical trials. We examined whether study participation status in addition to commonly known predictors for pCR improves prediction of pCR. Similar analyses were conducted for the mastectomy rate outcome measure. Finally, survival analyses were also conducted as part of an exploratory analysis.
Study participation was an independent predictor of pCR in addition to commonly known predictors. Adjusted odds ratio (OR) for trial participants versus non-participants was 1.53 (95% CI 1.03-2.28). Additionally, study participation improved the prediction of mastectomy risk. The adjusted OR for trial participants versus non-participants was 0.62 (95% CI 0.42-0.90). Subgroup-specific differences concerning the impact of study participation could not be shown for either pCR or mastectomy rate. Survival comparisons could not be conducted due to large differences in follow-up data in patients participating in clinical trials versus those who did not participate; however, pCR was a predictor of prognosis in both groups.
Patients taking part in neoadjuvant chemotherapy clinical trials have a higher pCR rate and a lower mastectomy risk than patients not participating in clinical trials for their cancer care. This finding is a supporting factor for trial participation in neoadjuvant chemotherapy trials.
临床试验可以在癌症患者的治疗方案获得批准并适用于所有患者之前，为他们提供新颖的、更先进的和/或新的治疗方法。虽然已经有几项研究探讨了临床试验参与对预后的影响，但这些研究尚未得出明确的结论。因此，我们选择测试临床试验参与对新辅助化疗后病理完全缓解（pCR）和乳房切除术率的影响。
在这项回顾性研究中，选择了 2001 年至 2014 年间接受新辅助化疗的所有患者。共纳入了 1038 例具有完整治疗、患者和肿瘤特征的患者。其中 260 例接受了临床试验治疗。我们检查了研究参与状态除了常见的 pCR 预测因素外，是否能改善 pCR 的预测。对乳房切除术率这一结局指标也进行了类似的分析。最后，还进行了生存分析作为探索性分析的一部分。
研究参与是除常见预测因素外，pCR 的独立预测因素。与非参与者相比，试验参与者的调整后优势比（OR）为 1.53（95%置信区间 1.03-2.28）。此外，研究参与提高了乳房切除术风险的预测。与非参与者相比，试验参与者的调整后 OR 为 0.62（95%置信区间 0.42-0.90）。对于 pCR 或乳房切除术率，无法显示研究参与的亚组特异性差异。由于临床试验参与者和未参与者之间的随访数据存在较大差异，因此无法进行生存比较；然而，在两组中，pCR 都是预后的预测因素。
与未参加癌症治疗临床试验的患者相比，参加新辅助化疗临床试验的患者具有更高的 pCR 率和更低的乳房切除术风险。这一发现为参加新辅助化疗临床试验提供了支持。
