Surgical Health Outcomes & Research Enterprise, Department of Surgery, University of Rochester Medical Center, Rochester, NY, USA.
Community Outreach & Engagement, Wilmot Cancer Center, University of Rochester Medical Center, Rochester, NY, USA.
J Gastrointest Surg. 2022 Feb;26(2):453-465. doi: 10.1007/s11605-021-05192-x. Epub 2021 Nov 9.
The generalizability of outcomes from randomized controlled trials (RCTs) in oncology is a frequent concern. Given the prevalence and multidisciplinary management of rectal cancer, understanding the generalizability of rectal cancer RCTs is critical to surgical oncologists.
An exhaustive literature review identified 100 non-metastatic rectal cancer RCTs published in English over the past 10 years investigating surgery, chemotherapy, or radiotherapy. In order to evaluate the representativeness of these RCTs compared to the USA and each continent's rectal cancer populations, demographic characteristics were stratified by surgical versus chemoradiotherapy (CRT) trial and by continent then compared with the National Cancer Database and CANCER TODAY using chi-squared and Welch's t-tests.
Of the 100 trials identified, 65% enrolled significantly younger patients, and 38% enrolled a significantly greater proportion of males than the US rectal cancer population. These demographic differences were more prominent among CRT trials than surgical trials. Half of all trials enrolled patients who were on average more than 7 years younger and enrolled a 5% greater proportion of males than their respective continental rectal cancer populations. Patients enrolled in trials had more advanced cancers than their corresponding continental populations. Sociodemographic data was rarely reported.
Patients enrolled in trials were younger, predominantly male, and had advanced stage cancer when compared to the rectal cancer population. Sociodemographic variables are underreported, further limiting equal participation in clinical trials. Future rectal cancer RCTs should strive to recruit representative samples. To enhance recruitment of women and underrepresented minorities, tailored recruitment strategies must be implemented.
肿瘤学中随机对照试验（RCT）结果的推广性是一个常见的关注点。鉴于直肠癌的普遍性和多学科管理，了解直肠癌 RCT 的推广性对于外科肿瘤学家至关重要。
通过全面的文献回顾，确定了过去 10 年发表的 100 项非转移性直肠癌 RCT，这些 RCT 研究了手术、化疗或放疗。为了评估这些 RCT 与美国和每个大陆的直肠癌人群的代表性，根据手术与放化疗（CRT）试验和大陆对这些 RCT 进行分层，然后使用卡方检验和 Welch's t 检验与国家癌症数据库和《癌症今日》进行比较。
在确定的 100 项试验中，65%的试验显著纳入了更年轻的患者，38%的试验显著纳入了更多的男性患者，而这一比例高于美国直肠癌患者人群。这些人口统计学差异在 CRT 试验中比手术试验中更为明显。所有试验中有一半纳入的患者平均年龄要小 7 岁以上，并且纳入的男性比例比各自的大陆直肠癌人群高 5%。与相应的大陆人群相比，试验中纳入的患者癌症更为晚期。很少有试验报告社会人口统计学数据。
与直肠癌患者人群相比，试验纳入的患者更年轻，主要是男性，且癌症处于更晚期。社会人口统计学变量报告不足，进一步限制了临床试验的平等参与。未来的直肠癌 RCT 应努力招募有代表性的样本。为了增加女性和代表性不足的少数族裔的参与，必须实施有针对性的招募策略。
