PDF(Pubmed)
Abstract：
OBJECTIVE: Randomized controlled trials (RCTs) are the gold standard for evaluating the comparative efficacy and safety of new cancer therapies. However, enrolling patients in control arms of clinical trials can be challenging for rare cancers, particularly in the context of precision oncology and targeted therapies. External Control Arms (ECAs) are a potential solution to address these challenges in clinical research design. We conducted a scoping review to explore the use of ECAs in oncology.
METHODS: We systematically searched four databases, namely MEDLINE, EMBASE, Web of Science, and Scopus. We screened titles, abstracts, and full texts for eligible articles focusing on patients undergoing therapy for cancer, employing ECAs, and reporting clinical outcomes.
RESULTS: Of the 629 articles screened, 23 were included in this review. The earliest included studies were published in 1996, while most studies were published in the past 5 years. 44% (10/23) of ECAs were employed in blood-related cancer studies. Geographically, 30% (7/23) of studies were conducted in the United States, 22% (5/23) in Japan, and 9% (2/23) in South Korea. The primary data sources used to construct the ECAs involved pooled data from previous trials (35%, 8/23), administrative health databases (17%, 4/23) and electronic medical records (17%, 4/23). While 52% (12/23) of the studies employed methods to align treatment and ECAs characteristics, 48% (11/23) lacked explicit strategies.
CONCLUSIONS: ECAs offer a valuable approach in oncology research, particularly when alternative designs are not feasible. However, careful methodological planning and detailed reporting are essential for meaningful and reliable results.
METHODS: We systematically searched four databases, namely MEDLINE, EMBASE, Web of Science, and Scopus. We screened titles, abstracts, and full texts for eligible articles focusing on patients undergoing therapy for cancer, employing ECAs, and reporting clinical outcomes.
RESULTS: Of the 629 articles screened, 23 were included in this review. The earliest included studies were published in 1996, while most studies were published in the past 5 years. 44% (10/23) of ECAs were employed in blood-related cancer studies. Geographically, 30% (7/23) of studies were conducted in the United States, 22% (5/23) in Japan, and 9% (2/23) in South Korea. The primary data sources used to construct the ECAs involved pooled data from previous trials (35%, 8/23), administrative health databases (17%, 4/23) and electronic medical records (17%, 4/23). While 52% (12/23) of the studies employed methods to align treatment and ECAs characteristics, 48% (11/23) lacked explicit strategies.
CONCLUSIONS: ECAs offer a valuable approach in oncology research, particularly when alternative designs are not feasible. However, careful methodological planning and detailed reporting are essential for meaningful and reliable results.
摘要：
目的：随机对照试验（RCTs）是评价癌症新疗法疗效和安全性的金标准。然而,将患者纳入临床试验的控制组可能对罕见癌症具有挑战性，特别是在精确肿瘤学和靶向治疗的背景下。外部控制臂（ECA）是解决临床研究设计中这些挑战的潜在解决方案。我们进行了范围审查，以探讨ECA在肿瘤学中的应用。
方法：我们系统地检索了四个数据库，即MEDLINE,EMBASE,WebofScience,还有Scopus.我们筛选了标题，摘要,以及针对接受癌症治疗的患者的合格文章的全文，雇用ECA，并报告临床结果。
结果：在筛选的629篇文章中，这次审查中包括23人。最早的纳入研究发表于1996年，而大多数研究发表于过去5年。44％（10/23）的ECA用于血液相关癌症研究。地理上,30%（7/23）的研究是在美国进行的，22%（5/23）在日本，9％（2/23）在韩国。用于构建ECA的主要数据源涉及来自先前试验的汇总数据(35%，8/23），行政健康数据库(17%，4/23）和电子病历（17%，4/23）。虽然52%(12/23)的研究采用了调整治疗和ECA特征的方法，48%(11/23)缺乏明确的策略。
结论：ECA为肿瘤学研究提供了一种有价值的方法，特别是当替代设计是不可行的。然而,仔细的方法规划和详细的报告对于有意义和可靠的结果至关重要。
方法：我们系统地检索了四个数据库，即MEDLINE,EMBASE,WebofScience,还有Scopus.我们筛选了标题，摘要,以及针对接受癌症治疗的患者的合格文章的全文，雇用ECA，并报告临床结果。
结果：在筛选的629篇文章中，这次审查中包括23人。最早的纳入研究发表于1996年，而大多数研究发表于过去5年。44％（10/23）的ECA用于血液相关癌症研究。地理上,30%（7/23）的研究是在美国进行的，22%（5/23）在日本，9％（2/23）在韩国。用于构建ECA的主要数据源涉及来自先前试验的汇总数据(35%，8/23），行政健康数据库(17%，4/23）和电子病历（17%，4/23）。虽然52%(12/23)的研究采用了调整治疗和ECA特征的方法，48%(11/23)缺乏明确的策略。
结论：ECA为肿瘤学研究提供了一种有价值的方法，特别是当替代设计是不可行的。然而,仔细的方法规划和详细的报告对于有意义和可靠的结果至关重要。
