Levine Cancer Institute at the Carolinas HealthCare System, 1021 Morehead Medical Drive, Suite 2100, Charlotte, NC 28204, United States.
NRG Oncology/Gynecologic Oncology Group Statistics & Data Center, Roswell Park Cancer Institute, Buffalo, NY 14263, United States.
Gynecol Oncol. 2018 Jun;149(3):605-611. doi: 10.1016/j.ygyno.2018.04.008. Epub 2018 Apr 24.
Performing clinical trials in rare gynecologic cancers presents specific challenges. Strategies for improving accrual and modifications in clinical trial design are outlined.
The literature was reviewed in order to present statistical designs pertinent to the study of rare gynecologic cancers. The experience of the Gynecologic Oncology Group/NRG Oncology is outlined as it relates to rare gynecologic cancer clinical trial development.
Significant progress has been made in studying rare tumors, both nationally and in gynecologic oncology, but challenges inherent to the study of uncommon diseases remain. Important components of these trials include establishing the standard of care, utilizing the appropriate clinical trial design to effectively answer the question in the trial, accurately estimating sample size, choosing modified and realistic endpoints, and avoiding pitfalls specific to rare tumors. Adaptive trial design and statistical modifications are important components of clinical trial design in rare tumors.
Strategies for effective study of rare gynecologic cancers must be implemented when designing clinical trials for these patients.
在罕见妇科癌症中开展临床试验具有特殊的挑战。本文介绍了提高入组率和临床试验设计修改的策略。
为了提出与罕见妇科癌症研究相关的统计学设计，我们对文献进行了综述。概述了妇科肿瘤学组/ NRG 肿瘤学在罕见妇科癌症临床试验开发方面的经验。
在研究罕见肿瘤方面，无论是在全国范围内还是在妇科肿瘤学领域，都取得了重大进展，但研究罕见疾病固有的挑战仍然存在。这些试验的重要组成部分包括确立标准治疗方法，利用适当的临床试验设计有效地回答试验中的问题，准确估计样本量，选择修改后的、切合实际的终点，并避免罕见肿瘤的特有陷阱。适应性试验设计和统计学修改是罕见肿瘤临床试验设计的重要组成部分。
在为这些患者设计临床试验时，必须实施针对罕见妇科癌症的有效研究策略。
