Modernizing Clinical Trial Eligibility Criteria: Recommendations of the American Society of Clinical Oncology-Friends of Cancer Research Brain Metastases Working Group
Modernizing Clinical Trial Eligibility Criteria: Recommendations of the American Society of Clinical Oncology-Friends of Cancer Research Brain Metastases Working Group
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DOI：
10.1200/jco.2017.74.0761
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发表时间：
2017-11-20
影响因子：
45.3
通讯作者：
Kim, Edward S.
中科院分区：
文献类型：
--
作者：
Lin, Nancy U.;Prowell, Tatiana;Kim, Edward S.
目的：扩大试验资格以改善累积性和可及性并更好地反映拟治疗人群已被视为优先事项。从历史上看，由于临床试验所有阶段的限制性资格，对脑转移患者的研究一直不足。方法2016年，经过文献检索和一系列电话会议，召开了一次多方利益相关者研讨会。我们的工作组专注于制定关于将脑转移患者纳入临床试验的共识建议，作为包括最低年龄、HIV状态和器官功能障碍在内的更广泛努力的一部分。工作组试图平衡保护患者安全、促进获得研究性治疗和确保试验完整性的需求。根据讲习班的投入，准则得到了进一步完善和最后确定。工作组确定了三个关键人群：已治疗/稳定的脑转移患者，定义为在研究开始时接受过脑转移治疗且中枢神经系统疾病放射学稳定的患者；活动性脑转移，在研究开始时定义为新发和/或进展性脑转移；还有那些患有脑膜轻症的人。在大多数情况下，工作组鼓励将已治疗/稳定的脑转移患者纳入临床试验。为活动性脑转移患者制定了一个关键考虑因素框架。对于患有轻脑膜疾病的患者，如果预期中枢神经系统活动并且与特定疾病类型相关，建议在早期和后期试验中纳入单独的队列。结论在许多情况下，扩大脑转移患者的资格是合理的，并可能加速在这一高临床需求领域开发有效的治疗方法。(C) 2017年由美国临床肿瘤学会出版
PurposeBroadening trial eligibility to improve accrual and access and to better reflect intended-to-treat populations has been recognized as a priority. Historically, patients with brain metastases have been understudied, because of restrictive eligibility across all phases of clinical trials.MethodsIn 2016, after a literature search and series of teleconferences, a multistakeholder workshop was convened. Our working group focused on developing consensus recommendations regarding the inclusion of patients with brain metastases in clinical trials, as part of a broader effort that encompassed minimum age, HIV status, and organ dysfunction. The working group attempted to balance the needs of protecting patient safety, facilitating access to investigational therapies, and ensuring trial integrity. On the basis of input at the workshop, guidelines were further refined and finalized.ResultsThe working group identified three key populations: those with treated/stable brain metastases, defined as patients who have received prior therapy for their brain metastases and whose CNS disease is radiographically stable at study entry; those with active brain metastases, defined as new and/or progressive brain metastases at the time of study entry; and those with leptomeningeal disease. In most circumstances, the working group encourages the inclusion of patients with treated/stable brain metastases in clinical trials. A framework of key considerations for patients with active brain metastases was developed. For patients with leptomeningeal disease, inclusion of a separate cohort in both early-phase and later-phase trials is recommended, if CNS activity is anticipated and when relevant to the specific disease type.ConclusionExpanding eligibility to be more inclusive of patients with brain metastasis is justified in many cases and may speed the development of effective therapies in this area of high clinical need. (C) 2017 by American Society of Clinical Oncology