Survival in Patients With Brain Metastases: Summary Report on the Updated Diagnosis-Specific Graded Prognostic Assessment and Definition of the Eligibility Quotient
Survival in Patients With Brain Metastases: Summary Report on the Updated Diagnosis-Specific Graded Prognostic Assessment and Definition of the Eligibility Quotient
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DOI：
10.1200/jco.20.01255
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发表时间：
2020-11-10
影响因子：
45.3
通讯作者：
Mehta, Minesh P.
中科院分区：
文献类型：
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作者：
Sperduto, Paul W.;Mesko, Shane;Mehta, Minesh P.
传统观点认为，脑转移患者没有资格参加临床试验，因为他们担心生存率低会掩盖其他有希望的治疗方法的好处。我们的小组以前发表了诊断特异性分级预后评估（GPA）。使用分子标志物和新发现的预后因素的更大当代队列的更新已经发表。这项工作的目的是在一个单一的报告，以指导治疗的选择，分层研究，并定义一个合格商数，以扩大eligibility.METHODSA多机构数据库的6,984例新诊断的脑转移瘤患者进行多变量分析的预后因素和治疗与每个主要网站的生存。重要因素被用来定义更新的GPA。GPA为4.0和0.0分别与最佳和最差预后相关。显著的预后因素因诊断而异，新的预后因素被确定。这些因素被纳入更新后的GPA中，亚组之间存在稳健的分离（P <0.01）。生存率有所提高，但差异很大，从7-47个月，3-36个月，5-34个月，3-17个月，和4-35个月，分别为非小细胞肺癌，乳腺癌，黑色素瘤，胃肠道和肾癌脑转移患者的GPA差异很大。更新后的GPA（可在brainmetgpa.com免费获得）提供了一个准确的工具，用于估计生存率，个性化治疗和分层临床试验。不应排除脑转移患者，应鼓励入组，这些试验应按GPA分层，以确保这些试验进行适当的比较。此外，我们建议扩大入选资格，允许入组既往接受过治疗的脑转移患者，这些患者有50%或更高的额外生存年的概率（资格商数> 0.50）。
PURPOSEConventional wisdom has rendered patients with brain metastases ineligible for clinical trials for fear that poor survival could mask the benefit of otherwise promising treatments. Our group previously published the diagnosis-specific Graded Prognostic Assessment (GPA). Updates with larger contemporary cohorts using molecular markers and newly identified prognostic factors have been published. The purposes of this work are to present all the updated indices in a single report to guide treatment choice, stratify research, and define an eligibility quotient to expand eligibility.METHODSA multi-institutional database of 6,984 patients with newly diagnosed brain metastases underwent multivariable analyses of prognostic factors and treatments associated with survival for each primary site. Significant factors were used to define the updated GPA. GPAs of 4.0 and 0.0 correlate with the best and worst prognoses, respectively.RESULTSSignificant prognostic factors varied by diagnosis and new prognostic factors were identified. Those factors were incorporated into the updated GPA with robust separation (P < .01) between subgroups. Survival has improved, but varies widely by GPA for patients with non-small-cell lung, breast, melanoma, GI, and renal cancer with brain metastases from 7-47 months, 3-36 months, 5-34 months, 3-17 months, and 4-35 months, respectively.CONCLUSIONMedian survival varies widely and our ability to estimate survival for patients with brain metastases has improved. The updated GPA (available free at brainmetgpa.com) provides an accurate tool with which to estimate survival, individualize treatment, and stratify clinical trials. Instead of excluding patients with brain metastases, enrollment should be encouraged and those trials should be stratified by the GPA to ensure those trials make appropriate comparisons. Furthermore, we recommend the expansion of eligibility to allow for the enrollment of patients with previously treated brain metastases who have a 50% or greater probability of an additional year of survival (eligibility quotient > 0.50).