Estimation of the Percentage of US Patients With Cancer Who Benefit From Genome-Driven Oncology
Estimation of the Percentage of US Patients With Cancer Who Benefit From Genome-Driven Oncology
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DOI：
10.1001/jamaoncol.2018.1660
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发表时间：
2018-08-01
期刊：
影响因子：
28.4
通讯作者：
Prasad, Vinay
中科院分区：
文献类型：
--
作者：
Marquart, John;Chen, Emerson Y.;Prasad, Vinay
到目前为止，基因组驱动的癌症治疗的好处还没有被量化。目的我们试图估计从2006年到2018年，美国有资格接受美国食品和药物管理局(FDA)批准的基因组驱动治疗并从中受益的晚期或转移性癌症患者的年度百分比。设计、背景和参与者使用公开的横断面数据进行回顾性横断面研究，这些数据包括：(1)晚期或转移性癌症患者的人口学特征；(2)FDA从2006年1月到2018年1月批准的癌症药物数据；(3)药物标签反应的测量和持续时间；以及(4)已发表的报告估计各种基因组异常的频率，用于估计在研究期间有资格接受基因组驱动治疗并将从其受益的患者的百分比。主要结果和测量每年符合并受益于基因组靶向和基因组信息治疗的美国癌症患者的估计百分比、基因组信息适应症的应答率和反应持续时间。结果从2006年1月1日到2018年1月31日，共有31种药物、38种FDA批准的适应症符合我们的基因组靶向或基因组信息治疗纳入标准。2006年，有资格接受基因组靶向治疗的患者估计数为28 72人(转移性癌症患者总数为564 830人，占5.09%，95%可信区间为5.03%-5.14%)。到2018年，这一数字已增加到60人中的50 811人(640人，或8.33%(95%CI，8.26%-8.40%)。2006年，基因组信息治疗的合格患者数量为5人(564 830人中有301人，或10.50%(95%CI，10.42%-10.58%))。2018年，可为60名患者中的94 157人(640人，或15.44%(95%CI，15.35%-15.53%)的转移性癌症患者提供基因组信息治疗。2006年，估计受益于基因组靶向治疗的癌症患者的百分比为0.70%(95%CI，0.68%-0.72%)，2018年，这一比例增加到4.90%(95%CI，4.85%-4.95%)。2006年，基因组知情治疗的受益百分比估计为1.31%(95%可信区间，1.28%-1.34%)，2018年增至6.62%(95%可信区间，6.56%-6.68%)。截至2018年1月，所有基因组信息药物的中位总缓解率为54%，中位缓解期为29.5个月。结论和相关性尽管符合基因组驱动治疗条件的患者数量随着时间的推移而增加，但这些药物帮助了少数晚期癌症患者。为了加速精确肿瘤学的进展，应该开发新的基因组疗法的试验设计，并寻求广泛的药物开发组合，包括免疫治疗和细胞毒方法。
IMPORTANCE To date, the benefit of genome-driven cancer therapy has not been quantified.OBJECTIVE We sought to estimate the annual percentage of patients in the United States with advanced or metastatic cancer who could be eligible for and benefit from US Food and Drug Administration (FDA)-approved genome-driven therapy from 2006 to 2018.DESIGN, SETTING, AND PARTICIPANTS Retrospective cross-sectional study using publically available data of (1) demographic characteristics of patients with advanced or metastatic cancer; (2) FDA data on cancer drugs approved from January 2006 through January 2018; (3) measures of response and duration of response from drug labels; and (4) published reports estimating the frequency of various genomic aberrations used to estimate what percentage of patients would have been eligible for and would have benefited from genome-driven therapy during the studied period.MAIN OUTCOMES AND MEASURES Estimated percentage of US patients with cancer eligible for and benefiting from genome-targeted and genome-informed therapy by year, response rate of genome-informed indications, and duration of response.RESULTS A total of 31 drugs with 38 FDA-approved indications met our inclusion criteria for genome-targeted or genome-informed therapy from January 1, 2006, through January 31, 2018. The estimated number of patients eligible for genome-targeted therapy in 2006 was 28 72(of a total 564 830 patients with metastatic cancer, or 5.09% (95% CI, 5.03%-5.14%). By 2018, this number had increased to 50 811 of 60(640, or 8.33%(95% CI, 8.26%-8.40%). For genome-informed therapy in 2006, the eligible number of patients was 5(301 of 564 830, or 10.50% (95% CI, 10.42%-10.58%). In 2018, genome-informed treatment could be offered to 94 157 of 60(640, or 15.44%(95% CI, 15.35%-15.53%) of patients with metastatic cancer. The percentage of patients with cancer estimated to benefit from genome-targeted therapy in 2006 was 0.70% (95% CI, 0.68%-0.72%), and in 2018, it had increased to 4.90% (95% CI, 4.85%-4.95%). For genome-informed treatment in 2006, the percentage estimated to benefit was 1.31% (95% CI, 1.28%-1.34%), and in 2018, it had increased to 6.62%(95% CI, 6.56%-6.68%). The median overall response rate for all genome-informed drugs through January 2018 was 54%, and the median duration of response was 29.5 months.CONCLUSIONS AND RELEVANCE Although the number of patients eligible for genome-driven treatment has increased over time, these drugs have helped a minority of patients with advanced cancer. To accelerate progress in precision oncology, novel trial designs of genomic therapies should be developed, and broad portfolios of drug development, including immunotherapeutic and cytotoxic approaches, should be pursued.