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本文引用的文献

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N Engl J Med. 2011 Oct 6;365(14):1273-83. doi: 10.1056/NEJMoa0910383.
2
Genomics education for health care professionals in the 21st century.21世纪面向医疗保健专业人员的基因组学教育。
JAMA. 2011 Sep 7;306(9):989-90. doi: 10.1001/jama.2011.1245.
3
Clinical development of cabazitaxel for the treatment of castration-resistant prostate cancer.卡巴他赛在去势抵抗性前列腺癌治疗中的临床开发。
Clin Med Insights Oncol. 2011;5:163-9. doi: 10.4137/CMO.S6566. Epub 2011 May 24.
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Abiraterone and increased survival in metastatic prostate cancer.阿比特龙与转移性前列腺癌患者的生存获益
N Engl J Med. 2011 May 26;364(21):1995-2005. doi: 10.1056/NEJMoa1014618.
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Identification of potential surrogate end points in randomized clinical trials of aggressive and indolent non-Hodgkin's lymphoma: correlation of complete response, time-to-event and overall survival end points.侵袭性和惰性非霍奇金淋巴瘤随机临床试验中潜在替代终点的鉴定:完全缓解、时间相关终点和总生存终点的相关性。
Ann Oncol. 2011 Jun;22(6):1392-1403. doi: 10.1093/annonc/mdq615. Epub 2011 Jan 25.
6
Making the investigational oncology pipeline more efficient and effective: are we headed in the right direction?提高肿瘤学研究管道的效率和效果:我们是否走在正确的道路上?
Clin Cancer Res. 2010 Dec 15;16(24):5956-62. doi: 10.1158/1078-0432.CCR-10-1279.
7
ALK inhibition for non-small cell lung cancer: from discovery to therapy in record time.ALK 抑制治疗非小细胞肺癌:从发现到治疗,创纪录的时间。
Cancer Cell. 2010 Dec 14;18(6):548-51. doi: 10.1016/j.ccr.2010.11.033.
8
EML4-ALK mutations in lung cancer that confer resistance to ALK inhibitors.非小细胞肺癌中导致对 ALK 抑制剂耐药的 EML4-ALK 突变。
N Engl J Med. 2010 Oct 28;363(18):1734-9. doi: 10.1056/NEJMoa1007478.
9
Anaplastic lymphoma kinase inhibition in non-small-cell lung cancer.间变性淋巴瘤激酶抑制在非小细胞肺癌中的作用。
N Engl J Med. 2010 Oct 28;363(18):1693-703. doi: 10.1056/NEJMoa1006448.
10
Prednisone plus cabazitaxel or mitoxantrone for metastatic castration-resistant prostate cancer progressing after docetaxel treatment: a randomised open-label trial.多西他赛治疗后进展的转移性去势抵抗性前列腺癌患者中,泼尼松联合卡巴他赛或米托蒽醌治疗的随机开放标签试验。
Lancet. 2010 Oct 2;376(9747):1147-54. doi: 10.1016/S0140-6736(10)61389-X.

从概念验证到临床确认:靶向药物不断发展的临床试验设计

Proof of concept to clinical confirmation: evolving clinical trial designs for targeted agents.

作者信息

Finn Laura, Tan Winston

机构信息

Hematology/Oncology, Mayo Clinic Florida, Jacksonville, FL 32224, USA.

出版信息

ISRN Oncol. 2012;2012:478607. doi: 10.5402/2012/478607. Epub 2012 Jun 3.

DOI:10.5402/2012/478607
PMID:22701804
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3371752/
Abstract

No single therapy benefits the majority of patients in the practice of oncology as responses differ even among patients with similar tumor types. The variety of response to therapy witnessed while treating our patients supports the concept of personalized medicine using patients' genomic and biologic information and their clinical characteristics to make informed decisions about their treatment. Personalized medicine relies on identification and confirmation of biologic targets and development of agents to target them. These targeted agents tend to focus on subsets of patients and provide improved clinical outcomes. The continued success of personalized medicine will depend on the expedited development of new agents from proof of concept to confirmation of clinical efficacy.

摘要

在肿瘤学实践中,没有单一疗法能使大多数患者受益,因为即使是肿瘤类型相似的患者,其反应也存在差异。在治疗患者过程中所见到的对治疗反应的多样性,支持了使用患者的基因组和生物学信息及其临床特征来做出关于其治疗的明智决策的个性化医疗概念。个性化医疗依赖于生物靶点的识别与确认以及针对这些靶点的药物研发。这些靶向药物往往针对特定患者亚群,并能改善临床疗效。个性化医疗的持续成功将取决于从概念验证到临床疗效确认的新型药物的加速研发。